UCSF Benioff Children's Hospital Oakland
Hematology/Oncology Department
UCSF Benioff Children's Hospital Oakland


Recent Faculty Publications (2018-2021)

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PEER-REVIEWED ARTICLES ABSTRACTS INVITED LECTURES/ARTICLES/BOOKS/CHAPTERS PATENTS NEWS MEDIA AWARDS

PEER-REVIEWED ARTICLES:

  1. Abrams DI, Couey P, Dixit N, Sagi V, Hagar W, Vichinsky E, Kelly ME, Connett JE, Gupta K. Effect of Inhaled Cannabis for Pain in Adults With Sickle Cell Disease: A Randomized Clinical Trial. JAMA Netw Open 2020 Jul 1; 3(7):e2010874. PMC7368173 Epub 2020/07/18. URL.
  2. Adesina OO, Jenkins IC, Wu QV, Fung EB, Narla RR, Lipkin EW, Mahajan K, Konkle BA, Kruse-Jarres R. Urinary cross-linked carboxyterminal telopeptide, a bone resorption marker, decreases after vaso-occlusive crises in adults with sickle cell disease. Blood Cells Mol Dis 2020 Feb; 80(102369). Epub 2019/11/05. URL.
  3. Adesina OO, Neumayr LD. Osteonecrosis in sickle cell disease: an update on risk factors, diagnosis, and management. Hematology Am Soc Hematol Educ Program 2019 Dec 6; 2019(1):351-8. PMC6913430 interests. Epub 2019/12/07. URL.
  4. Agrawal AK, Robertson S, Litwin L, Tringale E, Treadwell M, Hoppe C, Marsh A. Virtual reality as complementary pain therapy in hospitalized patients with sickle cell disease. Pediatr Blood Cancer 2019 Feb; 66 2:e27525. URL.
  5. Agrawal AK, Aguilar A, Feusner J. Unifying the diagnosis of isolated central nervous system relapse in acute lymphoblastic leukaemia based on minimal residual disease testing. Br J Haematol 2018. URL
  6. Akbari M, Patel R, Carrao V, Hagar W, Vichinsky E, Chuang SK. Evaluation of Mandible Fractures in Patients With Sickle Cell Anemia-A Nationwide Study . J Oral Maxillofac Surg 2019 Jul; 77(7):1418-22. Epub 2019/02/02. URL.
  7. Allard HM, Calvelli L, Weyhmiller MG, Gildengorin G, Fung EB. Vertebral Bone Density Measurements by DXA are Influenced by Hepatic Iron Overload in Patients with Hemoglobinopathies. J Clin Densitom 2019 Jul - Sep; 22(3):329-37. PMC6447456 Epub 2018/08/21. URL.
  8. Antwi-Boasiako C, Andemariam B, Colombatti R, Asare EV, Strunk C, Piccone CM, Manwani D, Boruchov D, Farooq F, Urbonya R, Wilson S, Boatemaa GD, Perrotta S, Sainati L, Rivers A, Rao S, Zempsky W, Ekem I, Sey F, Segbefia C, Inusa B, Tartaglione I, Campbell AD. A study of the geographic distribution and associated risk factors of leg ulcers within an international cohort of sickle cell disease patients: the CASiRe group analysis. Ann Hematol 2020 Sep; 99(9):2073-9. PMC7430152 Epub 2020/05/08. URL
  9. Bakkour S, Saa P, Groves JA...Vichinsky E et al. NHLBI Recipient Epidemiology and Donor Evaluation Study-IV-Pediatric (REDS-IV-P). Minipool Testing for SARS-CoV-2 RNA in United States Blood Donors. 2021 (in progress).
  10. Ballas SK, Connes P. Vichinsky, E (collaborator), Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia. Rheological properties of sickle erythrocytes in patients with sickle cell anaemia: the effect of hydroxyurea, fetal hemoglobin and alpha-thalassaemia. Eur J Haematol 2018:101(6)798-803. Epub 2018/09/12. URL.
  11. Ballas SK, Kuypers F, Gordeuk V, Hankins JS, Thompson A, Vichinsky E. Is it Time to Rethink Hemoglobin Thresholds in Sickle Cell Disease? Am J Hematol 2021 (submitted).
  12. Bansal D, Lal A. Iron Metabolism, Hemolytic Anemia, and Thalassemia. Indian J Pediatr 2020 Jan; 87(1):56-7. Epub 2019/12/13. URL.
  13. Bansal D, Lal A. Taking Stock of Hemophilia, Immune Thrombocytopenia, and Bone Marrow Failure. Indian J Pediatr 2020 Feb; 87(2):132-3. Epub 2020/01/05. URL.
  14. Barredo JC, Hastings C, Lu X, Devidas M, Chen Y, Armstrong D, Winick N, Wood BL, Yanofsky R, Loh M, Gastier-Foster JM, Jorstad DT, Marcus R, Ritchey K, Carrol WL, Hunger SP. Isolated late testicular relapse of B-cell acute lymphoblastic leukemia treated with intensive systemic chemotherapy and response-based testicular radiation: A Children's Oncology Group study. Pediatr Blood Cancer 2018;65 5:e26928. URL
  15. Belisario AR, Carneiro-Proietti AB, Sabino EC, Araujo A, Loureiro P, Maximo C, Flor-Park MV, Rodrigues D, Ozahata MC, McClure C, Mota RA, Gomes Moura IC, Custer B, Kelly S, Recipient Epidemiology and Donor Evaluation Study (REDS-III) International Component Brazil. Hb S/beta-Thalassemia in the REDS-III Brazil Sickle Cell Disease Cohort: Clinical, Laboratory and Molecular Characteristics. Hemoglobin 2020 Mar 16:1-9. URL.
  16. Belisario AR, Blatyta PF, Vivanco D, Oliveira CDL, Carneiro-Proietti AB, Sabino EC, de Almeida-Neto C, Loureiro P, Maximo C, de Oliveira Garcia Mateos S, Flor-Park MV, de Oliveira Werneck Rodrigues D, Afonso Mota R, Goncalez TT, Hoffmann TJ, Kelly S, Custer B, Recipient Epidemiology and Donor Evaluation Study (REDS-III) International Component Brazil. Association of HIV infection with clinical and laboratory characteristics of sickle cell disease. BMC Infect Dis 2020 Aug 27; 20(1):638. PMC7457248 Epub 2020/08/29. URL
  17. Berliner C, Wang ZJ, Singer ST, Grosse R, McDonough RV, Padua E, Weyhmiller M, Vichinsky E, Adam G, Yamamura J, Fischer R, Schoennagel BP. Anterior pituitary volume in patients with transfusion dependent anemias: volumetric approaches and relation to pituitary R2. 2021 (in progress).
  18. Blatyta PF, Kelly S, Sabino E, Preiss L, Mendes F, Carneiro-Proietti AB, Werneck Rodrigues DO, Mota R, Loureiro P, Maximo C, Park M, Mendrone-Jr A, Goncalez TT, de Almeida Neto C, Custer B, National Heart, Lung Blood Institute: Recipient Epidemiology Donor Evaluation Study (REDS III) International Component--Brazil. Prevalence of serologic markers of transfusion and sexually transmitted infections and their correlation with clinical features in a large cohort of Brazilian patients with sickle cell disease. Transfusion 2020 Feb; 60(2):343-50. URL.
  19. Blatyta PF, Kelly S, Goncalez TT, Carneiro-Proietti AB, Salomon T, Miranda C, Sabino E, Preiss L, Maximo C, Loureiro P, Custer B, de Almeida-Neto C, NHLBI Recipient Epidemiology Donor Evaluation Study-III International Component, Brazil. Characterization of HIV risks in a Brazilian sickle cell disease population. BMC Public Health 2020 Oct 23; 20(1):1606. PMC7585195 Epub 2020/10/25. URL
  20. Bose N, Carlberg K, Sensabaugh G, Erlich H, Calloway C. Target capture enrichment of nuclear SNP markers for massively parallel sequencing of degraded and mixed samples. Forensic Sci Int Genet 2018;34:186-96. URL
  21. Brazauskas R, Scigliuolo GM, Wang HL, Cappelli B, Ruggeri A, Fitzhugh CD, Hankins JS, Kanter J, Meerpohl JJ, Panepinto JA, Rondelli D, Shenoy S, Walters MC, Wagner JE, Tisdale JF, Gluckman E, Eapen M. Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease. Blood 2020 Jul 30; 136(5):623-6. PMC7393258 Epub 2020/06/11. URL.
  22. Brown RC, Cruz K, Kalfa TA, Kuypers FA, Saraf SL, Estepp JH, Smart LR, Malik P, Lerman M, Mayer R, Ribadeneira MD, Forsyth S, Schroeder P, Wu E, Kelly P, Telen MJ. FT-4202, an allosteric activator of pyruvate kinase-r, demonstrates proof of mechanism and proof of concept after asingle dose and after multiple daily doses in a phase 1 study of patients with sickle cell disease. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):SUPPL 1:19-20. URL
  23. Bseikri M, McCann JC, Lal A, Fong E, Graves K, Goldrich A, Block D, Gildengoren GL, Mietus-Snyder M, Shigenaga M, Suh J, Hardy K, Ames BN. A novel nutritional intervention improves lung function in overweight/obese adolescents with poorly controlled asthma: the Supplemental Nutrition in Asthma Control (SNAC) pilot study. The FASEB Journal 2018:fj.201700338. URL.
  24. Bukini D, deVries J, Treadwell M, Anie K, Dennis-Antwi J, Kamga KK, McCurdy S, Ohene-Frempong K, Makani J, Wonkam A. Exploring the Role of Shared Decision Making in the Consent Process for Pediatric Genomics Research in Cameroon, Tanzania, and Ghana. AJOB Empir Bioeth 2019 Jul-Sep; 10(3):182-9. Epub 2019/08/06. URL.
  25. Byer L, Kline CN, Coleman C, Allen IE, Whitaker E, Mueller S. A systematic review and meta-analysis of outcomes in pediatric, recurrent ependymoma. J Neurooncol 2019 Sep; 144(3):445-52. URL.
  26. Cabana MD, Kanter J, Marsh AM, Treadwell MJ, Rowland M, Stemmler P, Bardach NS. Barriers to Pediatric Sickle Cell Disease Guideline Recommendations. Glob Pediatr Health 2019 6(2333794X19847026. PMC6501475 Epub 2019/05/21. URL.
  27. Cabana MD, Marsh A, Treadwell MJ, Stemmler P, Rowland M, Bender MA, Bhasin N, Chung JH, Hassell K, Abdul Rashid NFN, Wong TE, Bardach NS. Improving Preventive Care for Children With Sickle Cell Anemia: A Quality Improvement Initiative. Pediatr Qual Saf 2021 Jan-Feb; 6(1):e379. PMC7781296 this article. Epub 2021/01/08. URL.
  28. Campbell AD, Colombatti R, Andemariam B, Strunk C, Tartaglione I, Piccone CM, Manwani D, Asare EV, Boruchov D, Farooq F, Urbonya R, Boatemaa GD, Perrotta S, Sainati L, Rivers A, Rao S, Zempsky W, Sey F, Segbefia C, Inusa B, Antwi-Boasiako C. An Analysis of Racial and Ethnic Backgrounds Within the CASiRe International Cohort of Sickle Cell Disease Patients: Implications for Disease Phenotype and Clinical Research. Journal of racial and ethnic health disparities 2021 8(1):99-106. URL
  29. Cappellini MD, Viprakasit V, Taher AT, Georgiev P, Kuo KHM, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Forni GL, Perrotta S, Khelif A, Lal A, Kattamis A, Vlachaki E, Origa R, Aydinok Y, Bejaoui M, Ho PJ, Chew LP, Bee PC, Lim SM, Lu MY, Tantiworawit A, Ganeva P, Gercheva L, Shah F, Neufeld EJ, Thompson A, Laadem A, Shetty JK, Zou J, Zhang J, Miteva D, Zinger T, Linde PG, Sherman ML, Hermine O, Porter J, Piga A, Believe Investigators. A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent beta-Thalassemia. N Engl J Med 2020 Mar 26; 382(13):1219-31. URL.
  30. Carlberg KT, Singer ST, Vichinsky EP. Fertility and Pregnancy in Women with Transfusion-Dependent Thalassemia. Hematol Oncol Clin North Am 2018;32 2:297-315. Epub 2018/02/21. URL
  31. Casella JF, Barton BA, Kanter J, Black LV, Majumdar S, Inati A, Wali Y, Drachtman RA, Abboud MR, Kilinc Y, Fuh BR, Al-Khabori MK, Takemoto CM, Salman E, Sarnaik SA, Shah N, Morris CR, Keates-Baleeiro J, Raj A, Alvarez OA, Hsu LL, Thompson AA, Sisler IY, Pace BS, Noronha SA, Lasky JL, 3rd, de Julian EC, Godder K, Thornburg CD, Kamberos NL, Nuss R, Marsh AM, Owen WC, Schaefer A, Tebbi CK, Chantrain CF, Cohen DE, Karakas Z, Piccone CM, George A, Fixler JM, Singleton TC, Moulton T, Quinn CT, de Castro Lobo CL, Almomen AM, Goyal-Khemka M, Maes P, Emanuele M, Gorney RT, Padgett CS, Parsley E, Kronsberg SS, Kato GJ, Gladwin MT. Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease: A Randomized Clinical Trial. JAMA 2021 Apr 20; 325(15):1513-23. Epub 2021/04/21. URL.
  32. Carneiro-Proietti ABF, Kelly S, Miranda Teixeira C, Sabino EC, Alencar CS, Capuani L, Salomon Silva TP, Araujo A, Loureiro P, Maximo C, Lobo C, Flor-Park MV, Rodrigues DOW, Mota RA, Goncalez TT, Hoppe C, Ferreira JE, Ozahata M, Page GP, Guo Y, Preiss LR, Brambilla D, Busch MP, Custer B, International Component of the NHLBI Recipient Epidemiology Donor Evaluation Study. Clinical and genetic ancestry profile of a large multi-centre sickle cell disease cohort in Brazil. Br J Haematol 2018 Sep; 182(6):895-908. Epub 2018/07/22. URL.
  33. Chao AK, Meyer JA, Lee AG, Hecht A, Tarver T, Van Ziffle J, Koegel AK, Golden C, Braun BS, Sweet-Cordero EA, Smith CC, Dvorak CC, Loh ML, Stieglitz E. Fusion driven JMML: a novel CCDC88C-FLT3 fusion responsive to sorafenib identified by RNA sequencing. Leukemia 2020 Feb; 34(2):662-6. PMC6995757 Epub 2019/09/13. URL.
  34. Chapin J, Cohen AR, Neufeld E, Vichinsky E, Giardina PJ, Boudreaux J, Lee BC, Kenney K, Trimble S, Thompson AA. An Update on the U.S. Adult Thalassemia Population: A Report from the CDC Thalassemia Treatment Centers An Update on the U.S. Adult Thalassemia Population: A Report from the CDC Thalassemia Treatment Centers. 2021 (submitted).
  35. Choi YJ, Saba JD. Sphingosine phosphate lyase insufficiency syndrome (SPLIS): A novel inborn error of sphingolipid metabolism. Adv Biol Regul 2019 Jan; 71(128-40. PMC6347531 Epub 2018/10/03. URL.
  36. Chung JE, Magis W, Vu J, Heo SJ, Wartiovaara K, Walters MC, Kurita R, Nakamura Y, Boffelli D, Martin DIK, Corn JE, DeWitt MA. CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells. PLoS One 2019 14(1):e0208237. PMC6333401 Epub 2019/01/16. URL.
  37. Chung JH, Phalke N, Hastings C, Killilea DW, Feusner JH, Fung EB. Zinc Deficiency and its Relationship with Treatment Related Toxicity in Children with Cancer. Pediatr Blood Cancer 2021 (accepted 27-Apr-21).
  38. Cintho-Ozahata M, Page GP, Guo Y, Ferreira JE, Dinardo CL, Carneiro-Proietti ABF, Loureiro P, Mota RA, Rodrigues DOW, Belisario AR, Maximo C, Flor-Park MV, Custer B, Kelly S, Sabino EC, International Component of the NHLBI Recipient Epidemiology Donor Evaluation Study. Clinical and Genetic Predictors of Priapism in Sickle Cell Disease: Results from the Recipient Epidemiology and Donor Evaluation Study III Brazil Cohort Study. J Sex Med 2019 Dec; 16(12):1988-99. PMC6904926 Epub 2019/11/02. URL.
  39. Clayton-Jones D, Matthie N, Treadwell M, Field JJ, Mager A, Sawdy R, George Dalmida S, Leonard C, Koch KL, Haglund K. Social and Psychological Factors Associated With Health Care Transition for Young Adults Living With Sickle Cell Disease. J Transcult Nurs 2019 Dec 31:1043659619896837. Epub 2020/01/01. URL.
  40. Coleman C, Stoller S, Grotzer M, Stucklin AG, Nazarian J, Mueller S. Pediatric hemispheric high-grade glioma: targeting the future. Cancer Metastasis Rev 2020 Jan 27. URL.
  41. Cooney T, Fisher PG, Tao L, Clarke CA, Partap S. Pediatric neuro-oncology survival disparities in California. J Neuro-Oncol 2018;138 1:83-97. URL.
  42. Cooney T, Yeo KK, Kline C, Prados M, Haas-Kogan D, Chi S, Mueller S. Neuro-Oncology Practice Clinical Debate: targeted therapy vs conventional chemotherapy in pediatric low-grade glioma. Neuro Oncol Pract 2020 Jan; 7(1):4-10. PMC7104878. URL.
  43. Cronin RM, Hankins JS, Byrd J, Pernell BM, Kassim A, Adams-Graves P, Thompson A, Kalinyak K, DeBaun M, Treadwell M. Risk factors for hospitalizations and readmissions among individuals with sickle cell disease: results of a U.S. survey study. Hematology 2019 Dec; 24 1:189-98. URL.
  44. Cronin RM, Hankins JS, Byrd J, Pernell BM, Kassim A, Adams-Graves P, Thompson AA, Kalinyak K, DeBaun MR, Treadwell M. Modifying factors of the health belief model associated with missed clinic appointments among individuals with sickle cell disease. Hematology 2018:1-9. URL
  45. Cronin RM, Yang M, Hankins JS, Byrd J, Pernell BM, Kassim A, Adams-Graves P, Thompson AA, Kalinyak K, DeBaun M, Treadwell M. Association between hospital admissions and healthcare provider communication for individuals with sickle cell disease. Hematology 2020 Dec; 25(1):229-40. PMC7440685 Epub 2020/06/20. URL
  46. Crosby LE, Walton A, Shook LM, Ware RE, Treadwell M, Saving KL, Britto M, Peugh J, McTate E, Oyeku S, Nwankwo C, Brinkman WB. Development of a Hydroxyurea Decision Aid for Parents of Children With Sickle Cell Anemia. J Pediatr Hematol Oncol 2019 Jan; 41 1:56-63. URL.
  47. Crotty E, Downey K, Ferrerosa L, Flores C, Hegde B, Raskin S, Hwang E, Vitanza N, Okada H. Considerations when treating high-grade pediatric glioma patients with immunotherapy. Expert Rev Neurother 2021 Feb; 21(2):205-19. PMC7880880 Epub 2020/11/24. URL
  48. Cullers A, King JC, Van Loan M, Gildengorin G, Fung EB. Effect of prenatal calcium supplementation on bone during pregnancy and 1 y postpartum. Am J Clin Nutr 2019 Jan 1; 109(1):197-206. PMC6900564 Epub 2019/01/17. URL.
  49. Daak AA, Dampier CD, Fuh B, Kanter J, Alvarez OA, Black LV, McNaull MA, Callaghan MU, George A, Neumayr L, Hilliard LM, Sancilio F, Rabinowicz AL, Heeney MM. Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial). Blood Adv 2018 Aug 14; 2(15):1969-79. PMC6093734 URL.
  50. Dandoy CE, Davies SM, Ahn KW, He Y, Kolb AE, Levine J, Bo-Subait S, Abdel-Azim H, Bhatt N, Chewing J, Gadalla S, Gloude N, Hayashi R, Lalefar NR, Law J, MacMillan M, O'Brien T, Prestidge T, Sharma A, Shaw P, Winestone L, Eapen M. Comparison of total body irradiation versus non- total body irradiation containing regimens for de novo acute myeloid leukemia in children. Haematologica 2020 Jun 18. Epub 2020/06/20. URL
  51. Dandoy CE, Rotz S, Alonso PB, Klunk A, Desmond C, Huber J, Ingraham H, Higham C, Dvorak CC, Duncan C, Schoettler M, Lehmann L, Cancio M, Killinger J, Davila B, Phelan R, Mahadeo KM, Khazal S, Lalefar N, Vissa M, Myers K, Wallace G, Nelson A, Khandelwal P, Bhatla D, Gloude N, Anderson E, Huo J, Roehrs P, Auletta JJ, Chima R, Lane A, Davies SM, Jodele S. A pragmatic multi-institutional approach to understanding transplant-associated thrombotic microangiopathy after stem cell transplant. Blood Adv 2021 Jan 12; 5(1):1-11. PMC7805323 Epub 2021/02/12. URL
  52. de Martino CC, Alencar CS, Loureiro P, Carneiro-Proietti ABF, Maximo CA, Mota RA, Rodrigues DOW, Gaburo Junior N, Kelly S, Sabino EC, International Component of the NHLBI Recipient Epidemiology Donor Evaluation Study. Use of an automated pyrosequencing technique for confirmation of sickle cell disease. PLoS One 2019 14(12):e0216020. PMC6907837 Epub 2019/12/13. URL.
  53. DeBaun M, Vichinsky E. Vaso-occlusive pain management in sickle cell disease. In: Mahoney DH, Schrier SL, Tirnauer J, eds. UpToDate. Topic 7144: Version 54.0; Oct 8, 2020. UpToDate, Inc., Waltham, MA. URL
  54. DeBaun M, Jordan LC, King AA, Schatz J, Vichinsky E, Fox CK, Mckinstry RC, Telfer P, Kraut MA, Daraz L, Kirkham F, Murad MH. American Society of Hematology 2020 guidelines for sickle cell disease: Prevention, diagnosis, and treatment of cerebrovascular disease in children and adults. Blood Adv 2020 Apr 28; 4(8):1554-88. PMC7189278. URL.
  55. Dennis-Antwi JA, Ohene-Frempong K, Anie KA, Dzikunu H, Agyare VA, Boadu RO, Antwi JS, Asafo MK, Anim-Boamah O, Asubonteng AK, Agyei S, Wonkam A, Treadwell MJ. Relation Between Religious Perspectives and Views on Sickle Cell Disease Research and Associated Public Health Interventions in Ghana. J Genet Couns 2018 Sep 1. PMC6395545 Epub 2018/09/02. URL.
  56. Dessie AS, Lanning M, Nichols T, Delgado EM, Hart LS, Agrawal AK. Patient Outcomes With Febrile Neutropenia Based on Time to Antibiotics in the Emergency Department. Pediatr Emerg Care 2020 Sep 15. Epub 2020/09/18. URL
  57. DiMartino LD, Baumann AA, Hsu LL, Kanter J, Gordeuk VR, Glassberg J, Treadwell MJ, Melvin CL, Telfair J, Klesges LM, King A, Wun T, Shah N, Gibson RW, Hankins JS. Sickle Cell Disease Implementation Consortium The sickle cell disease implementation consortium: Translating evidence-based guidelines into practice for sickle cell disease. Am J Hematol 2018 Dec; 93(12):E391-E5. PMC6503654 Epub 2018/09/12. URL.
  58. Dinardo CL, Kelly S, Dezan MR, Ribeiro IH, Castilho SL, Schimidt LC, Valgueiro MDC, Preiss LR, Custer B, Sabino EC, Westhoff CM, NHLBI Recipient Epidemiology Donor Evaluation Study III. Diversity of RH and transfusion support in Brazilian sickle cell disease patients with unexplained Rh antibodies. Transfusion 2019 Oct; 59(10):3228-35. PMC6785370 Epub 2019/08/14. URL.
  59. Dinardo CL, Oliveira TGM, Kelly S, Ashley-Koch A, Telen M, Schmidt LC, Castilho S, Melo K, Dezan MR, Wheeler MM, Johnsen JM, Nickerson DA, Jain D, Custer B, Pereira AC, Sabino EC, the NHLBI Recipient Epidemiology Donor Evaluation Study International Component-Brazil, the Outcome Modifying Genes in S. C. D. study, the NHLBI Trans-Omics for Precision Medicine Program Sickle Cell Disease Working Group. Diversity of variant alleles encoding Kidd, Duffy, and Kell antigens in individuals with sickle cell disease using whole genome sequencing data from the NHLBI TOPMed Program. Transfusion 2021 Feb; 61(2):603-16. Epub 2020/11/25.URL
  60. Eapen M, Brazauskas R, Walters MC, Bernaudin F, Bo-Subait K, Fitzhugh CD, Hankins JS, Kanter J, Meerpohl JJ, Bolanos-Meade J, Panepinto JA, Rondelli D, Shenoy S, Williamson J, Woolford TL, Gluckman E, Wagner JE, Tisdale JF. Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort study. Lancet Haematol 2019 Nov; 6(11):e585-e96. PMC6813907 Epub 2019/09/10. URL.
  61. El-Haj N, Hoppe CC. Newborn Screening for SCD in the USA and Canada. Int J Neonatal Screen 2018 4(4):36. URL.
  62. El-Mallawany NK, Kamiyango W, Villiera J, Slone JS, Kovarik CL, Campbell LR, Agrawal AK, Dittmer DP, Eason AB, Ahmed S, Schutze GE, Scheurer ME, Kazembe PN, Mehta PS. Proposal of a Risk-Stratification Platform to Address Distinct Clinical Features of Pediatric Kaposi Sarcoma in Lilongwe, Malawi. J Glob Oncol 2018 Sep; 4(1-7. PMC6180767 Epub 2017/12/23. URL.
  63. Faro E, Treadwell M, King A, Shook LM, Whiteman L, Ivy E, Hulihan M, Kavanagh P, Seik S, Oyeku S, Berns S. A National Measurement Framework to Assess and Improve Sickle Cell Care in Four US Regions. Public Health Reports 2020 (accepted).
  64. Farrell AT, Panepinto J, Desai AA, Kassim AA, Lebensburger J, Walters MC, Bauer DE, Blaylark RM, DiMichele DM, Gladwin MT, Green NS, Hassell K, Kato GJ, Klings ES, Kohn DB, Krishnamurti L, Little J, Makani J, Malik P, McGann PT, Minniti C, Morris CR, Odame I, Oneal PA, Setse R, Sharma P, Shenoy S. End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings. Blood Adv 2019 Dec 10; 3(23):4002-20. PMC6963248 Epub 2019/12/07. URL.
  65. Fearon A, Marsh A, Kim J, Treadwell M. Pediatric residents' perceived barriers to opioid use in sickle cell disease pain management. Pediatr Blood Cancer 2019 Feb; 66 2:e27535. PMC6301127. URL.
  66. Field J, Vichinsky E. Overview of the management and prognosis of sickle cell disease. In: Mahoney DH, DeBaun M, Tirnauer J, eds. UpToDate. Topic 7114 Version 74; Dec 9, 2020. UpToDate, Inc. Waltham, MD. URL
  67. Flor-Park MV, Kelly S, Preiss L, Custer B, Carneiro-Proietti ABF, Araujo AS, Loureiro P, Maximo C, Rodrigues DOW, Mota RA, Sabino EC, Rocha V, NHLBI Recipient Epidemiology Donor Evaluation Study. Identification and Characterization of Hematopoietic Stem Cell Transplant Candidates in a Sickle Cell Disease Cohort. Biol Blood Marrow Transplant 2019 Oct; 25(10):2103-9. Epub 2019/06/24. URL.
  68. Franck P, Postma C, Spaans A, Veuger M, de Kort G, Hudig C, Wijermans P, Kuypers F. Hereditary elliptocytosis: Variable clinical severity caused by 3 variants in the alpha-spectrin gene. Int J Lab Hematol 2018. URL
  69. Franck P, Buijs P, Meenhuis A, Dane M, Postma C, Spaans A, Gijsbertha N, Kuypers FA, Hudig C, Kerkhoffs JL. Haemoglobinopathy: Quantitative ektacytometry as a tool in clinical management of patients with sickle cell anemia and thalassemia. Br J Haematol 2020 (Submitted).
  70. Friedman D, Dozor A, Milner J, D’Souza M, Talano J, Moore TB, Shenoy S, Shi Q, Walters MC, Vichinsky E, Parsons SK, Braniecki S, Moorthy CR, Ayello J, Flower A, Morris E, Mahanti H, Fabricatore S, Klejmont L, van de Ven C, Baxter-Lowe LA, Cairo MS. Stable to Improved Cardiac and Pulmonary Function in Children with High Risk Sickle Cell Disease Following Haploidentical Stem Cell Transplantation. Bone Marrow Transplant 2021 (submitted).
  71. Fung EB, Ahmad T, Killilea D, Hussain R, Lal A. Zinc supplementation improves markers of glucose homeostasis in thalassemia. Br J Haematol 2020 Aug; 190(3):e162-e6. Epub 2020/06/04. URL
  72. Garcia Frausto E, Sivananthan A, Golden C, Szuminski M, Prado LNP, Lopez MP, Diaz V, Nieto D, Plenert E, Langevin AM, Dupuis LL. Translation of the Pediatric Nausea Assessment Tool (PeNAT) Into Spanish and Evaluating Understandability Among Spanish-Speaking Hispanic American Children and Adolescents Receiving Chemotherapy. Hisp Health Care Int 2021 Apr 15:15404153211003341. Epub 2021/04/16. URL
  73. Goktas P, Sukharevsky IO, Larkin S, Kuypers FA, Yalcin O, Altintas A. Image-Based Flow Cytometry and Angle-Resolved Light Scattering to Define the Sickling Process. Cytometry A 2019 May; 95(5):488-98. Epub 2019/04/14. URL.
  74. Goldberg EK, Neogi S, Lal A, Higa A, Fung E. Nutritional Deficiencies Are Common in Patients with Transfusion-Dependent Thalassemia and Associated with Iron Overload. J Food Nutr Res (Newark) 2018 6(10):674-81. PMC6296481 Epub 2018/12/21. URL.
  75. Goldberg EK, Fung EB. Precision of the Hologic DXA in the Assessment of Visceral Adipose Tissue. J Clin Densitom 2020 Oct - Dec; 23(4):664-72. PMC6754313 Epub 2019/04/18. URL.
  76. Guo Y, Busch MP, Seielstad M, Endres-Dighe S, Westhoff CM, Keating B, Hoppe C, Bordbar A, Custer B, Butterworth AS, Kanias T, Mast AE, Kleinman S, Lu Y, Page GP; National Heart, Lung Blood Institute Recipient Epidemiology Donor Evaluation Study III. Development and evaluation of a transfusion medicine genome wide genotyping array. Transfusion 2019 Jan; 59 1:101-11. URL.
  77. Hagar RW. Transfusional Iron Chelation Inches Forward. Acta Haematol 2019 142(3):123-4. Epub 2019/06/13. URL.
  78. Hankins JS, Shah N, DiMartino L, Brambilla D, Fernandez ME, Gibson RW, Gordeuk VR, Lottenberg R, Kutlar A, Melvin C, Simon J, Wun T, Treadwell M, Calhoun C, Baumann A, Potter MB, Klesges L, Bosworth H. Integration of Mobile Health Into Sickle Cell Disease Care to Increase Hydroxyurea Utilization: Protocol for an Efficacy and Implementation Study. JMIR Res Protoc 2020 2020/7/14; 9(7):e16319. URL
  79. Hastings C, Vieira C, Liu B, Bascon C, Gao C, Wang RY, Casey A, Hrynkow S. Expanded access with intravenous hydroxypropyl-beta-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis. Orphanet J Rare Dis 2019 Oct 21; 14(1):228. PMC6805667 Epub 2019/10/23. URL.
  80. Hochberg J, Zahler S, Geyer MB, Chen N, Krajewski J, Harrison L, Militano O, Ozkaynak MF, Cheerva AC, Talano J, Moore TB, Gillio AP, Walters MC, Baxter-Lowe LA, Hamby C, Cairo MS. The safety and efficacy of clofarabine in combination with high-dose cytarabine and total body irradiation myeloablative conditioning and allogeneic stem cell transplantation in children, adolescents, and young adults (CAYA) with poor-risk acute leukemia. Bone Marrow Transplant 2019 Feb; 54(2):226-235. Epub 2018/06/15. URL.
  81. Hodges JR, Phillips SM, Norell S, Nwosu C, Khan H, Luo L, Badawy SM, King A, Tanabe P, Treadwell M, Rojas Smith L, Calhoun C, Hankins JS, Porter J. Intentional and unintentional nonadherence to hydroxyurea among people with sickle cell disease: a qualitative study. Blood Adv 2020 Sep 22; 4(18):4463-73. PMC7509876 Epub 2020/09/18. URL
  82. Hoppe C. Methods for hemoglobin analysis and hemoglobinopathy testing. In: Mentzer WC, Tirnauer JS, eds. UpToDate. Topic 13941: Version 32.0; Nov. 18, 2019. UpToDate, Inc. Waltham, MA. URL.
  83. Hoppe C, Neumayr L. Sickle Cell Disease: Monitoring, Current Treatment, and Therapeutics Under Development. Hematol Oncol Clin North Am 2019 Jun; 33(3):355-71. Epub 2019/04/30. URL.
  84. Hord J, Shah M, Badawy SM, Matthews D, Hilden J, Wayne AS, Salsberg E, Leavey PS. Hastings, C (collaborator), American Society of Pediatric Hematology/Oncology Workforce Advisory Taskforce. The American Society of Pediatric Hematology/Oncology workforce assessment: Part 1-Current state of the workforce. Pediatr Blood Cancer 2018;65 2. URL
  85. Howard J, Ataga K, Brown C, Achebe M, Nduba V, El Beshlawy A, Hassab H, Agodoa I, Tonda M, Gray S, Lehrer-Graiwer J, Vichinsky E. Long-Term Efficacy and Safety of Voxelotor in Adolescents and Adults With Sickle Cell Disease in the HOPE Trial. Lancet Haematol 2021 (accepted).
  86. Hsieh MM, Bonner M, Pierciey FJ, Uchida N, Rottman J, Demopoulos L, Schmidt M, Kanter J, Walters MC, Thompson AA, Asmal M, Tisdale JF. Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease. Blood Adv 2020 May 12; 4(9):2058-63. PMC7218414 Epub 2020/05/13. URL.
  87. Inusa BPD, Colombatti R, Rees DC, Heeney MM, Hoppe CC, Ogutu B, Hassab HM, Zhou C, Yao S, Brown PB, Heath LE, Jakubowski JA, Abboud MR. Geographic Differences in Phenotype and Treatment of Children with Sickle Cell Anemia from the Multinational DOVE Study. J Clin Med 2019 Nov 17; 8(11). PMC6912763 URL.
  88. Inusa BPD, Stewart CE, Mathurin-Charles S, Porter J, Hsu LL, Atoyebi W, De Montalembert M, Diaku-Akinwumi I, Akinola NO, Andemariam B, Abboud MR, Treadwell M. Paediatric to adult transition care for patients with sickle cell disease: a global perspective. Lancet Haematol 2020 Apr; 7(4):e329-e41. URL.
  89. Islinger M, Costello JL, Kors S, Soupene E, Levine TP, Kuypers FA, Schrader M. The diversity of ACBD proteins – From lipid binding to protein modulators and organelle tethers. Biochimica et Biophysica Acta (BBA) - Molecular Cell Research 2020 2020/02/08/:118675. URL.
  90. Jamieson CHM, Millan MT, Creasey AA, Lomax G, Donohoe ME, Walters MC, Abedi M, Bota DA, Zaia JA, Adams JS, California Institute for Regenerative Medicine (CIRM) Alpha Stem Cell Clinic (ASCC) Network. CIRM Alpha Stem Cell Clinics: Collaboratively Addressing Regenerative Medicine Challenges. Cell Stem Cell 2018;22 6:801-5. URL.
  91. Jarisch A, Salzmann-Manrique E, Cario H, Grosse R, Soerensen J, Fischer R, Schulz A, Hammerstingl R, Wunderlich A, Bader P. Serum ferritin is not a reliable predictor to determine iron overload in thalassemia major patients post-hematopoietic stem cell transplantation. Eur J Haematol 2018 Dec; 101(6):791-7. URL.
  92. Kammen BF, Padua EM, Karakas SP, Hagar RW, Hitt DM, Pandya NK, Chung T. Clinical experience with two-point mDixon turbo spin echo as an alternative to conventional turbo spin echo for magnetic resonance imaging of the pediatric knee. Pediatr Radiol 2019 May; 49(6):791-800. Epub 2019/02/07. URL.
  93. Kanter J, Heath LE, Knorr J, Agbenyega ET, Colombatti R, Dampier C, Hassab H, Manwani D, Robitaille N, Brown PB, Jakubowski JA, Yao S, Hoppe C. Novel findings from the multinational DOVE study on geographic and age-related differences in pain perception and analgesic usage in children with sickle cell anaemia. Br J Haematol 2018. URL
  94. Kanter J, Gibson R, Lawrence RH, Smeltzer MP, Pugh NL, Glassberg J, Masese RV, King AA, Calhoun C, Hankins JS, Treadwell M. Perceptions of U.S. Adolescents and Adults with Sickle Cell Disease on Their Quality of Care. JAMA Network Open Hematology 2020 (accepted).
  95. Kanter J, Smith WR, Desai PC, Treadwell M, Andemariam B, Little J, Nugent D, Claster S, Manwani DG, Baker J, Strouse JJ, Osunkwo I, Stewart RW, King A, Shook LM, Roberts JD, Lanzkron S. Building access to care in adult sickle cell disease: defining models of care, essential components, and economic aspects. Blood Adv 2020 Aug 25; 4(16):3804-13. PMC7448595 Epub 2020/08/14. URL
  96. Kato GJ, Piel FB, Reid CD, Gaston MH, Ohene-Frempong K, Krishnamurti L, Smith WR, Panepinto JA, Weatherall DJ, Costa FF, Vichinsky EP. Sickle cell disease. Nat Rev Dis Primers 2018;4:18010. URL
  97. Kaur M, Brown M, Love TW, Thompson A, Treadwell M, Smith-Whitley K. Understanding sickle cell disease: impact of surveillance and gaps in knowledge. Blood Adv 2020 Feb 11; 4(3):496-8. Epub 2020/02/07. URL.
  98. Kayle M, Valle J, Paulukonis S, Holl JL, Tanabe P, French DD, Garg R, Liem RI, Badawy SM, Treadwell MJ. Impact of Medicaid expansion on access and healthcare among individuals with sickle cell disease. Pediatr Blood Cancer 2020 May; 67(5):e28152. PMC7096276. URL.
  99. Kelly S, Jacobs ES, Stone M, Keating SM, Lee TH, Chafets D, Heitman J, Dimapasoc M, Operskalski E, Hagar W, Vichinsky E, Busch MP, Norris PJ, Custer B, Recipient Epidemiology and Donor Evaluation Study (REDS-III). Influence of sickle cell disease on susceptibility to HIV infection. PLoS One 2020 15(4):e0218880. PMC7141606. URL.
  100. Kelly S, Rodeghier M, DeBaun MR. Automated exchange compared to manual and simple blood transfusion attenuates rise in ferritin level after 1 year of regular blood transfusion therapy in chronically transfused children with sickle cell disease. Transfusion 2020 Nov; 60(11):2508-16. Epub 2020/08/20. URL
  101. Kelly S, Belisario AR, Werneck Rodrigues DO, Carneiro-Proietti ABF, Goncalez TT, Loureiro P, Flor-Park MV, Maximo C, Mota RA, Dinardo C, Brambilla D, Preiss L, Sabino E, Custer B, Recipient Epidemiology and Donor Evaluation Study (REDS-III). Blood utilization and characteristics of patients treated with chronic transfusion therapy in a large cohort of Brazilian patients with sickle cell disease. Transfusion 2020 Aug; 60(8):1713-22. Epub 2020/06/25. URL
  102. Klopstock T, Tricta F, Neumayr L, Karin I, Zorzi G, Fradette C, Kmiec T, Buchner B, Steele HE, Horvath R, Chinnery PF, Basu A, Kupper C, Neuhofer C, Kalman B, Dusek P, Yapici Z, Wilson I, Zhao F, Zibordi F, Nardocci N, Aguilar C, Hayflick SJ, Spino M, Blamire AM, Hogarth P, Vichinsky E. Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study. Lancet Neurol 2019 Jul; 18(7):631-42. Epub 2019/06/17. URL.
  103. Knisely MR, Pugh N, Kroner B, Masese R, Gordeuk V, King AA, Smith SM, Gurney JG, Adams R, Wun T, Snyder A, Glassberg J, Shah N, Treadwell M. Sickle Cell Disease Implementation Consortium Patient-reported outcomes in sickle cell disease and association with clinical and psychosocial factors: Report from the sickle cell disease implementation consortium. Am J Hematol 2020 Sep; 95(9):1066-74. Epub 2020/05/26. URL
  104. Krishnamurti L, Neuberg DS, Sullivan KM, Kamani NR, Abraham A, Campigotto F, Zhang W, Dahdoul T, De Castro L, Parikh S, Bakshi N, Haight A, Hassell KL, Loving R, Rosenthal J, Smith SL, Smith W, Spearman M, Stevenson K, Wu CJ, Wiedl C, Waller EK, Walters MC. Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study. Am J Hematol 2019 Apr; 94(4):446-54. PMC6542639 Epub 2019/01/15. URL.
  105. Kutny MA, Geyer S, Laumann KM, Gregory J, Willman CL, Stock W, Larson RA, Powell BL, Feusner JH. Outcome for pediatric acute promyelocytic leukemia patients at Children's Oncology Group sites on the Leukemia Intergroup Study CALGB 9710 (Alliance). Pediatr Blood Cancer 2019 Mar; 66 3:e27542. URL.
  106. Lal A. Iron in Health and Disease: An Update. Indian J Pediatr 2020 Jan; 87(1):58-65. Epub 2019/09/15. URL.
  107. Lal A, Bansal D. Thalassemia: Common Clinical Queries in Management. Indian J Pediatr 2020 Jan; 87(1):75-81. Epub 2019/10/18. URL.
  108. Lal A, Wong TE, Andrews J, Balasa VV, Chung JH, Forester CM, Ikeda AK, Keel SB, Pagano MB, Puthenveetil G, Shah SJ, Yu JC, Vichinsky EP. Transfusion practices and complications in thalassemia. Transfusion 2018 Sep 27. URL.
  109. Lal A, Wong T, Keel SB, Pagano MB, Chung JE, Kamdar A, Rao L, Ikeda AK, Puthenveetil G, Shah S, Yu J, Vichinsky E. The Transfusion Management of Beta Thalassemia in the United States. Transfusion 2021 (submitted).
  110. Leavey PJ, Hilden JM, Matthews D, Dandoy C, Badawy SM, Shah M, Wayne AS, Hord J. Hastings, C (collaborator), American Society of Pediatric Hematology/Oncology Workforce Advisory Taskforce. The American Society of Pediatric Hematology/Oncology workforce assessment: Part 2-Implications for fellowship training. Pediatr Blood Cancer 2018 Feb; 65(2). URL.
  111. Lee JC, Villanueva-Meyer JE, Ferris SP, Cham EM, Zucker J, Cooney T, Gilani A, Kleinschmidt-DeMasters BK, Trembath D, Mafra M, Chiang J, Ellison DW, Cho SJ, Horvai AE, Van Ziffle J, Onodera C, Devine P, Grenert JP, de Voijs CMA, van Blokland WTM, de Leng WWJ, Ploegmakers MJ, Flucke U, Pekmezci M, Bollen AW, Tihan T, Koelsche C, von Deimling A, Wesseling P, Solomon DA, Perry A. Clinicopathologic and molecular features of intracranial desmoplastic small round cell tumors. Brain Pathol 2019 Dec 14. URL.
  112. Lee JY, Saba J. et al. The Oncogene AF1q Promotes Tumorigenesis and Suppresses Senescence Through a Direct Physical Interaction with the DOT1L H3K79 Methyltransferase. 2020 (In Process).
  113. Lee JC, Villanueva-Meyer JE, Ferris SP, Sloan EA, Hofmann JW, Hattab EM, Williams BJ, Guo H, Torkildson J, Florez A, Van Ziffle J, Onodera C, Grenert JP, Cho SJ, Horvai AE, Jones DTW, Pfister SM, Koelsche C, von Deimling A, Korshunov A, Perry A, Solomon DA. Primary intracranial sarcomas with DICER1 mutation often contain prominent eosinophilic cytoplasmic globules and can occur in the setting of neurofibromatosis type 1. Acta Neuropathol 2019 Mar; 137(3):521-5. PMC6408274 URL.
  114. Lew G, Chen Y, Lu X, Rheingold SR, Whitlock JA, Devidas M, Hastings CA, Winick NJ, Carroll WL, Wood BL, Borowitz MJ, Pulsipher MA, Hunger SP. Outcomes after late bone marrow and very early central nervous system relapse of childhood B-Acute lymphoblastic leukemia: a report from the Children's Oncology Group phase III study AALL0433. Haematologica 2021 Jan 1; 106(1):46-55. PMC7776266 Epub 2020/02/01. URL.
  115. Li C, Mathews V, Kim S, George B, Hebert K, Jiang H, Li C, Zhu Y, Keesler DA, Boelens JJ, Dvorak CC, Agarwal R, Auletta JJ, Goyal RK, Hanna R, Kasow K, Shenoy S, Smith AR, Walters MC, Eapen M. Related and unrelated donor transplantation for beta-thalassemia major: Results of an international survey. Blood Adv 2019 Sep 10; 3(17):2562-70. PMC6737407 Epub 2019/09/01. URL.
  116. Lopez GY, Van Ziffle J, Onodera C, Grenert JP, Yeh I, Bastian BC, Clarke J, Oberheim Bush NA, Taylor J, Chang S, Butowski N, Banerjee A, Mueller S, Kline C, Torkildson J, Samuel D, Siongco A, Raffel C, Gupta N, Kunwar S, Mummaneni P, Aghi M, Theodosopoulos P, Berger M, Phillips JJ, Pekmezci M, Tihan T, Bollen AW, Perry A, Solomon DA. The genetic landscape of gliomas arising after therapeutic radiation. Acta Neuropathol 2019 Jan; 137(1):139-50. PMC6589431 URL.
  117. Lown EA, Banerjee A, Vittinghoff E, Dvorak CC, Hartogensis W, Melton A, Mangurian C, Hu H, Shear D, Adcock R, Morgan M, Golden C, Hecht FM. Acupressure to Reduce Treatment-Related Symptoms for Children With Cancer and Recipients of Hematopoietic Stem Cell Transplant: Protocol for a Randomized Controlled Trial. Glob Adv Health Med 2019 8(2164956119870444. PMC6696841 Epub 2019/08/28. URL.
  118. Lucas CG, Villanueva-Meyer JE, Whipple N, Oberheim Bush NA, Cooney T, Chang S, McDermott M, Berger M, Cham E, Sun PP, Putnam A, Zhou H, Bollo R, Cheshier S, Poppe MM, Fung KM, Sung S, Glenn C, Fan X, Bannykh S, Hu J, Danielpour M, Li R, Alva E, Johnston J, Van Ziffle J, Onodera C, Devine P, Grenert JP, Lee JC, Pekmezci M, Tihan T, Bollen AW, Perry A, Solomon DA. Myxoid glioneuronal tumor, PDGFRA p.K385-mutant: clinical, radiologic, and histopathologic features. Brain Pathol 2019 Oct 14. URL.
  119. Lund TC, Doherty TM, Eisengart JB, Freese RL, Rudser KD, Fung EB, Miller BS, White KK, Orchard PJ, Whitley CB, Polgreen LE. Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I. JIMD Rep 2021 Mar; 58(1):89-99. PMC7932872 Epub 2021/03/18. URL
  120. Mack SJ, Udell J, Cohen F, Osoegawa K, Hawbecker SK, Noonan DA, Ladner MB, Goodridge D, Trachtenberg EA, Oksenberg JR, Erlich HA. High resolution HLA analysis reveals independent class I haplotypes and amino-acid motifs protective for multiple sclerosis. Genes Immun 2019 Apr; 20(4):308-26. PMC6035897 Epub 2018/01/09.URL
  121. Maggio A, Vitrano A, Meloni A, Pollina WA, Karimi M, El-Beshlawy A, Hajipour M, Di-Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar A, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Adekile K, Bouesseau MC, Christou S, Dardanoni G, Fang J, Fucharoen S, Hermine O, Hishamshah I, Moi P, Al-Jarrash S, Pistoia L, Pitrolo L, Rigano P, Sacco M, Shah F, Srivastava A, Viprakasit V, Yassin M, Vichinsky E. Evidence for three distinct classes of phenotype severity in beta-thalassaemia. Lancet 2020; SSRN preprint Feb 15, 2019). URL.
  122. Magis W, DeWitt MA, Wyman SK, Vu JT, Heo S-J, Shao SJ, Hennig F, Romero ZG, Campo-Fernandez B, McNeill M, Rettig GR, Sun Y, Lau PJ, Wang Y, Behlke MA, Kohn DB, Boffelli D, Walters MC, Corn JE, Martin DIK. High-level correction of the sickle mutation amplified in vivo during erythroid differentiation. bioRxiv 2019 (Submitted preprint):432716. URL
  123. Martin KW, WeaverN, Alhasan K, Gumus E, Sullivan BR, Zenker M, Hildebrandt F, Saba JD. MRI Spectrum of Brain Involvement in Sphingosine-1-Phosphate Lyase Insufficiency Syndrome. AJNR Am J Neuroradiol 2020 Oct; 41(10):1943-8. PMC7661081 Epub 2020/08/29. URL
  124. Mathur AK, Stewart Lewis ZA, Warren PH, Walters MC, Gifford KA, Xing J, Goodrich NP, Bennett R, Brownson A, Ellefson J, Felan G, Gray B, Hays RE, Klein-Glover C, Lagreco S, Metzler N, Provencher K, Walz E, Warmke K, Merion RM, Ojo AO. Best practices to optimize utilization of the National Living Donor Assistance Center for the financial assistance of living organ donors. Am J Transplant 2020 Jan; 20(1):25-33. Epub 2019/11/05. URL
  125. McMahon CM, Ferng T, Canaani J, Wang ES, Morrissette JJD, Eastburn DJ, Pellegrino M, Durruthy-Durruthy R, Watt CD, Asthana S, Lasater EA, DeFilippis R, Peretz CAC, McGary LHF, Deihimi S, Logan AC, Luger SM, Shah NP, Carroll M, Smith CC, Perl AE. Clonal Selection with RAS Pathway Activation Mediates Secondary Clinical Resistance to Selective FLT3 Inhibition in Acute Myeloid Leukemia. Cancer Discov 2019 Aug; 9(8):1050-63. URL.
  126. Michlitsch J, Larkin S, Vichinsky E, Kuypers FA. Highlight article: Vincristine-induced anemia in hereditary spherocytosis. Exp Biol Med (Maywood) 2019 Jul; 244(10):850-4. PMC6643194 Epub 2019/06/05. URL.
  127. Michlitsch JG, Romao RLP, Gleason JM, Braga LH, Allen L, Gupta A, Lorenzo AJ. Local control for vaginal botryoid rhabdomyosarcoma with pre-rectal transperineal surgical resection and autologous buccal graft vaginal replacement: A novel, minimally invasive, radiation-sparing approach. J Pediatr Surg 2018 Jul; 53(7):1374-80. URL.
  128. Minkley M, MacLeod P, Anderson CK, Nashmi R, Walter PB. Loss of tyrosine hydroxylase, motor deficits and elevated iron in a mouse model of phospholipase A2G6-associated neurodegeneration (PLAN). Brain Res 2020 Dec 1; 1748(147066. Epub 2020/08/21. URL
  129. Mittal N, Loughran T, Jr., Rivers A. T-cell large granular lymphocytic leukemia successfully treated with oral cyclosporine in a child: Case report and review of literature. Pediatr Blood Cancer 2020 Dec; 67(12):e28487. Epub 2020/06/27. URL
  130. Morris CR, Mauger DT, Suh JH, Phipatanakul W, Sheehan WJ, Moy JN, Paul IM, Szefler SJ, Jackson DJ, Fitzpatrick AM. National Institutes of Health/National Heart, Lung and Blood Institute, AsthmaNet Glutathione and arginine levels: Predictors for acetaminophen-associated asthma exacerbation? J Allergy Clin Immunol 2018. URL
  131. Munung NS, Nembaware V, de Vries J, Bukini D, Tluway F, Treadwell M, Sangeda RZ, Mazandu G, Jonas M, Paintsil V, Nnodu OE, Balandya E, Makani J, Wonkam A. Establishing a Multi-Country Sickle Cell Disease Registry in Africa: Ethical Considerations. Front Genet 2019 10(943). PMC6795756 Epub 2019/10/28. URL.
  132. Munoz F, Knisley M, Bhasin N. Cortical vein thrombosis in an infant with delayed vitamin K deficiency bleeding. Pediatr Blood Cancer 2020 Sep; 67(9):e28366. Epub 2020/06/04. URL
  133. Musallam KM, Vitrano A, Meloni A, Pollina WA, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Sankaran VG, Vichinsky E, Taher AT, Maggio A. Survival and causes of death in 2033 patients with non-transfusion-dependent beta-thalassemia. Haematologica 2021 Apr 22. Epub 2021/04/23. URL
  134. Neumayr LD, Hoppe CC, Brown C. Sickle cell disease: current treatment and emerging therapies. Am J Manag Care 2019 Nov; 25(18 Suppl):S335-S43. URL.
  135. Niihara Y, Miller ST, Kanter J, Lanzkron S, Smith WR, Hsu LL, Gordeuk VR, Viswanathan K, Sarnaik S, Osunkwo I, Guillaume E, Sadanandan S, Sieger L, Lasky JL, Panosyan EH, Blake OA, New TN, Bellevue R, Tran LT, Razon RL, Stark CW, Neumayr LD, Vichinsky EP, Investigators of the Phase 3 trial of L-Glutamine in Sickle Cell Disease. A Phase 3 Trial of L-Glutamine in Sickle Cell Disease. N Engl J Med 2018;379 3:226-35. URL.
  136. Nobuta H, Yang N, Ng YH, Marro SG, Sabeur K, Chavali M, Stockley JH, Killilea DW, Walter PB, Zhao C, Huie P, Jr., Goldman SA, Kriegstein AR, Franklin RJM, Rowitch DH, Wernig M. Oligodendrocyte Death in Pelizaeus-Merzbacher Disease Is Rescued by Iron Chelation. Cell Stem Cell 2019 Oct 3; 25(4):531-41 e6. URL.
  137. Nofal R, Zeinali L, Sawaf H. Splenic infarction induced by epstein-barr virus infection in a patient with sickle cell trait. J Paediatr Child Health 2019 Feb; 55(2):249-51. URL.
  138. Nordstrom M, Felton E, Sear K, Tamrazi B, Torkildson J, Gauvain K, Haas-Kogan DA, Chen J, Buono BD, Banerjee A, Samuel D, Saloner D, Tian B, Roddy E, Hess C, Fullerton H, Mueller S. Large Vessel Arteriopathy After Cranial Radiation Therapy in Pediatric Brain Tumor Survivors. J Child Neurol 2018;33 5:359-66. PMC5873308 URL
  139. Nunes K, Aguiar VRC, Silva M, Sena AC, de Oliveira DCM, Dinardo CL, Kehdy FSG, Tarazona-Santos E, Rocha VG, Carneiro-Proietti ABF, Loureiro P, Flor-Park MV, Maximo C, Kelly S, Custer B, Weir BS, Sabino EC, Porto LC, Meyer D. How Ancestry Influences the Chances of Finding Unrelated Donors: An Investigation in Admixed Brazilians. Front Immunol 2020 11(584950. PMC7677137 Epub 2020/11/27. URL
  140. Oliveira CDL, Kelly S, de Almeida-Neto C, Carneiro-Proietti AB, Camargos Piassi FC, Salomon T, Flor-Park MV, Maximo C, Rodrigues DW, Mota RA, Teixeira CM, Loureiro P, Sabino EC, Custer B, Recipient Epidemiology Donor Evaluation Study International Component, Brazil. Quality of life in pre-adolescent children with sickle cell disease in Brazil. Pediatr Hematol Oncol 2019 Nov; 36(8):457-67. PMC6872937 Epub 2019/09/07. URL
  141. Onengut-Gumuscu S, Chen WM, Robertson CC, Bonnie JK, Farber E, Zhu Z, Oksenberg JR, Brant SR, Bridges SL, Jr., Edberg JC, Kimberly RP, Gregersen PK, Rewers MJ, Steck AK, Black MH, Dabelea D, Pihoker C, Atkinson MA, Wagenknecht LE, Divers J, Bell RA, Youth SfDi, Type 1 Diabetes Genetics C, Erlich HA, Concannon P, Rich SS. Type 1 Diabetes Risk in African-Ancestry Participants and Utility of an Ancestry-Specific Genetic Risk Score. Diabetes Care 2019 Mar; 42(3):406-15. PMC6385701 Epub 2019/01/20. URL
  142. Pariury H, Golden C, Huh WW, Cham E, Chung T, Hayes-Jordan A. Pediatric ovarian angiosarcoma treated with systemic chemotherapy and cytoreductive surgery with heated intraperitoneal chemotherapy: Case report and review of therapy. Pediatr Blood Cancer 2019 Jul; 66(7):e27753. Epub 2019/04/13. URL.
  143. Pariury H, Willhoite J, Michlitsch J, Agrawal A. Potassium supplementation mitigates corticosteroid-induced neuropsychiatric effects in pediatric oncology patients. Pediatr Hematol Oncol 2019 Oct; 36(7):445-50. Epub 2019/09/21. URL.
  144. Pekmezci M, Villanueva-Meyer JE, Goode B, Van Ziffle J, Onodera C, Grenert JP, Bastian BC, Chamyan G, Maher OM, Khatib Z, Kleinschmidt-DeMasters BK, Samuel D, Mueller S, Banerjee A, Clarke JL, Cooney T, Torkildson J, Gupta N, Theodosopoulos P, Chang EF, Berger M, Bollen AW, Perry A, Tihan T, Solomon DA. The genetic landscape of ganglioglioma. Acta Neuropathol Commun 2018 Jun 7; 6(1):47. PMC5992851 URL.
  145. Polgreen LE, Lund TC, Braunlin E, Tolar J, Miller BS, Fung E, Whitley CB, Eisengart JB, Northrop E, Rudser K, Miller WP, Orchard PJ. Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation. Pediatr Res 2020 Jan; 87(1):104-11. PMC6960090 Epub 2019/08/23. URL.
  146. Rajpurkar M, Alonzo TA, Wang YC, Gerbing RB, Gamis AS, Feusner JH, Gregory J, Kutny MA. Risk Markers for Significant Bleeding and Thrombosis in Pediatric Acute Promyelocytic Leukemia; Report From the Children's Oncology Group Study AAML0631. J Pediatr Hematol Oncol 2019 Jan; 41 1:51-5. URL.
  147. Rivers A. Cognition affected in young children with serum ferritin <17 µg/L. J Pediatr 2020 May; 220(264-7. Epub 2020/04/27. URL
  148. Rivers A, Molokie R, Lavelle D. A new target for fetal hemoglobin reactivation. Haematologica 2019 Dec; 104(12):2325-7. PMC6959183 Epub 2019/12/04. URL
  149. Rivers A, Vaitkus K, Jagadeeswaran R, Ruiz MA, Ibanez V, Ciceri F, Cavalcanti F, Molokie RE, Saunthararajah Y, Engel JD, DeSimone J, Lavelle D. Oral administration of the LSD1 inhibitor ORY-3001 increases fetal hemoglobin in sickle cell mice and baboons. Exp Hematol 2018 Nov; 67(60-4.e2. Epub 2018/08/21. URL
  150. Rivers A, Jagadeeswaran R, Lavelle D. Potential role of LSD1 inhibitors in the treatment of sickle cell disease: a review of preclinical animal model data. Am J Physiol Regul Integr Comp Physiol 2018 Oct 1; 315(4):R840-r7. PMC6734057 Epub 2018/08/02. URL
  151. Ruhl AP, Sadreameli SC, Allen JL, Bennett DP, Campbell AD, Coates TD, Diallo DA, Field JJ, Fiorino EK, Gladwin MT, Glassberg JA, Gordeuk VR, Graham LM, Greenough A, Howard J, Kato GJ, Knight-Madden J, Kopp BT, Koumbourlis AC, Lanzkron SM, Liem RI, Machado RF, Mehari A, Morris CR, Ogunlesi FO, Rosen CL, Smith-Whitley K, Tauber D, Terry N, Thein SL, Vichinsky E, Weir NA, Cohen RT, Klings ES. Identifying Clinical and Research Priorities in Sickle Cell Lung Disease: An Official American Thoracic Society Workshop Report. Ann Am Thorac Soc 2019 Sep; 16(9):e17-e32. PMC6812163 Epub 2019/08/31. URL.
  152. Saba JD. Fifty years of lyase and a moment of truth: sphingosine phosphate lyase from discovery to disease. J Lipid Res 2019 Mar; 60(3):456-63. PMC6399507 Epub 2019/01/13. URL.
  153. Saba JD. et al. Efficacy of AAV9-mediated SGPL1 gene transfer in a mouse model of S1P lyase insufficiency syndrome. JCI Insight 2021 (in press) Mar 23, 2021.
  154. Schulte RR, Madiwale MV, Flower A, Hochberg J, Burke MJ, McNeer JL, DuVall A, Bleyer A. Levocarnitine for asparaginase-induced hepatic injury: a multi-institutional case series and review of the literature. Leuk Lymphoma 2018:1-9. URL
  155. Serjeant GR, Vichinsky E. Variability of homozygous sickle cell disease: The role of alpha and beta globin chain variation and other factors. Blood Cells Mol Dis 2018;70:66-77. URL
  156. Shah N, Treadwell M, Vichinsky E. Trends in transitioning adolescents with sickle cell disease from pediatric to adult care. Pediatr Blood Cancer 2021 (submitted).
  157. Shenoy S, Gaziev J, Angelucci E, King A, Bhatia M, Smith A, Bresters D, Haight AE, Duncan CN, de la Fuente J, Dietz AC, Baker KS, Pulsipher MA, Walters MC. Late Effects Screening Guidelines after Hematopoietic Cell Transplantation (HCT) for Hemoglobinopathy: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT. Biol Blood Marrow Transplant 2018. URL
  158. Shenoy S, Walters MC, Ngwube A, Soni S, Jacobsohn D, Chaudhury S, Grimley M, Chan K, Haight A, Kasow KA, Parikh S, Andreansky M, Connelly J, Delgado D, Godder K, Hale G, Nieder M, Pulsipher MA, Trachtenberg F, Neufeld E, Kwiatkowski JL, Thompson AA. Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial. Biol Blood Marrow Transplant 2018. URL
  159. Shih SY, Bose N, Goncalves ABR, Erlich HA, Calloway CD. Applications of Probe Capture Enrichment Next Generation Sequencing for Whole Mitochondrial Genome and 426 Nuclear SNPs for Forensically Challenging Samples. Genes (Basel) 2018;9 1. PMC5793200. URL.
  160. Shukla P, Goswami S, Keizer RJ, Winger BA, Kharbanda S, Dvorak CC, Long-Boyle J. Assessment of a Model-Informed Precision Dosing Platform Use in Routine Clinical Care for Personalized Busulfan Therapy in the Pediatric Hematopoietic Cell Transplantation (HCT) Population. Front Pharmacol 2020 11(888. PMC7351521 Epub 2020/07/28. URL
  161. Singer ST, Fischer R, Allen I, Lal A, Vichinsky E, Yuan Q, Wang ZJ. Pituitary iron and factors predictive of fertility status in transfusion dependent thalassemia. Haematologica 2020 Oct 29; Epub 2020/10/31. URL.
  162. Sloan EA, Cooney T, Oberheim Bush NA, Buerki R, Taylor J, Clarke JL, Torkildson J, Kline C, Reddy A, Mueller S, Banerjee A, Butowski N, Chang S, Mummaneni PV, Chou D, Tan L, Theodosopoulos P, McDermott M, Berger M, Raffel C, Gupta N, Sun PP, Li Y, Shah V, Cha S, Braunstein S, Raleigh DR, Samuel D, Scharnhorst D, Fata C, Guo H, Moes G, Kim JYH, Koschmann C, Van Ziffle J, Onodera C, Devine P, Grenert JP, Lee JC, Pekmezci M, Phillips JJ, Tihan T, Bollen AW, Perry A, Solomon DA. Recurrent non-canonical histone H3 mutations in spinal cord diffuse gliomas. Acta Neuropathol 2019 Nov; 138(5):877-81. PMC6818961 URL.
  163. Slone JS, Slone AK, Wally O, Semetsa P, Raletshegana M, S. A, Force LM, Chinyundo K, Margolin J, Agrawal AK, Anderson AR, Scheurer ME, Mehta PS. Establishing a Pediatric Hematology-Oncology Program in Botswana. J Glob Oncol 2018:4; 1-9. URL
  164. Solomon DA, Korshunov A, Sill M, Jones DTW, Kool M, Pfister SM, Fan X, Bannykh S, Hu J, Danielpour M, Li R, Johnston J, Cham E, Cooney T, Sun PP, Oberheim Bush NA, McDermott M, Van Ziffle J, Onodera C, Grenert JP, Bastian BC, Villanueva-Meyer JE, Pekmezci M, Bollen AW, Perry A. Myxoid glioneuronal tumor of the septum pellucidum and lateral ventricle is defined by a recurrent PDGFRA p.K385 mutation and DNT-like methylation profile. Acta Neuropathol 2018 Aug; 136(2):339-43. URL.
  165. Smith WR, Sisler IY, Johnson S, Lipato TJ, Newlin JS, Owens ZS, Morgan AM, Treadwell MJ, Polak K. Lessons Learned from Building a Pediatric-to-Adult Sickle Cell Transition Program. South Med J 2019 Mar; 112(3):190-7. PMC6590675 Epub 2019/03/05. URL.
  166. Soupene E, Kuypers FA. ACBD6 protein controls acyl chain availability and specificity of the N-myristoylation modification of proteins. J Lipid Res 2019 Mar; 60(3):624-35. PMC6399508 Epub 2019/01/16. URL.
  167. Soupene E, Schatz UA, Rudnik-Schoneborn S, Kuypers FA. Requirement of the acyl-CoA carrier ACBD6 in myristoylation of proteins: Activation by ligand binding and protein interaction. PLoS One 2020 15(2):e0229718. PMC7046191. URL.
  168. Steele C, Sinski A, Asibey J, Hardy-Dessources MD, Elana G, Brennan C, Odame I, Hoppe C, Geisberg M, Serrao E, Quinn CT. Point-of-care screening for sickle cell disease in low-resource settings: A multi-center evaluation of HemoTypeSC, a novel rapid test. Am J Hematol 2019 Jan; 94 1:39-45. PMC6298816. URL.
  169. Stilp AM, Emery LS, Broome JG, Buth EJ, Khan AT, Laurie CA, Wang FF, Wong Q, Chen D, D'Augustine CM, Heard-Costa NL, Hohensee CR, Johnson WC, Juarez LD, Liu J, Mutalik KM, Raffield LM, Wiggins KL, de Vries PS, Kelly TN, Kooperberg C, Natarajan P, Peloso GM, Peyser PA, Reiner AP, Arnett DK, Aslibekyan S, Barnes KC, Bielak LF, Bis JC, Cade BE, Chen MH, Correa A, Cupples LA, de Andrade M, Ellinor PT, Fornage M, Franceschini N, Gan W, Ganesh SK, Graffelman J, Grove ML, Guo X, Hawley NL, Hsu WL, Jackson RD, Jaquish CE, Johnson AD, Kardia SLR, Kelly S, Lee J, Mathias RA, McGarvey ST, Mitchell BD, Montasser ME, Morrison AC, North KE, Nouraie SM, Oelsner EC, Pankratz N, Rich SS, Rotter JI, Smith JA, Taylor KD, Vasan RS, Weeks DE, Weiss ST, Wilson CG, Yanek LR, Psaty BM, Heckbert SR, Laurie CC. A System for Phenotype Harmonization in the NHLBI Trans-Omics for Precision Medicine (TOPMed) Program. Am J Epidemiol 2021 Apr 16. Epub 2021/04/17. URL.
  170. Suh JH, Degagne E, Gleghorn EE, Setty M, Rodriguez A, Park KT, Verstraete SG, Heyman MB, Patel AS, Irek M, Gildengorin GL, Hubbard NE, Borowsky AD, Saba JD. Sphingosine-1-Phosphate Signaling and Metabolism Gene Signature in Pediatric Inflammatory Bowel Disease: A Matched-case Control Pilot Study. Inflamm Bowel Dis 2018 May 18; 24(6):1321-34. PMC5986285 Epub 2018/05/23. URL.
  171. Taliun D, Harris DN, Kessler MD, Carlson J, Szpiech ZA, Torres R, Taliun SAG, Corvelo A, Gogarten SM, Kang HM, Pitsillides AN, LeFaive J, Lee SB, Tian X, Browning BL, Das S, Emde AK, Clarke WE, Loesch DP, Shetty AC, Blackwell TW, Smith AV, Wong Q, Liu X, Conomos MP, Bobo DM, Aguet F, Albert C, Alonso A, Ardlie KG, Arking DE, Aslibekyan S, Auer PL, Barnard J, Barr RG, Barwick L, Becker LC, Beer RL, Benjamin EJ, Bielak LF, Blangero J, Boehnke M, Bowden DW, Brody JA, Burchard EG, Cade BE, Casella JF, Chalazan B, Chasman DI, Chen YI, Cho MH, Choi SH, Chung MK, Clish CB, Correa A, Curran JE, Custer B, Darbar D, Daya M, de Andrade M, DeMeo DL, Dutcher SK, Ellinor PT, Emery LS, Eng C, Fatkin D, Fingerlin T, Forer L, Fornage M, Franceschini N, Fuchsberger C, Fullerton SM, Germer S, Gladwin MT, Gottlieb DJ, Guo X, Hall ME, He J, Heard-Costa NL, Heckbert SR, Irvin MR, Johnsen JM, Johnson AD, Kaplan R, Kardia SLR, Kelly T, Kelly S, Kenny EE, Kiel DP, Klemmer R, Konkle BA, Kooperberg C, Kottgen A, Lange LA, Lasky-Su J, Levy D, Lin X, Lin KH, Liu C, Loos RJF, Garman L, Gerszten R, Lubitz SA, Lunetta KL, Mak ACY, Manichaikul A, Manning AK, Mathias RA, McManus DD, McGarvey ST, Meigs JB, Meyers DA, Mikulla JL, Minear MA, Mitchell BD, Mohanty S, Montasser ME, Montgomery C, Morrison AC, Murabito JM, Natale A, Natarajan P, Nelson SC, North KE, O'Connell JR, Palmer ND, Pankratz N, Peloso GM, Peyser PA, Pleiness J, Post WS, Psaty BM, Rao DC, Redline S, Reiner AP, Roden D, Rotter JI, Ruczinski I, Sarnowski C, Schoenherr S, Schwartz DA, Seo JS, Seshadri S, Sheehan VA, Sheu WH, Shoemaker MB, Smith NL, Smith JA, Sotoodehnia N, Stilp AM, Tang W, Taylor KD, Telen M, Thornton TA, Tracy RP, Van Den Berg DJ, Vasan RS, Viaud-Martinez KA, Vrieze S, Weeks DE, Weir BS, Weiss ST, Weng LC, Willer CJ, Zhang Y, Zhao X, Arnett DK, Ashley-Koch AE, Barnes KC, Boerwinkle E, Gabriel S, Gibbs R, Rice KM, Rich SS, Silverman EK, Qasba P, Gan W, Papanicolaou GJ, Nickerson DA, Browning SR, Zody MC, Zollner S, Wilson JG, Cupples LA, Laurie CC, Jaquish CE, Hernandez RD, O'Connor TD, Abecasis GR, NHLBI Trans-Omics for Precision Medicine Consortium. Sequencing of 53,831 diverse genomes from the NHLBI TOPMed Program. Nature 2021 Feb; 590(7845):290-9. PMC7875770 Epub 2021/02/12. URL
  172. Temple WC, Vo KT, Matthay KK, Balliu B, Coleman C, Michlitsch J, Phelps A, Behr S, Zapala MA. Association of image-defined risk factors with clinical features, histopathology, and outcomes in neuroblastoma. Cancer Med 2021 Apr; 10(7):2232-41. PMC7982630 Epub 2020/12/15. URL
  173. Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarre C, Beuzard Y, Chretien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M. Gene Therapy in Patients with Transfusion-Dependent beta-Thalassemia. N Engl J Med 2018;378 16:1479-93. URL
  174. Tisdale JF, Pierciey FJ, Jr., Bonner M, Thompson AA, Krishnamurti L, Mapara MY, Kwiatkowski JL, Shestopalov I, Ribeil JA, Huang W, Asmal M, Kanter J, Walters MC. Safety and feasibility of hematopoietic progenitor stem cell collection by mobilization with plerixafor followed by apheresis vs bone marrow harvest in patients with sickle cell disease in the multi-center HGB-206 trial. Am J Hematol 2020 May 13. Epub 2020/05/14. URL.
  175. Tolbert VP, Dvorak CC, Golden C, Vissa M, El-Haj N, Perwad F, Matthay KK, Vo KT. Risk Factors for Transplant-Associated Thrombotic Microangiopathy after Autologous Hematopoietic Cell Transplant in High-Risk Neuroblastoma. Biol Blood Marrow Transplant 2019 Oct; 25(10):2031-9. Epub 2019/06/15. URL.
  176. Tong S, Vichinsky E. Iron Deficiency: Implications Before Anemia. Pediatr Rev 2021 Jan; 42(1):11-20. Epub 2021/01/03. URL.
  177. Treadwell M. Transition from pediatric to adult care: Sickle cell disease. In: Schrier SL, Tirnauer J, eds. UpToDate. Topic 112919: Version 11.0; Dec 5, 2019. UpToDate, Inc. Waltham, MA. URL.
  178. Treadwell M.(working group member), Sickle Cell Disease Ontology Working Group. The Sickle Cell Disease Ontology: enabling universal sickle cell-based knowledge representation. Database 2019. URL.
  179. Treadwell M, Eufemia J, Hessler D, Chen AF, Mushiana S, Potter M, Vichinsky E. Community-based needs assessment to inform strategies to reduce sickle cell disease healthcare disparities. Journal of Clinical Outcomes Management 2020 (in peer review).
  180. Treadwell M, Jacob E, Hessler D, Chen AF, Chen Y, Mushiana S, Potter M, Vichinsky E. Health Care Disparities Among Adolescents and Adults With Sickle Cell Disease: A Community-Based Needs Assessment to Inform Intervention Strategies. Journal of Clinical Outcomes Management 2020 27(5):213-28. URL
  181. Trobaugh-Lotrario AD, Lopez-Terrada D, Li P, Feusner JH. Hepatoblastoma in patients with molecularly proven familial adenomatous polyposis: Clinical characteristics and rationale for surveillance screening. Pediatr Blood Cancer 2018:e27103. URL
  182. Vance Utset L, Ivy Z, Willen SM, Rodeghier M, Watt A, Schilling L, Jenkins CL, Pepper S, Speller-Brown B, Darbari DS, Majumdar S, Adisa O, DeBaun MR. Inhaled corticosteroid use to prevent severe vaso-occlusive episode recurrence in children between 1 and 4 years of age with sickle cell disease: a multicenter feasibility trial. Am J Hematol 2018 Aug; 93(4):E101-E3. PMC5842125 Epub 2018/01/13. URL
  183. Vichinsky E. Pregnancy in women with sickle cell disease. In: Schrier SL, Simpson L, Tirnauer J, Barss V, eds. UpToDate. Topic 7139: Version 31.0; Apr 3, 2018. UpToDate, Inc. Waltham, MA. URL.
  184. Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J. for the Hope Trial Investigators A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. N Engl J Med 2019 Aug 8; 381(6):509-19. Epub 2019/06/15. URL.
  185. Vichinsky E, Cohen A, Thompson AA, Giardina PJ, Lal A, Paley C, Cheng WY, McCormick N, Sasane M, Qiu Y, Kwiatkowski JL. Epidemiologic and clinical characteristics of nontransfusion-dependent thalassemia in the United States. Pediatr Blood Cancer 2018:e27067. URL
  186. Vichinsky E. Overview of the clinical manifestations of sickle cell disease. In: Schrier SL, Tirnauer JS, eds. UpToDate. Topic 7119; Version 48; Dec 14, 2020. UpToDate, Inc. Waltham, MD. URL
  187. Vichinsky E. Overview of Variant Sickle Cell Syndromes. In: Mahoney DH, Tirnauer J, eds. UpToDate. Topic 7115 Version 21; Nov 24, 2020. UpToDate, Inc. Waltham, MA. URL
  188. Vichinsky E. Sickle cell trait. In: Mahoney DH, Tirnauer J, eds. UpToDate. Topic 7145: Version 54; Dec 2, 2020. UpToDate, Inc. Waltham, MA. URL
  189. Vichinsky E, Mahoney DH. Diagnosis of sickle cell disorders. In: Schrier SL, Tirnauer J, eds. UpToDate. Topic 7113: Version 35; Sept 25, 2020. UpToDate, Inc. Waltham, MA. URL.
  190. Vichinsky E. Disease-modifying therapies for prevention of vaso-occlusive pain in sickle cell disease. In: DeBaun M, Tirnauer J, eds. UpToDate. Topic 126298: Version 12; Oct 27, 2020. UpToDate, Inc., Waltham, MA. https://www.uptodate.com/contents/disease-modifying-therapies-for-prevention-of-vaso-occlusive-pain-in-sickle-cell-disease
  191. Vichinsky E. Vichinsky E. Alpha Thalassemia Major: Prenatal and Postnatal Management. In: UpToDate. April 2021 (accepted). UpToDate, Inc. Waltham, MA.
  192. Vissa M, Vichinsky E. Voxelotor for the treatment of sickle cell disease. Expert Rev Hematol 2021 Mar 4:1-9. Epub 2021/02/20. URL.
  193. Vitrano A, Meloni A, Addario Pollina W, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Hussain Ansari S, Filosa A, Ricchi P, Ceci A, Daar S, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Vichinsky E, Maggio A. A complication risk score to evaluate clinical severity of thalassaemia syndromes. Br J Haematol 2021 Feb; 192(3):626-33. Epub 2020/11/21. URL.
  194. Vitrano A, Meloni A, Addario Pollina W, Karimi M, El-Beshlawy A, Di Marco V, Filosa A, Ricchi P, Ceci A, Daar S, Titi Singer S, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Vichinsky E, Sankaran V, Musallam K, Maggio A. Random forest clustering identifies three classes of Beta-thalassemia with distinct clinical severity (IWG-THAL-CLUST3R). Blood 2021 (submitted) Feb.
  195. Wang H, Long-Boyle J, Winger BA, Nicolaides T, Mueller S, Prados M, Ivaturi V. Population Pharmacokinetics of Vemurafenib in Children With Recurrent/Refractory BRAF Gene V600E-Mutant Astrocytomas. J Clin Pharmacol 2020 Sep; 60(9):1209-19. Epub 2020/06/02. URL
  196. Watkins B, Qayed M, McCracken C, Bratrude B, Betz K, Suessmuth Y, Yu A, Sinclair S, Furlan S, Bosinger S, Tkachev V, Rhodes J, Tumlin AG, Narayan A, Cribbin K, Gillespie S, Gooley TA, Pasquini MC, Hebert K, Kapoor U, Rogatko A, Tighiouart M, Kim S, Bresee C, Choi SW, Davis J, Duncan C, Giller R, Grimley M, Harris AC, Jacobsohn D, Lalefar N Norkin M, Farhadfar N, Pulsipher MA, Shenoy S, Petrovic A, Schultz KR, Yanik GA, Waller EK, Levine JE, Ferrara JL, Blazar BR, Langston A, Horan JT, Kean LS. Phase II Trial of Costimulation Blockade With Abatacept for Prevention of Acute GVHD. J Clin Oncol 2021 Jan 15:JCO2001086. Epub 2021/01/16.URL
  197. Willen SM, Rodeghier M, DeBaun MR. Asthma in children with sickle cell disease. Curr Opin Pediatr 2019 Jun; 31(3):349-56. Epub 2019/05/16. URL
  198. Willen SM, Cohen R, Rodeghier M, Kirkham F, Redline SS, Rosen C, Kirkby J, DeBaun MR. Age is a predictor of a small decrease in lung function in children with sickle cell anemia. Am J Hematol 2018 Mar; 93(3):408-15. PMC5803370 Epub 2017/12/12. URL
  199. Willen SM, DeBaun MR. The Epidemiology and Management of Lung Diseases in Sickle Cell Disease: Lessons Learned from Acute and Chronic Lung Disease in Cystic Fibrosis. Pediatr Clin North Am 2018 Jun; 65(3):481-93. Epub 2018/05/29. URL
  200. Willen SM, Rodeghier M, Rosen CL, DeBaun MR. Sleep disordered breathing does not predict acute severe pain episodes in children with sickle cell anemia. Am J Hematol 2018 Aug; 93(4):478-85. PMC5842111 Epub 2017/12/22. URL
  201. Willen SM, Rodeghier M, Strunk RC, Bacharier LB, Rosen CL, Kirkham FJ, DeBaun MR, Cohen RT. Aeroallergen sensitization predicts acute chest syndrome in children with sickle cell anaemia. Br J Haematol 2018 Feb; 180(4):571-7. PMC5945280 Epub 2018/01/25. URL
  202. Winger BA, Cortopassi WA, Garrido Ruiz D, Ding L, Jang K, Leyte-Vidal A, Zhang N, Esteve-Puig R, Jacobson MP, Shah NP. ATP-Competitive Inhibitors Midostaurin and Avapritinib Have Distinct Resistance Profiles in Exon 17-Mutant KIT. Cancer Res 2019 Aug 15; 79(16):4283-92. PMC6697585 Epub 2019/07/05. URL
  203. Yoon HS, Lee JH, Michlitsch J, Garcia-Carega M, Jeng M. Langerhans Cell Histiocytosis of the Gastrointestinal Tract: Evidence for Risk Organ Status. J Pediatr 2019 Sep; 212(66-72 e3. Epub 2019/07/08. URL.
  204. Yu H, Cohler Peretz CA, Matsunaga A, Agrawal AK. Vaginal Bleeding and Otorrhea in a 6-year-old Girl. Pediatr Rev 2020 Dec; 41(12):648-51. Epub 2020/12/03. URL
  205. Zhao P, Aguilar AE, Lee JY, Paul LA, Suh JH, Puri L, Zhang M, Beckstead J, Witkowski A, Ryan RO, Saba JD. Sphingadienes show therapeutic efficacy in neuroblastoma in vitro and in vivo by targeting the AKT signaling pathway. Invest New Drugs 2018. URL
  206. Zhao P, Liu ID, Hodgin JB, Benke PI, Selva J, Torta F, Wenk MR, Endrizzi JA, West O, Ou W, Tang E, Goh DL, Tay SK, Yap HK, Loh A, Weaver N, Sullivan B, Larson A, Cooper MA, Alhasan K, Alangari AA, Salim S, Gumus E, Chen K, Zenker M, Hildebrandt F, Saba JD. Responsiveness of sphingosine phosphate lyase insufficiency syndrome to vitamin B6 cofactor supplementation. J Inherit Metab Dis 2020 Mar 31. URL.
  207. Zhao P, Tassew GB, Lee JY, Oskouian B, Munoz DP, Hodgin JB, Watson GL, Tang F, Wang JY, Luo J, Yang Y, King SM, Krauss RM, Keller N, Saba JD. Efficacy of AAV9-mediated SGPL1 gene transfer in a mouse model of S1P lyase insufficiency syndrome. JCI Insight 2021 Mar 23. Epub 2021/03/24.

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ABSTRACTS:

  1. Abdel-Azim H, Dvorak CC, Bunin NJ, Lalefar N, Anderson E, Flower A, Talano J-A, Ahn KW, Chaudhury S, Kitko CL, Monos D, Wing L, Pulsipher MA. High OS, PFS and EFS with Low Rates of Rejection and GVHD after KIR-Favorable abCD3/CD19 Depleted Haploidentical HCT in Children with ALL/AML/MDS: Primary Analysis of the Pediatric Transplantation and Cellular Therapy Consortium ONC1401 Trial. TCT Meetings of ASTCT and CIBMTR. Feb 8-12, 2021. Transplantation and Cellular Therapy 2021 (27):3, Supplement:S5-S6. URL
  2. Abdel-Azim H, Dvorak CC, Bunin N, Walters MC, Talano JAM, Anderson EJ, Malvar J, Chaudhury S, Cairo MS, Kitko CL, Monos DS, Wing L, Pulsipher MA. Next Generation Sequence Minimal Residual Disease (NGS-MRD) Predicts Event Free Survival (EFS) Irrespective of Hematopoietic Cell Transplantation (HCT) Conditioning Regimen, Graft ΑΒ T and B-Cell Depletion or Patient Weight Category in Children with Acute Lymphoblastic Leukemia (ALL). American Society for Transplantation and Cellular Therapy; Houston, TX; Feb 20-24, 2020. Biology of Blood and Marrow Transplantation 2020 (26):3:S90-S2. URL
  3. Adesina OO, Jenkins IC, Wu QV, Fung EB, Mahajan K, Kruse-Jarres R. Urinary cross-linked carboxyterminal telopeptide decreases with resolution of painful vaso-occlusive crises in adults with sickle cell disease: Results from the Sickle Cell Pain Markers (SCPM) study. 60th ASH Annual Meeting & Exposition. San Diego, CA; Dec 1-4, 2018. Blood 2018.
  4. Agrawal A, Chen A, Chen E, Yu H, Long LS. Feasibility of inpatient low-dose ketamine infusion for pain control in hematology/oncology patients. American Society of Pediatric Hematology Oncology (ASPHO). New Orleans, LA; May 1-4, 2019. Pediatric Blood and Cancer 2019 (66):S186-S7. URL.
  5. Alvarez OA, Daak AA, Heeney MM, Fuh B, Dampier CD, Kanter J, V. BL, McNaul MA, Callaghan MU, George A, Neumayr L, Hilliard LM, Rabinowicz AL. Effect of Long Term Treatment with SC411 on Sickle Cell Crisis Rate in Children with Sickle Cell Disease. 46th Annual National Convention for Sickle Cell Disease. Baltimore, MD; Oct 10-13, 2018. Sickle Cell Disease Association of America (SCDAA).
  6. Andemariam B, Scheele W, Gordeuk V, Howard J, Kanter J, Eleftheriou P, Pancham S, Hagar R, Clarke L, Blyden G, Johnson K, Lisbon E, Ballal R. IMR-687, a highly selective phosphodiesterase 9 inhibitor (PDE9I), increases F-cells and fetal hemoglobin in a ph-2a interim analysis. 25th Congress of the European Hematology Association. June 11, 2020; Virtual edition. HemaSphere 2020 (4):106. URL
  7. Anurathapan U, Locatelli F, Kwiatkowski JL, Rasko JEJ, Schiller GJ, Porter J, Sauer MG, Thrasher AJ, Chabannon C, Elliot H, Deary B, Chen Y, Tao G, Asmal M, Thompson AA, Walters MC. Lentiglobin Gene Therapy for Transfusion-Dependent ?-Thalassemia: Outcomes from the Phase 1/2 Northstar and Phase 3 Northstar-2 Studies. Transplantation and Cellular Therapy Meetings. Feb 20-24, 2019; Houston, Texas. Biology of Blood and Marrow Transplantation 2019 (25):3:S66-S7. URL.
  8. Asmal M, Kanter J, Tisdale JF, Mapara MY, Kwiatkowski JL, Krishnamurti L, Schmidt M, Miller AL, Pierciey FJ, Huang W, Ribeil JA, Thompson AA, Walters MC. Resolution of sickle cell disease manifestations in patients treated with LentiGlobin gene therapy: Updated results from the phase 1/2 HGB-206 Group C study. 60th Annual Scientific Meeting of the British Society for Haematology. April 27-29, 2020; Birmingham, UK. British Journal of Haematology 2020 (189):33. URL
  9. Baker JR, Treadwell M, Brown M, Claster S, Vora S, Vichinsky E, Nugent D. Mobilizing Regional Sickle Cell Health Systems Improvements: Real world experiment results from HRSA's Pacific Sickle Cell Regional Collaborative 2014-2018. 12th Annual Sickle Cell Disease Research and Educational Symposium. Foundation for Sickle Cell Disease Research; Washington, DC: June 2018.
  10. Belisario AR, Blatyta PF, Vivanco D, Oliveira CDL, Carneiro-Proietti AB, Sabino EC, Almeida-Neto C, Loureiro P, Maximo C, Mateos SOG, Flor-Park MV, Rodrigues DOW, Mota RA, Goncalez TT, Hoffmann TJ, Kelly S, Custer B. Association of HIV infection with clinical and laboratory characteristics of sickle cell disease. Hematology, Transfusion and Cell Therapy 2020 (42)S2:36. URL
  11. Boye-Doe A, Brown E, Puri-Sharma C, Chawla A, Field JJ, Neumayr LD, Padrino S, Desai P, Manwani D, Lanzkron SM, Little JA. The Grndad Registry: Contemporary Natural History Data and an Analysis of Real-World Patterns of Use and Limitations of Disease Modifying Therapy in Adults with SCD. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):Supplement 1:34-6. URL
  12. Breese MR, Shah AT, Lee AG, Tanasa B, Leung SG, Spillinger A, Liu HY, Behroozfard I, Dinh P, Hazard FK, Rangaswami A, Spunt SL, Lacayo NJ, Cooney T, Michlitsch JG, Agrawal AK, Sweet-Cordero EA. Integrative analysis of whole-genome and RNA sequencing in high-risk pediatric malignancies. AACR Annual Meeting 2019. March 29-April 3, 2019; Atlanta, GA. Cancer Research 2019 (79):13. URL.
  13. Bronte-Hall L, Andemariam B, Gershwin B, Lugthart S, Mant T, Howard J, Fok H, Eleftheriou P, Hagar RW, Mason J, Ballal R, Lufkin J, Lisbon E. Benefits and safety of long-term use of IMR-687 as monotherapy or in combination with a stable dose of hydroxyurea (HU) in 2 adult sickle cell patients. 62nd Annual ASH Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):SUPPL 1:29-30. URL
  14. Brown C, Hoppe C, Inati A, Abboud MR, Wang W, Liem R, Woods G, Hsu LL, Gordeuk VR, Piccone C, Drachtman RA, Fong E, Dixon S, Tonda M, Washington C, Lehrer-Graiwer J. Efficacy and Safety of 1500 mg Voxelotor in a Phase 2a Study (GBT440-007) in Adolescents with Sickle Cell Disease. 60th ASH Annual Meeting & Exposition. San Diego, CA; Dec 1-4, 2018. Blood 2018 (132):Suppl 1:509. URL.
  15. Brown C, Hoppe C, Inati A, Abboud M, Wang W, Liem R, Woods G, Hsu L, Gordeuk V, Piccone C, Drachtman R, Fong E, Dixon S, Tonda M, Washington C, Lehrer-Graiwer J. Phase 2A study (GBT440-007) of voxelotor in adolescents with sickle cell disease. 2019 ASPHO Conference. New Orleans, LA; May 1-4, 2019. Pediatric Blood and Cancer 2019 (66):S241-S2. URL
  16. Cairo M, Parsons SK, Braniecki S, Talano J-A, Moore TB, Shi Q, Keever-Taylor CA, Weinberg RS, Grossman BJ, Dozor AJ, Friedman D, McKinstry RC, Verbsky J, Moorthy CR, Weidner RA, Rodday AM, Ayello J, Semidei-Pomales M, Flower AM, Chu Y, Morris E, Mahanti H, Fabricatore S, Militano O, Klejmont L, van de Ven C, Baxter-Lowe LA, Vichinsky E, Walters MC, Shenoy S. Significantly Improved Long Term Health Related Quality of Life (HRQL) and Neurocognition Following Familial Haploidentical Stem Cell Transplantation (HISCT) Utilizing CD34 Enrichment and Mononuclear (CD3) Addback in High Risk Patients with Sickle Cell Disease (SCD). 60th ASH Annual Meeting & Exposition. San Diego, CA; Dec 1-4, 2018. Blood 2018 (132):Suppl 1:162. URL
  17. Campbell AD, Colombatti R, Andemariam B, Strunk CJ, Tartaglione I, Piccone CM, Manwani D, Asare EV, Farooq F, Urbonya R, Dankwah G, Perrotta S, Sainati L, Rivers A, Rao S, Zempsky W, Sey F, Segbefia CI, Inusa BPD, Antwi-Boasiako C. An analysis of racial and ethnic backgrounds within the casire international cohort of sickle cell disease patients: Implications for disease phenotype and clinical research. 61st Annual ASH Meeting & Exposition. Dec. 7-10, 2019; Orlando, FL. Blood 2019 (134 ):S1. URL
  18. Cappellini MD, Viprakasit V, Taher A, Georgiev P, Kuo KH, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Forni G, Perrotta S, Khelif A, Lal A, Kattamis A, Vlachaki E, Origa R, Aydinok Y, Bejaoui M, Ho PJ, Chew LP, Bee PC, Lim SM, Lu MY, Tantiworawit A, Ganeva P, Gercheva L, Shah F, Neufeld EJ, Laadem A, Shetty JK, Zou J, Miteva D, Zinger T, Linde PG, Sherman ML, Hermine O, Porter J, Piga A. The Believe Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions. 60th ASH Annual Meeting & Exposition; San Diego, CA; Dec 1-4, 2018.
  19. Cappellini MD, Hermine O, Piga A, Viprakasit V, Georgiev P, Kuo KHM, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Forni GL, Perrotta S, Khelif A, Lal A, Kattamis A, Shah F, Porter J, Laadem A, Shetty JK, Kuo WL, Zhang J, Miteva D, Zinger T, Sinsimer D, Louis C, Linde PG, Taher AT. Assessment of response to luspatercept by B-globin genotype in adult patients with B-thalassemia in the believe trial. 25th Congress of the European Hematology Association. June 11, 2020; Virtual edition. HemaSphere 2020 (4):108-9. URL
  20. Chawla A, Kanter J, Mapara M, Kwiatkowski J, Krishnamurti L, Schmidt M, Miller A, Pierciey F, Shi W, Ribeil JA, Asmal M, Thompson A, Walters M, Tisdale J. Evolution of the HGB-206 study design for evaluating lentiglobin gene therapy in sickle cell disease. 2019 ASPHO Conference. New Orleans, LA; May 1-4, 2019. Pediatric Blood and Cancer 2019 (66):S242-S3. URL
  21. Chiruvolu N, Patel A, Rowley C, Treadwell MJ, Vichinsky EP, Pakbaz Z. A pilot adult sickle cell hematology clinic in California's inland empire improves patient outcome. 61st Annual ASH Meeting & Exposition. San Diego, CA; Nov 30-Dec 1, 2019. Blood 2019 (134):S1. URL.
  22. Coleman C, Cooney T, Perry A, Guo H, Solomon D, Torkildson J. Prolonged Survival and Eventual Death By Disseminated Pulmonary Metastasis in a Pediatric Patient with Diffuse Pontine H3.1K27M Glioma: A Case Report. International Symposium on Pediatric Neuro-Oncology (ISPNO). Denver, CO; June 29 - July 3, 2018. 2018
  23. Coleman C, Cooney T, Perry A, Guo H, Solomon D, Torkildson J. Prolonged Survival and Eventual Death By Disseminated Pulmonary Metastasis in a Pediatric Patient with Diffuse Pontine H3.1K27M Glioma: A Case Report. ASPHO 31st Annual Meeting. Pittsburgh, PA; May 2-5, 2018. Pediatric Blood & Cancer 65(S1); e27057. URL
  24. Cooper-Sood JB, Marsh A, Naprawa J, Hoppe C, Agrawal A. Adjuvant low-dose ketamine in acute pediatric sickle cell vaso-occlusive episodes. Pediatric American Societies (PAS) 2018 Meeting. Toronto, Ontario, Canada; May 5-8, 2018.
  25. Daak AA, Heeney MM, Dampier CD, Fuh B, Kanter J, Alvarez OA, V. BL, McNaul MA, Callaghan MU, George A, Neumayr L, Hilliard LM, Rabinowicz AL. SC411 for Treatment of Children with Sickle Cell Disease: A Phase 2, Randomized, Double-Blind, Dose-Finding, Placebo-Controlled, Parallel-Group, Multi-Center Study (SCOT trial). 46th Annual National Convention for Sickle Cell Disease. Baltimore, MD; Oct 10-13, 2018. Sickle Cell Disease Association of America (SCDAA).
  26. Daak A, Heeney M, Dampier C, Fuh B, Kanter J, Alvarez O, Black V, McNaull M, Callaghan M, George A, Neumayr L, Hilliard L, Sancilio F, Rabinowicz A. Effects of SC411 (altemiatm) on blood cell membrane omega-3 index and select sickle cell disease biomarkers in the scot trial: A phase 2 randomized, doubleblind, placebo-controlled, parallel-group, multi-center study. ASPHO 31st Annual Meeting. Pittsburgh, PA; May 2-5, 2018. Pediatric Blood and Cancer 2018 (65):S122-S3. URL.
  27. Daak A, Heeney M, Dampier C, Fuh B, Kanter J, Alvarez O, Black V, McNaull M, Callaghan M, George A, Neumayr L, Hilliard L, Sancilio F, Rabinowicz A. Clinical effect of SC411 (Altemia TM) on children with sickle cell disease in the SCOT trial: A phase 2 randomized, double-blind, placebo-controlled, parallel-group, dose-finding multi-center study. ASPHO 31st Annual Meeting. Pittsburgh, PA; May 2-5, 2018. Pediatric Blood and Cancer 2018 (65):S8. URL.
  28. Dandoy CE, Rotz SR, Alonso PB, Lane A, Higham C, Dvorak CC, Duncan CN, Schoettler ML, Lehmann LE, Cancio M, Killinger J, Davila B, Phelan R, Prasad S, Mahadeo KM, Khazal SJ, Lalefar NR, Vissa M, Klunk A, Bhatla D, Gloude NJ, Anderson EJ, Huo JS, Roehrs PA, Auletta JJ, Davies SM, Chima R, Jodele S. Incidence and Outcomes of Patients with Thrombotic Microangiopathy after Transplant: Results of Prospective Screening through a Multi-Institutional Collaborative. American Society for Transplantation and Cellular Therapy. Houston, TX: Feb 20-24, 2020. Biology of Blood and Marrow Transplantation 2020 (26):3:S92. URL.
  29. Dandoy CE, Rotz S, Alonso PB, Higham C, Dvorak CC, Duncan C, Schoettler M, Lehmann L, Cancio M, Killinger J, Davila B, Kasprzyk ZA, Phelan R, Mahadeo K, Khazal S, Steiner M, Smith A, Lalefar N, Vissa M, Bhatla D, Gloude N, Anderson E, Huo J, Roehrs P, Auletta J, Davies S, Chima R, Jodele S. Transplant associated thrombotic microangiopathy after transplant: A PBMTC/palisi multi-center collaborative. 2019 ASPHO Conference. New Orleans, LA; May 1-4, 2019. Pediatric Blood and Cancer 2019 (66):S290. URL
  30. Davini M, Hastings C, Feusner J. Routine imaging for detection of relapse and assessing treatment response in alpha feto protein (AFP) positive hepatoblastoma: Is it really necessary? ASPHO 31st Annual Meeting. Pittsburgh, PA; May 2-5, 2018. Pediatric Blood & Cancer 2018 (65):S1:e27057, #525. URL.
  31. Demaree B, Delley C, Peretz C, Vasudevan H, Ruff D, Ooi A, Smith CC, Abate A. Combined Single-Cell DNA Genotyping and Protein Quantification (DAb-seq) in Acute Myeloid Leukemias Reveals Distinct Immunophenotypic Subsets Among Pathogenic Clones. 61st Annual ASH Meeting & Exposition. Orlando, FL; Sept 7-10, 2019. Blood 2019 (134):Supplement_1:poster 2088. URL.
  32. Edwards-Elliott R, Cooper R, Allen S, Rivers A. Peer patient advocates development of educational material for adolescent sickle cell patients. American Society Pediatric Hematology Oncology (ASPHO) Conference. April 20-23, 2021; Portland, OR. 2021.
  33. Estepp JH, Kalfa T, Saraf S, Kim H, Biernat L, Malik P, Cruz K, Brown RC, Fessler R, Kuypers FA, Wilson L, Ribadeneira M, Forsyth S, Schroeder P, Wu E, Kelly P, Telen MJ. Phase 1 single (SAD) and multiple ascending dose (MAD) study of the safety, pharmacokinetics (PK) and pharmacodynamics (PD) of FT-4202, a pkractivator, in healthy and sickle cell disease subjects. 25th Congress of the European Hematology Association June 11-22, 2020 (Virtual). HemaSphere 2020 (4):709-10. URL
  34. Fradette C, Klopstock T, Neumayr L, Karin I, Zorzi G, Kmiec T, Buchner B, Steele H, Horvath R, Chinnery P, Basu A, Kupper C, Neuhofer C, Zibordi F, Nardocci N, Stilman A, Spino M, Vichinsky E, Tricta F. Safety of iron chelation in patients with brain iron overload. 2019 International Congress, International Parkinson and Movement Disorder Society. Sept 22-26, 2019. Nice, France. Mov Disord 2019 (34):S40-S1. URL
  35. Fradette C, Klopstock T, Vichinsky E, Neumayr L, Karin I, Zorzi G, Kmiec T, Buchner B, Steele HE, Horvath R, Chinnery P, Basu A, Kupper C, Neuhofer C, Kalman B, Dusek P, Zibordi F, Nordocci N, Aguilar C, Hayflick S, Hogarth P, Blamire A, Stilman A, Spino M, Tricta F. Long-term safety and efficacy of deferiprone in patients with pantothenate kinase-associated neurodegeneration (PKAN). 8th Congress of the International Bioiron Society. .Heidelberg, Germany: May 5-19, 2019.
  36. Gollapudi A, Lamble A, Walters M, Winestone L, Aguayo-Hiraldo P, Baezconde-Garbanati L, Coker T, Dave H, Dornsife D, Keating A, Merino D, Ramsey B, Park J, Agrawal A. Equitable access to car-t cell trials: An analysis of patient demographic and socioeconomic factors at a single institution. 2020 ASPHO Conference. Pediatric Blood and Cancer 2020 (67):SUPPL 2. URL
  37. Gordeuk VR, Kroner B, Pugh N, Hankins JS, Kutlar A, King AA, Shah N, Kanter J, Glassberg J, Treadwell M. Hydroxyurea use and outcomes of pregnancy in sickle cell disease. 62nd Annual ASH Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):SUPPL 1:33. URL
  38. Hankin D, Murphy P. Distress screening implementation and outcomes in a pediatric survivorship program. American Psychosocial Oncology Society (APOS) Annual Meeting. Tucson, AZ; Feb 22-23, 2018.
  39. Hastings C. Update on phase I/II clinical trial of hydroxyprpoyl betacyclodextrin in treatment of Niemann-Pick type C disease. National Niemann-Pick Disease Foundation 2019 Conference. Bloomington, MN; Feb. 4-8, 2019.
  40. Hastings C, Liu B, Hurst B, Murray B, Hrynkow SH. Initial Pharmacokinetic Data from Clinical Trials using Trappsol Cyclo Intravenously in Niemann-Pick Disease Type C Patients. 15th Annual World Lysosomal Disease Symposium. Orlando, FL; Feb 4-8, 2019.
  41. Hastings C, Gowans LK. Heterogeneity in school re-entry programs for children returning to school following treatment for cancer. Pediatric Blood and Cancer 2018 (65):S189. ASPHO 31st Annual Meeting, Pittsburgh, PA; May 2-5, 2018. URL.
  42. Hastings C, Liu B, Hurst B, Hrynkow SH. Trappsol Cyclo hydroxypropyl betacyclodextrin administered intravenously in patients with Niemann-Pick type C disease reduces cholesterol in liver tissue. 16th Annual World Lysosomal Disease Symposium. Orlando, FL; Feb 10-13, 2020.
  43. Hermine O, Cappellini MD, Taher AT, Coates TD, Viprakasit V, Voskaridou E, Lal A, Liew HK, Perrotta S, Khelif A, Kattamis A, Shetty JK, Zhang G, Tian Y, Miteva D, Zinger T, Tang D, Backstrom JT, Porter JB. Longitudinal Effect of Luspatercept Treatment on Iron Overload and Iron Chelation Therapy (ICT) in Adult Patients (Pts) with Beta-Thalassemia in the Believe Trial. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):Supplement 1:47-8. URL
  44. Hongeng S, Thompson A, Kwiatkowski J, Rasko J, Schiller G, Vichinsky E, Kletzel M, Ho PJ, Anurathapan U, Schmidt M, Cavazzana M, Leboulch P, Deary B, Chen Y, Asmal M, Walters M. Long-term safety and efficacy of lentiglobin gene therapy in patients with transfusion-dependent Beta-thalassemia following completion of the phase 1/2 Northstar study. The 45th Annual Meeting of the European Society for Blood and Marrow Transplantation: Physicians Poster Session. March 24-27, 2019; Frankfurt, Germany. Bone Marrow Transplantation 2019 (54):352-4. URL
  45. Hongeng S, Thompson AA, Kwiatkowski JL, Locatelli F, Porter JB, Rasko J, Cavazzana M, Sauer MG, Thrasher AJ, Thuret I, Lal A, Kulozik AE, Yannaki E, Schmidt M, Chen Y, Guo R, Liu W, Colvin RA, Walters MC. Efficacy and Safety of Betibeglogene Autotemcel (beti-cel; LentiGlobin for Beta-thalassemia) Gene Therapy in 60 Patients with Transfusion-Dependent Beta-Thalassemia (TDT) Followed for up to 6 Years Post-Infusion. TCT Meetings of ASTCT and CIBMTR. Feb 8-12, 2021. Transplantation and Cellular Therapy 2021 (27):3, Supplement:S1. URL
  46. Hood AM, Johnson Y, Nwankwo C, Shook LM, Hildenbrand AK, King AA, Smith-Whitley K, Badawy SM, Thompson AA, Creary SE, Piccone CM, Neumayr LD, Raphael JL, Yates AM, Rana S, Reader SK, Meier ER, Sobota A, Crosby LE. Clinical practice patterns for hydroxyurea initiation in young children with sickle cell disease. 61st Annual ASH Meeting & Exposition. Orlando, FL: Sept 7-10, 2019. Blood 2019 (134);S1. URL
  47. Hood AM, Strong H, Nwankwo C, Johnson Y, Mara CA, Shook LM, Brinkman W, Real FJ, Klein M, King AA, Calhoun C, Smith-Whitley K, Creary SE, Britto MT, Saving KL, Piccone CM, Raphael JL, Volanakis E, Hildenbrand AK, Reader SK, Rana S, Neumayr LD, Yates AM, Badawy SM, Thompson AA, Sobota A, Meier ER, Quinn CT, Crosby LE. Addressing recruitment challenges in the engage-HU trial inyoung children with sickle cell disease. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):SUPPL 1:26-7. URL
  48. Hoppe C, Ataga K, Ware R, Howard J, Tonda M, Ganju J, Lehrer J. Novel trial design to evaluate oral voxelotor for the treatment of sickle cell disease: Protocol of the phase 3 hemoglobin oxygen affinity modulation to inhibit sickle hemoglobin polymerization (HOPE) trial (GBT440-031). ASPHO 31st Annual Meeting. Pittsburgh, PA; May 2-5, 2018. Pediatric Blood & Cancer 2018 (65):S122. URL.
  49. Hoppe C, Inati A, Brown C, Wang W, Hsu L, Gordeuk V, Liem R, Woods G, Piccone C, Fong E, Balaratnam G, Dixon S, Tonda M, Washington C, Yaron Y, Lehrer J. Initial results from a cohort in a phase 2A study (GBT440-007) evaluating adolescents with sickle cell disease treated with multiple doses of voxelotor, a sickle hemoglobin polymerization inhibitor. ASPHO 31st Annual Meeting. Pittsburgh, PA; May 2-5, 2018. Pediatric Blood and Cancer 2018 (65):S91-S2. URL
  50. Howard J, Vichinsky E, Knight-Madden J, Tonda M, Washington C, Tong B, Lehrer-Graiwer J, Gordeuk VR. Correlation of voxelotor exposure with hemoglobin response and measures of hemolysis in patients from the HOPE study. 61st Annual ASH Meeting & Exposition. Orlando, FL: Sept 7-10, 2019. Blood 2019 (134):S1. URL.
  51. Howard J, Vichinsky E, Tonda M, Washington C, Tong B, Lehrer-Graiwer J, Gordeuk VR. Correlation of Voxelotor Exposure with Hemoglobin Response and Measures of Hemolysis in Patients from the HOPE Study. EHA Annual Congress. Frankfurt, Germany; June 11-14, 2020.
  52. Howard J, Ataga KI, Brown RC, Achebe M, Nduba V, El-Beshlawy A, Hassab H, Agodoa I, Tonda M, Gray S, Lehrer-Graiwer J, Vichinsky E. Efficacy and safety of voxelotor in adolescents and adults with sickle cell disease: Hope trial 72-week analysis. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):SUPPL 1:19. URL
  53. Hrynkow SH, Hurst B, Sharma R, Paucar-Arce M, Staretz-Chacham O, Liu B, Hastings C. Initial Pharmacokinetic Data from Clinical Trials using Trappsol Cyclo Intravenously in Niemann-Pick Disease Type C Patients. Brains 4 Brains conference. Frankfurt, Germany; Jan. 2019
  54. Jagadeeswaran R, Ibanez V, Hong LK, Molokie R, Diamond AM, Rivers A. Selenium levels in SCD patients and impact of selenium deficiency on a SCD mouse model. American Society Pediatric Hematology Oncology (ASPHO) Conference. April 20-23, 2021; Portland, OR. 2021.
  55. Kalfa TA, Kuypers FA, Telen MJ, Malik P, Konstantinidis DG, Estepp JH, Kim HJ, Saraf SL, Wilson L, Ribadeneira MD, Forsyth S, Schroeder P, Drake A, Polyanskaya O, Kelly P, Biernat L. Phase 1 single (SAD) and multiple ascending dose (MAD) studies of the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of FT-4202, an allosteric activator of pyruvate kinase-R, in healthy and sickle cell disease subjects. 61st Annual ASH Meeting & Exposition Dec 7-10, 2019; Orlando, Florida. Blood 2019 (134):S1:616. URL
  56. Kanter J, Tisdale JF, Kwiatkowski JL, Krishnamurti L, Mapara MY, Schmidt M, Miller AL, Pierciey FJ, Shi W, Ribeil J-A, Walters MC, Thompson AA. Outcomes for Initial Patient Cohorts with up to 33 Months of Follow-up in the Hgb-206 Phase 1 Trial. 60th ASH Annual Meeting & Exposition. San Diego, CA; Dec 1-4, 2018. Blood 2018 (132):Suppl 1:1080-. URL.
  57. Kanter J, Tisdale JF, Mapara MY, Kwiatkowski JL, Krishnamurti L, Schmidt M, Bonner M, Pierciey FJ, Huang W, Ribeil JA, Thompson AA, Walters MC. Outcomes in patients treated with lentiglobin for sickle cell disease (SCD) gene therapy: Updated results from the phase 1/2 HGB-206 group c study. 25th Congress of the European Hematology Association. June 11-21, 2020; Virtual Edition. HemaSphere 2020 (4):102-3. URL
  58. Kanter J, Tisdale JF, Mapara MY, Kwiatkowski JL, Krishnamurti L, Chen R, Gallagher ME, Ding Y, Goyal S, Paramore C, Thompson AA, Walters MC. Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):Supplement 1:10. URL
  59. Kelly S, Dinardo C, Deng X, Belisario A, Proietti AC, Loureiro P, Mota RA, Flor-Park MV, Maximo C, Sabino E, Custer B. Changes in gene expression in response to red blood cell transfusions in chronically transfused sickle cell disease patients. AABB Annual Meeting San Antonio, TX, October 19-22, 2019. Transfusion 2019 (59):83A. URL
  60. Kline C, Felton E, Byer L, Stoller S, Torkildson J, Gauvain K, Samuel D, Tong E, Liu J, Fullerton H, Dubal D, Mueller S. ApoE4 as a genetic predictor for neurocognitive outcomes in pediatric brain tumor survivors. 18th International Symposium on Pediatric Neuro-Oncology (ISPNO). Denver, CO; June 30-July 3, 2018. Neuro-Oncology 2018 (20):i167. URL.
  61. Klopstock T, Tricta F, Neumayr L, Karin I, Zorzi G, Fradette C, Kmiec T, Buchner B, Steele H, Horvath R, Chinnery P, Basu A, Kupper C, Neuhofer C, Dusek P, Zhao F, Zibordi F, Nardocci N, Aguilar C, Hayflick S, Spino M, Blamire A, Hogarth P, Vichinsky E. A randomized trial of Deferiprone for Pantothenate Kinase-Associated Neurodegeneration (PKAN). 70th Annual American Academy of Neurology Meeting; Los Angeles, CA; April 21-27, 2018.
  62. Klopstock T, Tricta F, Fradette C, Spino M, Buchner B, Karin I, Neumayr L, Aguilar C, Zhao F, Kmiec T, Nardocci N, Zorzi G, Steele H, Horvath R, Chinnery P, Hogarth P, Hayflick SJ, Blamire A, Vichinsky E. A randomized trial of Deferiprone for Pantothenate Kinase-Associated Neurodegeneration (PKAN). 4th Congress of the European Academy of Neurology Lisbon, Portugal, June 2018. European Journal of Neurology 2018 (25):S2:34.
  63. Klopstock T, Tricta F, Neumayr L, Karin I, Zorzi G, Fradette C, Kmiec T, Buechner B, Steele H, Horvath R, Chinnery P, Basu A, Kupper C, Neuhofer C, Dusek P, Zhao F, Zibordi F, Nardocci N, Aguilar C, Hayflick S, Spino M, Blamire A, Hogarth P, Vichinsky E. A randomized trial of deferiprone for pantothenate kinase-associated neurodegeneration. International Congress of Parkinson's Disease and Movement Disorders. Oct 5-9, 2018; Hong Kong. Movement Disorders 2018 (33):S2:S219. URL
  64. Kuo KHM, Layton DM, Uhlig K, Lynch M, Liu L, Vichinsky EP. Mitapivat (AG-348), an oral PK-R activator, in adults with non-transfusion dependent thalassemia: A phase 2, open-label, multicenter study in progress. 61st ASH Annual Meeting & Exposition. Orlando, FL; Dec 7-10, 2019. Blood 2019 (134):Supplement_1:2249. URL
  65. Kuo K, Layton DM, Lal A, Al-Samkari H, Tai F, Lynch M, Uhlig K, Vichinsky EP. Proof of concept for the oral pyruvate kinase activator mitapivat (ag-348) in adults with non-transfusion-dependent thalassemia: first results from an ongoing, phase 2, open-label, multicenter study. 5th Annual Congress of the European Hematology Association (EHA). Frankfurt, Germany; June 11-14, 2020. Hemasphere 2020 (4):109-10. URL
  66. Kuo K, Layton DM, Lal A, Al-Samkari H, Tai F, Lynch M, Uhlig K, Vichinsky E. Proof of Concept for the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non-Transfusion-Dependent Thalassemia: Interim Results from an Ongoing, Phase 2, Open-Label, Multicenter Study. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020; poster. URL
  67. Kuo K, Layton DM, Lal A, Al-Samkari H, Tong B, Lynch M, Uhlig K, Vichinsky EP, MacKenzie TC. Phase 2, Open-Label, Multicenter Study of the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non-Transfusion-Dependent Alpha- and Beta-Thalassemia. EHA Virtual Congress, June 9-17, 2021.
  68. Kwiatkowski JL, Thompson AA, Rasko JEJ, Hongeng S, Schiller GJ, Anurathapan U, Cavazzana M, Ho PJ, Schmidt M, Kletzel M, Vichinsky EP, Deary B, Chen Y, Petrusich A, Walters MC. Long-term clinical outcomes of lentiglobin gene therapy for transfusion-dependent ?-thalassemia in the northstar (HGB-204) study. 60th Annual ASH Meeting & Exposition. San Diego, CA; Nov 30-Dec 1, 2018. Blood 2019 (134). URL.
  69. Kwiatkowski JL, Walters MC, Hongeng S, Locatelli F, Rasko JEJ, Cavazzana M, Chen Y, Colvin RA, Thompson AA. Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent ß-Thalassemia: Results in Patients with up to 6 Years of Follow-up. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):Supplement 1:51-2. URL
  70. Kwiatkowski J, Walters M, Locatelli F, Lal A, Yannaki E, Kulozik A, Sauer M, Porter J, Thuret I, Hongeng S, Thrasher A, Olson T, Schneiderman J, Elliot H, Tao G, Liu W, Colvin R, Thompson A. Lentiglobin gene therapy in pediatrics, adolescents, adults with transfusion-dependent Beta-thalassemia. American Society of Pediatric Hematology Oncology (ASPHO) 2020. Online. Pediatric Blood and Cancer 2020 (67):SUPPL 2. URL
  71. Lal A, Avrin W, Kolotovska V, Calvelli L, Weyhmiller M. Advances in Biomagnetic Liver Susceptometry Allow the Measurement of Liver Iron Concentration with a Room Temperature Sensor. 60th ASH Annual Meeting & Exposition. San Diego, CA; Dec 1-4, 2018.
  72. Lal A, Piga A, Viprakasit V, Maynard J, Kattamis A, Yaeger D, Byrnes B, Chawla L, Tidmarsh G. A Phase 1, Open-Label Study to Determine the Safety, Tolerability, and Pharmacokinetics of Escalating Doses of LJPC-401 (Synthetic Human Hepcidin) in Patients With Iron Overload. 23rd Congress of the European Hematology Association (EHA). Stockholm, Sweden; June 14-17, 2018. Oral presentation. HemaSphere 2018 (2):396-7.
  73. Lal A, Sheth S, Gilbert S, Kwiatkovvski J. Thalassemia Management Checklists: Quick Reference Guides to Reduce Disparities in the Care of Patients with Transfusion-Dependent Thalassemia. 60th ASH Annual Meeting & Exposition; San Diego, CA; Dec 1-4, 2018.
  74. Lal A, Guo H, Weyhmiller M, Marsh A, Vichinsky E. Impact of variable ferritin values using different assay methods on the diagnosis of iron disorders. Eighth Congress of the International BioIron Society (IBIS). Heidelberg, Germany; May 5-10, 2019.
  75. Lal A, Fischer R, Vichinsky E, Weyhmiller M. Trends in iron overload over past two decades: Results from the natural history of iron burden study with the squid biosusceptometer. 61st Annual ASH Meeting & Exposition. Orlando, FL; Dec 7-10, 2019. Blood 2019 (134):S1. URL.
  76. Lal A, Voskaridou E, Flevari P, Taher A, Chew LP, Valone F, Gupta S, Viprakasit V. A hepcidin mimetic, PTG-300, demonstrates pharmacodynamic effects indicating reduced iron availability in transfusion-dependent beta-thalassemia subjects. 25th Congress of the European Hematology Association Virtual Edition, 2020. HemaSphere 2020 (4):110. URL
  77. Leavey PJ, Hilden JM, Matthews D, Dandoy C, Badawy SM, Shah M, Wayne AS, Hord J. Hastings, C (collaborator), American Society of Pediatric Hematology/Oncology Workforce Advisory Taskforce. The American Society of Pediatric Hematology/Oncology workforce assessment: Part 2-Implications for fellowship training. ASPHO 31st Annual Meeting; Pittsburgh, PA; May 2-5, 2018. Pediatr Blood Cancer 2018;65 2. URL
  78. Li C, Mathews V, George B, Kim S, Hebert K, Jiang H, Li C, Zhu Y, Keesler DA, Agarwal R, Boelens JJ, Dvorak CC, Auletta J, Goyal RK, Hanna R, Kasow KA, Margolis D, Shenoy S, Walters MC, Eapen M. Related and Unrelated Donor Transplantation for ? Thalassemia Major: Results of an International Survey. 60th ASH Annual Meeting & Exposition. San Diego, CA; Dec 1-4, 2018. Blood 2018 (132):Suppl 1:308. URL.
  79. Lianoglou BR, Gonzalez V, Velez JG, Norton ME, Chen B, Finley B, Hirata G, Ogasawara K, MacKenzie TC, Brown RN, Sanchez-Embrey E, Shaw SW, Foe M, Bitanga M, Chien M, Stumbaugh T, Thompson AA, Viprakasit V, Volanakis EJ, Vichinsky E. Impact of in utero transfusions in fetuses with hydrops fetalis due to alpha thalassemia. 40th Annual Meeting Society for Maternal Fetal Medicine. Dallas, TX; Feb 3-8, 2020. American Journal of Obstetrics and Gynecology 2020 (222):1:S300-S1. URL.
  80. Locatelli F, Walters MC, Kwiatkowski JL, Porter J, Sauer M, Thuret I, Hongeng S, Kulozik A, Lal A, Thrasher AJ, Yannaki E, Elliot H, Tao G, Asmal M, Thompson AA. LentiGlobin Gene Therapy for Patients with Transfusion-Dependent ?-thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies. 60th ASH Annual Meeting & Exposition. San Diego, CA; Dec 1-4, 2018.
  81. MacKenzie TC, Frascoli M, Sper R, Lianoglou BR, Velez JG, Dvorak CC, Kharbanda S, Vichinsky E. In utero stem cell transplantation in patients with alpha thalassemia major: Interim results of a phase 1 clinical trial. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):SUPPL 1:1. URL
  82. Madiwale MV, Kim J, Feusner J, Raphael R. Yield of screening echocardiogram during treatment and in the early post-therapy period in childhood cancer. ASPHO 31st Annual Meeting. Pittsburgh, PA; May 2-5, 2018. Pediatric Blood & Cancer 65(S1); e27057, #652. URL
  83. Maggio A, Vitrano A, Meloni A, Pollina WA, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Vichinsky E. Evidence for three distinct classes of phenotype severity in beta thalassaemia. 25th Congress of the European Hematology Association. Virtual Edition, June 11-21, 2020. HemaSphere 2020 (4):1059-60. URL
  84. Maggio A, Vitrano A, Meloni A, Addario Pollina W, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Singer ST, Borgio JF, Pepe A, Scondotto S, Dardanoni G, Sacco M, Pistoia L, Barone R, Bonifazi F, Pitrolo L, Vichinsky EP. Three distinct groups of phenotype severity in beta-thalassemia. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):SUPPL 1:15-6. URL
  85. Major K, Gardin K, Cherbini K, Hale L, Hagar W, Hoehner C, Marsh A, Schouten D, Treadwell M. Supporting transition from pediatric to adult care for individuals with sickle cell disease: transition workshop. 46th Annual National Convention for Sickle Cell Disease. Baltimore, MD; Oct 10-13, 2018. Sickle Cell Disease Association of America (SCDAA).
  86. Mapara MY, Tisdale JF, Kanter J, Kwiatkowski JL, Krishnamurti L, Schmidt M, Miller AL, Pierciey FJ, Shi W, Ribeil JA, Asmal M, Thompson AA, Walters MC. Lentiglobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from Hgb-206. Transplantation and Cellular Therapy Meetings. Feb 20-24, 2019; Houston, Texas. Biology of Blood and Marrow Transplantation 2019 (25):3:S64-S5. URL.
  87. Masese RV, Bulgin D, Knisely M, Preiss L, Stevenson E, Hankins JS, Treadwell M, King AA, Gordeuk VR, Kanter J, Gibson R, Glassberg JA, Tanabe P, Shah N. Sex based differences in sickle cell disease. 62nd Annual ASH Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):SUPPL 1:37. URL
  88. Masese RV, Bulgin D, Preiss L, Knisely M, Stevenson E, Hankins JS, Treadwell M, King AA, Gordeuk VR, Kanter J, Gibson R, Glassberg JA, Tanabe P, Shah N. Predictors of maternal morbidity among participants enrolled in the sickle cell disease implementation consortium registry. 62nd Annual ASH Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):SUPPL 1:3. URL
  89. Michlitsch J, Larkin S, Vichinsky E, Kuypers FA. Highlight article: Vincristine-induced anemia in hereditary spherocytosis. Exp Biol Med (Maywood) 2019 Jul; 244(10):850-4. PMC6643194 Epub 2019/06/05. URL.
  90. Milner J, Friedman D, Dozor AJ, D'Souza MD, Talano J-A, Moore TB, Shenoy S, Shi Q, Keever-Taylor CA, Weinberg RS, Grossman B, Walters MC, Vichinsky EP, Parsons SK, Braniecki S, McKinstry R, Verbsky J, Moorthy CR, Ayello J, Flower A, Chu Y, Morris E, Mahanti H, Fabricatore S, Militano O, Klejmont L, van de Ven C, Baxter-Lowe LA, Cairo MS. Stable to Improved Long Term Cardiac and Pulmonary Function in Children, Adolescents, and Young Adults with High Risk Sickle Cell Disease Following Myeloimmunoablative Conditioning and Familial Haploidentical Stem Cell Transplantation. TCT Meetings of ASTCT and CIBMTR. Feb 8-12, 2021. Transplantation and Cellular Therapy 2021 (27):3, Supplement:S316-S7. URL
  91. Minkley M, Jackson A, Smith D, Borchers C, Vichinsky E, Nashmi R, Walter PB, MacLeod PM. Neurodegeneration with brain iron accumulation: PLA2G6-associated dystonia-parkinsonism: Clinical and animal studies. 50th European Society of Human Genetics Conference. Bangkok, Thailand; August 26-27, 2019. European Journal of Human Genetics 2019 (26):402. URL.
  92. Ojo O, Litcher-Kelly L, Mazar I, Klooster B, Ollis S, Chaston E, Roeder A, Treadwell M, Reasner D. Measurement of sickle cell disease symptoms from the patient perspective: Considerations for clinical trials. 60th ASH Annual Meeting & Exposition. San Diego, CA; Dec 1-4, 2018. Blood 2018 (132):Suppl 1:3571.
  93. Olson T, Walters M, Kwiatkowski J, Hongeng S, Rasko J, Porter J, Sauer M, Thrasher A, Thuret I, Schiller G, Elliot H, Deary B, Chen Y, Tao G, Asmal M, Locatelli F, Thompson A. Lentiglobin gene therapy in transfusion-dependent B-thalassemia patients with non-ß0/ß0 genotypes. 2019 ASPHO Conference. New Orleans, LA; May 1-4, 2019. Pediatric Blood and Cancer 2019 (66):S1-S2. URL
  94. Peretz C, McGary L, Kumar T, Jackson H, Jacob J, Durruthy-Durruthy R, Zhang C, Levis MJ, Perl AE, Leung AYH, Smith CC. Single cell sequencing reveals evolution of tumor heterogeneity of acute myeloid leukemia on quizartinib. 61st Annual ASH Meeting & Exposition. Orlando, FL: Sept 7-10, 2019. Blood 2019 (134):S1. Poster. URL.
  95. Polgreen LE, Miller BS, Fung EB, White K, Rudser K, Petryk A, Whitley CB. Growth, bone, and joint outcomes in non-neuronopathic mucopolysaccharidosis type II over 10?years. Society for Inherited Metabolic Disorders. Seattle, WA: April 6-9, 2019. Molecular Genetics and Metabolism 2020 (129):2:S130. URL.
  96. Porter J, Shah F, Drasar E, Hill QA, Viprakasit V, Taher A, Georgiev P, Kuo KHM, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Perrotta S, Khelif A, Lal A, Kattamis A, Laadem A, Shetty JK, Linde PG, Hermine O, Piga A, Cappellini MD. Effects of luspatercept on iron overload and impact on responders to luspatercept: Results from the BELIEVE Trial. 60th Annual Scientific Meeting of the British Society for Haematology. April 27-29, 2020; Birmingham, UK. British Journal of Haematology 2020 (189):136-7. URL
  97. Porter J, Thompson A, Walters MC, Kwiatkovvski J, Hongeng S, Sauer M, Thrasher A, Thuret I, Tao G, Colvin R, Locatelli F. Improvement in erythropoiesis in patients with transfusion-dependent Beta-thalassemia following treatment with Betibeglogene Autotemcel (Lentiglobin for Beta-thalassemia) in the Phase 3 HGB-207 Study. 25th Congress of the European Hematology Association. June 11-21, 2020; Virtual Edition. HemaSphere 2020 (4):S296. URL
  98. Ramasamy J, Ibanez V, Al-Qadi K, Zhang H, Afranie-Sakyi J, Molokie RE, Lavelle D, Rivers A. Cathepsin B, a Negative Regulator of Autophagy, Identified As a Novel Therapeutic Drug Target in Sickle Cell Disease. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):Supplement 1:32. URL
  99. Raphael R, Hankin D, Gelgoot E, Salman N. Transition of care for young adult survivors of childhood cancer. ASPHO 31st Annual Meeting. Pittsburgh, PA; May 2-5, 2018. Pediatric Blood & Cancer 65(S1); e27057, #657. URL
  100. Rasko JEJ, Thompson AA, Kwiatkowski JE, Hongeng S, Schiller GJ, Anurathapan U, Cavazzana M, Ho J, Schmidt M, Kletzel M, Leboulch P, Vichinsky E, Deary B, Asmal M, Walters MC. Clinical outcomes following Lentiglobin gene therapy for transfusion-dependent B-thalassemia in the Northstar HGB-204 Study. International Society Cell & Gene Therapy( ISCT) Europe. Florence, Italy; Sept 12-14, 2018.
  101. Rasko JEJ, Thompson AA, Kwiatkowski JL, Hongeng S, Schiller GJ, Anurathapan U, Cavazzana M, Ho PJ, Schmidt M, Kletzel M, Leboulch P, Vichinsky E, Deary B, Chen Y, Asmal M, Walters MC. Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent Beta-Thalassemia Following Completion of the Northstar HGB-204 Study. 60th ASH Annual Meeting & Exposition. San Diego, CA; Dec 1-4, 2018. Blood 2018 (132):Suppl 1:167. URL.
  102. Schneiderman J, Thompson AA, Walters MC, Kwiatkowski JL, Kulozik AE, Sauer MG, Porter JB, Thuret I, Hongeng S, Lal A, Thrasher AJ, Yannaki E, Elliot H, Tao G, Liu W, Colvin RA, Locatelli F. Interim Results from the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy (LentiGlobin) for the Treatment of Transfusion-Dependent Beta-Thalassemia. American Society for Transplantation and Cellular Therapy. Houston, TX; Feb 20-24, 2020. Biology of Blood and Marrow Transplantation 2020 (26):3:S87-S8. URL.
  103. Schwab ME, Lianoglou BR, Ganno D, Gonzalez-Velez JM, Vichinsky E, MacKenzie TC. Long-Term Neurodevelopmental Outcomes After Fetal Transfusions for Hydrops Fetalis. American Pediatric Surgical Association Annual Meeting. May 20-22, 2021 (virtual).
  104. Sears M, Lanzkron S, Hoppe C, Field JJ, Desai PC, Williams E, Little J. Iron Overload Is Under-Recognized and Under-Treated in SCD: A Report from the Grndad Registry. 60th ASH Annual Meeting & Exposition. San Diego, CA; Dec 1-4, 2018. Blood 2018 (132):Suppl 1:158. URL
  105. Sharma R, Paucar-Acre M, Staretz-Chacham O, Hastings C, Hurst B, Murray B, Hrynkow SH. Initial safety and efficacy findings for a phase I/II trial of hydroxypropyl-beta-cyclodextrins administered intravenously in patients with Niemann-Pick Type C disease. 15th Annual World Lysosomal Disease Symposium. Orlando, FL; Feb 4-8, 2019.
  106. Smith WR, Sisler IY, Johnson S, Lipato T, Newlin JS, Owens ZS, Morgan AM, Treadwell MJ, Polak K. Lessons Learned from Building a Pediatric-to-Adult Sickle Cell Transition Program. 2019 Indiana Sickle Cell Conference: Time for Improved Emotional Health. Indianapolis, IN; April 26, 2019. South Med J 2019 (112):3:190-97. URL.
  107. Sowemimo-Coker SO, Kuypers F, Larkin S, Tung G, Dunham A. Effects of hypoxic red blood cells on sickling kinetics of red blood cells from patients with sickle cell disease. AABB Annual Meeting. San Antonio, TX, Oct 19-22, 2019. Transfusion 2019 (59):157A. URL.
  108. Taher AT, Viprakasit V, Cappellini MD, Hermine O, Georgiev P, Kuo KHM, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Forni GL, Perrotta S, Khelif A, Lal A, Kattamis A, Shah F, Porter J, Laadem A, Shetty JK, Kuo WL, Zhang J, Miteva D, Zinger T, Sinsimer D, Louis C, Linde PG, Piga A. Assessment of longer-term efficacy and safety in the phase 3 believe trial of luspatercept to treat anemia in patients (pts) with beta-thalassemia. 25th Congress of the European Hematology Association; Virtual Edition, 2020. HemaSphere 2020 (4):717-8. URL
  109. Thompson AA, Kwiatkowski JL, Porter JB, Hongeng S, Yannaki E, Kulozik AE, Sauer MG, Thrasher AJ, Thuret I, Lal A, Guo R, Liu W, Colvin RA, Walters MC, Locatelli F. Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent Beta-Thalassemia. 62nd ASH Annual Meeting & Exposition. Dec 5-8,, 2020. Blood 2020 (136):Supplement 1:52-4. URL
  110. Thompson AA, Walters MC, Mapara MY, Kwiatkowski JL, Krishnamurti L, Aygun B, Kasow KA, Rifkin-Zenenberg S, Schmidt M, DelCarpini J, Pierciey FJ, Jr., Miller AL, Gallagher ME, Chen R, Goyal S, Kanter J, Tisdale JF. Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):Supplement 1:16-7. URL
  111. Thompson A, Krishnamurti L, Mapara M, Kwiatkowski J, Tisdale J, Schmidt M, Miller A, Pierciey F, Huang W, Ribeil JA, Kanter J, Walters M. Lentiglobin gene therapy for patients with sickle cell disease: Updated results from study hgb-206. 2020 ASPHO Conference (online). Pediatric Blood and Cancer 2020 (67):SUPPL 2. URL
  112. Tisdale JF, Kanter J, Hsieh M, Lakshmanan K, Kwiatkowski J, Kamble RT, Von Kalle C, Miller A, Pierciey FJ, Shi W, Asmal M, Thompson AA, Walters MC. Single-agent plerixafor mobilization to collect autologous stem cells for use in gene therapy for severe sickle cell disease. Feb 21, 2018 (poster).
  113. Tisdale JF, Kanter J, Mapara MY, Kwiatkowski JL, Krishnamurti L, Schmidt M, Miller AL, Pierciey FJ, Shi W, Ribeil J-A, Asmal M, Thompson AA, Walters MC. Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study. 60th ASH Annual Meeting & Exposition. San Diego, CA; Dec 1-4, 2018. Blood 2018 (132):Suppl 1:1026. URL.
  114. Truong B, Agrawal A, Winestone L. Language barriers and incidence of febrile neutropenia among pediatric and young-adult patients with acute lymphoblastic leukemia. SIOP Annual Conference. Oct 14-17, 2020; Ottawa, Canada. Pediatric Blood and Cancer 2020 (67):SUPPL 2. URL
  115. Uhlig K, Vichinsky E, Kuo K, Layton M, Hua L, Mix C. Design of a Phase 2, Open Label, Multicenter Study of MITAPIVAT (AG348) in Adults with Non-Transfusion Dependent Thalassemia. 24th Congress of the European Hematology Association. Amsterdam, Netherlands; June 13-16, 2019. HemaSphere 2019 (3):1065-6.
  116. Vichinsky EP, Telfer P, Inati A, Tonda M, Tong B, Agodoa I, Lehrer-Graiwer J, Ataga KI. Incidence of Vaso-occlusive Crisis Does Not Increase with Achieving Higher Hemoglobin Levels on Voxelotor Treatment or After Discontinuation: Analyses of the HOPE Study. EHA Annual Congress. Frankfurt, Germany; June 11-14, 2020. HemaSphere 2020 (4):Supplement 1:706.
  117. Vichinsky EP, Telfer P, Inati A, Tonda M, Tong B, Agodoa I, Lehrer-Graiwer J, Ataga KI. Incidence of vaso-occlusive crisis does not increase with achieving higher hemoglobin levels on voxelotor treatment or after discontinuation: Analyses of the hope study. 60th Annual ASH Meeting & Exposition. San Diego, CA; Nov 30-Dec 1, 2018. Blood 2019 (134):S1. URLURL.
  118. Vichinsky E, Hoppe C, Howard J, Ataga K, Nduba V, El Beshlawy A, Diuguid D, Al Kindi S, Brown C, Hassab H, Telfer P, Tsitsikas D, Unal S, Kanter J, Abboud M, Gordeuk V, Lehrer-Graiwer J, Sherman C, Tonda M, Intondi A, Yaron Y, Ware R. Voxelotor in adolescents and adults with sickle cell disease: Results of the phase 3 HOPE trial. 2019 American Society of Pediatric Hematology/Oncology Conference (ASPHO). New Orleans, LA; May 1-4, 2019. Pediatric Blood and Cancer 2019 (66):S2:S242; poster 713. URL.
  119. Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El Beshlawy A, Hassab H, Achebe MM, Al-Kindi S, Brown C, Diuguid DL, Telfer P, Dimitris TA, El Ghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J. Results from the Randomized Placebo-Controlled Phase 3 Hope Trial of Voxelotor in Adults and Adolescents with Sickle Cell Disease. 24th Congress of the European Hematology Association. Amsterdam, Netherlands; June 13-16, 2019.
  120. Vichinsky EP, Telfer P, Inati A, Tonda M, Tong B, Agodoa I, Lehrer-Graiwer J, Ataga KI. Incidence of vaso-occlusive crisis does not increase with achieving higher hemoglobin levels on voxelotor treatment or after discontinuation: Analyses of the HOPE study. 60th Annual ASH Meeting & Exposition. San Diego, CA; Nov 30-Dec 1, 2018. Blood 2019 (134):S1. URL.
  121. Vichinsky E, Gordeuk V, Telfer P, Inati A, Tonda M, Gray S, Agodoa I, Ataga K. Higher hemoglobin levels achieved with voxelotor are associated with lower vaso-occlusive crisis incidence: 72-week analysis from the HOPE Study. 62nd ASH Annual Meeting & Exposition; Dec 5-8, 2020. Blood 2020 (136):S1:31-2. URL
  122. Vissa M, Michlitsch J. A unique case of pediatric ALL with FUS-ERG rearrangement undergoing lineage switch to AML. American Society of Pediatric Hematology Oncology (ASPHO). New Orleans, LA; May 1-4, 2019. Pediatric Blood and Cancer 2019 (66):S52-S3. URL.
  123. Vissa M, Soupene E, Larkin SK, Neumayr LD, Vichinsky EP, Kuypers FA. Differential acetone extraction of total and hemoprotein-unbound heme to quantify heme binding capacity of plasma in patients with sickle cell disease: The role of heme scavengers. 62nd ASH Annual Meeting and Exposition. Dec 5-8, 2020. Blood 2020 (136):SUPPL 1:15. URL
  124. Vitrano A, Meloni A, Pollina WA, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Singer ST, Borgio JF, Pepe A, Scondotto S, Dardanoni G, Di Maggio R, Sacco M, Vichinsky EP, Maggio A. Development of a severity score system for thalassemia syndromes. 60th Annual ASH Meeting & Exposition. San Diego, CA; Nov 30-Dec 1, 2018. Blood 2019 (134):S1. URL.
  125. Vo KT, Michlitsch J, Shah AT, Long-Boyle J, Kim MO, Gustafson WC, Sweet-Cordero EA, Matthay KK, DuBois SG. Phase 1 multicenter trial to assess the maximum tolerated dose, safety, pharmacokinetics, and pharmacodynamics of pazopanib in combination with irinotecan and temozolomide (PAZIT) for children and young adults with advanced sarcoma. ASCO annual meeting. Chicago, IL, June 2018. J Clin Oncol 36(suppl; abstr TPS10576; poster #248b.
  126. Vo KT, Michlitsch J, Shah AT, Reid JM, Buhrow SA, Graham EM, Hollinger F, Zapala MA, Long-Boyle J, Kim MO, Matthay KK, DuBois SG. Phase 1 Trial of Pazopanib in Combination with Irinotecan and Temozolomide (PAZIT) for Children and Young Adults with Advanced Sarcoma. ASCO annual meeting. May 29-31, 2020. Poster.
  127. Walters MC, Kwiatkowski JL, Rasko JE, Hongeng S, Schiller GJ, Anurathapan U, Cavazzana M, Ho J, Schmidt S, Kletzel M, Leboulch P, Vichinsky E, Deary B, Chen Y, Petrusich A, Thompson AA. Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent Beta-Thalassemia (TDT) Following Completion of the Northstar (Hgb-204) Study. 24th Congress of the European Hematology Association. Amsterdam, Netherlands; June 13-16, 2019.
  128. Walters M, Kwiatkovvski J, Rasko J, Hongeng S, Schiller GJ, Anurathapan U, Cavazzana M, Ho PJ, Von Kalle C, Kletzel M, Leboulch P, Vichinsky E, Deary B, Asmal M, Thompson AA. Safety following autologous transplantation with lentiglobin gene therapy for transfusion-dependent Beta thalassemia (TDT) in the Northstar Hgb-204 Study. Feb 23, 2018 (oral presentation).
  129. Walters MC, Kwiatkowski JL, Porter JB, Hongeng S, Yannaki E, Kulozik AE, Sauer MG, Thrasher AJ, Thuret I, Lal A, Guo R, Liu W, Colvin RA, Locatelli F, Thompson AA. Safety and Efficacy Outcomes in Pediatric Patients with Transfusion-Dependent Beta-Thalassemia (TDT) Receiving Betibeglogene Autotemcel (beti-cel; LentiGlobin for Beta-thalassemia) Gene Therapy in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies. TCT Meetings of ASTCT and CIBMTR. Feb 8-12, 2021. Transplantation and Cellular Therapy 2021 (27):3, Supplement:S112-S4. URL
  130. Walters MC, Kanter J, Kwiatkowski JL, Krishnamurti L, Mapara MY, Schmidt M, Miller AL, Pierciey JFJ, Bonner M, Huang W, Ribeil JA, Thompson AA, Tisdale JF. Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy (GT): Updated Results in Group C Patients from the Phase 1/2 Hgb-206 Study. American Society for Transplantation and Cellular Therapy. Houston, TX; Feb 20-24, 2020. Biology of Blood and Marrow Transplantation 2020 (26):3:S1-S2. URL
  131. Walters MC, Chui DHK, Farrell JJ, Lal A, Locatelli F, Kwiatkowski JL, Porter JB, Sauer MG, Thuret I, Hongeng S, Kulozik AE, Thrasher AJ, Yannaki E, Yang J, Whitney D, Petrusich A, Colvin RA, Thompson AA. Response of Patients with Transfusion-Dependent Beta-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for Beta-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):Supplement 1:1-3. URL
  132. Walters MC, Thompson AA, Mapara MY, Kwiatkowski JL, Krishnamurti L, Aygun B, Kasow KA, Rifkin-Zenenberg S, Schmidt M, DelCarpini J, Pierciey FJ, Miller A, Chen R, Goyal S, Kanter J, Tisdale JF. Resolution of Serious Vaso-Occlusive Pain Crises: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy. TCT Meetings of ASTCT and CIBMTR. Feb 8-12, 2021. Transplantation and Cellular Therapy 2021 (27):3, Supplement:S12-S3. URL
  133. Washington C, Savic R, Green M, Inati A, Estepp J, Brown C, Hoppe C, Wang W, Woods G, Balaratnam G, Dixon S, Fong E, Hutchaleelaha A, Tonda M, Lehrer J. The pharmacokinetics of voxelotor following single doses in pediatric patients with sickle cell disease The pharmacokinetics of voxelotor following single doses in pediatric patients with sickle cell disease. ASPHO 31st Annual Meeting; Pittsburgh, PA; May 2-5, 2018. Pediatric Blood and Cancer 2018;65:S94. URL.
  134. Washington C, Green M, Inati A, Estepp JH, Brown C, Abboud M, Drachtman R, Gordeuk VR, Hoppe CC, Hsu L, Liem R, Piccone C, Wang W, Woods G, Goldstein B, Dixon S, Fong E, Hutchaleelaha A, Tonda M, Lehrer J. Pharmacokinetics (PK) of Voxelotor (GBT44) using population pharmacokinetic (PPK) and physiologically based pharmacokinetic (PBPK) modeling in pediatric subjects with sickle cell disease (SCD). 23rd Congress of the European Hematology Association (EHA). Stockholm, Sweden; June 14-17, 2018.
  135. Washington C, Goldstein B, Dixon S, Hoppe C, Ware RE, Lehrer J. Voxelotor dose extrapolation for a phase 3, randomized, double blind, placebo-controlled study in pediatric patients with sickle cell disease (GBT440-032, HOPE KIDS 2). 23rd Congress of the European Hematology Association (EHA). Stockholm, Sweden; June 14-17, 2018.
  136. Washko JK, Treadwell M, Marsh A, Bardach N, Cabana M. Results of a national provider survey in sickle cell disease management. ASPHO 31st Annual Meeting. Pittsburgh, PA; May 2-5, 2018. Pediatric Blood & Cancer 2018 (65):S1:e27057. URL.
  137. Wen F, Rock A, Salomon-Andonie J, Kurban G, Niu X, Wang S, Zhang X, Gordeuk VR, Zhang Y, Nouraie SM, Gladwin MT, Ashley-Koch A, Telen MJ, Custer B, Kelly S, Dinardo CL, Sabino E, Wong Q, Taylor JG, Nekhai S. Genome wide association analysis of iron overload in the trans-omics for precision medicine (TOPMED) sickle cell disease cohorts. 62nd Annual ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):SUPPL 1:52. URL
  138. Weyhmiller M, Lal A, Arwin W, Cheng A. Room temperature sensors for biomagnetic liver susceptometry: Comparisons with the Oakland ferritometer. Eighth Congress of the International BioIron Society (IBIS). Heidelberg, Germany; May 5-10, 2019.
  139. Weyhmiller M, Lal A, Vichinsky E, Fischer R, Paulson D, Pratt K. The SQUID biosusceptometer at Oakland: Sixteen years of experience Eighth Congress of the International BioIron Society (IBIS). Heidelberg, Germany; May 5-10, 2019.
  140. Williams E, Brown E, Manwani D, Desai P, Field JJ, Neumayr LD, Padrino S, Clay ELJ, Cong Z, Agodoa I, Hoppe C, Lanzkron SM, Little JA. Chronic kidney disease is under-screened in SCD and mild albuminuria is associated with a drop in hemoglobin: A report from the grndad sickle cell registry. 61st Annual ASH Meeting & Exposition. Dec 7-10, 2019; Orlando, Florida. Blood 2019 (134):S1. URL
  141. Xia-Zhu D, Ward N, Peretz CAC, Hagar W, Michlitsch J, Hastings C. Clots and cancer. CHORI Summer Research Symposium. Oakland, CA; August 2018
  142. Yaeger D, Piga A, Lal A, Kattamis A, Salman S, Byrnes B, Tidmarsh G, Chawla L. A phase 1, placebo-controlled study to determine the safety, tolerability, and pharmacokinetics of escalating subcutaneous doses of LJPC-401 (synthetic human hepcidin) in healthy adults. 23rd Congress of the European Hematology Association. Stockholm, Sweden, June 14-17, 2018. HemaSphere 2018 (2):188. URL.
  143. Yannaki E, Locatelli F, Kulozik AE, Porter JB, Thuret I, Sauer MG, Lal A, Liu W, Colvin RA, Thompson AA, Kwiatkowski JL. Betibeglogene autotemcel (Lentiglobin) in patients with transfusion-dependent b-thalassemia and b0/b0, b+IVS-I-110/b+IVS-I-110, or b0/b+ivs-i-110 genotypes: Updated results from the HGB-212 study. 25th Congress of the European Hematology Association Virtual Edition, 2020. HemaSphere 2020 (4):692-3. URL
  144. Yoon HS, Michlitsch J, Jeng M. Langerhans cell histiocytosis: gastrointestinal involvement in children. ASPHO 31st Annual Meeting. Pittsburgh, PA; May 2-5, 2018. Pediatric Blood & Cancer 65(S1); e27057, #758. URL
  145. Yu H, Peretz CC, Matsunaga A, Agrawal A. Pre-B acute lymphoblastic leukemia in a child presenting with vaginal bleeding and otorrhea. American Society of Pediatric Hematology Oncology (ASPHO). New Orleans, LA; May 1-4, 2019. Pediatric Blood and Cancer 2019 (66):S40-S1. URL.

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INVITED LECTURES/ARTICLES/BOOKS/CHAPTERS

Books:

  • Earles A, Lessing S, Vichinsky EP, eds. A Parent's Handbook for Sickle Cell Disease, Part II: Birth to 6 to 18 Years of Age. California Department of Public Health, Genetic Diseases Branch; 1st ed 1994; 2019 (3rd edition in progress).
  • Lessing S, Vichinsky E, eds. A Parent's Handbook for Sickle Cell Disease: Part 1: Birth to 6 Years of Age. California Department of Health, Genetic Disease Branch; 2019 (3rd edition in progress).

Chapters/Articles:

  • Bhasin N, Vichinsky E. Sickle Cell Disease. In: Hoffbrand AV, Higgs DR, eds. Postgraduate Haematology, 8th ed: John Wiley & Sons, Ltd 2021 (in progress).
  • Carlberg KT, Hastings C. Hyperleukocytosis. In: Cabana MD, ed. The 5-Minute Pediatric Consult. 8th edition. ed. Philadelphia: Wolters Kluwer; 2019. URL.
  • Carlberg KT, Hastings C. Leukocytosis. In: Cabana MD, ed. The 5-Minute Pediatric Consult. 8th edition. ed. Philadelphia: Wolters Kluwer; 2019. URL.
  • Fritch-Lila S, Singer ST, Vichinsky EP. Thalassemia Disorders in the Adolescent Female. In: Srivaths LV, ed. Hematology in the Adolescent Female. 1st ed. Springer; 2020 (In Press).
  • Hastings C. The ASPHO 2019 distinguished career award goes to Dr. Elliott P. Vichinsky. Pediatr Blood Cancer 2019 Jun; 66 Suppl 2(e27689. Epub 2019/03/03. URL.
  • Jamieson CHM, Millan MT, Creasey AA, Lomax G, Donohoe ME, Walters MC, Abedi M, Bota DA, Zaia JA, Adams JS. CIRM Alpha Stem Cell Clinics: Collaboratively Addressing Regenerative Medicine Challenges. Cell Stem Cell 2018 2018/06/01/; 22(6):801-5. URL
  • Kidwell KM, Treadwell M, Crosby LE. Pediatric Sickle Cell Disease. In: Modi AC, Driscoll KA, eds. Adherence and self-management in pediatric populations. 1st ed. San Diego: Academic Press (Elsevier); 2020: p.185-201.
  • Kuypers FA. Cell Membranes. In: Goodman SR, ed. Goodman's Medical Cell Biology. 4th ed., Elsevier; 2020 (In Press).
  • Lal A. Thalassemia Management Checklists: Monitoring Deferasirox Therapy. Cooley's Anemia Foundation, Health Resources and Services Administration (HRSA) 2018. URL.
  • Lal A. Challenges in chronic transfusion for patients with thalassemia. Am Soc Hematol Educ ProgramDec 4; 2020(1):160-6. PMC7727587 Epub 2020/12/05. URL
  • Lalefar N, Walters MC. Hematopoietic Cell Transplantation for non-malignant disorders. In: Appelbaum F, ed. Wintrobe's Clinical Hematology. 14th ed. Philadelphia, PA: Wolters Kluwer/Lippincott Williams & Wilkins; 2019.
  • Leonard A, Tisdale JF, Lakshmanan K, Walters MC. Hematopoietic Stem Cell Transplantation in Sickle Cell Disease. 2020 (in press).
  • Marsh A, Matsunaga A. Neonatal Alloimmune Thrombocytopenia. In: Cabana MD, Goldsby RE, eds. The 5 Minute Pediatric Consult. 8th ed: Wolters Kluwer; 2018 (in press).
  • Minkowitz B, Sawyer A, Fung EB, Dvorzhinskiy A, Lane JM. The Answer is Vitamin D! From Pediatrics to Geriatrics in Orthopaedics. Instr Course Lect 2018 Feb 15; 67(529-41. Epub 2018/02/15. URL.
  • Murphy P, Raphael R. Cancer Therapy Late Effects. In: Cabana MD, Goldsby RE, eds. The 5 Minute Pediatric Consult. 8th ed: Wolters Kluwer; 2018 (in press).
  • Peretz CAC, Feusner J. Germ Cell Tumors. In: Cabana MD, ed. The 5-minute pediatric consult. 8th edition. ed. Philadelphia: Wolters Kluwer; 2019:xli, 1111 pages. URL.
  • Rich SS, Erlich H, Concannon P. Genetics of Type 1 Diabetes. In: Cowie CC, Casagrande SS, Menke A, et al., eds. Diabetes in America. Bethesda (MD); 2018. URL
  • Romero Z, DeWitt M, Walters MC. Promise of gene therapy to treat sickle cell disease. Expert Opin Biol Ther2018 Nov; 18(11):1123-36. Epub 2018/10/17. URL.
  • Saba J. Sphingosine phosphate lyase insufficiency syndrome. NIH National Center for Advancing Translational Sciences. 2020; URL.
  • Strunk C, Campbell A, Colombatti R, Andemariam B, Kesse-Adu R, Treadwell M, Inusa BPD. Annual Academy of Sickle Cell and Thalassaemia (ASCAT) conference: a summary of the proceedings. BMC Proc 2020 Dec 16; 14(Suppl 20):21. PMC7739449 Epub 2020/12/17. URL
  • Treadwell M, Baker JR, Brown M, Vora S. for the California Sickle Cell Planning Initiative stakeholders. California Sickle Cell Action Plan. Dec. 2018. URL.
  • Treadwell M, Jacob E, Hessler D, Chen A, Chen O, Mushiana S, Potter M, Vichinsky E. Sickle Cell Care Coordination Initiative: Community-based needs assessment to inform strategies to reduce healthcare disparities in Northern California. Jan. 2019.
  • Vichinsky E, Mekontso-Dessap A. Acute Chest Syndrome. In: Gladwin MT, ed. Sickle Cell Disease. McGraw-Hill, 1st ed; 2021 (In Press).
  • Walters MC. Sickle marrow: double, double toil and trouble. Blood 2020 Jun 4; 135(23):2017-8. Epub 2020/06/05. URL.
  • Zlotnicki M, Hastings C. Acute Lymphoblastic Leukemia. In: Cabana MD, Goldsby RE, eds. The 5 Minute Pediatric Consult. 8th ed: Wolters Kluwer; 2018 (in press).

Editorial:

  • Walters MC. Induction of Fetal Hemoglobin by Gene Therapy. N Engl J Med 2021 Jan 21; 384(3):284-5. Epub 2021/01/21. URL

Commentary:

  • Agrawal AK, Michlitsch J, Golden C, Hastings CA, Raphael R, Feusner JH. Nelarabine in Pediatric and Young Adult T-Cell Acute Lymphoblastic Leukemia-Clearly Beneficial? J Clin Oncol 2021 Feb 20; 39(6):694. Epub 2021/01/15. URL
  • Morris CR, Vichinsky EP. RE: L-Glutamine for Sickle Cell Disease: Some Answers to Your Questions by Quinn CT. Blood. July 30, 2018 132;7. URL
  • Musallam K, Vitrano A, Meloni A, Pollina WA, Karimi M, Beshlawy A, Hajipour M, Di Marco V, Ansari S, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Sankaran V, Vichinsky E, and the International Working Group on Thalassemia (IWG-THAL). Survival and causes of death in 2033 patients with non-transfusion-dependent ß-thalassemia (NTDT). Haematologica 2021 (submitted).
  • Stewart J-AT, Willen SM, Cohen R, Rodeghier M, Kirkham F, Redline SS, Rosen C, Kirkby J, DeBaun MR. BMI percentile is an independent predictor of increase in lung function in children with sickle cell anemia. American Journal of Hematology 2019 2019/05/01; 94(5):E136-E8. URL
  • Willen SM, McNeil JB, Rodeghier M, Kerchberger VE, Shaver CM, Bastarache JA, Steinberg MH, DeBaun MR, Ware LB. Haptoglobin genotype predicts severe acute vaso-occlusive pain episodes in children with sickle cell anemia. Am J Hematol 2020 Apr; 95(4):E92-E5. PMC7343605 Epub 2020/01/11. URL

Lectures (INCOMPLETE):

2018

  • Agrawal A.
    1. CAR-T therapy in relapsed ALL/NHL. Pediatric Hematology/Oncology Conference (invited talk); Kaiser Permanente Oakland, 2018.
    2. CAR-T therapy in relapsed ALL/NHL. Immunotherapy Conference (invited talk); UCSF Benioff Children’s Hospital San Francisco, 2018.
  • Calloway C.
    1. Probe capture next-generation sequencing system for forensic and clinical applications. Thermofisher; Oyster Point, CA; Feb 12, 2018.
    2. Forensic and Clinical Applications to Next-Generation Sequencing. Molecular Techniques ETX278 Course; Mar 12, 2018, UC Davis, CA.
    3. Probe capture next-generation sequencing system for forensic and clinical applications. Natera; San Carlos, CA; May 10, 2018.
  • Fung E.
    1. 5th Pediatric Bone Health Symposium. Invited speaker; San Francisco, CA; 2018.
    2. Achondroplasia BMN 111 Study Train the Trainers. Invited speaker, BioMarin, Chicago, IL; 2018.
    3. Cooley's Anemia Family Conference. Invited speaker; Atlanta, GA; 2018.
    4. Thriving Not Just Surviving. Invited speaker, Experimental Biology; Chicago, IL; 2018.
  • Hagar RW, Vichinsky E, Hoppe C, Walters M, Bell M, Marsh A, Treadwell M, Martin K, Kurio G, Neumayr L, Garcia A, Kelly S, Hoehner C, Harmatz P, Fung E, Major K, Lal A.
    1. HELP-SC (Health Education and Liaison Program for Sickle Cell) Adult Boot Camp [Seminar]: UCSF Benioff Children's Hospital Oakland; Apr 30-May 4, 2018.
  • Hastings C.
    1. National Niemann-Pick Disease Foundation 26th Annual Conference, Louisville, KY
    2. Webinar, National Niemann-Pick Disease Foundation
  • Hoppe CC.
    1. Bioverativ medical advisory board meeting. Bethesda, MD; 2018 (invited speaker).
  • Lal A.
    1. A Phase 1, Open-Label Study to Determine the Safety, Tolerability, and Pharmacokinetics of Escalating Doses of LJPC-401 (Synthetic Human Hepcidin) in Patients With Iron Overload. 23rd Congress of the European Hematology Association (EHA). Stockholm, Sweden; June 14-17, 2018.
  • Saba J.
    1. Dendritic cells are the metabolic gatekeepers of thymic egress through regulation of S1P gradients. Gordon Research Conference on Glycolipid and Sphingolipid Biology (invited talk). Galveston, TX; Feb 2018.
    2. The role of S1P lyase in lymphocyte trafficking: from mouse models to human primary immunodeficiency. Children’s National Medical Center Department of Immunology and Cancer seminar series (guest lecturer). Washington, DC; June 2018.
    3. SPL Insufficiency Syndrome (SPLIS) -- a novel inborn error of sphingolipid metabolism -- reveals the critical role of S1P in the pathogenesis of several diseases. 59th Advances in Biological Regulation Symposium (invited talk). Bologna, Italy; Oct 2018.
  • Treadwell M.
    1. Public private partnerships to create meaningful impact on sickle cell disease in Africa in the next decade -- Expert Discussion. 71st World Health Assembly; May 22, 2018; World Health Organization, Geneva, Switzerland.
    2. Sickle Cell Disease Implementation Consortium. NHLBI Sickle Cell Clinical Research Meetings; Rockville, MD; Aug 14, 2018.
    3. New approaches to therapies for sickle cell disease. Cayenne Wellness Sickle Cell Educational Symposium; Sacramento, CA; Sept 6, 2018.
    4. Biopsychosocial-spiritual model of coping in sickle cell disease. Annual Sickle Cell Disease and Thalassaemia Conference; London, UK; Oct 23, 2018.
  • Vichinsky E.
    1. Martin Luther King Jr. Celebration and Healthcare Fair. Keynote speaker, Pittsburg, CA.; Jan 15, 2018.
    2. Training the Healthcare Community in Sickle Cell Disease. UC Davis, Sacramento, CA; Jan 27, 2018.
    3. Challenges with SCD and Sickle Cell Trait: Follow-up in California. California Sickle Cell State Planning Initiative; Sacramento, CA; Jan 27, 2018.
    4. Disparities in Treatment of Sickle Cell Disease. Keynote speaker; Northern California Sickle Cell Social Justice Rally; Pittsburg, CA; Apr 7, 2018.
    5. Adult Neuro-cognitive Outcomes. Seminar presenter: Neurological consequences of Sickle Cell Disease Conference. Emory University, Atlanta, GA; March 8-9, 2018.
    6. Northern California Sickle Cell Patient Panel. Keynote speaker, Pittsburgh, CA; Apr 7, 2018.
    7. HELP-SC (Health Education and Liaison Program for Sickle Cell) Adult Boot Camp. Seminar presenter; UCSF Benioff Children's Hospital Oakland; Apr 30-May 4, 2018.
    8. Alpha Thalassemia. Workshop Chair and speaker. American Society of Pediatric Hematology Oncology, Pittsburgh, PA; May 2-5, 2018.
    9. Chronic Transfusion is the Treatment of Choice for Secondary Stroke in Sickle Cell Disease. American Society of Pediatric Hematology Oncology, Pittsburgh, PA; May 2-5, 2018.
    10. Advances in the management of Alpha Thalassemia Major, including preclinical and Phase I studies involving in utero stem cell therapy. American Society of Pediatric Hematology Oncology, Pittsburgh, PA; May 3, 2018.
    11. Alpha Thalassemia: a growing public health disease with changing landscape of in utero and post-natal therapy. American Society of Pediatric Hematology Oncology, Pittsburgh, PA; May 3, 2018.
    12. Overview of Thalassemia. Agios Pharmaceuticals, San Mateo, CA; July 18, 2018.
    13. Speaker, Thalassemia Adoption Clinic Dinner; July 18, 2018, Oakland, CA.
    14. Oxford Union Style Debate with audience participation on HCT for SCD. Symposium on Curative Therapies in SCD; Bethesda, MD; Aug 13, 2018.
    15. Sickle Cell Therapeutics: Is it time for combination therapy? Invited presentation; NIH Annual Sickle Cell Disease meeting; Bethesda, MD; Aug 15, 2018.
    16. Alpha Thalassemia--A rapidly growing public health disease with changing landscape in population screening, in-utero transfusion therapy, novel targeted molecules, and promising stem cell therapy options. UCSF Health Executive Council; guest speaker; Aug. 29, 2018, San Francisco, CA.
    17. Sickle Cell Trait and Sickle Cell Complications. Sickle Cell Disease Educational Seminar; Sept 6-7, 2018, UC Davis, CA.
    18. 10th annual Sickle Cell California Symposium. Keynote speaker, Oakland, CA; Sept 9-10, 2018.
    19. Multiorgan failure and aging in sickle cell disease. 7th Annual SCD Therapeutics Conference; Global Blood Therapeutics, New York, NY; Sep 12-13, 2018.
    20. Overview of Thalassemia. San Mateo, CA; Sept. 24, 2018.
    21. Sickle Cell Workshop Day 1. Preceptorship symposium for Novartis employees; Children's Hospital Oakland Research Institute; Oct 18, 2018, Oakland, CA.
    22. Sickle Cell Workshop Day 2. Preceptorship symposium for Novartis employees; Children's Hospital Oakland Research Institute; Oct 19, 2018, Oakland, CA.
    23. Seminar on Hemoglobin BARTs Hydrops Fetalis. Keynote speaker; Rady Children's Hospital; Nov 1, 2018, San Diego, CA.
    24. Alpha Thalassemia: a rapidly growing public health disease with changing landscape of in-utero and post-natal therapy. Nov 1-2, 2018, San Diego, CA.
    25. State of Sickle Cell Disease in the United States. Keynote speaker; Sickle Cell Disease Education and Awareness Symposium; Nov 15, 2018; Children's Hospital Oakland Research Institute.
    26. Emerging Therapies in Hemoglobinopathies. Frontiers in Medicine, Inquiry CIC1 Lecture; Nov. 27, 2018, UCSF, San Francisco, CA.
    27. Hb S Polymerization Inhibitors. Invited speaker, ASH Symposium Sickle Cell Disease Forum: Innovation in Drug Development and Emerging Science; Nov 30, 2018, San Diego, CA.
    28. Results from Part A of the Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization (HOPE) Trial (GBT 440-031), a placebo-controlled randomized study evaluating Voelotor (GBT 440) in adults and ad9olescents with sickle cell disease. Best of ASH 2018, 60th ASH Annual Meeting & Exposition; Dec 3, 2018; San Diego, CA.
    29. Sickle Cell Disease Forum: Innovation in Drug Development and Emerging Science. Activity Chair and Monitor, CME certified symposium; 60th ASH Annual Meeting & Exposition; Dec 3, 2018; San Diego, CA.
  • Walters M, Vichinsky E.
    1. Oxford Union Style Debate with audience participation on HCT for SCD. Symposium on Curative Therapies in SCD. Bethesda, MD; Aug 13, 2018.
  • Walters M.
    1. Bioverativ medical advisory board meeting. Bethesda, MD, 2018.
    2. Chair, NHLBI Sickle Cell Disease Advisory Committee meeting. Bethesda, MD; Jan 26, 2018.
    3. Gene Therapy for Hemoglobinopathies. ASBMT Tandem Meeting, pediatric symposium (invited speaker), Salt Lake City, UT; Feb 20-24, 2018.
    4. Gene Therapy for Hemoglobinopathies. Hematology/Oncology/BMT; Feb 22, 2018; UCSF Benioff Children's Hospital, Oakland, CA.
    5. Rare Disease: Sickle Cell Disease/1507 Haplo HCT. ASBMT Tandem Meeting, pediatric symposium (invited speaker), Salt Lake City, UT; Feb 20-24, 2018.
    6. Single-Agent Plerixafor Mobilization to Collect Autologous Stem Cells for Use in Gene Therapy for Severe Sickle Cell Disease. ASBMT Tandem Meeting, pediatric symposium (invited speaker), Salt Lake City, UT; Feb 20-24, 2018.
    7. Safety Following Autologous Transplantation with Lentiglobin Gene Therapy for Transfusion-Dependent ?-Thalassemia (TDT) in the Northstar Hgb-204 Study. ASBMT Tandem Meeting, pediatric symposium (invited speaker), Salt Lake City, UT; Feb 20-24, 2018.
    8. Single-agent plerixafor mobilization to collect autologous stem cells for use in gene therapy for severe sickle cell disease. European Blood & Marrow Transplant meeting (oral presentation), Lisbon, Portugal; March 19, 2018.
    9. Update on ongoing clinical trials/studies. Symposium on Curative Therapies in SCD. Bethesda, MD; Aug 13, 2018.
    10. Oxford Union Style Debate with audience participation on HCT for SCD.Symposium on Curative Therapies in SCD, Bethesda, MD; Aug 13, 2018.

2019

  • Hastings C
    1. International Niemann-Pick Disease Alliance (INPDA) 6th Biennial “Face to face” meeting, June 14016, 2019, Lyon, France
    2. National Niemann-Pick Disease Foundation 27th Annual Conference, Bloomington, MN
    3. Webinar, National Niemann-Pick Disease Foundation
  • Saba J
    1. External Advisory Panel, NIDDK Intramural Programs. Oct 10, 2019.
  • Vichinsky E.
    1. POST-ASH San Diego 2018. Novita dal Meeting della Società Americana di Ematologia; Feb 14-16, 2019, Bologna, Italy.
    2. Deferiprone Trial Update. 10th Annual International NBIA Disorders Association Family Conference; May 30, 2019, Deerfield, IL.
    3. NHLBI Annual SCD Research Meeting; Aug. 26-28, 2019, Bethesda, MD.
    4. Moving from Science Fiction to Clinical Reality: Gene Therapy for Beta-Hemoglobinopathies. Chair. Hosted by Medscape, Friday Satellite Symposia at the 61st Annual ASH Meeting and Exposition. Dec. 6, 2019, Orlando, FL.
    5. Alpha Thalassemia Major Trial Update. Cooley's Anemia Foundation, Medical Advisory Board Luncheon. Dec. 8, 2019, Orlando, FL.
    6. Sickle Cell Disease: Diagnoses, Screening and Treatment. Sickle Cell Peer Exchange, MDMagazine HCPLive Educational Event, 61st Annual ASH Meeting and Exposition; Dec 8, 2019; Orlando, FL.

2020

  • Fung EB.
    1. Bone Health in Thalassemia. ECHO Webinar for Western States CareProviders of Patients with Thalassemia; March 24, 2020.
  • Michlitsch J.
    1. Phase 1 Trial of Pazopanib in Combination with Irinotecan and Temozolomide (PAZIT) for Children and Young Adults with Advanced Sarcoma. ASCO annual meeting. May 29-31, 2020. Selected for poster discussion.
  • Saba J.
    1. Gordon Conference on Glycolipid and Sphingolipid Biology: A Holistic Approach to Understanding Simple and Complex Sphingolipids. Session Chair; Luca, Italy; March 22-27, 2020.
    2. University of Missouri Graduate Program in Molecular Pathogenesis and Therapeutics. Speaker; Columbia, MO; March 18, 2020.
    3. Keystone Symposia on Lipidomics of Health and Disease. Speaker; Whistler, BC, Canada; March 22-26, 2020.
  • Treadwell M.
    1. Transforming Sickle Cell Disease Care in the U.S. Vision and Priorities for the Next Decade. Presentation on behalf of the HRSA Sickle Cell Disease Treatment Demonstration Regional Collaborative Program Regional Leads; Feb 5, 2020. Briefing with Admiral Brett Giroir (HHS/OASH), Washington DC.
    2. Adverse Childhood Experiences: Relation to Clinical and Patient Reported Outcomes for Adults with Sickle Cell Disease. Presentation; Feb 26, 2020; NHLBI Sickle Cell Disease Implementation Consortium Steering Committee Meeting. Bethesda, MD.
    3. Sickle Cell + COVID-19 + Mental Health. Interview; March 26, 2020 The Sickle Cell Podcast, Sickle Cell 101.
    4. Mental health across the lifecourse in sickle cell disease. Presentation; April 28, 2020; Education and Mentoring to BRing Access to CarE for SCD (EMBRACE) Sickle Cell Project ECHO Telementoring.
    5. Cultivating Calm in the Midst of COVID-19. Presentation; May 5, 2020, Sickle Cell Disease Association of America Webinar.
  • Vichinsky E.
    1. Postnatal Management of Alpha Thalassemia Major: Improving Comprehensive Medical Care for Thalassemia; Project ECHO (Extension for Community Healthcare Outcomes); Thalassemia Western Consortium; Oakland, CA; March 26, 2020.
    2. Historical Timeline; World Sickle Cell Day podcast; Cayenne Wellness Center; June 19, 2020.
    3. The Brain: From Silent Infarcts to Dementia. Sickle Cell Boot Camp, UCSF Benioff Children's Hospital Oakland, Oct 2020.
    4. Emerging Therapies in Hemoglobinopathies. Frontiers in Medicine Core Curriculum Inquiry Series, UCSF, Nov. 23, 2020.
  • Walters M.
    1. Invited plenary speaker, Saudi Society for Blood Disorders, Riyadh, KSA.
    2. Invited talk; Feb 2020, BMT-CTN steering committee meeting.
    3. Invited speaker, Frontiers in Child Health Research, UCSF Feb 2020
    4. Invited Speaker, Fanconi Anemia Research Fund webinar, May 2020
    5. Chair, NHLBI Sickle Cell Advisory Committee Meeting, June 2020
    6. EBMT gene therapy for hemoglobin disorder. Invited plenary speaker, Invited Faculty; June 2020, Leiden, Netherlands. Saudi Society for Blood Disorders, Riyadh, KSA.

2021

  • Saba J.
    1. Detection and treatment of sphingosine phosphate lyase insufficiency syndrome. Presentation; July25-30, 2021, Gordon Conference on Molecular and Cellular Biology of Lipids, Waterville Valley NH.
  • Treadwell M.
    1. Online presentation/panel discussion; Women Leaders in the DEI Movement; UCSF Health Equity Council. March 12, 2021.
  • Vichinsky E.
    1. Advances in Alpha Thalassemia Major: No Longer a Fatal Disease Postnatal transfusion protocols, UCSF Center for Maternal-Fetal Precision Medicine, CIRM, CAF, TIF. Jan 8-9, 2021
    2. Vichinsky E. Advances in management of SCD: Agents on the Horizon. Virtual webinar; May 8, 2021; New Paradigms in the Management of Hemoglobinopathies: 2021 Update.
  • Walters M.
    1. "Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy (GT): Updated Results in Group C Patients from the Phase 1/2 Hgb-206 Study". TCT Meetings of ASTCT and CIBMTR. Feb 2021
    2. "Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent Beta-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy". TCT Meetings of ASTCT and CIBMTR. Feb 2021
    3. Town Hall Briefing: "Gene Therapy and Sickle Cell Disease". Feb 24, 2021, Cayenne Wellness Center and Children's Foundation. URL

PATENTS

  • Kirgiz I, Calloway C. Apparatus and Method for Collecting Touch DNA Using FTA Material. Serial Number: PCT/US2017/051650. University of California, Davis; (UC-2016-191-2FP). PCT International Patent Application; international Filing date: Sept 14, 2017. Approved for Phase II; 2/22/2018. Patent number 10,431,330 issued 10/1/2019.
  • Lal A, Robertson S, Vichinsky E. Method and System to Provide Patient Information and Facilitate Care of a Patient. Serial Number: PCT/US2017/051650. University of California, Davis; (UC-2016-191-2FP). PCT International Patent Application; international Filing date: Sept 14, 2017. Approved for Phase II; 2/22/2018. Patent number 10,431,330 issued 10/1/2019.

Profiles:

Back to Top

MEDIA:

2021

  1. Postnatal transfusion protocols. Video presentation by Dr. Elliott Vichinsky, Advances in Alpha Thalassemia Major: No Longer a Fatal Disease, UCSF Center for Maternal Fetal Medicine, Jan. 9, 2021.
  2. Emerging Therapies in the Management of Sickle Cell Disease:HCP Live, Jan. 21, 2021. Video presentations featuring Dr. Elliott Vichinsky.
    Episode 1: Sickle Cell Disease Prevalence and Pathophysiology.
    Episode 2: Complications of Sickle Cell Disease.
    Episode 3: Sickle Cell Disease QoL and Life Expectancy.
  3. UC Consortium Launches First Clinical Trial Using CRISPR to Correct Gene Defect That Causes Sickle Cell Disease. By Lorna Fernandes, UCSF News, March 30, 2021.
  4. Surviving with Joy. The journey of 5 families with Alpha Thalassemia Major. UCSF Fetal Treatment Center; March 2021.
  5. UC consortium launches first clinical trial using CRISPR to correct sickle cell gene. EurekAlert!, AAAS; March 30, 2021.
  6. First CRISPR Trial for Patients with Severe Sickle Cell Disease to Begin. ASH Clinical News, April 11, 2021.

2020

  1. Overview of Sickle Cell Disease. HCP Live Network, Jan. 20, 2020. Dr. Vichinsky - panel member.
  2. Clinical Presentations Associated With Sickle Cell Disease. HCP Live Network, Jan. 22, 2020. Dr. Vichinsky - panel member.
  3. NHLBI Guidelines for the Screening and Diagnosis of SCD. HCP Live Network, Jan 27, 2020. Dr. Vichinsky - panel member.
  4. Early Interventions for the Treatment of Sickle Cell Disease. HCP Live Network, Jan 29, 2020. Dr. Vichinsky - panel member.
  5. Blood Transfusion Therapy to Treat Sickle Cell Disease. HCP Live Network, Feb 3, 2020. Dr. Vichinsky - panel member.
  6. Hematopoietic Stem Cell Transplant in SCD. HCP Live Network, Feb 5, 2020. Dr. Vichinsky - panel member.
  7. OSUSTAIN Trial: Crizanlizumab in Reducing Painful Crisis. HCP Live Network, Feb 10, 2020. Dr. Vichinsky - panel member.
  8. Clinical Implications of Using Voxelotor To Treat Sickle Cell Disease. HCP Live Network, Feb 17, 2020. Dr. Vichinsky - panel member.
  9. Mom helps infant daughter ring bell after beating brain cancer. By Kelly Taylor Hayes, FOX TV, Jan. 29, 2020.
  10. ASH 2020 Clinical Guidelines: Sickle Cell Disease and Cerebrovascular Disease . By Caleb Rans, PharmD; Hematology Advisor, May 20, 2020.
  11. Christopher Newton and Elliott Vichinsky Honored as Endowed Physicians by UCSF Benioff Children's Hospital Oakland. By Richard Barg; June 26, 2020.
  12. Elliott Vichinsky, MD: Voxelotor and New HOPE Data for Sickle Cell. DOC TALK Podcast; by Kevin Kunzmann. HCP Live, Dec. 8, 2020.
  13. Cheat Codes -- A Sickle Cell Podcast: Sickle Cell Folklore with Elliott Vichinsky MD. Bloodstream Media, Episode 25; December 22, 2020.

2019

  1. Sickle Cell Disease Forum: Innovation in Drug Development and Emerging Science. HemOnc Today Education Lab, Jan.29, 2019.
  2. CIRM and NHLBI Collaborating to Fund Cell and Gene Therapies for Sickle Cell Disease. By Ana Pena, May 7, 2019. Sickle Cell Anemia News
  3. Deferiprone trial update. 10th Annual International NBIA Disorders Association Family Conference. Deerfield, IL. May 30, 2019 (video of presentation).
  4. Scientists Slow Progression of Debilitating Neurodegenerative Disorder: Chelation Study on PKAN Has Implications for Treating Alzheimer's, Parkinson's Disease and Multiple Sclerosis. By Lorna Fernandes, UCSF News, June 13, 2019.
  5. Voxelotor increases hemoglobin levels, reduces hemolysis markers in sickle cell disease. Healio, Hem Onc Today, June 14, 2019.
  6. Patients with Sickle Cell Increase Healthy Blood Cells in New Drug Trial: Phase 3 HOPE Trial Finds Improved Anemia, a Major Risk Factor for Organ Failure and Early Death. By Lorna Fernandes. UCSF News, June 17, 2019.
  7. German & Oakland-Based Researchers Slow Progression of Pantothenate Kinase-Associated Neurodegeneration. Trial Site News; June 17, 2019.
  8. Deferiprone Trial Update. 2019 NBIA Conference; Presentation by Dr. Elliott Vichinsky, Jun 27, 2019.
  9. Deferiprone trial results produce positive findings for some with PKAN. NBIA Disorders Association, August 2019.
  10. "Sickle Cell". The Tote and Pears Podcast; Season2: Episode 17, August 2019. Patient Cameron's story of receiving a sibling-donor bone marrow transplant at BCHO.
  11. Voxelotor Increases Hemoglobin, Decreases Hemolysis in Patients with Sickle Cell Disease. ASH Clinical News. August 1, 2019
  12. A new sickle cell drug could soon get U.S. approval. But does it work?. By Meredith Wadman, Science Magazine, Sept. 17, 2019.
  13. Hope, frustration mark new era of sickle cell disease. By Erin Allday, San Francisco Chronicle, Sept. 23, 2019.
  14. Tippi, Elliott and Elianna's Story: Little Girl Thrives After Fetal Stem Cell Transplant. UCSF Foundation, Sept 23, 2019.
  15. Young dreamer's early vision still influences his field: At a memorial symposium for blood geneticist George Stamatoyannopoulos, speakers told how he helped set their course in research.. UW Medicine Newsroom, Sept 26, 2019.
  16. Sickle Cell Drug Raises Hopes And Doubts: FDA Grants Speedy Review of Eagerly Awaited Treatment, Despite Unproven Clinical Benefit. By Meredith Wadman, Science, Sept 20, 2019.
  17. What Does the Future Hold for Gene Therapy in Nonmalignant Hematology? ASH Clinical News, Oct. 2019
  18. Update: FDA Issues Speedy Approval of Innovative Sickle Cell Drug. By Meredith Wadman, Science, Nov. 26, 2019
  19. Moving From Science Fiction to Clinical Reality: Gene Therapy for Beta-Hemoglobinopathies. CME presentation by Dr. Elliott Vichinsky, 12/23/2019.

2018

  1. The Bay Area's Top Doctors of 2018 (includes Feusner J, Vichinsky E). San Francisco Magazine, 1/11/2018.
  2. Reno twins with rare disease at center of a multimillion-dollar lawsuit against pharmaceutical giants: . By Siobhan McAndrew, Reno Gazette Journal, Jan 12, 2018. (Story about the Niemann-Pick twins cared for by Dr. Caroline Hastings).
  3. 48 Hours: Theo's Story. Children's Hospital Oakland, Vimeo, 1/30/2018. (Diagnosis of a child with T-cell leukemia, featuring Dr. Holly Paiury and Dr. Caroline Hastings and a multi-specialty team of physicians)
  4. Certificate of Excellence awarded to Dr. Carla Golden and research staff for highest NCORP enrolling institution, Children's Oncology Group (COG) NCORP Steering Committee, 4/11/2018.
  5. How being a parent to a child with cancer changes your life. PBS News Hour, May 17, 2018. (Interview with parents of a child with rhabdomyosarcoma treated at BCHO)
  6. UCSF Welcomes In-Utero Stem Cell Transplant Baby: Fetal Treatment May One Day Cure Multiple Disorders Months Before Birth. By Suzanne Leigh, UCSF News Center, 5/25/2018. (The first patient enrolled in the world's first clinical trial of stem cell transplant to a fetus with alpha thalassemia major -- at UCSF Benioff Children's Hospitals)
  7. Five Blood Transfusions, One Bone Marrow Transplant -- All Before Birth. By Denise Grady, The New York Times, 5/25/2018.
  8. Ebb in flow: Sickle cell disease draws multiple challenges to the surface. CMA Today, May/June 2018. (Dr. Elliott Vichinsky was interviewed for this article.)
  9. Baby born in world's first in utero stem cell transplant trial. By Suzanne Leigh, UCSF News Center, 6/4/2018.
  10. Lifeline in the womb. By Denise Grady, The Statesman, June 5, 2018. (More about the Alpha Thalassemia major baby, featuring Dr. Elliott Vichinsky and Dr. Tippi MacKenzie).
  11. Ebb in flow: Sickle cell disease draws multiple challenges to the surface. CMA Today, May/June 2018. (Dr. Elliott Vichinsky was interviewed for this article.)
  12. In recognition of World Sickle Cell Day, the CDC released videos on transition from pediatric to adult care featuring young adults with sickle cell disease and staff from the Northern California Comprehensive Sickle Cell Center:
  13. Toward a Cure for Sickle Cell: How Doctors Are Fighting a Crippling Disease. By Mitzi Baker, UCSF News Center, 6/20/2018. (Dr. Mark Walters and colleagues at UCSF Benioff Children's Hospitals -- at the leading edge of advancements in bone marrow transplantation, gene therapy and cutting-edge CRISPR-Cas9 gene editing technology to cure sickle cell disease.)
  14. Nutrition bar improves lung function in overweight, obese teens with asthma. Healio.com. (Dr. Lal et al., the 'CHORI bar') July 20, 2018.
  15. The Realness: The Untold Story of Albert "Prodigy" Johnson, Episode 4. WNYC Studios, July 17, 2018. (Audio documentary about a rapper with sickle cell disease. E. Vichinsky was consulting expert).
  16. The Realness: The Untold Story of Albert "Prodigy" Johnson, Episode 5. WNYC Studios, July 30, 2018. (Audio documentary about a rapper with sickle cell disease. E. Vichinsky was consulting expert).
  17. What's Wrong With The Twins?. Season Two of HLN's "Something's Killing Me" with BD Wong. Airdate: Aug 19, 2018 on CNN at 8pm ET/PT. This episode will feature Dr. Caroline Hastings and her patients with Niemann-Pick Type C Disease.
  18. Sickle Cell: The Last Health-Care Frontier for Black Lives. by Momo Chang, East Bay Express, Sep 12, 2018.
  19. Children's Hospital Oakland at forefront of sickle cell treatment, research. By Jon Kawamoto, Bay Area News Group; 9/17/2018.
  20. Children's Hospital Oakland at forefront of sickle cell treatment, research: UCSF Benioff Children's Hospital Oakland, Vallejo family work as team for 11-year-old boy's treatment . By Jon Kawamoto, Bay Area News Group. The Mercury News; Sept. 17, 2018.
  21. UCSF Doctors Work to Eliminate Sickle Cell Disease. UCSF Benioff Children's Hospital Oakland, 11/21/2018.

RECENT FACULTY AWARDS AND HONORS

DATEFACULTY NAMEINSTITUTION AWARD NAME
2021Nura El-Haj, MDASPHOEarly Career Award
2021Julie Saba, MDUCSFCatalyst Award for gene therapy to treat children with SPL insufficiency syndrome
2020Caroline Hastings, MDUCSF Benioff Children's Hospital OaklandCareer Achievement Award
2020Frans Kuypers, PhDUCSF Benioff Children's Hospital OaklandScientific Achievement Award
2020Lynne Neumayr, MDUCSF Benioff Children's Hospital OaklandScientific Achievement Award
2020Mark Walters, MDCIBMTRBest Abstract Award, Feb 2020 Transplantation and Cellular Therapy Meetings of ASTCT & CIBMTR
2020Elliott Vichinsky, MDUCSFEndowed Clinical Care Professorship
2019Anu Agrawal, MD UCSF President's Innovation Fund (PIF) Understanding the Benefit of Integrative Modalities for Complex Ped/Young Adult Hem Onc Patients $67,200
2019BCHO, represented by Carla Golden, MD NCI Community Oncology Research Program (NCORP) Highest Enrolling NCORP Institution for 2019
2019 Carla Golden, MD NCI Community Oncology Research Program (NCORP) 2019 BATI-NCORP Co-Principal Investigator Highest Accruing NCORP Physician to LTE studies
2019 Shannon Kelly, MD UCSF President's Innovation Fund (PIF) Implementation of Ultrasound-Guided Peripheral IV Access in the Day Hospital and Apheresis $65,000
2019 Lynne Neumayr, MD UCSF Benioff Children's Hospital Oakland Career Achievement Award 2019
2019 Cheryl Peretz, MD ASH American Society of Hematology Ahstract Achievement Award
2019 Sylvia Titi Singer, MD UCSF President's Innovation Fund (PIF) Next-Generation Sequencing for Targeted Molecular Screening and Clinical Genotyping in Hemoglobinopathies $75,000
2019 Marsha Treadwell, PhD Representative Barbara Lee, 13th Congressional District Certificate of Special Congressional Recognition
2019 Elliott Vichinsky, MD American Society of Pediatric Hematology/Oncology (ASPHO) Distinguished Career Award
2019 Elliott Vichinsky, MD UCSF Division Chief Incentive Award
2018 Marsha Treadwell, PhD The John Jordon Foundation Jordan Fund Endowed Chair
2018 Mark Walters, MD UCSF Benioff Children's Hospital Oakland Career Achievement Award

updated 5/4/2021


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