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


Recent Faculty Publications (2017 - 2018)

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

PEER-REVIEWED ARTICLES:

  1. Abla O, Ribeiro RC, Testi AM, Montesinos P, Creutzig U, Sung L, Di Giuseppe G, Stephens D, Feusner JH, Powell BL, Hasle H, Kaspers GJL, Dalla-Pozza L, Lassaletta A, Tallman MS, Locatelli F, Reinhardt D, Lo-Coco F, Hitzler J, Sanz MA. Predictors of thrombohemorrhagic early death in children and adolescents with t(15;17)-positive acute promyelocytic leukemia treated with ATRA and chemotherapy. Ann Hematol 2017;96 9:1449-56. URL
  2. Abraham A, Hsieh M, Eapen M, Fitzhugh C, Carreras J, Keesler D, Guilcher G, Kamani N, Walters MC, Boelens JJ, Tisdale J, Shenoy S. National Institutes of Health; Center for International Blood Marrow Transplant Research Relationship between Mixed Donor-Recipient Chimerism and Disease Recurrence after Hematopoietic Cell Transplantation for Sickle Cell Disease. Biol Blood Marrow Transplant 2017;23 12:2178-83. PMC5782809 URL
  3. Agrawal AK, Robertson S, Litwin L, Tringale E, Treadwell M, Hoppe C, Marsh A. Immersive virtual reality as a complementary pain therapy in hospitalized pediatric patients with sickle cell disease. 2018 Pediatr Blood Cancer (SUBMITTED).
  4. 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
  5. Arnold SD, Brazauskas R, He N, Li Y, Aplenc R, Jin Z, Hall M, Atsuta Y, Dalal J, Hahn T, Khera N, Bonfim C, Majhail NS, Diaz MA, Freytes CO, Wood WA, Savani BM, Kamble RT, Parsons S, Ahmed I, Sullivan K, Beattie S, Dandoy C, Munker R, Marino S, Bitan M, Abdel-Azim H, Aljurf M, Olsson RF, Joshi S, Buchbinder D, Eckrich MJ, Hashmi S, Lazarus H, Marks DI, Steinberg A, Saad A, Gergis U, Krishnamurti L, Abraham A, Rangarajan HG, Walters M, Lipscomb J, Saber W, Satwani P. Clinical risks and healthcare utilization of haematopoietic cell transplantation for sickle cell disease in the U.S. using merged databases. Haematologica Nov. 2017. URL
  6. Badawy SM, Black V, Meier ER, Myers KC, Pinkney K, Hastings C, Hilden JM, Zweidler-McKay P, Stork LC, Johnson TS, Vaiselbuh SR. Early career mentoring through the American Society of Pediatric Hematology/Oncology: Lessons learned from a pilot program. Pediatr Blood Cancer 2017;64 3. PMC5685518 URL
  7. 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; Epub ahead of print. Sep 11. URL.
  8. 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
  9. 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
  10. 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.
  11. Cairo MS, Talano J, Moore TB, Shi Q, Keever-Taylor C, Weinberg R, Grossman B, Walters MC, Vichinsky E, Parsons SK, Braniecki S, Dozor AJ, Friedman D, McKinstry R, Verbsky J, Moorthy CR, Ayello J, Semidei-Pomales M, Flower A, Chu YM, E. , Mahanti H, Fabricatore S, Militano O, Klejmont L, van de Ven C, Baxter-Lowe LA, Shenoy S. Familial Haploidentical Stem Cell Transplantation in Patients with Sickle Cell Disease. N Engl J Med 2018 (SUBMITTED).
  12. Carlberg K, Bose N, Lal A, Erlich H, Calloway C. Noninvasive Prenatal Test for β-thalassemia and Sickle Cell Disease using Probe Capture Enrichment and Next Generation Sequencing of Cell-Free DNA in Maternal Plasma. PLOS One 2018 SUBMITTED.
  13. Chung JE, Magis W, Vu J, Heo S-J, Wartiovaara K, Walters MC, Kurita R, Nakamura Y, Boffelli D, Martin DI, Corn JE, Dewitt MA. CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells. bioRxiv (pre-print; May 31, 2018). URL.
  14. Cooney T, Wei MC, Rangaswami A, Xu L, Sage J, Hazard FK. CD47 is not Over-Expressed in Fibrolamellar Hepatocellular Carcinoma. Ann Clin Lab Sci 2017;47 4:395-402. URL
  15. Cooney T, Lane A, Bartels U, Bouffet E, Goldman S, Leary SES, Foreman NK, Packer RJ, Broniscer A, Minturn JE, Shih CS, Chintagumpala M, Hassall T, Gottardo NG, Dholaria H, Hoffman L, Chaney B, Baugh J, Doughman R, Leach JL, Jones BV, Fouladi M, Warren KE, Monje M. Contemporary survival endpoints: an International Diffuse Intrinsic Pontine Glioma Registry study. Neuro Oncol 2017;19 9:1279-80. PMC5570207 URL
  16. 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.
  17. 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
  18. 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;2 15:1969-79. URL
  19. Dietz AC, Duncan CN, Alter BP, Bresters D, Cowan MJ, Notarangelo L, Rosenberg PS, Shenoy S, Skinner R, Walters MC, Wagner J, Baker KS, Pulsipher MA. The Second Pediatric Blood and Marrow Transplant Consortium International Consensus Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation: Defining the Unique Late Effects of Children Undergoing Hematopoietic Cell Transplantation for Immune Deficiencies, Inherited Marrow Failure Disorders, and Hemoglobinopathies. Biol Blood Marrow Transplant 2017;23 1:24-9. PMC5267609 URL
  20. Dowling MM, Quinn CT, Ramaciotti C, Kanter J, Osunkwo I, Inusa B, Iyer R, Kwiatkowski JL, Johnson C, Rhodes M, Owen W, Strouse JJ, Panepinto JA, Neumayr L, Sarnaik S, Plumb PA, Dlamini N, Kirkham F, Hynan LS, PFAST Investigators. Increased prevalence of potential right-to-left shunting in children with sickle cell anaemia and stroke. Br J Haematol 2017;176 2:300-8. PMC5239723 URL
  21. 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 2017:JGO1700054. URL
  22. Fearon A, Marsh A, Kim J, Treadwell M. Pediatric residents' perceived barriers to opioid use in sickle cell pain management. 2018 Pediatr Blood Cancer (SUBMITTED).
  23. Field JJ, Majerus E, Ataga KI, Vichinsky EP, Schaub R, Mashal R, Nathan DG. NNKTT120, an anti-iNKT cell monoclonal antibody, produces rapid and sustained iNKT cell depletion in adults with sickle cell disease. PLoS One 2017;12 2:e0171067. PMC5289534 URL
  24. Field JJ, Majerus E, Gordeuk VR, Gowhari M, Hoppe C, Heeney MM, Achebe M, George A, Chu H, Sheehan B, Puligandla M, Neuberg D, Lin G, Linden J, Nathan DG. Randomized phase 2 trial of regadenoson for treatment of acute vaso-occlusive crises in sickle cell disease. Blood Adv 2017;1 20:1645-9.PMC5728341. URL
  25. Fitzhugh CD, Walters MC. The case for HLA-identical sibling hematopoietic stem cell transplantation in children with symptomatic sickle cell anemia The case for HLA-identical sibling hematopoietic stem cell transplantation in children with symptomatic sickle cell anemia. Blood Adv 2017;1 26:2563-7. PMC5728630 interests. URL
  26. 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
  27. Ganz T, Jung G, Naeim A, Ginzburg Y, Pakbaz Z, Walter PB, Kautz L, Nemeth E. Immunoassay for human serum erythroferrone. Blood 2017;130 10:1243-6. URL
  28. Gluckman E, Cappelli B, Bernaudin F, Labopin M, Volt F, Carreras J, Pinto Simoes B, Ferster A, Dupont S, de la Fuente J, Dalle JH, Zecca M, Walters MC, Krishnamurti L, Bhatia M, Leung K, Yanik G, Kurtzberg J, Dhedin N, Kuentz M, Michel G, Apperley J, Lutz P, Neven B, Bertrand Y, Vannier JP, Ayas M, Cavazzana M, Matthes-Martin S, Rocha V, Elayoubi H, Kenzey C, Bader P, Locatelli F, Ruggeri A, Eapen M. Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood 2017;129 11:1548-56. PMC5356458 URL
  29. Hahn C, Tyka K, Saba JD, Lenzen S, Gurgul-Convey E. Overexpression of sphingosine-1-phosphate lyase protects insulin-secreting cells against cytokine toxicity. J Biol Chem 2017;292 49:20292-304. PMC5724014 URL
  30. Harmatz PR, Mengel E, Geberhiwot T, Muschol N, Hendriksz CJ, Burton BK, Jameson E, Berger KI, Jester A, Treadwell M, Sisic Z, Decker C. Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open-label, phase 2 study. Am J Med Genet A 2017;173 2:375-83. PMC5298029 URL
  31. Heath LE, Heeney MM, Hoppe CC, Adjei S, Agbenyega T, Badr M, Masera N, Zhou C, Brown PB, Jakubowski JA, Dampier C. Successful utilization of an electronic pain diary in a multinational phase 3 interventional study of pediatric sickle cell anemia. Clin Trials 2017:1740774517723307. URL
  32. 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 June 13, 2018. URL.
  33. Hoppe C, Jacob E, Styles L, Kuypers F, Larkin S, Vichinsky E. Simvastatin reduces vaso-occlusive pain in sickle cell anaemia: a pilot efficacy trial. Br J Haematol 2017;177 4:620-9. PMC5435522 URL
  34. 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
  35. Ikon N, Shearer J, Liu J, Tran JJ, Feng S, Kamei A, Beckstead JA, Kiss RS, Weers PM, Ren G, Ryan RO. A facile method for isolation of recombinant human apolipoprotein A-I from E. coli. Protein Expr Purif 2017;134:18-24. PMC5479713 URL
  36. Jakubowski JA, Hoppe CC, Zhou C, Smith BE, Brown PB, Heath LE, Inusa B, Rees DC, Small DS, Gupta N, Yao S, Heeney MM, Kanter J. Real-time dose adjustment using point-of-care platelet reactivity testing in a double-blind study of prasugrel in children with sickle cell anaemia. Thromb Haemost 2017;117 03:580-8. URL
  37. 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.
  38. 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
  39. 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
  40. Keller S, Yang M, Treadwell MJ, Hassell KL. Sensitivity of alternative measures of functioning and wellbeing for adults with sickle cell disease: comparison of PROMIS(R) to ASCQ-Me. Health Qual Life Outcomes 2017;15 1:117. PMC5455105 URL
  41. Kesselheim JC, Agrawal AK, Bhatia N, Cronin A, Jubran R, Kent P, Kersun L, Rao AN, Rose M, Savelli S, Sharma M, Shereck E, Twist CJ, Wang M. Measuring pediatric hematology-oncology fellows' skills in humanism and professionalism: A novel assessment instrument. Pediatr Blood Cancer 2017;64 5. URL
  42. Khurana M, Fung EB, Vichinsky EP, Theil EC. Dietary nonheme iron is equally bioavailable from ferritin or ferrous sulfate in thalassemia intermedia. Pediatr Hematol Oncol 2017;34 8:455-67. URL
  43. Kirgiz IA, Calloway C. Increased recovery of touch DNA evidence using FTA paper compared to conventional collection methods. J Forensic Leg Med 2017;47:9-15. URL.
  44. Kline CN, Joseph NM, Grenert JP, van Ziffle J, Talevich E, Onodera C, Aboian M, Cha S, Raleigh DR, Braunstein S, Torkildson J, Samuel D, Bloomer M, Campomanes AGA, Banerjee A, Butowski N, Raffel C, Tihan T, Bollen AW, Phillips JJ, Korn WM, Yeh I, Bastian BC, Gupta N, Mueller S, Perry A, Nicolaides T, Solomon DA. Targeted next-generation sequencing of pediatric neuro-oncology patients improves diagnosis, identifies pathogenic germline mutations, and directs targeted therapy. Neuro Oncol 2017;19 5:699-709. PMC5464451 URL
  45. Klopstock T, Tricta F, Neumayr L, Karin I, Zorzi G, Fradette C, Kmiec T, Büchner B, Steele HE, Horvath R, Chinnery PF, Basu A, Küpper 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. Lancet 2018 SUBMITTED.
  46. Krishnamoorthy A, Tavoosi N, Chan GKL, Liu J, Ren G, Cavigiolio G, Ryan RO. Effect of curcumin on amyloid-like aggregates generated from methionine-oxidized apolipoprotein A-I. FEBS Open Bio 2018;8 2:302-10. PMC5794470 URL
  47. Krishnamoorthy A, Witkowski A, Ryan RO. Nutlin-3a Nanodisks Induce p53 Stabilization and Apoptosis in a Subset of Cultured Glioblastoma Cells. J Nanomed Nanotechnol 2017;8 4. PMC5619870 URL
  48. Kumar A, Zamora-Pineda J, Degagne E, Saba JD. S1P Lyase Regulation of Thymic Egress and Oncogenic Inflammatory Signaling. Mediators Inflamm 2017;2017:7685142. PMC5733215 URL
  49. Kutny MA, Alonzo TA, Gerbing RB, Wang YC, Raimondi SC, Hirsch BA, Fu CH, Meshinchi S, Gamis AS, Feusner JH, Gregory JJ, Jr. Arsenic Trioxide Consolidation Allows Anthracycline Dose Reduction for Pediatric Patients With Acute Promyelocytic Leukemia: Report From the Children's Oncology Group Phase III Historically Controlled Trial AAML0631. J Clin Oncol 2017;35 26:3021-9. URL
  50. Lal A, Wong T, Andrews J, Balasa V, Chung J, Forester C, Ikeda A, Keel S, Pagano M, Puthenveethil G, Shah S, Yu J, Vichinsky E. Transfusion practices and complications in thalassemia. Transfusion 2018 (ACCEPTED).
  51. 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;65 2. URL
  52. Lovric S, Goncalves S, Gee HY, Oskouian B, Srinivas H, Choi WI, Shril S, Ashraf S, Tan W, Rao J, Airik M, Schapiro D, Braun DA, Sadowski CE, Widmeier E, Jobst-Schwan T, Schmidt JM, Girik V, Capitani G, Suh JH, Lachaussee N, Arrondel C, Patat J, Gribouval O, Furlano M, Boyer O, Schmitt A, Vuiblet V, Hashmi S, Wilcken R, Bernier FP, Innes AM, Parboosingh JS, Lamont RE, Midgley JP, Wright N, Majewski J, Zenker M, Schaefer F, Kuss N, Greil J, Giese T, Schwarz K, Catheline V, Schanze D, Franke I, Sznajer Y, Truant AS, Adams B, Desir J, Biemann R, Pei Y, Ars E, Lloberas N, Madrid A, Dharnidharka VR, Connolly AM, Willing MC, Cooper MA, Lifton RP, Simons M, Riezman H, Antignac C, Saba JD, Hildebrandt F. Mutations in sphingosine-1-phosphate lyase cause nephrosis with ichthyosis and adrenal insufficiency. J Clin Invest 2017;127 3:912-28. PMC5330730 URL
  53. McGann PT, Hoppe C. The pressing need for point-of-care diagnostics for sickle cell disease: A review of current and future technologies. Blood Cells Mol Dis 2017. URL
  54. Michlitsch JG, Romao RLP, Gleason JM, Braga LH, Allen L, Gupta A, Lorenzo AJ. Local control for vaginal botryoid rhabdomyosarcoma with prerectal transperineal surgical resection and autologous buccal graft vaginal replacement: A novel, minimally invasive, radiation-sparing approach. J Pediatr Surg 2017. URL
  55. Minniti CP, Vichinsky E. Lifespan care in SCD: Whom to transition, the patients or the health care system? Am J Hematol 2017;92 6:487-9. URL
  56. Mitroi DN, Karunakaran I, Graler M, Saba JD, Ehninger D, Ledesma MD, van Echten-Deckert G. SGPL1 (sphingosine phosphate lyase 1) modulates neuronal autophagy via phosphatidylethanolamine production. Autophagy 2017;13 5:885-99. PMC5446076 URL
  57. Morshed RA, Lau D, Sun PP, Ostling LR. Spinal drop metastasis from a benign fourth ventricular choroid plexus papilloma in a pediatric patient: case report. J Neurosurg Pediatr 2017;20 5:471-9. URL
  58. 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
  59. 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.
  60. 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
  61. Paulukonis ST, Feuchtbaum LB, Coates TD, Neumayr LD, Treadwell MJ, Vichinsky EP, Hulihan MM. Emergency department utilization by Californians with sickle cell disease, 2005-2014. Pediatr Blood Cancer 2017;64 6. PMC5403550 URL
  62. Raleigh DR, Tomlin B, Buono BD, Roddy E, Sear K, Byer L, Felton E, Banerjee A, Torkildson J, Samuel D, Horn B, Braunstein SE, Haas-Kogan DA, Mueller S. Survival after chemotherapy and stem cell transplant followed by delayed craniospinal irradiation is comparable to upfront craniospinal irradiation in pediatric embryonal brain tumor patients. J Neurooncol 2017;131 2:359-68. URL
  63. Rivera A, Vandorpe DH, Shmukler BE, Gallagher DR, Fikry CC, Kuypers FA, Brugnara C, Snyder LM, Alper SL. Erythrocytes from hereditary xerocytosis patients heterozygous for KCNN4 V282M exhibit increased spontaneous Gardos channel-like activity inhibited by senicapoc. Am J Hematol 2017;92 6:E108-E10. URL
  64. Romero-Garcia Z, DeWitt M, Walters MC. Promise of gene therapy to treat sickle cell disease. Expert Onin Biol Ther 2018 SUBMITTED.
  65. Saba J, Logan AC. Obinutuzumab-induced serum sickness following salvage therapy for chronic lymphocytic leukemia. Clin Case Rep 2017;5 6:891-3. PMC5457998 URL
  66. 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
  67. 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
  68. Sharma SD, Fischer R, Schoennagel BP, Nielsen P, Kooijman H, Yamamura J, Adam G, Bannas P, Hernando D, Reeder SB. MRI-based quantitative susceptibility mapping (QSM) and R2* mapping of liver iron overload: Comparison with SQUID-based biomagnetic liver susceptometry. Magn Reson Med 2017;78 1:264-70. PMC5303570 URL
  69. Shenoy S, Angelucci E, Arnold SD, Baker KS, Bhatia M, Bresters D, Dietz AC, De La Fuente J, Duncan C, Gaziev J, King AA, Pulsipher MA, Smith AR, Walters MC. Current Results and Future Research Priorities in Late Effects after Hematopoietic Stem Cell Transplantation for Children with Sickle Cell Disease and Thalassemia: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant 2017;23 4:552-61. URL
  70. 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
  71. 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
  72. 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.
  73. 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 20184:1-9. URL
  74. Songdej D, Babbs C, Higgs DR, Singer T (collaborator), Vichinsky E (collaborator), BHFS International Consortium. An international registry of survivors with Hb Bart's hydrops fetalis syndrome. Blood 2017;129 10:1251-9. PMC5345731 URL
  75. Soupene E, Larkin SK, Kuypers FA. Depletion of HDL3 high density lipoprotein and altered functionality of HDL2 in blood from sickle cell patients. Exp Biol Med (Maywood) 2017:1535370217706966. PMC5476340 URL
  76. Suh JH, Makarova AM, Gomez JM, Paul LA, Saba JD. An LC/MS/MS method for quantitation of chemopreventive sphingadienes in food products and biological samples. J Chromatogr B Analyt Technol Biomed Life Sci 2017;1061-1062:292-9. URL
  77. Thakar MS, Bonfim C, Walters MC, Storb R, Pasquini R, Burroughs L, Sandmaier BM, Woolfrey A, Kiem HP. Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia. Bone Marrow Transplant 2017;52 4:570-3. PMC5382060 URL
  78. 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
  79. Tong S, Vichinsky E. Iron Deficiency: the implications before anemia. Pediatrics in Review 2018 SUBMITTED.
  80. Treadwell MJ, Makani J, Ohene-Frempong K, Ofori-Acquah S, McCurdy S, de Vries J, Bukini D, Dennis-Antwi J, Kamga Karen K, Mbekenga C, Wonkam ET, Tangwa G, Royal CD, Wonkam A, and members of the H3Africa Consortium. Stakeholder Perspectives on Public Health Genomics Applications for Sickle Cell Disease: A Methodology for a Human Heredity and Health in Africa (H3Africa) Qualitative Research Study. OMICS: A Journal of Integrative Biology 2017;21 6:323-32. URL.
  81. 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
  82. Trobaugh-Lotrario AD, Meyers RL, O'Neill AF, Feusner JH. Unresectable hepatoblastoma: current perspectives. Hepat Med 2017;9:1-6. PMC5293365 URL
  83. Vichinsky E. Chronic organ failure in adult sickle cell disease. Hematology Am Soc Hematol Educ Program 2017;2017 1:435-9. URL
  84. 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
  85. Vichinsky E, El-Beshlawy A, Al Zoebie A, Kamdem A, Koussa S, Chotsampancharoen T, Bruederle A, Gilotti G, Han J, Elalfy M. Long-term safety and efficacy of deferasirox in young pediatric patients with transfusional hemosiderosis: Results from a 5-year observational study (ENTRUST). Pediatr Blood Cancer 2017;64 9. URL
  86. Vohr SH, Gordon R, Eizenga JM, Erlich HA, Calloway CD, Green RE. A phylogenetic approach for haplotype analysis of sequence data from complex mitochondrial mixtures. Forensic Sci Int Genet 2017;30:93-105. URL.
  87. Wajcman H, Azimi M, Cui J, Hoppe C, Flamini M, Ho C, Reddy S. Hemoglobinopathy testing: the significance of accuracy and pitfalls in HbA2 determination. Int J Lab Hematol 2017;39 1:e23-e7. URL
  88. Walter PB, Minkley M, Vichinsky E, MacLeod P, Borchers C. Identification of novel biomarkers for patients with NBIA being treated with deferiprone: PKAN biomarker study. 2018 (SUBMITTED).
  89. Ward SL, Turpin A, Spicer AC, Treadwell MJ, Church GD, Flori HR. Long-Term Pulmonary Function and Quality of Life in Children After Acute Respiratory Distress Syndrome: A Feasibility Investigation. Pediatr Crit Care Med 2017;18 1:e48-e55. PMC5221949 URL
  90. Wehbe MS, Yamamura J, Fischer R, Grosse R, Berliner C, Graessner J, Lund G, Adam G, Schoennagel BP. Left atrial active contractile function parameters assessed by cardiac MR are sensitive to myocardial iron. J Magn Reson Imaging 2017;45 2:535-41. URL
  91. Winstead M, Sun PP, Martin K, Earl J, Neumayr L, Hoppe C, Vichinsky E. Encephaloduroarteriosynangiosis (EDAS) in young patients with cerebrovascular complications of sickle cell disease: Single-institution experience. Pediatr Hematol Oncol 2017;34 2:100-6. URL
  92. 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

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ABSTRACTS:

  1. 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.
  2. 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).
  3. 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.
  4. Calloway CD, Bose N, Gordon R, Taylor C, Shih S, Almada G, Sensagbaugh G, Erlich H. Mitochondrial genome and nuclear SNP probe capture next-generation sequencing system for analyzing degraded and mixed DNA samples. 28th Annual International Symposium on Human Identification (ISHI); poster presentation. Seattle, WA; Oct 4, 2017.
  5. 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.
  6. Carlberg K, Feusner JH. Hyperhydration alone in children with acute lymphoblastic leukemia (ALL) at very low risk for developing tumor lysis syndrome (TLS). ASPHO 30th Annual Meeting. Montreal, Quebec, Canada; April 26-29, 2017. Pediatric Blood & Cancer 64(S1); e26591, Poster #323. URL
  7. 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
  8. 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.
  9. Cooney T, Tran DC, Onar-Thomas A, Norris RE. Pediatric oncology phase 1 trial efficiency in the era of targeted and biological therapies. J Clin Oncol 35(15); e14100. URL.
  10. 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.
  11. 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) 2017 Meeting. San Francisco, CA; May 6-9, 2017.
  12. 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).
  13. 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 65(S1); e27057, #525. URL
  14. Fearon A, Marsh A, Kim J, Treadwell M. Pediatric residents' comfort level and perceived barriers toward the use of opioids in the treatment of severe vaso-occlusive pain episodes for patients with sickle cell disease. Rosalind Franklin Medical School Medical Student Research Symposium. Chicago, IL; Oct 18, 2017.
  15. Foe M, Treadwell MJ, Yamashita R, Lal A. Factors Related to Iron Chelation Therapy Adherence in Young Adults with Thalassemia: A Framework from the Patient Perspective. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 2080. URL
  16. Grosse R, Hainmann I, Berliner C, Weyhmiller M, Fung E, Schoennagel B, Yamamura J, Nielsen P, Fischer R. Pancreatic iron and fat, early indicators of impaired glucose metabolism? European Iron Club, Zurich, Switzerland, 2018; poster #49.
  17. 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.
  18. Hoppe CC, Jakubowski JA, Foster WM, Heath LE, Pillai S, Pallav B. Candidate Gene Associations with Vaso-Occlusive Crisis in Children with Sickle Cell Anemia Enrolled in the Multinational DOVE Trial. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 962 URL
  19. Hoppe CC, Inati AC, Brown C, Wang W, Gordeuk VR, Liem R, Woods G, Piccone CM, Fong E, Balaratnam G, Dixon S, Tonda ME, Washington CB, 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 GBT440, a HbS Polymerization Inhibitor. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 689. URL
  20. 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 65(S122). URL.
  21. Hrynkow SH, Hastings C, Liu B, Sharma R, Wang RY, Plummer E, Englehart D, Bascon C, Gao C, Fine NS. Intravenous hydroxyl-propyl-β-cyclodextrin trials and Compassionate use in Niemann-Pick Type C Disease. World Lysosomal Storage Diseases Annual 17th Annual Meeting. San Diego, CA; Feb 13-17, 2017. Metabolic Genetics Metabolism.
  22. Kanter J, Walters MC, Hsieh M, Krishnamurti L, Kwiatkowski JL, Kamble R, von Kalle C, Joseney-Antoine M, Pierciey FJ, Shi W, Asmal M, Thompson AA, Tisdale JF. Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 527. URL
  23. Kanter J, Marsh A, Treadwell M, Bardach N, Cabana M. Results of a national provider survey in sickle cell disease management. ASPHO 31st Annual Meeting. Pittsburgh, PA; May 5, 2018. Pediatric Blood & Cancer 65(S1); e27057. URL
  24. Karin I, Buechner B, Neuhofer C, Kuepper C, Darling A, Perez-Duenas B, Dusek P, Stanković I, Svetel M, Rohani M, Chinnery P, Steele H, Horvath R, Gregory A, Hogarth P, Hayflick SJ, Vichinsky E, Neumayr L, Zibordi F, Zorzi G, Nardocci N, Kmiec T, Klopstock T. Treat iron-related childhood-onset neurodegeneration (TIRCON) work package 1: International patient registry for Neurodegeneration with Brain Iron Accumulation (NBIA). European Journal of Neurology 24; Suppl 1(557). URL.
  25. Kutny M, Alonzo T, Gerbing RB, Feusner J. Arsenic trioxide consolidation results in excellent survival in young children as well as older children and adolescents with newly diagnosed acute promyelocytic leukema: a report from the Children's Oncology Group Study AAML0631. 7th International Symposium on Acute Promyelocytic Leukemia. Rome, Italy; Sept 24-27, 2017. oral presentation.
  26. Kutny MA, Alonzo TA, Gerbing RB, Wang YC, Aplenc R, Gamis AS, Feusner J, Woods WG, Meshinchi S, Johnston D. Outcomes based on CNS disease status in pediatric acute myeloid leukemia and the role of peripheral blood contamination in diagnostic lumbar punctures; A report from the children's oncology group studies AAML0531 and AAML1031. Blood 130(Suppl 1). URL.
  27. Kutny M, Alonzo T, Gerbing RB, Feusner J. Toxicity in pediatric patients treated with All-trans retinoic acid and arsenic trioxide induction: a report from the Children's Oncology Group Study AAML1331. 7th International Symposium on Acute Promyelocytic Leukemia. Rome, Italy; Sept 24-27, 2017. oral presentation.
  28. Kwiatkowski JL, Thompson AA, 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, Walters MC. Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 360. URL
  29. 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.
  30. 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.
  31. 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.
  32. Lal A, Weyhmiller M, Vichinsky E. Threshold ferritin values to predict control of liver iron burden in thalassemia. Seventh Congress of the International BioIron Society (IBIS). Los Angeles, CA; May 7-11, 2017. American Journal of Hematology 92(8); E178-E506, poster #57. URL
  33. Lane S, Skowron M, Wong A, Yongblah K, Ehlting J, Walter PB. Isolation and Chelation Activity of Plant Phenolic Root Exudates As Potential Clinical Iron Chelators. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 3505. URL
  34. Lesser M, Nguyen L, Fung EB. Retrospective analysis on the effect of vitamin D supplementation on bone mineral density in pediatric leukemic patients who underwent allogenic bone marrow transplant. Experimental Biology; Chicago, 2017.
  35. 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 β-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.
  36. Madiwale MV, Schulte RR, Hochberg J, MnNeer J, Messinger YH, Bleyer A. Asparaginase-induced hepatic injury and role of levocarnitine in management: A multi-institutional case series. ASPHO 30th Annual Meeting. Montreal, Quebec, Canada; April 26-29, 2017. Pediatric Blood & Cancer 64(S1); e26591, Poster #333. URL
  37. 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
  38. 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).
  39. Mei J, Yamashita R, Lal A, Olivieri NF, Vichinsky E. Community Survey of Knowledge of Thalassemia. 59th ASH Annual Meeting. Atlanta GA; Dec 9-12, 2017. Blood 130(Suppl 1); 5580. URL
  40. Michlitsch J, Vichinsky E, Larkin S, Kuypers F. Vincristine-induced Anemia in Hereditary Spherocytosis. ASPHO 31st Annual Meeting. Pittsburgh, PA; May -5, 2018. Pediatric Blood & Cancer 65(S1); e27057, #076. URL
  41. Minkley M, Sweeters N, Kasravi S, Ferguson J, Higa A, Jackson A, Smith D, Borchers C, Nashmi R, Macleod P, Neumayr LD, Vichinsky E, Walter PB. Systemic Biomarkers Show Elevated Oxidative Stress and Chronic Inflammation in Two Disorders of Neurodegeneration with Brain Iron Accumulation (NBIA). In: 59th ASH Annual Meeting; 2017; Atlanta, GA; Dec 9-12, 2017: Blood;130:Suppl 1;943. URL
  42. Neumayr L, Shah P, Rowland M, Smith K, Moscicki AB, Treadwell M. Documenting sickle cell disease health-related quality of life in the electronic medical record. 2017 Health Measures User Conference. Chicago, IL; Sept 27-18, 2017.
  43. Nguyen J, Carlberg K, Calloway CD. The Effects of Storage Temperature and Duration on the Extraction Yield of Cell-Free DNA. CIRM SPARK Trainee Meeting. Pasadena, CA; Aug 2017.
  44. 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.
  45. Pariury H, Wilhoite J, Michlitsch J, Agrawal AK. Potassium supplementation mitigates corticosteroid-induced neuropsychiatric effects in pediatric patients with acute lymphoblastic leukemia. 49th Congress of the International Society of Paediatric Oncology (SIOP). Washington, DC; Oct 2017. poster presentation.
  46. Paulukonis ST, Vichinsky E, Treadwell M, Hulihan M. County Level Surveillance of Sickle Cell Disease Population, Healthcare Utilization, and Fragmentation to Support Improvements in Access to Care. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 3369. URL
  47. Paulukonis ST, Hagar RW. Mortality Among California's Sickle Cell Data Collection Cohort, 2005-2015. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 2133. URL
  48. Polgreen LE, White K, Rudser KD, Menk JS, Miller BS, Fung E, Eisengart J, Lund TC, Orcard P. Defining clinical measures of skeletal disease severity in Mucopolysaccharidosis type I. LDN World Conference 2017.
  49. Porter J, Russell K, Rupff R, Treadwell M, Ferris M, Hankins JS, Wang W. Provider Perspectives of Necessary Factors in Sickle Cell Disease Health Care Transition: Results from an Online Survey. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 2140. URL
  50. Puri L, Hankins J, Hillenbrand CM, Flanagan JM, Kang G, Bi W, Tipirneni-Sajja A, Villavicencio M, Loeffler R. Genetic Modifiers of Iron Overload in Sickle Cell Disease. ASPHO 30th Annual Meeting. Montreal, Quebec, Canada; April 26-29, 2017. Pediatric Blood & Cancer 64(S1); e26591, Poster #161. URL
  51. 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
  52. Rasko J, Thompson A, Kwiatkowski J, Hongeng S, Schiller GJ, Anurathapan U, Cavazzana-Calvo M, Ho P, Von Kalle C, Kletzel M, Leboulch P, Vichinsky E, Petrusich A, Asmal M, Walters M. Lentiglobin gene therapy for transfusiondependent B-thalassemia: Update from the northstar HGB-204 phase 1/2 clinical study. Cytotherapy 2017; 19(5); S19.
  53. 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.
  54. Sears M, Ewing C, Lanzkron S, Field JJ, Hoppe CC, Little JA. A Prospective Multi-Centered Registry Is Feasible in Sickle Cell Disease: Globin Regional Network for Data and Discovery (GRNDaD). 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 3375. URL
  55. Shah P, Smith K, Rowland M, Moscicki B, Treadwell M, Neumayr L. Electronic Medical Record Documentation of Quality of Life for Patients with Sickle Cell Disease. Pediatric American Societies (PAS) 2017 Meeting. San Francisco, CA; May 6-9, 2017. Poster presentation.
  56. Singer ST, Fischer R, Yuan Q, Lal A, Allen IE, Vichinsky E, Wang ZJ. Pituitary Iron: Effect on Pituitary Volume and Function in Transfusion Dependent Thalassemia. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 3502. URL
  57. Slater N, Weyhmiller M, Evan P, Porter J, Higa A, Ng V, Sweeters N, Price J, Manji A, Killea D, Neumayr L, Lal A, Harmatz P, Vichinsky E, Walter P. Cardiac iron burden is correlated with innate immune markers, body mass index, and labile iron plasma in Beta Thalassemia Major patients undergoing combined chelation therapy. Seventh Congress of the International BioIron Society (IBIS). Los Angeles, CA; May 7-11, 2017. American Journal of Hematology 92(8); E178-E506, poster #137. URL
  58. Talano J-A, Moore TB, Keever-Taylor CA, Shenoy S, Walters MC, Parsons SK, Dozor AJ, Friedman D, Shi Q, Braniecki S, Grossman BJ, Weinberg RS, Vichinsky E, Chu Y, Morris E, Fabricatore S, Ayello J, Baxter-Lowe LA, Cairo MS. Promising Results at 1 Year Follow-up Following Familial Haploidentical (FHI) T-Cell Depleted (TCD) with CD34 Enrichment and T-Cell (CD3) Addback Allogeneic Stem Cell Transplantation in Patients with High-Risk Sickle Cell Disease (SCD) (IND 14359). 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 4602. URL
  59. 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).
  60. Tisdale JF, Pierciey FJ, Kamble R, Kanter J, Krishnamurti L, Kwiatkowski JL, Thompson AA, Shestopalov I, Bonner M, Joseney-Antoine M, Asmal M, Walters MC. Successful Plerixafor-Mediated Mobilization, Apheresis, and Lentiviral Vector Transduction of Hematopoietic Stem Cells in Patients with Severe Sickle Cell Disease. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 990. URL
  61. Treadwell M, Werner E. Sickle Cell Disease Implementation Consortium. Health Measures User Conference. Chicago, IL; Sept 27-28, 2017.
  62. Vichinsky E, El-Beshlawy A, Alzoebia A, Kamdem A, Koussa S, Chotsampancharoen T, Bruederle A, Han J, Mohsen E. Long-term iron chelation therapy with deferasirox in pediatric patients with transfusional hemosiderosis (ENTRUST). Seventh Congress of the International BioIron Society (IBIS). Los Angeles, CA; May 7-11, 2017. American Journal of Hematology 92(8); E178-E506, poster #203. URL
  63. Vichinsky E, El-Beshlawy A, Alzoebia A, Koussa S, Chotsampancharoen T, Bruederle A, Han J, Elalfy M. Long-term Therapy with Deferasirox in Young Pediatric Patients with Transfusional Hemosiderosis Completing up to 5 Years of Treatment in the Observational Entrust Study. ASPHO 30th Annual Meeting. Montreal, Quebec, Canada; April 26-29, 2017. Pediatric Blood & Cancer 64(S1); e26591, Poster #242. URL
  64. Vichinsky E, Hoppe C, Howard J, Ataga K, Nduba V, El Beshlawy A, Diuguid DL, Al-Kindi S, Brown C, Hassab H, Telfer P, Tsitsikas DA, Unal S, Kanter J, Abboud MA, Gordeuk VR, Lehrer-Graiwer J, Sherman C, Tonda M, Intondi A, Yaron Y, Ware RE. Results from Part A of the Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization (HOPE) Trial (GBT440-031), a placebo-controlled randomized study evaluating Voxelotor (GBT440) in adults and adolescents with sickle cell disease. 60th ASH Annual Meeting & Exposition. San Diego, CA; Dec 1-4, 2018.
  65. 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.
  66. Walters MC, Hongeng S, Kwiatkowski JL, Locatelli F, Porter JB, Sauer MG, Thrasher AJ, Thuret I, Yannaki E, Elliot H, Gayron M, Asmal M, Thompson AA. Results from the Hgb-207 (Northstar-2) Trial: A Phase 3 Study to Evaluate Safety and Efficacy of Lentiglobin Gene Therapy for Transfusion-Dependent β Thalassemia (TDT) in Patients with Non-β0/β0 Genotypes. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 526 URL
  67. 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).
  68. Washington CB, Green M, Inati AC, Brown C, Hoppe CC, Wang W, Balaratnam G, Dixon S, Fong E, Hutchaleelaha A, Tonda ME, Lehrer J. The Pharmacokinetics (PK) of GBT440 Following Single Doses in Pediatric Patients with Sickle Cell Disease (SCD). 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 980. URL
  69. 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.
  70. 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.
  71. 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.
  72. Watkins B, Qayed M, Bratrude B, Betz K, Brown M, Rhodes J, Sinclair S, Suessmuth Y, Yu A, Hebert K, Pasquini MC, Choi SW, Davis J, Duncan C, Giller R, Grimley M, Harris AC, Jacobsohn DA, Lalefar N, Norkin M, Pulsipher MA, Shenoy S, Blazar B, Langston A, Horan JT, Kean L. T Cell Costimulation Blockade with Abatacept Nearly Eliminates Early Severe Acute Graft Versus Host Disease after HLA-Mismatched (7/8 HLA Matched) Unrelated Donor Transplant, with a Favorable Impact on Disease-Free and Overall Survival. 59th ASH Annual Meeting. Atlanta, GA; Dec 9-12, 2017. Blood 130(Suppl 1); 212. URL
  73. Weyhmiller M, Buac N, Fung EB, Lal A, Fischer R. Effects of bone marrow iron on bone mineral density assessments: A phantom study. Seventh Congress of the International BioIron Society (IBIS). Los Angeles, CA; May 7-11, 2017. American Journal of Hematology 92(8); E178-E506, poster #50. URL
  74. Weyhmiller M, Buac N, Fung EB, Lal A. Iron: a vertebral bone marrow phantom menace? BioIron Meeting. Los Angeles, CA; May 7-11, 2017.
  75. 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 (EHA). Stockholm, Sweden; June 14-17, 2018.
  76. Yamamura J, Fischer R, Schonnage B, Grosse R, Nielsen P. Pancreatic iron in patients with hemochromatosis: Not as rare as cardiac iron. Seventh Congress of the International BioIron Society (IBIS). Los Angeles, CA; May 7-11, 2017. American Journal of Hematology 92(8); E178-E506, poster #151. URL
  77. 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
  78. Zlotnicki M, Peretz C, Agrawal A. Negative initial bone marrow aspirate does not definitively rule out acute lymphoblastic leukemia: A case series. ASPHO 30th Annual Meeting. Montreal, Quebec, Canada; April 26-29, 2017. Pediatric Blood & Cancer 64(S1); e26591, Poster #932. URL

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INVITED LECTURES/ARTICLES/BOOKS/CHAPTERS

Books:

  • Lessing S, Vichinsky E, eds. A Parent's Handbook for Sickle Cell Disease: Part 1: Birth to 6 Years of Age. 2nd ed: California Department of Health, Genetic Disease Branch; 2018 (in press).

Chapters/Articles:

  • 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. URL
  • Carlberg K, Hastings C. Hyperleukocytosis. In: Cabana MD, Goldsby RE, eds. The 5 Minute Pediatric Consult. 8th ed: Wolters Kluwer; 2018 (in press).
  • Carlberg K, Hastings C. Leukocytosis. In: Cabana MD, Goldsby RE, eds. The 5 Minute Pediatric Consult. 8th ed: Wolters Kluwer; 2018 (in press).
  • DeBaun M, Vichinsky E. Vaso-occlusive pain management in sickle cell disease. In: Mahoney DH, Schrier SL, Tirnauer J, eds. UpToDate. Topic 7144: Version 34.0; Feb 12, 2018. UpToDate, Inc., Waltham, MA. URL
  • DeBaun M, Vichinsky E. Red blood cell transfusion in sickle cell disease. In: Schrier SL, Mahoney DH, Tirnauer J, eds. UpToDate. Topic 90145: Version 29; Dec 19, 2017. UpToDate, Inc. Waltham, MA. URL
  • Field J, Vichinsky E, DeBaun M. Overview of the management and prognosis of sickle cell disease. In: Schrier SL, Mahoney DH, Tirnauer J, eds. UpToDate. Topic 7114 Version 53.0; Oct 23, 2017. UpToDate, Inc. Waltham, MD. URL
  • Fischer R. Evaluation of Iron Overload by Non-Invasive Measurement Techniques. In: Crichton R, Ward RJ, Hider RC, eds. Metal Chelation in Medicine. The Royal Society of Chemistry; 2017.
  • Hoppe C. Methods for hemoglobin analysis and hemoglobinopathy testing. In: Mentzer WC, Tirnauer JS, eds. UpToDate; Topic 13941: Version 27.0; May 4, 2018. UpToDate, Inc., Waltham, MA. URL.
  • Lal A. Thalassemia Management Checklists: Monitoring Deferasirox Therapy. Cooley's Anemia Foundation, Health Resources and Services Administration (HRSA) 2018. 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; 2018 (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. JAAOCS publication, Instr Course Lecture 2018;67:529-547.
  • 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 CA, Feusner J. Germ Cell Tumors. In: Cabana MD, Goldsby RE, eds. The 5 Minute Pediatric Consult. 8th ed: Wolters Kluwer; 2018 (in press).
  • Singer ST. Fertility Issues in Transfusion-Dependent Thalassemia Patients: Pathophysiology, Assessment, and Management. In: Woodruff TK, ed. Pediatric and Adolescent Oncofertility: Best Practices and Emerging Technologies. 1st ed., chapter 13. New York, NY: Springer; 2016: p.209-30. URL.
  • Treadwell MJ. Transition from pediatric to adult care: Sickle cell disease. In: Schrier SL, Tirnauer J, eds. UpToDate. Topic 112919: Version 5.0; Jan 18, 2018. UpToDate, Inc. Waltham, MA. URL.
  • Vichinsky E, Mahoney DH. Diagnosis of sickle cell disorders. In: Schrier SL, Tirnauer J, eds. UpToDate. Topic 7113: Version 26.0; Feb 26, 2018. UpToDate, Inc. Waltham, MA. URL
  • Vichinsky E. Overview of the clinical manifestations of sickle cell disease. In: Schrier SL, Tirnauer JS, eds. UpToDate. Topic 7119 Version 40.0; Nov 8, 2017. UpToDate, Inc. Waltham, MD. URL
  • Vichinsky E. Overview of Variant Sickle Cell Syndromes. In: Mahoney DH, Tirnauer J, eds. UpToDate. Topic 7115 Version 17.0; Jan 9, 2018. UpToDate, Inc. Waltham, MA. URL
  • 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
  • Vichinsky E. Sickle cell trait. In: Mahoney DH, Tirnauer J, eds. UpToDate. Topic 7145: Version 44.0; Jan 9, 2018. UpToDate, Inc. Waltham, MA. URL
  • Yogen S, Vichinsky E. Sickle Cell Disease: Clinical Features and Management. In: Hoffmann R, Benz EJ, Silberstein LE, Heslop H, Weitz J, Anastasi J, eds. Hematology: Basic Principles and Practice. Chapter 42 7th ed: Elsevier; 2017. 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).

Lectures:

  • Agrawal A. CAR-T therapy in relapsed ALL/NHL. Pediatric Hematology/Oncology Conference (invited talk); Kaiser Permanente Oakland, 2018.
  • Agrawal A. CAR-T therapy in relapsed ALL/NHL. Immunotherapy Conference (invited talk); UCSF Benioff Children’s Hospital San Francisco, 2018.
  • Agrawal A. Global concerns in pediatric pain medicine. Interventional Cancer Pain Symposium (invited talk). Memorial Sloan Kettering Cancer Center, Sept 8-9, 2017.
  • Agrawal A. Supportive Care. Workshop for Nurses and Physicians on Pediatric Oncology Care (invited talk); Ho Chi Minh City Medicine and Pharmacy University, 2017.
  • Carlberg K, Bose N, Lal A, Erlich H, Calloway C. Development of a Noninvasive Prenatal Test for β-Hemoglobinopathies Utilizing NGS and a Novel Probe Capture Panel to Analyze Cell-Free Fetal DNA in Maternal Plasma. Non-Invasive Prenatal Testing Symposium; Cambridge Healthtech Institute's Next Generation Dx Summit; Aug 18, 2017, Washington, DC; URL
  • Calloway C. Development of a Probe Capture NGS System for Forensics. Forensic Technology Center of Excellence (FTCOE) Webinar; Mar 28, 2017. URL.
  • Calloway C. Forensic and Clinical Applications to Next-Generation Sequencing. Molecular Techniques ETX278 Course; Mar 12, 2018, UC Davis, CA.
  • Calloway C. Probe capture next-generation sequencing system for forensic and clinical applications. Thermofisher; Oyster Point, CA; Feb 12, 2018.
  • Calloway C. Probe capture next-generation sequencing system for forensic and clinical applications. Natera; San Carlos, CA; May 10, 2018.
  • Erlich H. The Analysis of Mixtures Using NGS: Applications to Non-Invasive Prenatal Testing for Beta-Thalassemia and Sickle Cell Anemia. 10th International Society for Applied Biological Sciences (ISABS) Conference; Dubrovnik, Croatia; June 2017. URL.
  • Fung E. 5th Pediatric Bone Health Symposium. Invited speaker; San Francisco, CA; 2018.
  • Fung E. Achondroplasia BMN 111 Study Train the Trainers. Invited speaker, BioMarin, Chicago, IL; 2018.
  • Fung E. Cooley's Anemia Family Conference. Invited speaker; Atlanta, GA; 2018.
  • Fung E. 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. 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.
  • Hankin DC, Raphael R, Feusner J. Long-term follow-up for young adult survivors of pediatric cancer: Successful transition of care. Symposium presentation: American Psychosocial Oncology Society (APOS) Conference, Orlando, FL; Feb 15-18, 2017.
  • Hastings C, Liu B. Overview of Phase I Clinical Trial with Trappsol®Cyclo™ for Niemann-Pick Type C. National Niemann-Pick Disease Conference; Aug 11, 2017, Chicago, IL.
  • Hoppe CC. Bioverativ medical advisory board meeting. Bethesda, MD; 2018 (invited speaker).
  • Hoppe CC. Global Blood Therapeutics Key Opinion Leader Meeting. Atlanta, GA; Dec 11, 2017 (invited speaker).
  • Hoppe CC. Hemoglobinopathy Testing: The Role of the Reference Laboratory. Bio-Rad Laboratories 7th Hemoglobin Update Meetings, New Delhi (Kolkata, Hyderabad, Chennai); July 7 (July 8-11), 2017 (invited speaker).
  • Hoppe CC. Initial Results from a Cohort in a Phase 2a Study (GBT440-007) Evaluating Adolescents with Sickle Cell Disease Treated with Multiple Doses of GBT440, a HbS Polymerization Inhibitor. Highlights of ASH, 59th ASH Annual Meeting, Atlanta, GA; Dec 11, 2017 (invited speaker).
  • Hoppe CC. Interim Results from a Cohort in a Phase 2a Study Evaluating Adolescents with SCD Treated with Multiple Doses of Voxelotor (GBT440). 59th ASH Annual Meeting, Atlanta, GA; Dec 11, 2017 (invited speaker).
  • Hoppe CC. Point of Care Testing for Sickle Cell Disease. Sickle Cell in Focus Conference, Kingston, Jamaica; Oct 26, 2017 (invited speaker).
  • Hoppe CC. Thalassemias: Challenges in Screeing and Confirmatory Testing. 15th Annual Newborn Screening Convention, Manilla, Phillipines; Oct 4, 2017 (invited speaker).
  • Lal A. 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.
  • Neumayr L, Shah P, Rowland M, Smith K, Moscicki AB, Treadwell M. Documenting sickle cell disease health-related quality of life in the electronic medical record. Health Measures User Conference, Chicago, IL; Sept 27-28, 2017.
  • Raphael R. Rare Childhood Cancers. Nevada Cancer Control Summit, Reno, NV Nov 6, 2017.
  • Saba J. 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.
  • Saba J. Doubling time: lessons from a cancer insider. Champions Against Cancer (invited talk); Oct 12-13, 2017.
  • Saba J. Mutations in SGPL1 as the cause of a novel inborn primary immunodeficiency syndrome coupled with nephropathy, neuropathy and adrenal insufficiency. FASEB Science Research Conference on Lysophospholipid and Related Mediators: From Bench to Clinic (keynote speaker). New Orleans, LA; Aug 2017.
  • Saba J. 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.
  • Saba J. S1P lyase insufficiency causes immunodeficiency, nephropathy, neuropathy and adrenal insufficiency. 15th International Conference on Bioactive Lipids in Cancer, Inflammation and Related Diseases (invited talk). Puerto Vallarta, Mexico; Oct 2017.
  • Saba J. Sphingadienes: novel dietary sphingolipids with anti-cancer properties in colon cancer and other malignancies. NIH Office of Dietary Supplements (ODS) Seminar Series: 2017 Emphasis on Scientific Rigor (invited talk); Mar 22, 2017.
  • Saba J. Sphingosine-1-phosphate signaling and metabolism gene signature in pediatric inflammatory bowel disease: a matched-case control pilot study. NIH Metabolomics Program Seminar (invited talk, webinar); Oct 5, 2017.
  • Saba J. 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. Biopsychosocial-spiritual model of coping in sickle cell disease. Annual Sickle Cell Disease and Thalassaemia Conference; London, UK; Oct 23, 2018.
  • Treadwell M, Cavazzana M. Cure for sickle cell disease or thalassemia is the priority over comprehensive care: Yes! Academy for Sickle Cell and Thalassemia: Guy's and St Thomas NHS Trust Annual Meeting; London, UK; Oct 13, 2017.
  • Treadwell M. Disparities in healthcare: sickle cell disease. Alameda County Public Health Commission. San Leandro, CA; Nov 9, 2017.
  • Treadwell M. Evaluating and addressing challenges to optimal sickle cell disease care: Health Literate Care Model. Centers for Medicare and Medicaid Services Region 9 webinar; Apr 19, 2017. URL.
  • Treadwell M. Evaluating and addressing challenges to optimal sickle cell disease care: Health Literate Care Model. Centers for Medicare and Medicaid Services Region 9 webinar; Nov 15, 2017.
  • Treadwell M. New approaches to therapies for sickle cell disease. Cayenne Wellness Sickle Cell Educational Symposium; Sacramento, CA; Sept 6, 2018.
  • Treadwell M. Patient reported outcome measures in sickle cell disease. 46th Annual National Convention for Sickle Cell Disease; Sickle Cell Disease Association of America (SCDAA), Baltimore, MD; Oct 25, 2017.
  • Treadwell M. 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.
  • Treadwell M, Paulukonis S. Sickle cell care coordination initiative: Linking the community, providers, and systems. San Francisco Bay Clinical Research Network; San Francisco, CA; May 10, 2017.
  • Treadwell M. Sickle Cell Disease Implementation Consortium. NHLBI Sickle Cell Clinical Research Meetings; Rockville, MD; Aug 14, 2018.
  • Treadwell M. Sickle cell data collection impact: Turning data into action. CDC Foundation; Atlanta, GA; Dec 6, 2017.
  • Treadwell M, Field J. Transition from pediatric to adult care in sickle cell disease. Sickle Treatment Outcomes Research in the Midwest (webinar); May 17, 2017.
  • Vichinsky E. 10th annual Sickle Cell California Symposium. Keynote speaker, Oakland, CA; Sept 9-10, 2018.
  • Vichinsky E. Alpha Thalassemia. Workshop Chair and speaker. American Society of Pediatric Hematology Oncology, Pittsburgh, PA; May 2-5, 2018.
  • Vichinsky E. 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. San Francisco, CA; Aug. 29, 2018.
  • Vichinsky E. 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.
  • Vichinsky E. Glutamine therapy in sickle cell disease: results of a phase 3 trial. Invited presentation, NIH Annual Sickle Cell Disease meeting; Bethesda, MD, 2018.
  • Vichinsky E. Martin Luther King Jr. Celebration and Healthcare Fair. Keynote speaker, Pittsburg, CA.; Jan 15, 2018.
  • Vichinsky E. Multiorgan failure and aging in sickle cell disease. 7th Annual SCD Therapeutics Conference; Global Blood Therapeutics, New York, NY; Sep 12-13, 2018.
  • Vichinsky E. Neurological Consequences of Sickle Cell Disease in Adult Patients. Neuro-sickle Symposium, Emory University, Atlanta, GA; March 2018.
  • Vichinsky E. Northern California Sickle Cell Patient Panel. Keynote speaker, Pittsburgh, CA; Apr 7, 2018.
  • Vichinsky E. Overview of Thalassemia. Agios Pharmaceuticals, San Mateo, CA; July 18, 2018.
  • Vichinsky E. Training the Healthcare Community in Sickle Cell Disease. UC Davis, Sacramento, CA; Jan 27, 2018.
  • Vichinsky E. Chronic Organ Failure in Adult Sickle Cell Disease. ASH Education Session at the 59th ASH Annual Meeting, Atlanta, GA; Dec 9, 2017.
  • Vichinsky E. Fetal Therapy for Alpha Thalassemia Major. Kaiser Genetics Meeting, Oakland, CA; Jan 26, 2017.
  • Vichinsky E. Fetal Therapy for Alpha Thalassemia Major, HRSA Thalassemia Annual Meeting, Oakland, CA: Oct 6, 2017.
  • Vichinsky E, Morris CR. L-Glutamine in Sickle Cell Disease. Invited presentation: Sickle Cell Therapeutics: Is it time for combination therapy? National Institutes of Health; Bethesda, MD, Aug. 15, 2018.
  • Vichinsky E. Management of Sickle Cell Disease: Present and Future. American Society of Hematology Education Day, Atlanta, GA; Dec. 9-11, 2017.
  • Vichinsky E. Oakland Center Finds Sickle Cell Treatment Success;. Interview by Jenny Gold for series, “You, Me, and Them: Experiencing Discrimination in America”; Health News, NPR Weekend Edition, Nov 5, 2017; URL
  • Vichinsky E. Oxford Union Style Debate with audience participation on HCT for SCD. Symposium on Curative Therapies in SCD. Bethesda, MD; Aug 13, 2018.
  • Vichinsky E. Pros and cons of stem cell therapy in sickle cell disease. Invited presentation, NIH Annual Sickle Cell Disease meeting; Bethesda, MD, 2018.
  • Vichinsky E. A randomized, double-blind, placebo-controlled trial of deferiprone in patients with panthothenate kinase-associated neurodegeneration (PKAN). NBIA Disorders Association Family Conference, Deerfield, IL; June 2, 2017.
  • Vichinsky E. Sickle Cell Care Coordination Initiative: Linking the Community, Providers and Systems. Kaiser Joint Meeting, Oakland, CA: Oct 26, 2017.
  • Vichinsky E. Sickle Cell Patients Endure Discrimination, Poor Care And Shortened Lives. Interview by Jenny Gold for series, “You, Me, and Them: Experiencing Discrimination in America”; Health News, NPR Weekend Edition, Nov 4, 2017; URL
  • Vichinsky E. Sickle Cell Trait, and Sickle Cell Complications. Sickle Cell Disease Educational Seminar at UC Davis. Cayenne Wellness; Sacramento; Sept 6-7, 2018.
  • Vichinsky E. Stem cell therapy vs. transfusion for strokes in sickle cell disease. Plenary invited debate. ASPHO 31st Annual Meeting, Pittsburgh, PA; May 2018.
  • Vichinsky E. Invited talk, Thalassemia Adoption Clinic Dinner. UCSF Benioff Children's Hospital Oakland, CA; July 18, 2018.
  • Walters M. 2nd Annual Cell & Gene Therapy Symposium Invited presentation, Vellore, India, Sept 5-10, 2017.
  • Walters M. 5th Annual Re-writing Genomes Symposium. QB3, invited speaker, UC Berkeley, CA; Aug 18, 2017.
  • Walters M. Bioverativ medical advisory board meeting. Bethesda, MD, 2018.
  • Walters M. CBBS Meeting. Invited speaker, Lake Tahoe, CA; May 5, 2017.
  • Walters M. Chair, NHLBI Sickle Cell Disease Advisory Committee meeting. Bethesda, MD; Jan 26, 2018.
  • Walters M. CIRM Light a Spark to High School Students Summer Program. Invited presentation, CHORI, Oakland, CA; July 26, 2017.
  • Walters M. Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study. ASBMT Tandem Meeting (invited speaker), Orlando, FL; Feb 20-26, 2017.
  • Walters M. CRISPRCon. Invited panelist, UC Berkeley, CA; Aug 17, 2017.
  • Walters M. European Hematology Association Meeting. Oral presentation, Madrid, Spain, June 24-26, 2017.
  • Walters M. Foundation for Research in Sickle Cell Disease meeting. Hollywood, FL, 2017.
  • Walters M. Gene Therapy for Hemoglobinopathies. ASBMT Tandem Meeting, pediatric symposium (invited speaker), Salt Lake City, UT; Feb 20-24, 2018.
  • Walters M. Gene Therapy for Hemoglobinopathies. Hematology/Oncology/BMT; Feb 22, 2018; UCSF Benioff Children's Hospital, Oakland, CA.
  • Walters M. Global Blood Therapeutics. Invited speaker, So. San Francisco, CA; Feb 1, 2017.
  • Walters M. Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease. ASBMT Tandem Meeting (invited speaker), Orlando, FL; Feb 20-26, 2017.
  • Walters M. NHLBI annual sickle cell disease clinical research meeting. Invited speaker, Bethesda, MD; Mar 29-31, 2017.
  • Walters M. NHLBI Sickle Cell Disease Advisory Committee. Bethesda, MD; Mar 29, 2017.
  • Walters M. Oxford Union Style Debate with audience participation on HCT for SCD. Symposium on Curative Therapies in SCD. Bethesda, MD; Aug 13, 2018.
  • Walters M. Promising Results at 1 Year Follow-up Following Familial Haploidentical (FHI) T-Cell Depleted (TCD) with CD34 Enrichment and T-Cell (CD3) Addback Allogeneic Stem Cell Transplantation in Patients with High-Risk Sickle Cell Disease (SCD) (IND 14359). ASBMT Tandem Meeting (invited speaker), Orlando, FL; Feb 20-26, 2017.
  • Walters M. Rare Disease: Sickle Cell Disease/1507 Haplo HCT. ASBMT Tandem Meeting, pediatric symposium (invited speaker), Salt Lake City, UT; Feb 20-24, 2018.
  • Walters M. Results from the Hgb-207 (Northstar-2) Trial: A Phase 3 Study to Evaluate Safety and Efficacy of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in Patients with Non-β0/β0 Genotypes ASBMT Tandem Meeting (invited speaker), Orlando, FL; Feb 20-26, 2017.
  • Walters M. Safety Following Autologous Transplantation with Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in the Northstar Hgb-204 Study. ASBMT Tandem Meeting, pediatric symposium (invited speaker), Salt Lake City, UT; Feb 20-24, 2018.
  • Walters M. Sangamo Biosciences. Invited speaker, 2017.
  • Walters M. Saudi Scientific Society of Blood & Marrow Transplantation (invited presentation), Riyadh, Saudi Arabia, May 11, 2017.
  • Walters M. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. ASBMT Tandem Meeting (invited speaker), Orlando, FL; Feb 20-26, 2017.
  • Walters M. Siebel Institutes workshop. Invited presentation, UC Berkeley/Stanford, Berkeley, CA; Jan 11, 2017.
  • Walters M. 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.
  • Walters M. 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.
  • Walters M. STELLAR annual advisory board meeting. Emory University; Oct 27-29, 2017.
  • Walters M. Successful Plerixafor-Mediated Mobilization, Apheresis, and Lentiviral Vector Transduction of Hematopoietic Stem Cells in Patients with Severe Sickle Cell Disease. ASBMT Tandem Meeting (invited speaker), Orlando, FL; Feb 20-26, 2017.
  • Walters M. University of Pittsburgh Grand Rounds. Invited speaker, Pittsburgh, PA; Nov 2, 2017.
  • Walters M. Update on ongoing clinical trials/studies. Symposium on Curative Therapies in SCD. Bethesda, MD; Aug 13, 2018.

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.
  • Lal A, Robertson S, Vichinsky E. Method and System to Provide Patient Information and Facilitate Care of a Patient. Application no. 14/161,526. Children's Hospital & Research Center Oakland. Current status: Request for continued review; 2/22/2018.

Profiles:

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MEDIA:

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. Transition from pediatric to adult sickle cell care. Stepping Up: Kevin’s Decision, and Stepping Up: Calvanay’s Advice. Centers for Disease Control and Prevention (CDC). June 14, 2018. (In recognition of World Sickle Cell Day, the CDC released videos on transition from pediatric to adult care that feature young adults with sickle cell disease and staff from the Northern California Comprehensive Sickle Cell Center.)
  12. 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.)
  13. Nutrition bar improves lung function in overweight, obese teens with asthma. Healio.com. (Dr. Lal et al., the 'CHORI bar') July 20, 2018.
  14. 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).
  15. 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).
  16. New study shows L-glutamine decreases sickle cell pain Crises, hospitalizations. By Melinda Krigel, UCSF News Center, July 24, 2018. (Dr. Elliott Vichinsky interviewed about a paper he co-authored.)
  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. Jazz and her journey with sickle cell disease. By Elise Carlaccini, Sr. Creative Producer and Jon Miller, Senior Donor Relations Writer. Under production by Corduroy Media, August 2018. (A video production featuring a patient with sickle cell, and her grandmother).
  19. Sickle Cell Patient Stories. By Amy Weitz, UCSF Public Affairs. Under production, August 2018. (Amy is working to use the media--newspaper, radio, possibly TV--to help raise public awareness about sickle cell disease by telling patient stories. She is also looking to create a "top 10" list of things to promote and sustain wellness for patients with sickle cell).
  20. Sickle Cell Photoshoot Project by Margaret Stutt, Stewardship Officer, UCSF Benioff Children's Hospitals Foundation. In production, August 2018. (A patient-clinician photography session in the Hematology Clinic featuring twins and another patient with sickle cell disease. These photos will be used for grant/gift reporting and future proposals).
  21. GBT brings together sickle cell disease influencers at 7th Annual Sickle Cell Disease (SCD) Therapeutics Conference. By GlobeNewswire, Sept 6, 2018.
  22. Sickle Cell: The Last Health-Care Frontier for Black Lives. By Momo Chang, East Bay Express, 9/12/2018. (Features BCHO Sickle Cell Center, patients and physicians).
  23. 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.
  24. Scientists Are Developing New Ways to Treat Disease With Cells, Not Drugs. By Alice Park, Time; Sept, 13, 2018. (The UCSF alpha thalassemia fetal transplant trial to extract the cells from a pregnant woman to treat a developing fetus in utero is just one of several innovative uses of stem cells to treat a growing list of diseases with cells instead of drugs.)

2017

  1. Bay Area Top Doctors 2017 (includes Feusner J, Vichinsky E). San Francisco Magazine, 12/23/2016.
  2. GBT Announces Presentation of Six Studies at ASH Supporting Voxelotor Sickle Cell Disease (SCD) Program. Global Blood Therapeutics Inc. (GBT), press release and investor webcast; Dec. 11, 2017. (case study of 7 patients with severe sickle cell disease treated with voxelotor, an experimental drug approved by the FDA for single-patient compassionate access.)
  3. GBT Announces New Phase 2a Data at ASH for Voxelotor in Adolescents with Sickle Cell Disease (SCD). Global Blood Therapeutics Inc. (GBT), press release and investor webcast; Dec. 9, 2017. Highlighted in ASH press briefing Dec. 11, 2017.
  4. Perspectives: Newsletter of the Northern California Thalassemia Center. December 2017. (BCHO thalassemia newsletter, featured on our web site, thalassemia.com)
  5. Outstanding Contributions Certificate, awarded to Dr. Carla Golden and Research Staff for outstanding contributions, National Cancer Institute, June 2017.
  6. Pioneer Award, presented to Dr. Marsha Treadwell by the Sickle Cell Disease Association of America, 10/27/2017. (Dr. Treadwell was one of 2 recipients of this year's Pioneer Award for her significant impact on the sickle cell community both nationally and globally as well as her lifelong overwhelming commitment to sickle cell disease.)
  7. In-Utero HSC Transplant with Fetal Transfusion Could Lead to Cures for Many Blood Diseases. UCSF Pediatric Focus, Sept. 2017.
  8. Novel Treatment Aims to Transform Protocol for Deadly Blood Disorder. By Suzanne Leigh, UCSF News Center, 8/30/17.

    (Enrollment announcements for the clinical trial that will test the safety of combining an in-utero hematopoietic stem cell transplant with a fetal transfusion of red blood cells at UCSF Benioff Children's Hospitals)
  9. In excruciating pain, sickle cell patients are shunted aside. by Sharon Begley, STAT, 9/18/2017. (A patient's story of the discrimination she has faced in seeking treatment for sickle cell vaso-occlusive pain at hospital emergency rooms; includes commentary by Dr. Keith Quirolo)
  10. While my son battles leukemia, the health care system is under assault. by Ari Levy, CNBC News, 6/27/2017. (Parent of a child with cancer treated at BCHO writes about the financial threat to hospitals, patients, and clinical research from current political agendas; features comments from Dr Carla Golden.)
  11. bluebird bio Announces Early Data from Phase 3 Northstar-2 (HGB-207) Study of LentiGlobinTM Drug Product at European Hematology Association (EHA) Annual Meeting. BusinessWire, 6/26/2017. (Early interim data from the Phase 3 clinical study of LentiGlobin drug product in patients with transfusion-dependent beta-thalassemia, presented by Dr. Mark Walters at the European Hematology Association meeting)
  12. Kamryn needs to find a match. It could be you. Be the Match, YouTube, 5/3/2017. (BCHO sickle cell patient Kamryn and her family urge the community to register as a bone marrow donor)
  13. Meet Leah Carroll, the 4-year-old girl inspiring the world as an "Overcomer". By Mireeya Villarreal, CBS NEWS, 3/27/2017. (BCHO patient with rare blood disorder treated by Dr. Alison Matsunaga sings along to an inspirational music video by Mandisa)
  14. Bronze Bambino Lifetime Achievement Award: Presented to Dr. James Feusner by the medical staff of UCSF Benioff Children's Hospital Oakland, 3/24/2017. (The Bronze Bambino is a lifetime achievement award presented annually to an outstanding member of the medical staff. It has been a tradition at Children's Hospital Oakland since 1967.)
  15. New Collaboration Launches to Advance Technology for Children's Health. By Nina Bai, UCSF News Center, 2/16/2017. (UCSF Benioff Children's Hospitals collaborate to form the Engineering for Children’s Health Initiative, including a study in Oakland of virtual reality technology to mitigate pain in children with sickle cell disease.)
  16. Virtual Reality Helps Kids Escape From Pain. Medical Expo e-Magazine, 2/1/2017. (More about the sickle cell disease virtual reality study. with comments from Dr. Anne Marsh)
  17. St Baldrick's 2017 Shave-a-thon. KTVU News, 3/13/2017. (News video coverage of the annual St. Baldrick's childhood cancer fundraiser at UCSF Benioff Children's Hospital Oakland)
  18. Children's Hospital Oakland staff use grant to tackle sickle cell disease. by Agatha Kereere, Oakland North, 1/26/2017. (Co-principal investigator Dr. Marsha Treadwell discusses the $4.1 million NHLBI grant awarded to UCSF Benioff Children’s Hospital Oakland for the Sickle Cell Care Coordination Initiative.)
  19. Oakland Center Finds Sickle Cell Treatment Success. NPR Weekend Edition with Jenny Gold, November 5, 2017. (Interview with BCHO sickle cell patient Derek, and Dr. Elliott Vichinsky)
  20. Sickle Cell Patients Endure Discrimination, Poor Care and Shortened Lives. NPR Weekend Edition with Jenny Gold, November 4, 2017. (Continuation of the interview with several BCHO sickle cell patients and Dr. Elliott Vichinsky)
  21. Sickle cell patients suffer discrimination, poor care -- and shorter lives. By Jenny Gold, Kaiser Health News, Nov 18, 2017. (Written story about the preceding two radio shows)
  22. Voxelotor improves hemoglobin levels, hemolysis markers in sickle cell disease. HemOnc Today; Dec. 9, 2017. (Dr. Carolyn Hoppe speaks at a press conference about the experimental drug voxelotor for sickle cell disease)
  23. Bioengineering and specialized therapies yield results for rare, challenging blood diseases. American Society of Hematology, Dec. 11, 2017. (Researchers -- including Dr. Carolyn Hoppe -- announce promising findings on innovative tools and therapies for hard-to-treat blood disorders including sickle cell disease, Beta thalassemia, and X-linked severe combined immunodeficiency (X-SCID).)
  24. Sickle-cell disease gets rush of gene-based research. by Michelle Fay Cortez, Chicago Tribune; Dec. 12, 2017. (Dr. Carolyn Hoppe, lead researcher on the voxelotor trial, is featured in a story about Bluebird bio, Inc. at the American Society of Hematology meeting.)

updated 9/18/2018


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