UCSF BENIOFF CHILDREN’S HOSPITAL OAKLAND

SICKLE CELL CENTER PUBLICATIONS

Fiscal Year 2021 (7/1/2020 – 6/30/2021)

 

PEER-REVIEWED ARTICLES

1.     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 2020-08-27; 20(1):638. PMID: 32854639  PMC7457248 Epub 2020/08/29. https://www.ncbi.nlm.nih.gov/pubmed/32854639

2.     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 2020-07-01; 3(7):e2010874. PMID: 32678452  PMC7368173 Epub 2020/07/18. https://www.ncbi.nlm.nih.gov/pubmed/32678452

3.     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 2020-09; 99(9):2073-9. PMID: 32377816  PMC7430152 Epub 2020/05/08. https://www.ncbi.nlm.nih.gov/pubmed/32377816

4.     Ballas SK, Kuypers FA, Gordeuk VR, Hankins JS, Thompson AA, Vichinsky E. Time to rethink haemoglobin threshold guidelines in sickle cell disease. Br J Haematol 2021 2021-06-15. PMID: 34131897  Epub 2021/06/17. https://www.ncbi.nlm.nih.gov/pubmed/34131897

5.     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 2020-10-23; 20(1):1606. PMID: 33097032  PMC7585195 Epub 2020/10/25. https://www.ncbi.nlm.nih.gov/pubmed/33097032

6.     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. Blood 2020 2020-07-30; 136(5):623-6. PMID: 32518950  PMC7393258 Epub 2020/06/11. https://www.ncbi.nlm.nih.gov/pubmed/32518950

7.     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 2021-01-08; 6(1):e379. PMID: 33409431  PMC7781296 this article. Epub 2021/01/08. https://www.ncbi.nlm.nih.gov/pubmed/33409431

8.     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. J Racial Ethn Health Disparities 2021 2021-02; 8(1):99-106. PMID: 32418182  PMC7669607 Epub 2020/05/18. https://www.ncbi.nlm.nih.gov/pubmed/32418182

9.     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 2021-04-20; 325(15):1513-23. PMID: 33877274  PMC8058640 Epub 2021/04/21. https://www.ncbi.nlm.nih.gov/pubmed/33877274

10.   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 Social and Psychological Factors Associated With Health Care Transition for Young Adults Living With Sickle Cell Disease. J Transcult Nurs 2021 2021-01-01; 32(1):21-9. PMID: 31889479  Epub 2020/01/01. https://www.ncbi.nlm.nih.gov/pubmed/31889479

11.   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 2020-12; 25(1):229-40. PMID: 32552526  PMC7440685 Epub 2020/06/20. https://www.ncbi.nlm.nih.gov/pubmed/32552526

12.   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 2021-01; 5(1):1-11. PMID: 33570619  PMC7805323 Epub 2021/02/12. https://www.ncbi.nlm.nih.gov/pubmed/33570619

13.   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. 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 2021-02; 61(2):603-16. PMID: 33231305  Epub 2020/11/25. https://www.ncbi.nlm.nih.gov/pubmed/33231305

14.   Faro E, Treadwell M, King A, Shook LM, Whiteman L, Ivy E, Hulihan M, Kavanagh P, Seik S, Oyeku S, Berns SA. A National Measurement Framework to Assess and Improve Sickle Cell Care in Four US Regions. Public Health Reports 2020 2020-07-08. PMID:  

15.   Friedman D, Dozor AJ, Milner J, D'Souza M, Talano JA, 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 2021-05-06. PMID: 33958740  Epub 2021/05/08. https://www.ncbi.nlm.nih.gov/pubmed/33958740

16.   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 2020-07-14; 9(7):e16319. PMID:  https://www.researchprotocols.org/2020/7/e16319

17.   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 2020-09-22; 4(18):4463-73. PMID: 32941646  PMC7509876 Epub 2020/09/18. https://www.ncbi.nlm.nih.gov/pubmed/32941646

18.   Hood AM, Strong H, Nwankwo C, Johnson Y, Peugh J, Mara CA, Shook LM, Brinkman WB, Real FJ, Klein MD, Hackworth R, Badawy SM, Thompson AA, Raphael JL, Yates AM, Smith-Whitley K, King AA, Calhoun C, Creary SE, Piccone CM, Hildenbrand AK, Reader SK, Neumayr L, Meier ER, Sobota AE, Rana S, Britto M, Saving KL, Treadwell M, Quinn CT, Ware RE, Crosby LE. Engaging Caregivers and Providers of Children With Sickle Cell Anemia in Shared Decision Making for Hydroxyurea: Protocol for a Multicenter Randomized Controlled Trial. JMIR Res Protoc 2021 2021-05-21; 10(5):e27650. PMID: 34018965  PMC8178738 Epub 2021/05/22. https://www.ncbi.nlm.nih.gov/pubmed/34018965

19.   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. Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol 2021 2021-05; 8(5):e323-e33. PMID: 33838113  Epub 2021/04/11. https://www.ncbi.nlm.nih.gov/pubmed/33838113

20.   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 2020-08-25; 4(16):3804-13. PMID: 32785684  PMC7448595 Epub 2020/08/14. https://www.ncbi.nlm.nih.gov/pubmed/32785684

21.   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 2020-11; 60(11):2508-16. PMID: 32812667  Epub 2020/08/20. https://www.ncbi.nlm.nih.gov/pubmed/32812667

22.   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 C. 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 2020-09; 95(9):1066-74. PMID: 32449965  PMC8141351 Epub 2020/05/26. https://www.ncbi.nlm.nih.gov/pubmed/32449965

23.   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 2021-03; 58(1):89-99. PMID: 33728251  PMC7932872 Epub 2021/03/18. https://www.ncbi.nlm.nih.gov/pubmed/33728251

24.   Luo L, King AA, Carroll Y, Baumann AA, Brambilla D, Carpenter CR, Colla J, Gibson RW, Gollan S, Hall G, Klesges L, Kutlar A, Lyon M, Melvin CL, Norell S, Mueller M, Potter MB, Richesson R, Richardson LD, Ryan G, Siewny L, Treadwell M, Zun L, Armstrong-Brown J, Cox L, Tanabe P. Electronic Health Record-Embedded Individualized Pain Plans for Emergency Department Treatment of Vaso-occlusive Episodes in Adults With Sickle Cell Disease: Protocol for a Preimplementation and Postimplementation Study. JMIR Res Protoc 2021 2021-04-16; 10(4):e24818. PMID: 33861209  PMC8087964 Epub 2021/04/17. https://www.ncbi.nlm.nih.gov/pubmed/33861209

25.   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 2020-11-27; 11(584950. PMID: 33240273  PMC7677137 Epub 2020/11/27. https://www.ncbi.nlm.nih.gov/pubmed/33240273

26.   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 2020-12-16; 14(Suppl 20):21. PMID: 33323100  PMC7739449 Epub 2020/12/17. https://www.ncbi.nlm.nih.gov/pubmed/33323100

27.   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 2020-09; 27(5):213-28. PMID:  https://www.mdedge.com/jcomjournal/article/228922/hematology/health-care-disparities-among-adolescents-and-adults-sickle

28.   Vissa M, Vichinsky E. Voxelotor for the treatment of sickle cell disease. Expert Rev Hematol 2021 2021-03; 14(3):253-62. PMID: 33602029  Epub 2021/02/20. https://www.ncbi.nlm.nih.gov/pubmed/33602029

ABSTRACTS

1.     Andemariam B, Bronte L, Gordeuk V, Howard J, Kanter J, Eleftheriou P, Pancham S, Hagar R, Clarke L, Blyden G, Lugthart S, Mant T, Fok H, Barroso F, Mason J, Yen J, Attie K, Lufkin J, Ballal R. The safety, pharmacokinetics & pharmacodynamic effects of IMR-687, a highly selective PDE inhibitor, in adults with sickle cell disease: phase-2a placebo-controlled & open-label extension studies.  26th Congress of the European Hematology Association. June 13-17,  2021 (Virtual).  Hemasphere  2021 (5):e566. 

2.     Belisário AR, Blatyta PF, Vivanco D, Oliveira CDL, Carneiro-Proietti AB, Sabino EC, Almeida-Neto C, Loureiro P, Máximo C, Mateos SOG, Flor-Park MV, Rodrigues DOW, Mota RA, Gonçalez 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.  https://www.sciencedirect.com/science/article/pii/S253113792030345X?via%3Dihub.

3.     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):S1:34-6.  https://doi.org/10.1182/blood-2020-138895.

4.     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 ASH Annual Meeting & Exposition. Dec 5-8, 2020.  Blood  2020 (136):S1:29-30.  http://dx.doi.org/10.1182/blood-2020-140540.

5.     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):S1:19-20.  http://dx.doi.org/10.1182/blood-2020-134269.

6.     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.  Pediatric Blood & Cancer  2021 (68):S3. 

7.     Fung E, Calvelli L, Cervantes M, Cadena K, Lal A. Bone Quality is Associated with Fragility Fracture in Patients with Hemoglobinopathies.  Annual Meeting of the American Society for Bone and Mineral Research. Virtual Event; September 11–15, 2020.  Journal of Bone and Mineral Research  2020 (35):SUPPL 1:241.  http://dx.doi.org/10.1002/jbmr.4206.

8.     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 ASH Annual Meeting & Exposition. Dec 5-8, 2020.  Blood  2020 (136):S1:33.  http://dx.doi.org/10.1182/blood-2020-143315.

9.     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 β-Thalassemia in the Believe Trial.  62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020.  Blood  2020 (136):S1:47-8.  https://doi.org/10.1182/blood-2020-136517.

10.  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):S1:26-7.  http://dx.doi.org/10.1182/blood-2020-141471.

11.  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):S1:19.  http://dx.doi.org/10.1182/blood-2020-140553.

12.   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.  Pediatric Blood & Cancer  2021 (68):S3. 

13.  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):S1:10-.  https://doi.org/10.1182/blood-2020-136193.

14.  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.   2020 (136):S1:poster.  https://ash.confex.com/ash/2020/webprogram/Paper136662.html.

15.   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.  26th Congress of the European Hematology Association (EHA). June 9-17, 2021 (virtual).   2021. 

16.   Kwiatkovvski J, Thompson AA, Walters MC, Krishnamurti L, Mapara M, Rifkin-Zenenberg S, Aygun B, Kasow K, Schmidt M, Nnamani M, Miller A, Gallagher ME, Chen R, Kim D, Goyal S, Kanter J, Tisdale J. Complete resolution of severe vaso-occlusive events and improved pathophysiology with lentiglobin gene therapy in sickle cell disease (SCD): ongoing phase 1/2 Hgb-206 Group C Study.  26th Congress of the European Hematology Association. June 13-17,  2021 (Virtual).  Hemasphere  2021 (5):e566. 

17.  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 β-Thalassemia: Results in Patients with up to 6 Years of Follow-up.  62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020.  Blood  2020 (136):S1:51-2.  https://doi.org/10.1182/blood-2020-135850.

18.  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):S1:1.  http://dx.doi.org/10.1182/blood-2020-142698.

19.  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):S1:15-6.  http://dx.doi.org/10.1182/blood-2020-139196.

20.  Mapara MY, Tisdale JF, Kanter J, Kwiatkowski JL, Krishnamurti L, Schmidt M, Miller AL, Pierciey FJ, Bonner M, Huang W, Ribeil JA, Thompson AA, Walters MC. Lentiglobin for sickle cell disease (SCD) gene therapy (GT): Updated results in group c patients from the phase 1/2 HGB-206 study.  The 46th Annual Meeting of the European Society for Blood and Marrow Transplantation. Aug 29-Sept 1 (virtual).  Bone Marrow Transplantation  2020 (55):78-9.  https://www.nature.com/articles/s41409-020-01119-3.

21.  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 ASH Annual Meeting & Exposition. Dec 5-8, 2020.  Blood  2020 (136):S1:37.  http://dx.doi.org/10.1182/blood-2020-140896.

22.  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 ASH Annual Meeting & Exposition. Dec 5-8, 2020.  Blood  2020 (136):S1:3.  http://dx.doi.org/10.1182/blood-2020-140743.

23.  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.  https://www.sciencedirect.com/science/article/pii/S266663672100405X.

24.  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):S1:32-.  https://doi.org/10.1182/blood-2020-140119.

25.  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):S1:16-7.  https://doi.org/10.1182/blood-2020-134940.

26.  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; poster 795.  https://ashpublications-org.ucsf.idm.oclc.org/blood/article/136/Supplement%201/31/470559/Higher-Hemoglobin-Levels-Achieved-with-Voxelotor.

27.  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 & Exposition. Dec 5-8, 2020.  Blood  2020 (136):S1:15.  http://dx.doi.org/10.1182/blood-2020-134717.

28.  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.  https://www.sciencedirect.com/science/article/pii/S2666636721000397.

29.  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 ASH Annual Meeting & Exposition. Dec 5-8, 2020.  Blood  2020 (136):S1:52.  http://dx.doi.org/10.1182/blood-2020-142809.

30.  Yannaki E, Locatelli F, Thrasher AJ, Tisdale JF, Orchard PJ, Duncan CN, Kühl JS, De Oliveira S, Sauer MG, Kulozik AE, Hongeng S, Mapara MY, Krishnamurti L, Hermine O, Blanche S, Aubourg P, Smith N, Shi W, Colvin RA, McNeil E, Ribeil JA, Cavazzana M, Walters MC, Williams DA. Safety of autologous hematopoietic stem cell transplantation with gene addition therapy for transfusion-dependent β-thalassemia, sickle cell disease, and cerebral adrenoleukodystrophy.  46th Annual Meeting of the European Society for Blood and Marrow Transplantation. Aug 29-Sept 1, 2020; .  Bone Marrow Transplantation  2020 (55):75-6.  http://dx.doi.org/10.1038/s41409-020-01119-3.

BOOK CHAPTERS

1.     King AA, Badawy SM, Panepinto J, Anie K, Jonassaint C, Treadwell M. Psychosocial Burden in Sickle Cell Disease. In: Gladwin MT, Kato GJ, Novelli EM, eds. Sickle Cell Disease 1st (April 23, 2021) ed. New York, NY: McGraw-Hill 2021:558-78. https://www.amazon.com/Sickle-Cell-Disease-Mark-Gladwin/dp/1260458598/ref=sr_1_1

2.     Leonard A, Tisdale JF, Krishnamurti L, Walters MC. Hematopoietic Stem Cell Transplantation in Sickle Cell Disease. In: Gladwin MT, Kato GJ, Novelli EM, eds. Sickle Cell Disease 1st (April 23, 2021) ed. New York, NY: McGraw-Hill 2021:492-527. https://www.amazon.com/Sickle-Cell-Disease-Mark-Gladwin/dp/1260458598/ref=sr_1_1

3.     Mekontso-Dessap A, Vichinsky E. Acute Chest Syndrome. In: Gladwin MT, Kato GJ, Novelli EM, eds. Sickle Cell Disease 1st (April 23, 2021) ed. New York, NY: McGraw-Hill 2021:228-43. https://www.amazon.com/Sickle-Cell-Disease-Mark-Gladwin/dp/1260458598/ref=sr_1_1

OTHER PUBLICATIONS

1.     Tong S, Vichinsky E. Iron Deficiency: Implications Before Anemia. Pediatr Rev 2021 2021-01; 42(1):11-20. PMID: 33386301  Epub 2021/01/03. https://www.ncbi.nlm.nih.gov/pubmed/33386301

2.     Walters MC. Induction of Fetal Hemoglobin by Gene Therapy. N Engl J Med 2021 2021-01-01; 384(3):284-5. PMID: 33471981  Epub 2021/01/21. https://www.ncbi.nlm.nih.gov/pubmed/33471981

3.     DeBaun M, Chou ST. Red blood cell transfusion in sickle cell disease: Indications and transfusion techniques. In: Mahoney DH, Vichinsky E, Tirnauer J, eds. UpToDate. Topic 90145: Version 41; May 4 2021. UpToDate, Inc. Waltham, MA. https://www.uptodate.com/contents/red-blood-cell-transfusion-in-sickle-cell-disease

4.     DeBaun M, Vichinsky E. Acute vaso-occlusive pain management in sickle cell disease. In: Mahoney DH, Tirnauer J, eds. UpToDate. Topic 7144: Version 59; July 1 2021.  UpToDate, Inc. Waltham, MA. https://www.uptodate.com/contents/vaso-occlusive-pain-management-in-sickle-cell-disease

5.     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 78; Dec 9 2020.  UpToDate, Inc. Waltham, MD. https://www.uptodate.com/contents/overview-of-the-management-and-prognosis-of-sickle-cell-disease

6.     Heeney M, Mahoney DH. The acute chest syndrome in children and adolescents with sickle cell disease. In: Mallory GB, Vichinsky E, Tirnauer J, eds. UpToDate. Topic 5913: Version 48.0; June 3 2020. UpToDate, Inc. Waltham, MA. https://www.uptodate.com/contents/the-acute-chest-syndrome-in-children-and-adolescents-with-sickle-cell-disease

7.     Treadwell M, Hankins JS, King A. Sickle cell disease (SCD) in adolescents and young adults (AYA): Transition from pediatric to adult care. In: Debaun M, Tirnauer J, eds. UpToDate. Topic 112919; Version 15.0; Oct 5, 2021.  UpToDate, Inc. Waltham, MA. https://www.uptodate.com/contents/sickle-cell-disease-scd-in-adolescents-and-young-adults-aya-transition-from-pediatric-to-adult-care

8.     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

9.     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. https://www.uptodate.com/contents/overview-of-the-clinical-manifestations-of-sickle-cell-disease

10.   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. http://www.uptodate.com/contents/variant-sickle-cell-syndromes

11.   Vichinsky E. Sickle Cell Trait. In: Mahoney DH, Tirnauer J, eds. UpToDate. Topic 7145: Version 54; Dec 2, 2020. UpToDate, Inc.  Waltham, MA. https://www.uptodate.com/contents/sickle-cell-trait

12.   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. https://www.uptodate.com/contents/diagnosis-of-sickle-cell-disorders

13.   Vichinsky E. Sickle cell disease: Pregnancy considerations. In: Simpson L, DeBaun M, Tirnauer J, Barss V, eds. UpToDate. Topic 7139: Version 42.0; Sept 29, 2021. UpToDate, Inc. Waltham, MA. https://www.uptodate.com/contents/sickle-cell-disease-pregnancy-considerations