Recent Publications (2020-2022)

PEER-REVIEWED ARTICLES:

  1. Bansal D, Lal A. Iron Metabolism, Hemolytic Anemia, and Thalassemia. Indian J Pediatr 2020 2021-01; 87(1):56-7. PMID: 31828597 Epub 2019/12/13. https://www.ncbi.nlm.nih.gov/pubmed/31828597
  2. Berliner C, Wang ZJ, Singer ST, Grosse R, McDonough RV, Padua E, Yuan Q, Weyhmiller M, James E, Vichinsky E, Adam G, Yamamura J, Bannas P, Fischer R, Schoennagel BP. Anterior Pituitary Volume in Patients with Transfusion Dependent Anemias: Volumetric Approaches and Relation to Pituitary MRIR2. Clin Neuroradiol 2021 2021-10-28. PMID: 34709410 Epub 2021/10/29. https://www.ncbi.nlm.nih.gov/pubmed/34709410
  3. Cappellini MD, Viprakasit V, Taher AT, Georgiev P, Kuo KHM, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Forni GL, Perrotta S, Khelif A, Lal A, Kattamis A, Vlachaki E, Origa R, Aydinok Y, Bejaoui M, Ho PJ, Chew LP, Bee PC, Lim SM, Lu MY, Tantiworawit A, Ganeva P, Gercheva L, Shah F, Neufeld EJ, Thompson A, Laadem A, Shetty JK, Zou J, Zhang J, Miteva D, Zinger T, Linde PG, Sherman ML, Hermine O, Porter J, Piga A. Believe Investigators A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent beta-Thalassemia. N Engl J Med 2020 2020-03-26; 382(13):1219-31. PMID: 32212518 https://www.ncbi.nlm.nih.gov/pubmed/32212518
  4. Chapin J, Cohen AR, Neufeld EJ, Vichinsky E, Giardina PJ, Boudreaux J, Le BC, Kenney K, Trimble S, Thompson AA. An update on the US adult thalassaemia population: a report from the CDC thalassaemia treatment centres. Br J Haematol 2022 Jan; 196(2):380-9. PMID: 34775608 Epub 2021/11/15. https://www.ncbi.nlm.nih.gov/pubmed/34775608
  5. Erlich HA, López-Peña C, Carlberg KT, Shih S, Bali G, Yamaguchi KD, Salamon H, Das R, Lal A, Calloway CD. Non-Invasive Prenatal Test for β-Thalassemia and Sickle Cell Disease Using Probe Capture Enrichment and Next-Generation Sequencing of DNA in Maternal Plasma. The Journal of Applied Laboratory Medicine 2021. PMID:  http://dx.doi.org/10.1093/jalm/jfab118
  6. Fung EB, Ahmad T, Killilea DW, Hussain R, Lal A. Zinc supplementation improves markers of glucose homeostasis in thalassaemia. Br J Haematol 2020 2020-06-24; 190(3):e162-e6. PMID: 32488893 Epub 2020/06/04. https://www.ncbi.nlm.nih.gov/pubmed/32488893
  7. Goldberg EK, Fung EB. Precision of the Hologic DXA in the Assessment of Visceral Adipose Tissue. J Clin Densitom 2020 2020-10; 23(4):664-72. PMID: 30992223 PMC6754313 Epub 2019/04/18. https://www.ncbi.nlm.nih.gov/pubmed/30992223
  8. Goldberg EK, Lal A, Fung EB. Nutrition in Thalassemia: A Systematic Review of Deficiency, Relations to Morbidity, and Supplementation Recommendations. J Pediatr Hematol Oncol 2021 2021-09-06. PMID: 34486568 Epub 2021/09/07. https://www.ncbi.nlm.nih.gov/pubmed/34486568
  9. Lal A. Challenges in chronic transfusion for patients with thalassemia. Hematology Am Soc Hematol Educ Program 2020 2020-12-04; 2020(1):160-6. PMID: 33275743 PMC7727587 Epub 2020/12/05. https://www.ncbi.nlm.nih.gov/pubmed/33275743
  10. Lal A. Iron in Health and Disease: An Update. Indian J Pediatr 2020 2020-01; 87(1):58-65. PMID: 31520313 Epub 2019/09/15. https://www.ncbi.nlm.nih.gov/pubmed/31520313
  11. Lal A, Bansal D. Thalassemia: Common Clinical Queries in Management. Indian J Pediatr 2020 2020-01; 87(1):75-81. PMID: 31620986 Epub 2019/10/18. https://www.ncbi.nlm.nih.gov/pubmed/31620986
  12. Lal A, Wong T, Keel S, Pagano M, Chung J, Kamdar A, Rao L, Ikeda A, Puthenveetil G, Shah S, Yu J, Vichinsky E. The transfusion management of beta thalassemia in the United States. Transfusion 2021 2021-08-29; 61(10):3027-39. PMID: 34453453 Epub 2021/08/29. https://www.ncbi.nlm.nih.gov/pubmed/34453453
  13. Locatelli F, Thompson AA, Kwiatkowski JL, Porter JB, Thrasher AJ, Hongeng S, Sauer MG, Thuret I, Lal A, Algeri M, Schneiderman J, Olson TS, Carpenter B, Amrolia PJ, Anurathapan U, Schambach A, Chabannon C, Schmidt M, Labik I, Elliot H, Guo R, Asmal M, Colvin RA, Walters MC. Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia. The New England Journal of Medicine 2022 386(5):415-27. PMID:  http://dx.doi.org/10.1056/NEJMoa2113206
  14. MacKenzie TC, Amid A, Angastiniotis M, Butler C, Gilbert S, Gonzalez J, Keller RL, Kharbanda S, Kirby-Allen MA, Koenig BA, Kyono W, Lal A, Lianoglou BR, Norton ME, Ogasawara KK, Panchalee T, Rosner M, Schwab ME, Thompson AA, Waye JS, Vichinsky E. Consensus statement for the perinatal management of patients with alpha thalassemia major. Blood Adv 2021 2021-11-09. PMID: 34749399 Epub 2021/11/09. https://www.ncbi.nlm.nih.gov/pubmed/34749399
  15. Musallam KM, Vitrano A, Meloni A, Addario Pollina S, Di Marco V, Hussain Ansari S, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Karimi M, El-Beshlawy A, Hajipour M, Bonifazi F, Vichinsky E, Taher AT, Sankaran VG, Maggio A. International Working Group on Thalassemia Primary HBB gene mutation severity and long-term outcomes in a global cohort of beta-thalassaemia. Br J Haematol 2022 Jan; 196(2):414-23. PMID: 34697800 Epub 2021/10/27. https://www.ncbi.nlm.nih.gov/pubmed/34697800
  16. Polgreen LE, Lund TC, Braunlin E, Tolar J, Miller BS, Fung E, Whitley CB, Eisengart JB, Northrop E, Rudser K, Miller WP, Orchard PJ. Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation. Pediatr Res 2020 2020-01; 87(1):104-11. PMID: 31434105 PMC6960090 Epub 2019/08/23. https://www.ncbi.nlm.nih.gov/pubmed/31434105
  17. Singer ST, Fischer R, Allen I, Lal A, Vichinsky E, Yuan Q, Wang ZJ. Pituitary iron and factors predictive of fertility status in transfusion dependent thalassemia. Haematologica 2021 2021-06-01; 106(6):1740-4. PMID: 33121232 PMC8168491 Epub 2020/10/31. https://www.ncbi.nlm.nih.gov/pubmed/33121232
  18. Vitrano A, Meloni A, Addario Pollina W, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Hussain Ansari S, Filosa A, Ricchi P, Ceci A, Daar S, Titi Singer S, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Vichinsky E, Maggio A. A complication risk score to evaluate clinical severity of thalassaemia syndromes. Br J Haematol 2021 2021-02; 192(3):626-33. PMID: 33216983 Epub 2020/11/21. https://www.ncbi.nlm.nih.gov/pubmed/33216983

 

ABSTRACTS:

  1. Cappellini MD, Hermine O, Piga A, Viprakasit V, Georgiev P, Kuo KHM, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Forni GL, Perrotta S, Khelif A, Lal A, Kattamis A, Shah F, Porter J, Laadem A, Shetty JK, Kuo WL, Zhang J, Miteva D, Zinger T, Sinsimer D, Louis C, Linde PG, Taher AT. Assessment of response to luspatercept by B-globin genotype in adult patients with B-thalassemia in the believe trial. 25th Congress of the European Hematology Association (EHA). June 11-22, 2020 (virtual). HemaSphere 2020 (4):108-9. http://dx.doi.org/10.1097/HS9.0000000000000404.
  2. 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.
  3. 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.
  4. 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 YO, Miteva D, Zinger T, Tang D, Backstrom JT, Porter JB. Assessment of the Longitudinal Effects of Luspatercept on Iron Overload and Iron Chelation Therapy Usage in Adults with ?-thalassaemia Enrolled in the Phase 3 BELIEVE Study. 61st Annual Scientific Meeting of the British Society for Haematology. April 25-28, 2021 (virtual). British Journal of Haematology 2021 (193):SUPPL 1:65-6. http://dx.doi.org/10.1111/bjh.17491.
  5. Hongeng S, Thompson AA, Kwiatkowski JL, Locatelli F, Porter JB, Rasko J, Cavazzana M, Sauer MG, Thrasher AJ, Thuret I, Lal A, Kulozik AE, Yannaki E, Schmidt M, Chen Y, Guo R, Liu W, Colvin RA, Walters MC. Efficacy and Safety of Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-thalassemia) Gene Therapy in 60 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed for up to 6 Years Post-Infusion. TCT Meetings of ASTCT and CIBMTR. Feb 8-12, 2021. Transplantation and Cellular Therapy 2021 (27):3, Supplement: S1. https://www.sciencedirect.com/science/article/pii/S2666636721000270.
  6. Kulozik AE, Kunz J, Thuret I, Thompson AA, Kwiatkowski JL, Porter JB, Hongeng S, Yannaki E, Sauer MG, Thrasher AJ, Lal A, Guo R, Colvin RA, Walters MC, Locatelli F.  Interim results of betibeglogene autotemcel gene-additiontherapy in pediatric patients with transfusion-dependent β-thalassemia (TDT) treated in the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies. Oncology Research and Treatment   2021 (44):SUPPL 2:8. http://dx.doi.org/10.1159/000518417.
  7. Kulozik A, Thuret I, Kwiatkovvski J, Thompson AA, Porter JB, Hongeng S, Yannaki E, Sauer M, Thrasher A, A. L, Guo R, Liu W, Colvin R, Walters MC, Locatelli F. Interim results of betibeglogene autotemcel gene therapy in pediatric patients with transfusion-dependent Beta-thalassemia (TDT) treated in the phase 3 Northstar-2 and Northstar-3 studies. 26th Congress of the European Hematology Association. June 13-17, 2021 (Virtual). Hemasphere 2021 (5):e566.
  8. Kulozik AE, Thuret I, Thompson AA, Kwiatkowski JL, Porter JB, Hongeng S, Yannaki E, Sauer MG, Thrasher AJ, Lal A, Du L, Colvin RA, Walters MC, Locatelli F. Betibeglogene autotemcel (beti-cel) gene therapy for the treatment of Transfusion-Dependent β-Thalassemia (TDT): Updated long-term efficacy and safety results. Virtual; June 9-17, 2021. 26th Annual Congress of the European Hematology Association, taking place June 9-17, 2021. Oncology Research and Treatment 2021 (44):SUPPL 2:8-9. http://dx.doi.org/10.1159/000518417.
  9. Kuo K, Layton DM, A. L, Al-Samkari H, Bhatia J, Tong B, Lynch M, Uhlig K, Vichinsky E. Results from a phase 2, open-label, multicenter study of the oral pyruvate kinase activator mitapivat in adults with non–transfusion dependent alpha- or beta-thalassemia. 26th Congress of the European Hematology Association. June 13-17, 2021 (Virtual). Hemasphere 2021 (5):e566.
  10. 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. Blood 2020 (136):S1:poster. https://ash.confex.com/ash/2020/webprogram/Paper136662.html.
  11. Kuo K, Layton DM, Lal A, Al-Samkari H, Tai F, Lynch M, Uhlig K, Vichinsky EP. Proof of concept for the oral pyruvate kinase activator mitapivat (ag-348) in adults with non–transfusion-dependent thalassemia: first results from an ongoing, phase 2, open-label, multicenter study. 25th Congress of the European Hematology Association (EHA). June 11-22, 2020 (virtual). Hemasphere 2020 (4):109-10. https://library.ehaweb.org/eha/2020/eha25th/295116/kevin.hm.kuo.proof.of.concept.for.the.oral.pyruvate.kinase.activator.mitapivat.html.
  12. 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.
  13. Kuo KHM, Layton DM, Lal A, Al-Samkari H, Bhatia J, Kosinski PA, Tong B, Lynch M, Uhlig K, Vichinsky E. Long-Term Efficacy and Safety of the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia. 63rd ASH Annual Meeting and Exposition. Atlanta, GA; Dec 11-14, 2021. Blood 2021 (138):S1:576. https://doi.org/10.1182/blood-2021-150386.
  14. Kuo KHM, Singer S, Sheth S, Al-Samkari H, Blyden G, Bronte-Hall L, Pace BS, Kutlar A, Kuypers FA, Faller AD, Nouraie SM, Terse P, Jin H, Xu X, Alimardanov A, Sytkowski AJ, Haugabook S, Perrine SP. The Benefits Trial of PB-04 to Evaluate Safety, PK, and Preliminary Efficacy in Inducing Fetal Globin Expression in Beta Thalassemia Intermedia and Sickle Cell Disease. 63rd ASH Annual Meeting and Exposition Atlanta, GA; Dec 11-14, 2021. Blood 2021 (138):S1:4159. http://dx.doi.org/10.1182/blood-2021-148909.
  15. Kwiatkowski JL, Locatelli F, Walters MC, Porter JB, Hongeng S, Yannaki E, Kulozik AE, Sauer MG, Thrasher AJ, Thuret I, Lal A, Guo R, Colvin RA, Gruppioni K, Thompson AA. Improvement in Health-Related Quality of Life Following Treatment with Betibeglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia Enrolled in Phase 3 Studies. 63rd ASH Annual Meeting and Exposition Atlanta, GA; Dec 11-14, 2021. Blood 2021 (138):S1:3085. http://dx.doi.org/10.1182/blood-2021-150392
  16. Kwiatkowski J, Walters M, Lal A., Locatelli F, Porter J, Hongeng S, Thuret I, Yannaki E, Kulozik A, Sauer M, Thrasher A, Guo R, Liu W, Colvin R, Thompson A. BETI-CEL gene therapy outcomes in pediatric patients with transfusion-dependent β-thalassemia. American Society Pediatric Hematology Oncology (ASPHO) Conference. April 20-23, 2021; Portland, OR. Pediatric Blood & Cancer 2021 (68):S3.
  17. Kwiatkowski J, Walters M, Locatelli F, Lal A, Yannaki E, Kulozik A, Sauer M, Porter J, Thuret I, Hongeng S, Thrasher A, Olson T, Schneiderman J, Elliot H, Tao G, Liu W, Colvin R, Thompson A. Lentiglobin gene therapy in pediatrics, adolescents, adults with transfusion-dependent Beta-thalassemia. 2020 ASPHO Conference. (online). Pediatric Blood and Cancer 2020 (67):SUPPL 2. http://dx.doi.org/10.1002/pbc.28321.
  18. 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.
  19. Lal A, Brown RCC, Coates TD, Kalfa TA, Kwiatkowski JL, Brevard J, Potter V, Wood KW, Sheth S. Trial in Progress: A Phase 2, Open-Label Study Evaluating the Safety and Efficacy of the PKR Activator Etavopivat (FT-4202) in Patients with Thalassemia or Sickle Cell Disease. 63rd ASH Annual Meeting and Exposition; Atlanta, GA; Dec 11-14, 2021. Blood 2021 (138):4162. http://dx.doi.org/10.1182/blood-2021-145752.
  20. Lal A, Voskaridou E, Flevari P, Taher A, Chew LP, Valone F, Gupta S, Viprakasit V. A hepcidin mimetic, PTG-300, demonstrates pharmacodynamic effects indicating reduced iron availability in transfusion-dependent beta-thalassemia subjects. 25th Congress of the European Hematology Association (EHA). June 11-22, 2020 (virtual). HemaSphere 2020 (4):110. http://dx.doi.org/10.1097/HS9.0000000000000404.
  21. Lianoglou BR, Gonzalez V, Velez JG, Norton ME, Chen B, Finley B, Hirata G, Ogasawara K, MacKenzie TC, Brown RN, Sanchez-Embrey E, Shaw SW, Foe M, Bitanga M, Chien M, Stumbaugh T, Thompson AA, Viprakasit V, Volanakis EJ, Vichinsky E. Impact of in utero transfusions in fetuses with hydrops fetalis due to alpha thalassemia. 40th Annual Meeting Society for Maternal Fetal Medicine. Dallas, TX; Feb 3-8, 2020. American Journal of Obstetrics and Gynecology 2020 (222):1:S300-S1. http://dx.doi.org/10.1016/j.ajog.2019.11.474.
  22. Locatelli F, Kwiatkowski J, Walters M, Hongeng S, Rasko J, Cavazzana M, Schmidt M, Chen Y, Colvin R, Thompson A, Locatelli F, Kwiatkowski J, Walters M, Hongeng S, Rasko J, Cavazzana M, Schmidt M, Chen Y, Colvin R, Thompson A. Durable Clinical Outcomes Following Betibeglogene Autotemcel (BETI-CEL) Gene Therapy with Up to 6 Years of Follow-Up in Patients with Transfusion-Dependent Β-Thalassemia (TDT). 47th Annual Meeting of the European Society for Blood and Marrow Transplantation. March 14-17, 2021 (virtual). Bone Marrow Transplantation 2021 (56):69-70. https://www.nature.com/articles/s41409-021-01342-6.pdf.
  23. Locatelli F, Kwiatkowski JL, Thompson AA, Yannaki E, Kulozik AE, Sauer MG, Porter JB, Thuret I, Hongeng S, Lal A, Thrasher AJ, Elliot H, Tao G, Liu W, Colvin RA, Walters MC. Clinical outcomes following autologous hematopoietic stem cell transplantation with lentiglobin gene therapy in the phase 3 Northstar-2 and Northstar-3 studies for transfusion-dependent β-thalassemia. The 46th Annual Meeting of the European Society for Blood and Marrow Transplantation. Aug 29-Sept1, 2020 (virtual). Bone Marrow Transplantation 2020 (55):71-2. http://dx.doi.org/10.1038/s41409-020-01119-3.
  24. Locatelli F, Kwiatkowski JL, Thompson AA, Yannaki E, Kulozik AE, Sauer MG, Porter JB, Thuret I, Hongeng S, Lal A, Thrasher AJ, Tao G, Liu W, Colvin RA, Walters MC. Clinical Outcomes Following Autologous Hematopoietic Stem Cell Transplantation with Betibeglogene Autotemcel Gene Therapy in the Phase 3 Northstar-2 and Northstar-3 Studies for Transfusiondependent b-Thalassemia (TDT). 22nd Congress of the European Hematology Association. June 11-21, 2020 (virtual). Haematologica 2020 (105):SUPPL 2:S36. https://journals.lww.com/hemasphere/citation/2020/06001/abstract_book__25th_congress_of_the_european.1.aspx.
  25. Locatelli F, Kwiatkowski JL, Thompson AA, Yannaki E, Kulozik AE, Sauer MG, Porter JB, Thuret I, Hongeng S, Lal A, Thrasher AJ, Tao G, Liu W, Colvin RA, Walters MC. Clinical Outcomes Following Autologous Hematopoietic Stem Cell Transplantation with Betibeglogene Autotemcel Gene Therapy in the Phase 3 Northstar-2 and Northstar-3 Studies for Transfusiondependent b-Thalassemia (TDT). XVI Congress of the Italian Society of Experimental Hematology. October 15-17, 2020; Napoli, Italy. Haematologica 2020 (105):SUPPL 2:S36. https://haematologica.org/issue/view/373.
  26. Locatelli F, Kwiatkowski JL, Walters MC, Porter JB, Hongeng S, Yannaki E, Kulozik AE, Sauer MG, Thrasher AJ, Thuret I, Lal A, Guo R, Liu W, Colvin RA, Thompson AA. Betibeglogene autotemcel in patients with transfusion-dependent β-thalassemia: Updated results from HGB-207 (NORTHSTAR-2) and HGB-212 (NORTHSTAR-3). 26th Congress of the European Hematology Association (EHA). June 9-27, 2021 (virtual). HemaSphere 2021 (5):SUPPL 2:91. http://dx.doi.org/10.1097/HS9.0000000000000566.
  27. 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.
  28. 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.
  29. Maggio A, Vitrano A, Meloni A, Pollina WA, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Vichinsky E. Evidence for three distinct classes of phenotype severity in betathalassaemia. 25th Congress of the European Hematology Association (EHA). June 11-22, 2020 (virtual). HemaSphere 2020 (4):1059-60. http://dx.doi.org/10.1097/HS9.0000000000000404.
  30. Porter J, Shah F, Drasar E, Hill QA, Viprakasit V, Taher A, Georgiev P, Kuo KHM, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Perrotta S, Khelif A, Lal A, Kattamis A, Laadem A, Shetty JK, Linde PG, Hermine O, Piga A, Cappellini MD. Effects of luspatercept on iron overload and impact on responders to luspatercept: Results from the BELIEVE Trial. 60th Annual Scientific Meeting of the British Society for Haematology. April 27-29, 2020; Birmingham, UK. British Journal of Haematology 2020 (189):136-7. http://dx.doi.org/10.1111/bjh.16638.
  31. Porter J, Thompson A, Walters MC, Kwiatkovvski J, Hongeng S, Sauer M, Thrasher A, Thuret I, Tao G, Colvin R, Locatelli F. Improvement in erythropoiesis in patients with transfusion-dependent Beta-thalassemia following treatment with Betibeglogene Autotemcel (Lentiglobin for Beta-thalassemia) in the Phase 3 HGB-207 Study. 25th Congress of the European Hematology Association (EHA). June 11-22, 2020 (virtual). HemaSphere 2020 (4):S296. https://journals.lww.com/hemasphere/Citation/2020/06001/Abstract_Book__25th_Congress_of_the_European.1.aspx
  32. Porter JB, Locatelli F, Kwiatkowski JL, Thompson AA, Yannaki E, Kulozik AE, Sauer MG, Thuret I, Hongeng S, Lal A, Thrasher AJ, Elliot H, Tao G, Liu W, Colvin RA, Walters MC. Clinical outcomes following autologous haematopoietic stem cell transplantation with LentiGlobin gene therapy in the phase 3 Northstar-2 and Northstar-3 studies for transfusion-dependent b-thalassaemia. British Society for Haematology Annual Scientific Meeting. Nov 9-14, 2020 (virtual). British Journal of Haematology 2020 (189):S1:39-40. http://dx.doi.org/10.1111/bjh.16638.
  33. Schneiderman J, Thompson AA, Walters MC, Kwiatkowski JL, Kulozik AE, Sauer MG, Porter JB, Thuret I, Hongeng S, Lal A, Thrasher AJ, Yannaki E, Elliot H, Tao G, Liu W, Colvin RA, Locatelli F. Interim Results from the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy (LentiGlobin) for the Treatment of Transfusion-Dependent Beta-Thalassemia. American Society for Transplantation and Cellular Therapy. Houston, TX; Feb 20-24, 2020. Biology of Blood and Marrow Transplantation 2020 (26):3:S87-S8. http://dx.doi.org/10.1016/j.bbmt.2019.12.588.
  34. Schwab ME, Lianoglou BR, Ganno D, Gonzalez-Velez JM, Vichinsky E, MacKenzie TC. Long-Term Neurodevelopmental Outcomes After Fetal Transfusions for Hydrops Fetalis. American Pediatric Surgical Association (APSA) Annual Meeting. May 20-22, 2021 (virtual).
  35. Taher AT, Viprakasit V, Cappellini MD, Hermine O, Georgiev P, Kuo KHM, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Forni GL, Perrotta S, Khelif A, Lal A, Kattamis A, Shah F, Porter J, Laadem A, Shetty JK, Kuo WL, Zhang J, Miteva D, Zinger T, Sinsimer D, Louis C, Linde PG, Piga A. Assessment of longer-term efficacy and safety in the phase 3 believe trial of luspatercept to treat anemia in patients (pts) with b-thalassemia. 25th Congress of the European Hematology Association (EHA). June 11-22, 2020 (virtual). HemaSphere 2020 (4):717-8. http://dx.doi.org/10.1097/HS9.0000000000000404.
  36. Thompson AA, Kwiatkowski JL, Porter JB, Hongeng S, Yannaki E, Kulozik AE, Sauer MG, Thrasher AJ, Thuret I, Lal A, Guo R, Liu W, Colvin RA, Walters MC, Locatelli F. Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia. 62nd ASH Annual Meeting & Exposition. Dec 5-8,, 2020. Blood 2020 (136):S1:52-4. https://doi.org/10.1182/blood-2020-135857.
  37. Thuret I, Thompson A, Kwiatkovvski J, Porter J, Hongeng S, Yannaki E, Kulozik A, Sauer M, Thrasher A, Lal A, Guo R, Liu W, Colvin R, Walters M. Interim Results of Betibeglogene Autotemcel Gene Therapy in Pediatric Patients with Transfusiondependent Β-Thalassemia (TDT) Treated in the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) Studies. American Society Pediatric Hematology Oncology (ASPHO) Conference. April 20-23, 2021; Portland, OR. Pediatric Blood & Cancer 2021 (68):S3.
  38. Thuret I, Thompson A, Kwiatkowski J, Porter J, Hongeng S, Yannaki E, Kulozik A, Sauer M, Thrasher A, Lal A, Guo R, Liu W, Colvin R, Walters M, Locatelli F. Interim Results of Betibeglogene Autotemcel Gene Therapy in Pediatric Patients with Transfusiondependent Β-Thalassemia (TDT) Treated in the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) Studies. 47th Annual Meeting of the European Society for Blood and Marrow Transplantation. March 14-17, 2021 (virtual). Bone Marrow Transplantation 2021 (56):64-5. http://dx.doi.org/10.1038/s41409-021-01342-6.
  39. Walters MC, Chui DHK, Farrell JJ, Lal A, Locatelli F, Kwiatkowski JL, Porter JB, Sauer MG, Thuret I, Hongeng S, Kulozik AE, Thrasher AJ, Yannaki E, Yang J, Whitney D, Petrusich A, Colvin RA, Thompson AA. Response of Patients with Transfusion-Dependent Beta-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for Beta-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers. 62nd ASH Annual Meeting & Exposition. Dec 5-8, 2020. Blood 2020 (136):S1:1-3. https://doi.org/10.1182/blood-2020-137642.
  40. Walters MC, Kwiatkowski JL, Porter JB, Hongeng S, Yannaki E, Kulozik AE, Sauer MG, Thrasher AJ, Thuret I, Lal A, Guo R, Liu W, Colvin RA, Locatelli F, Thompson AA. Safety and Efficacy Outcomes in Pediatric Patients with Transfusion-Dependent β-Thalassemia (TDT) Receiving Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-thalassemia) Gene Therapy in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies. TCT Meetings of ASTCT and CIBMTR. Feb 8-12, 2021. Transplantation and Cellular Therapy 2021 (27):3, Supplement:S112-S4. https://www.sciencedirect.com/science/article/pii/S2666636721001482.
  41. Walters MC, Locatelli F, Thrasher AJ, Tisdale JF, Orchard PJ, Duncan CN, Kühl JS, De Oliveira SN, Sauer MG, Kulozik AE, Yannaki E, Hongeng S, Mapara MY, Krishnamurti L, Hermine O, Blanche S, Aubourg P, Smith NJC, Shi W, Colvin RA, McNeil E, Ribeil JA, Cavazzana M, Williams DA. Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent Beta-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy. American Society for Transplantation and Cellular Therapy. Houston, TX; Feb 20-24, 2020. Biology of Blood and Marrow Transplantation 2020 (26):3:S38-S9. http://dx.doi.org/10.1016/j.bbmt.2019.12.104.
  42. Walters MC, Smith AR, Schiller GJ, Esrick EB, Williams DA, Gogoleva T, Rouy D, Cockroft BM, Vercellotti GM. Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia. 63rd Annual Meeting and Exposition. Atlanta, GA; Dec 11-14, 2021. Blood 2021 (138):S1:3974. http://dx.doi.org/10.1182/blood-2021-147907.
  43. Yannaki E, Locatelli F, Kulozik AE, Porter JB, Thuret I, Sauer MG, Lal A, Liu W, Colvin RA, Thompson AA, Kwiatkowski JL. Betibeglogene autotemcel (Lentiglobin) in patients with transfusion-dependent b-thalassemia and b0/b0, b+IVS-I-110/b+IVS-I-110, or b0/b+ivs-i-110 genotypes: Updated results from the HGB-212 study. 25th Congress of the European Hematology Association (EHA). June 11-22, 2020 (virtual). HemaSphere 2020 (4):692-3. http://dx.doi.org/10.1097/HS9.0000000000000404.
  44. Yannaki E, Locatelli F, Kwiatkowski JL, Walters MC, Porter JB, Hongeng S, Kulozik AE, Sauer MG, Thrasher AJ, Thuret I, Lal A, Cavazzana M, Rasko JE, Guo R, Liu W, Colvin RA, Thompson AA. Betibeglogene autotemcel gene therapy for the treatment of transfusion-dependent Β-Thalassemia: Updated long-term efficacy and safety results. 26th Congress of the European Hematology Association (EHA). June 9-27, 2021 (virtual). HemaSphere 2021 (5):SUPPL 2:86-7. http://dx.doi.org/10.1097/HS9.0000000000000566.
  45. 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. Fritch Lilla SA, Singer ST, Vichinsky EP. Thalassemia Disorders in the Adolescent Female. In: Srivaths LV, ed. Hematology in the Adolescent Female. Cham: Springer International Publishing; 2020:205-16. https://doi.org/10.1007/978-3-030-48446-0_19I
  2. Yamashita R, Haines D, Mednick L, Murphy W. Psychological Support. In: Cappellini MD, Farmakis D, Porter J, Taher A, eds. 2021 Guidelines for the Management of Transfusion Dependent Thalassemia (TDT). Chapter 15: Thalassemia International Federation; 2021. https://www.thalassemia.org/boduw/wp-content/uploads/2021/06/TIF-2021-Guidelines-for-Mgmt-of-TDT.pdf

OTHER PUBLICATIONS:

  1. El-Haj N, HarnEnz Z, Singer ST, Kim S, Cham E, Ling N, Karakas SP, Nguyen VA, Petru AM. From Microcytosis to Macrodiagnosis. Pediatrics 2021 Aug; 148(2). PMID: 34326177 Epub 2021/07/31. https://www.ncbi.nlm.nih.gov/pubmed/34326177
  2. Lal A, Lianoglou BR, Gonzalez-Velez JM. Alpha Thalassemia Major: Prenatal and Postnatal Management. In: Vichinsky E, ed. UpToDate. Topic 131582; Version 2.0; Oct 25, 2021. UpToDate, Inc. Waltham, MA. https://www-uptodate-com.ucsf.idm.oclc.org/contents/alpha-thalassemia-major-prenatal-and-postnatal-management
  3. Musallam KM, Vitrano A, Meloni A, Pollina WA, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Sankaran VG, Vichinsky E, Taher AT, Maggio A. Survival and causes of death in 2,033 patients with non-transfusion-dependent β-thalassemia. Haematologica 2021 2021-09-01; 106(9):2489-92. PMID: 33882642 Epub 2021/04/23.
  4. Musallam KM, Vitrano A, Meloni A, Pollina SA, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Sankaran VG, Vichinsky E, Taher AT, Maggio A. International Working Group on Thalassemia Risk of mortality from anemia and iron overload in nontransfusion-dependent beta-thalassemia. Am J Hematol 2022 Feb 1; 97(2):E78-E80. PMID: 34862982 Epub 2021/12/05. https://www.ncbi.nlm.nih.gov/pubmed/34862982
  5. Walters MC. Induction of Fetal Hemoglobin by Gene Therapy. N Engl J Med 2021 2021-01-21; 384(3):284-5. PMID: 33471981 Epub 2021/01/21. https://www.ncbi.nlm.nih.gov/pubmed/33471981

Rev. 2/14/2022