Beta thalassemia

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Tillman Benjamin-2.jpg
Benjamin Tillman, MD
Vanderbilt University
Nashville, TN, USA

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2 regimens on this page
2 variants on this page

All lines of therapy

Exagamglogene autotemcel monotherapy


Study Dates of enrollment Evidence
Locatelli et al. 2024 (CLIMB THAL-111) 2018-09-10 to NR Non-randomized phase 3 (RT)

Targeted therapy

One course


  1. CLIMB THAL-111: Locatelli F, Lang P, Wall D, Meisel R, Corbacioglu S, Li AM, de la Fuente J, Shah AJ, Carpenter B, Kwiatkowski JL, Mapara M, Liem RI, Cappellini MD, Algeri M, Kattamis A, Sheth S, Grupp S, Handgretinger R, Kohli P, Shi D, Ross L, Bobruff Y, Simard C, Zhang L, Morrow PK, Hobbs WE, Frangoul H; CLIMB THAL-111 Study Group. Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia. N Engl J Med. 2024 May 9;390(18):1663-1676. Epub 2024 Apr 24. link to original article PubMed NCT03655678

Luspatercept monotherapy


Study Dates of enrollment Evidence Comparator Comparative Efficacy
Cappellini et al. 2020 (BELIEVE) 2016-07 to 2017-06 Phase 3 (E-RT-esc) Placebo Superior primary endpoint

Growth factor therapy

21-day cycle for at least 16 cycles


  1. BELIEVE: 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 β-Thalassemia. N Engl J Med. 2020 Mar 26;382(13):1219-1231. link to original article contains dosing details in manuscript PubMed NCT02604433