Section editor
	Benjamin Tillman, MD Vanderbilt University Nashville, TN, USA LinkedIn

Last updated on 2024-07-23: 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 not reported	Non-randomized phase 3 (RT)

One course

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

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

Luspatercept (Reblozyl) 1 mg/kg SC once on day 1
- Dose can be increased based upon response (see paper for details)

21-day cycle for at least 16 cycles

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 dosing details in manuscript have been reviewed by our editors PubMed NCT02604433