Beta thalassemia

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*We have moved How I Treat articles to a dedicated page.
Last updated on 2024-12-02:3 regimens on this page
3 variants on this page
=All lines of therapy=
==Betibeglogene autotemcel monotherapy==
===Regimen===
Study 	Dates of enrollment 	Evidence
Locatelli et al. 2021 (HGB-207) 	2016-07 to 2020-01 	Non-randomized phase 3 (RT)
Kwiatkowski et al. 2024 (HGB-212)	2017-06-08 to 2020-03-12	Non-randomized phase 3 (RT)
====Targeted therapy====
*Betibeglogene autotemcel (Zynteglo)
One course
===References===
#HGB-207: 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. N Engl J Med. 2022 Feb 3;386(5):415-427. Epub 2021 Dec 11. link to original article EuroPMC NCT02906202
#HGB-212: Kwiatkowski JL, Walters MC, Hongeng S, Yannaki E, Kulozik AE, Kunz JB, Sauer MG, Thrasher AJ, Thuret I, Lal A, Tao G, Ali S, Thakar HL, Elliot H, Lodaya A, Lee J, Colvin RA, Locatelli F, Thompson AA. Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial. Lancet. 2024 Nov 8:S0140-6736(24)01884-1. link to original article EuroPMC NCT03207009
==Exagamglogene autotemcel monotherapy==
===Regimen===
Study 	Dates of enrollment 	Evidence
Locatelli et al. 2024 (CLIMB THAL-111)	2018-09-10 to not reported	Non-randomized phase 3 (RT)
====Targeted therapy====
*Exagamglogene autotemcel (Casgevy)
One course
===References===
#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 EuroPMC NCT03655678
==Luspatercept monotherapy==
===Regimen===
Study 	Dates of enrollment	Evidence 	Comparator	Comparative Efficacy
Cappellini et al. 2020 (BELIEVE)	2016-07 to 2017-06 	Phase 3 (E-RT-esc)	Placebo 	Higher percentage of patients who had a reduction in the transfusion burden of at least 33% from baseline during weeks 13 through 24 plus a reduction of at least 2 red-cell units over this 12-week interval (primary endpoint)Percentage: 21.4% vs 4.5%
====Growth factor therapy====
*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
===References===
# 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 EuroPMC NCT02604433
Category:Beta thalassemia regimens
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Category:Hemoglobinopathies