Beta thalassemia
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| Section editor | |
|---|---|
 |
Benjamin Tillman, MD Vanderbilt University Nashville, TN |
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Guidelines
"How I Treat"
- 2018: Taher & Cappellini How I manage medical complications of β-thalassemia in adults PubMed
All lines of therapy
Luspatercept monotherapy
Regimen
| Study | Dates of enrollment | Evidence | Comparator | Comparative Efficacy |
|---|---|---|---|---|
| Cappellini et al. 2020 (BELIEVE) | 2016-2017 | Phase 3 (E-RT-esc) | Placebo | Superior reduction in transfusion burden |
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 contains dosing details in manuscript PubMed Clinical Trial Registry