Beta thalassemia

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Benjamin Tillman, MD
Vanderbilt University
Nashville, TN
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All lines of therapy

Luspatercept monotherapy

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Regimen

Study Years of enrollment Evidence Comparator Comparative Efficacy
Cappellini et al. 2020 (BELIEVE) 2016-2017 Phase III (E-esc) Placebo Superior reduction in transfusion burden

Growth factor therapy

21-day cycle for at least 16 cycles

References

  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 verified protocol PubMed NCT02604433