Difference between revisions of "Beta thalassemia"
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|[https://doi.org/10.1056/nejmoa1910182 Cappellini et al. 2020 (BELIEVE)] | |[https://doi.org/10.1056/nejmoa1910182 Cappellini et al. 2020 (BELIEVE)] | ||
− | |2016-2017 | + | |2016-07 to 2017-06 |
| style="background-color:#1a9851" |Phase 3 (E-RT-esc) | | style="background-color:#1a9851" |Phase 3 (E-RT-esc) | ||
|[[#Placebo|Placebo]] | |[[#Placebo|Placebo]] |
Revision as of 13:15, 28 November 2023
Section editor | |
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Benjamin Tillman, MD Vanderbilt University Nashville, TN, USA |
- We have moved How I Treat articles to a dedicated page.
2 regimens on this page
2 variants on this page
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All lines of therapy
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 | Superior primary endpoint |
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 NCT02604433