Difference between revisions of "Beta thalassemia"
Jump to navigation
Jump to search
Warner-admin (talk | contribs) m (Text replacement - "{| class="wikitable" style="float:right; margin-left: 5px;" |- |back to top |}" to "") |
Warner-admin (talk | contribs) m (Text replacement - "'''contains verified protocol'''" to "'''contains dosing details in manuscript'''") |
||
| Line 37: | Line 37: | ||
'''21-day cycle for at least 16 cycles''' | '''21-day cycle for at least 16 cycles''' | ||
===References=== | ===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. [https://doi.org/10.1056/nejmoa1910182 link to original article] '''contains | + | # '''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. [https://doi.org/10.1056/nejmoa1910182 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/32212518/ PubMed] NCT02604433 |
[[Category:Beta thalassemia regimens]] | [[Category:Beta thalassemia regimens]] | ||
[[Category:Disease-specific pages]] | [[Category:Disease-specific pages]] | ||
[[Category:Hemoglobinopathies]] | [[Category:Hemoglobinopathies]] | ||
Revision as of 12:43, 29 August 2022
| Section editor | |
|---|---|
 |
Benjamin Tillman, MD Vanderbilt University Nashville, TN |
2 regimens on this page
2 variants on this page
|
All lines of therapy
Luspatercept monotherapy
Regimen
| Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
|---|---|---|---|---|
| Cappellini et al. 2020 (BELIEVE) | 2016-2017 | Phase 3 (E-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 NCT02604433