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 | |2016-2017 | ||
| − | | style="background-color:#1a9851" |Phase 3 (E-esc) | + | | style="background-color:#1a9851" |Phase 3 (E-RT-esc) |
|[[#Placebo|Placebo]] | |[[#Placebo|Placebo]] | ||
| style="background-color:#1a9850" |Superior reduction in transfusion burden | | style="background-color:#1a9850" |Superior reduction in transfusion burden | ||
| Line 34: | Line 34: | ||
'''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 dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/32212518/ PubMed] NCT02604433 | # '''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 | ||
Revision as of 01:06, 4 September 2022
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-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 NCT02604433