Beta thalassemia
Revision as of 12:24, 15 July 2024 by Warner-admin (talk | contribs) (Text replacement - "'''dosing details in manuscript have been reviewed'''" to "'''dosing details in manuscript have been reviewed by our editors'''")
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
Exagamglogene autotemcel monotherapy
Regimen
Study | Dates of enrollment | Evidence |
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Locatelli et al. 2024 (CLIMB THAL-111) | 2018-09-10 to NR | Non-randomized phase 3 (RT) |
References
- CLIMB THAL-111: Locatelli F, Lang P, Wall D, Meisel R, Corbacioglu S, Li AM, de la Fuente J, Shah AJ, Carpenter B, Kwiatkowski JL, Mapara M, Liem RI, Cappellini MD, Algeri M, Kattamis A, Sheth S, Grupp S, Handgretinger R, Kohli P, Shi D, Ross L, Bobruff Y, Simard C, Zhang L, Morrow PK, Hobbs WE, Frangoul H; CLIMB THAL-111 Study Group. Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia. N Engl J Med. 2024 May 9;390(18):1663-1676. Epub 2024 Apr 24. link to original article PubMed NCT03655678
Luspatercept monotherapy
Regimen
Study | Dates of enrollment | Evidence | Comparator | Comparative Efficacy |
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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 dosing details in manuscript have been reviewed by our editors PubMed NCT02604433