Difference between revisions of "Beta thalassemia"
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{{TOC limit|limit=3}} | {{TOC limit|limit=3}} | ||
=All lines of therapy= | =All lines of therapy= | ||
| + | ==Exagamglogene autotemcel monotherapy {{#subobject:8dgex1|Regimen=1}}== | ||
| + | <div class="toccolours" style="background-color:#eeeeee"> | ||
| + | ===Regimen {{#subobject:excbt2|Variant=1}}=== | ||
| + | {| class="wikitable sortable" style="width: 60%; text-align:center;" | ||
| + | !style="width: 33%"|Study | ||
| + | !style="width: 33%"|Dates of enrollment | ||
| + | !style="width: 33%"|[[Levels_of_Evidence#Evidence|Evidence]] | ||
| + | |- | ||
| + | |[https://doi.org/10.1056/nejmoa2309673 Locatelli et al. 2024 (CLIMB THAL-111)] | ||
| + | |2018-09-10 to NR | ||
| + | | style="background-color:#91cf61" |Non-randomized phase 3 (RT) | ||
| + | |- | ||
| + | |} | ||
| + | <div class="toccolours" style="background-color:#b3e2cd"> | ||
| + | ====Targeted therapy==== | ||
| + | *[[Exagamglogene autotemcel (Casgevy)]] | ||
| + | '''One course''' | ||
| + | </div></div> | ||
| + | ===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. [https://doi.org/10.1056/nejmoa2309673 link to original article] [https://pubmed.ncbi.nlm.nih.gov/38657265/ PubMed] [https://clinicaltrials.gov/study/NCT03655678 NCT03655678] | ||
| + | |||
==Luspatercept monotherapy {{#subobject:8dgac1|Regimen=1}}== | ==Luspatercept monotherapy {{#subobject:8dgac1|Regimen=1}}== | ||
<div class="toccolours" style="background-color:#eeeeee"> | <div class="toccolours" style="background-color:#eeeeee"> | ||
Revision as of 23:41, 14 May 2024
| Section editor | |
|---|---|
 |
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
|
All lines of therapy
Exagamglogene autotemcel monotherapy
Regimen
| Study | Dates of enrollment | Evidence |
|---|---|---|
| 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 |
|---|---|---|---|---|
| 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