Difference between revisions of "Beta thalassemia"

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*''We have moved [[How I Treat]] articles to a dedicated page.''
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|<div style="background-color: #fee0d1; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}" align="right"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] |?Regimen |limit=10000|format=sum}} [[Tutorial#Regimens|regimens]] on this page</b></font></div>
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|style="width:35%"|<big>[[User:Benjamintillman|Benjamin Tillman, MD]]<br>Vanderbilt University<br>Nashville, TN</big>
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<div style="background-color: #deebf6; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] |?Variant |limit=10000|format=sum}} [[Tutorial#Variants|variants]] on this page</b></font></div>
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=All lines of therapy=
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==Exagamglogene autotemcel monotherapy {{#subobject:8dgex1|Regimen=1}}==
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<div class="toccolours" style="background-color:#eeeeee">
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===Regimen {{#subobject:excbt2|Variant=1}}===
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!style="width: 33%"|Study
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!style="width: 33%"|Dates of enrollment
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!style="width: 33%"|[[Levels_of_Evidence#Evidence|Evidence]]
 
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|[https://doi.org/10.1056/nejmoa2309673 Locatelli et al. 2024 (CLIMB THAL-111)]
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|2018-09-10 to NR
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| style="background-color:#91cf61" |Non-randomized phase 3 (RT)
 
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====Targeted therapy====
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*[[Exagamglogene autotemcel (Casgevy)]]
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'''One course'''
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</div></div>
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===References===
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#'''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]
  
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=All lines of therapy=
 
 
==Luspatercept monotherapy {{#subobject:8dgac1|Regimen=1}}==
 
==Luspatercept monotherapy {{#subobject:8dgac1|Regimen=1}}==
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===Regimen {{#subobject:91cbt2|Variant=1}}===
 
===Regimen {{#subobject:91cbt2|Variant=1}}===
 
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!style="width: 20%"|Study
 
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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)]
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|2016-2017
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|2016-07 to 2017-06
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| style="background-color:#1a9851" |Phase 3 (E-esc)
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| style="background-color:#1a9851" |Phase 3 (E-RT-esc)
 
|[[#Placebo|Placebo]]
 
|[[#Placebo|Placebo]]
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| style="background-color:#1a9850" |Superior reduction in transfusion burden
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| style="background-color:#1a9850" |Superior primary endpoint
 
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<div class="toccolours" style="background-color:#b3e2cd">
 
====Growth factor therapy====
 
====Growth factor therapy====
 
*[[Luspatercept (Reblozyl)]] 1 mg/kg SC once on day 1
 
*[[Luspatercept (Reblozyl)]] 1 mg/kg SC once on day 1
 
**Dose can be increased based upon response (see paper for details)
 
**Dose can be increased based upon response (see paper for details)
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'''21-day cycle for at least 16 cycles'''
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</div></div>
  
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'''21-day cycle for at least 16 cycles'''
 
 
===References===
 
===References===
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# '''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 verified protocol''' [https://pubmed.ncbi.nlm.nih.gov/32212518/ PubMed] NCT02604433
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# '''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] [https://clinicaltrials.gov/study/NCT02604433 NCT02604433]
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[[Category:Beta thalassemia regimens]]
 
[[Category:Beta thalassemia regimens]]
 
[[Category:Disease-specific pages]]
 
[[Category:Disease-specific pages]]
 
[[Category:Hemoglobinopathies]]
 
[[Category:Hemoglobinopathies]]

Latest revision as of 11:35, 15 May 2024

Back to Top

Section editor
Tillman Benjamin-2.jpg
 Benjamin Tillman, MD
Vanderbilt University
Nashville, TN, USA
LinkedIn
  • 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)

Targeted therapy

One course

References

  1. 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

21-day cycle for at least 16 cycles

References

  1. 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
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