APOLLO-B, a study of patisiran in patients with transthyretin cardiac amyloidosis: primary long-term results from the open-label extension period
Circulation
; 148(Suppl.1)Nov. 7, 2023.
Artigo
em Inglês
| CONASS, Sec. Est. Saúde SP, SESSP-IDPCPROD, Sec. Est. Saúde SP
| ID: biblio-1519637
Biblioteca responsável:
BR79.1
ABSTRACT
INTRODUCTION:
The Phase 3 APOLLO-B study evaluates patisiran in patients (pts) with transthyretin (ATTR) cardiac amyloidosis over a 12-month (M) double-blind (DB) period, followed by an open-label extension (OLE) period when all pts receive patisiran (NCT03997383).Hypothesis:
Patisiran provides long-term benefit in pts with ATTR cardiac amyloidosis.Aims:
Describe safety and efficacy of patisiran during the APOLLO-B OLE (18M+).METHODS:
Pts (18-85 yrs) with ATTR cardiac amyloidosis and heart failure history were randomized 11 to patisiran or placebo (pbo). Pts completing DB period were eligible to receive patisiran in the OLE for ≤36M. Results summarized based on DB treatment arm. Exploratory assessments include change from study baseline (CFB) in 6-minute walk test (6MWT), KCCQ-OS, NT-proBNP, and troponin I.RESULTS:
In the DB period, 359 pts (pbo n=178; patisiran n=181) received study drug (median [range] age, 76.0 [41, 85] yrs; male, 89%; wtATTR, 80%; tafamidis at baseline, 25%); 334 (93%) entered the OLE. In patisiran arm, M12 and M18 results, respectively, were similar for each endpoint 6MWT and KCCQ-OS (mean [SEM] CFB) −8.09 [5.73] vs −9.21 [6.04] meters (m) and 0.60 [1.36] vs 0.22 [1.48]; NT-proBNP and troponin I (geometric mean fold-CFB [95%CI]) 1.10 [1.03, 1.17] vs 1.17 [1.07, 1.27] and 1.11 [1.05, 1.18] vs 1.09 [1.01, 1.17]). In pbo arm, patisiran initiation in OLE was associated with a slower rate of worsening or relative stability across endpoints; CFB at M12 vs M18, respectively 6MWT, −25.43 [5.61] vs −31.08 [5.45] m; KCCQ-OS, −3.41 [1.33] vs −4.02 [1.49]; NT-proBNP, 1.39 [1.28, 1.51] vs 1.53 [1.38, 1.71]; and troponin I, 1.29 [1.21, 1.38] vs 1.21 [1.13, 1.30]. Patisiran had an acceptable safety profile; no new concerns. OLE analyses are ongoing; updated data to be presented.CONCLUSIONS:
The M18 results provide evidence that beneficial effects observed in DB period on functional capacity, health status, and quality of life were maintained by continued treatment with patisiran during the OLE. Pbo-treated pts initiating patisiran at M12 showed slowed worsening or stabilization in most endpoints at M18. Early treatment initiation is important pbo-treated pts did not recover functional capacity or health lost prior to initiating OLE patisiran.
Texto completo:
Disponível
Coleções:
Bases de dados nacionais
/
Brasil
Base de dados:
CONASS
/
Sec. Est. Saúde SP
/
SESSP-IDPCPROD
Idioma:
Inglês
Revista:
Circulation
Ano de publicação:
2023
Tipo de documento:
Artigo
/
Congresso e conferência
Instituição/País de afiliação:
Alnylam Pharmaceuticals/US
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Boston Univ Sch of Med/US
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Columbia Univ Medical Ctr/US
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Instituto Dante Pazzanese de Cardiologia/BR
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Penn Presbyterian Medical Ctr/US
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Royal Free Hosp/US
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Unidade de Pesquisa Clínica UPC/BR
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Univ Federal Fluminense (UFF) & Hosp Procardíaco/BR
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Vanderbilt Univ Medical Ctr/US