Journal of comparative effectiveness research · 2022
How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison.
СтатьяПоддержка исследования: вне правительства СШАСистематический обзорНазвание и аннотация: Перевод в очереди
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Ribero Valerie Aponte, Daigl Monica, Martí Yasmina, Gorni Ksenija, Evans Rachel, Scott David Alexander, Mahajan Anadi, Abrams Keith R, Hawkins Neil
Аннотация
Aim: To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). Patients & methods: Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneity across studies. Results: In Type 1 SMA, studies of risdiplam and nusinersen included similar populations. Indirect comparison results found improved survival and motor function with risdiplam versus nusinersen. Comparison with onasemnogene abeparvovec in Type 1 SMA and with nusinersen in Types 2/3 SMA was challenging due to substantial differences in study populations; no concrete conclusions could be drawn from the indirect comparison analyses. Conclusion: Indirect comparisons support risdiplam as a superior alternative to nusinersen in Type 1 SMA.