How does risdiplam compare with other treatments for Types 1–3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison

To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneit...

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Published inJournal of comparative effectiveness research Vol. 11; no. 5; pp. 347 - 370
Main Authors Ribero, Valerie Aponte, Daigl, Monica, Martí, Yasmina, Gorni, Ksenija, Evans, Rachel, Scott, David Alexander, Mahajan, Anadi, Abrams, Keith R, Hawkins, Neil
Format Journal Article
LanguageEnglish
Published England Future Medicine Ltd 01.04.2022
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ISSN2042-6305
2042-6313
2042-6313
DOI10.2217/cer-2021-0216

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Summary:To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneity across studies. 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. Indirect comparisons support risdiplam as a superior alternative to nusinersen in Type 1 SMA. How does risdiplam compare with other treatments for Types 1–3 spinal muscular atrophy? A systematic literature review and indirect treatment comparison.
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ISSN:2042-6305
2042-6313
2042-6313
DOI:10.2217/cer-2021-0216