How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison
JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH(2022)
Abstract
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. Tweetable abstract 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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Key words
indirect treatment comparison,MAIC,nusinersen,onasemnogene abeparvovec,risdiplam,SMA,spinal muscular atrophy,STC
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