Open AccessHow does risdiplam compare with other treatments for Types 1–3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison
Author(s) -
Valerie Aponte-Ribero,
Monica Daigl,
Yasmina Martí,
Ksenija Gorni,
Rachel Evans,
David A. Scott,
Anadi Mahajan,
Keith R. Abrams,
Neil Hawkins
Publication year - 2022
Publication title -
journal of comparative effectiveness research
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.567
H-Index - 23
eISSN - 2042-6313
pISSN - 2042-6305
DOI - 10.2217/cer-2021-0216
Subject(s) - sma* , spinal muscular atrophy , medicine , motor function , physical medicine and rehabilitation , surgery , disease , mathematics , combinatorics
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.