Journal
JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH
Volume 11, Issue 5, Pages 347-370Publisher
FUTURE MEDICINE LTD
DOI: 10.2217/cer-2021-0216
Keywords
indirect treatment comparison; MAIC; nusinersen; onasemnogene abeparvovec; risdiplam; SMA; spinal muscular atrophy; STC
Categories
Ask authors/readers for more resources
Indirect comparisons indicate that risdiplam is superior to nusinersen in terms of improved survival and motor function in Type 1 SMA. However, concrete conclusions couldn't be drawn due to substantial differences in study populations for comparisons with onasemnogene abeparvovec in Type 1 SMA and nusinersen in Types 2/3 SMA.
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.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available