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

dataset

posted on 2024-05-03, 08:00 authored by Valerie Ribero, Monica Daigl, Yasmina Marti, Ksenija Gorni, Rachel Evans, David Scott, Anadi Mahajan, Keith Abrams, Neil Hawkins

These are peer-reviewed supplementary materials for the article 'How does risdiplam compare with other treatments for Types 1–3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison' published in the Journal of Comparative Effectiveness Research.

Supplementary figure 1
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Supplementary Table 1: PICOS framework and additional search criteria for the SLR.
Supplementary Table 2: List of data sources used in the SLR.
Supplementary Table 3: Key deviations from statistical analysis plan and justification
Supplementary Table 4: Clinical trials excluded from the SLR.
Supplementary Table 5: Availability of endpoints of interest in Type 1 SMA.
Supplementary Table 6: Baseline characteristics of FIREFISH pre- and post‑matching with STR1VE‑US (MAIC analysis).
Supplementary Table 7: STC model fit statistics of FIREFISH versus STR1VE-US.
Supplementary Table 8: BSID-III covariate estimates (STC FIREFISH vs STR1VE-US).
Supplementary Table 9: CHOP-INTEND covariate estimates (STC FIREFISH vs STR1VE-US).
Supplementary Table 10: Availability of endpoints of interest in Types 2 and 3 SMA.
Supplementary Table 11: Analyses of HFMSE outcomes in SUNFISH and CHERISH at Month 12.

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.