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

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 1Supplementary figure 2Supplementary figure 3Supplementary figure 4Supplementary figure 5Supplementary figure 6Supplementary 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 justificationSupplementary 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.

本为《比较疗效研究杂志》上发表之文章《Risdiplam与其它治疗脊髓性肌萎缩症类型1至3的疗效比较：一项系统文献综述及间接疗效比较》的同行评审补充材料。包括补充图1至补充图6，以及补充表1：系统文献综述的PICOS框架及附加搜索标准，补充表2：系统文献综述中使用的资料来源清单，补充表3：统计分析计划中的关键偏差及其合理性说明，补充表4：系统文献综述中排除的临床试验，补充表5：1型脊髓性肌萎缩症中感兴趣终点的可及性，补充表6：FIREFISH试验在STR1VE-US（MAIC分析）匹配前后的基线特征，补充表7：FIREFISH与STR1VE-US的STC模型拟合统计量，补充表8：STC FIREFISH与STR1VE-US的BSID-III协变量估计，补充表9：STC FIREFISH与STR1VE-US的CHOP-INTEND协变量估计，补充表10：2型和3型脊髓性肌萎缩症中感兴趣终点的可及性，补充表11：SUNFISH和CHERISH试验12个月时HFMSE结果的统计分析。研究目标：对risdiplam与其他经批准的脊髓性肌萎缩症（SMA）治疗方案进行间接疗效比较。患者与方法：将risdiplam临床试验的个体患者数据与已发表关于nusinersen和onasemnogene abeparvovec的研究的汇总数据相比较，考虑到研究间的异质性。结果：在1型SMA中，risdiplam和nusinersen的研究包含相似的患者群体。间接比较结果显示，与nusinersen相比，risdiplam可改善生存率和运动功能。在1型SMA中与onasemnogene abeparvovec的比较以及在2/3型SMA中与nusinersen的比较由于研究人群存在实质性差异而具有挑战性；间接比较分析中无法得出明确的结论。结论：间接比较支持risdiplam作为1型SMA中优于nusinersen的替代治疗方案。