Supplementary materials: Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data

These are peer-reviewed supplementary materials for the article 'Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data' published in the Journal of Comparative Effectiveness Research.Supplementary Table 1: PICOS(+) framework to identify trials for the SLR.Supplementary Table 2: Search in MEDLINE, 2022 SLR.Supplementary Table 3: Search in MEDLINE, 2023 SLR update.Supplementary Table 4: Search in EMBASE, 2022 SLR.Supplementary Table 5: Search in EMBASE, 2023 SLR update.Supplementary Table 6: Search in Cochrane Central Register of Controlled Trials.Supplementary Table 7: Search in Cochrane Database of Systematic Reviews.Supplementary Table 8: Search in PROSPERO.Supplementary Table 9: Search in ISRCTN.com.Supplementary Table 10: Search in DARE.Supplementary Table 11: Conferences searches, 2022 SLR.Supplementary Table 12: Conferences searches, 2023 SLR update.Supplementary Table 13: Search in ClinicalTrials.gov.Supplementary Table 14: Search on ICTRP.Supplementary Table 15: Search in EU Clinical Trials Register.Supplementary Table 16: Search in Google Scholar and CDER.Supplementary Table 17: List of included publications.Supplementary Table 18: Quality assessment according to RoB2 Tool: PROPEL and COMET.Supplementary Table 19: Quality assessment according to ROBINS-I Tool: NEO1/NEO-EXT and COMET OLE.Supplementary Table 20: Efficacy results of included trials at or around 1 year.Supplementary Figure 1: PRISMA flow chart for selection of studies.Supplementary Figure 2: Longitudinal efficacy results from included trials – 6MWD (m) change from baseline.Supplementary Figure 3: Longitudinal efficacy results from included trials – FVC (% predicted) change from baseline.Supplementary Figure 4: Forest plots for 6MWD (m) and FVC (% predicted), base case scenario, all models.Supplementary Figure 5: SUCRA scores, base case scenario, all models.Supplementary Figure 6: Forest plots for 6MWD and FVC change from baseline at Week 52, previous ERT duration scenarios.Supplementary Figure 7: Forest plots for 6MWD (m) and FVC (% predicted) at different weeks.Supplementary Table 21: Matching criterionAim: Late-onset Pompe disease is characterized by progressive loss of muscular and respiratory function. Until recently, standard of care was enzyme replacement therapy (ERT) with alglucosidase alfa. Secondgeneration ERTs avalglucosidase alfa (aval) and cipaglucosidase alfa with miglustat (cipa+mig) are now available. Without head-to-head trials comparing aval with cipa+mig, an indirect treatment comparison is informative and timely for understanding potential clinical differentiation. Materials & methods: A systematic literature review was performed to identify relevant studies on cipa+mig and aval. Using patient-level and aggregate published data from randomized controlled trials (RCTs) and phase I/II and open-label extension (OLE) trials, a multi-level network meta-regression was conducted, adjusting for various baseline covariates, including previous ERT duration, to obtain relative effect estimates on 6-minute walk distance (6MWD, meters [m]) and forced vital capacity (FVC, % predicted [pp]). Analyses of two networks were conducted: Network A, including only RCTs, and network B, additionally including single-arm OLE and phase I/II studies. Results: Network B (full evidence analysis) showed that cipa+mig was associated with a relative increase in 6MWD (mean difference 28.93 m, 95% credible interval [8.26–50.11 m]; Bayesian probability 99.7%) and FVC (2.88 pp [1.07–4.71 pp]; >99.9%) compared with aval. The comparison between cipa+mig and aval became more favorable for cipa+mig with increasing previous ERT duration for both end points. Analysis of network A showed that cipa+mig was associated with a relative decrease in 6MWD(-10.02m[-23.62 to 4.00 m]; 91.8%) and FVC (-1.45 pp [-3.01 to 0.07 pp]; 96.8%)compared with aval. Conclusion: Cipa+mig showed a favorable effect versus aval when all available evidence was used in the analysis.

本数据集为发表于《比较疗效研究杂志》上的文章《比较艾普加葡萄糖苷酶α联合米格鲁司他与其他酶替代疗法对晚发型庞贝病的疗效：一项利用患者层级和汇总数据的网络荟萃分析》的同行评审补充材料。补充表1：用于确定系统评价试验的PICOS(+)框架。补充表2：2022年系统评价的MEDLINE检索。补充表3：2023年系统评价更新中的MEDLINE检索。补充表4：2022年系统评价的EMBASE检索。补充表5：2023年系统评价更新中的EMBASE检索。补充表6：Cochrane中央对照试验注册库的检索。补充表7：Cochrane系统评价数据库的检索。补充表8：PROSPERO的检索。补充表9：ISRCTN.com的检索。补充表10：DARE的检索。补充表11：2022年系统评价的会议检索。补充表12：2023年系统评价更新中的会议检索。补充表13：ClinicalTrials.gov的检索。补充表14：ICTRP的检索。补充表15：欧盟临床试验登记库的检索。补充表16：Google Scholar和CDER的检索。补充表17：纳入文献清单。补充表18：根据RoB2工具进行的质量评估：PROPEL和COMET。补充表19：根据ROBINS-I工具进行的质量评估：NEO1/NEO-EXT和COMET OLE。补充表20：纳入试验在或接近1年时的疗效结果。补充图1：PRISMA流程图，用于研究的选择。补充图2：纳入试验的纵向疗效结果——从基线至6分钟步行距离（6MWD）的变化。补充图3：纳入试验的纵向疗效结果——从基线至肺活量（FVC）的变化。补充图4：基于基线情景的森林图，包括所有模型。补充图5：SUCRA分数，基于基线情景，所有模型。补充图6：基线至第52周，根据既往酶替代疗法（ERT）持续时间情景，6MWD和FVC变化森林图。补充图7：不同周次下6MWD（米）和FVC（预计百分比）的森林图。补充表21：匹配标准目的：晚发型庞贝病以肌肉和呼吸功能的逐渐丧失为特征。直到最近，标准治疗方案为酶替代疗法（ERT）使用艾葡萄糖苷酶α。第二代ERT包括艾葡萄糖苷酶α（aval）和艾普加葡萄糖苷酶α与米格鲁司他的联合疗法（cipa+mig）。由于缺乏aval与cipa+mig的头对头试验，进行间接治疗比较对于理解潜在的临床差异具有重要意义。材料与方法：进行了一项系统文献综述，以识别关于cipa+mig和aval的相关研究。使用来自随机对照试验（RCT）、I/II期和开放标签扩展（OLE）试验的患者层级和汇总已发表数据，进行多层级网络荟萃回归分析，调整各种基线协变量，包括既往ERT持续时间，以获得相对效应估计值，包括6分钟步行距离（6MWD，米[m]）和肺活量（FVC，预计百分比[pp]）。进行了两个网络的分析：网络A，仅包括RCT；网络B，除包括单臂OLE和I/II期研究。结果：网络B（全面证据分析）显示，与aval相比，cipa+mig与6MWD（平均差异28.93米，95%可信区间[8.26–50.11米]；贝叶斯概率99.7%）和FVC（2.88预计百分比[1.07–4.71预计百分比]；>99.9%）的相对增加相关。对于两个终点，既往ERT持续时间增加，cipa+mig与aval的比较结果对cipa+mig更有利。网络A的分析显示，与aval相比，cipa+mig与6MWD（-10.02米[-23.62至4.00米]；91.8%）和FVC（-1.45预计百分比[-3.01至0.07预计百分比]；96.8%）的相对减少相关。结论：当分析中使用所有可用证据时，cipa+mig与aval相比显示出有利的效果。