Supplementary data: Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA

These are peer-reviewed supplementary data for the article 'Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA' published in the Journal of Comparative Effectiveness Research.Supplementary Table 1: Poisson Regression Analysis ResultsSupplementary Table 2: Sensitivity Analyses Poisson Regressions in the Matched Treated and SoC PatientsAim: To evaluate treatment effects of eteplirsen among patients with Duchenne muscular dystrophy. Methods: Using real-world claims and electronic medical record data, this retrospective comparative analysis assessed eteplirsen-treated and control cohorts matched by age, disease progression state, and pre-index period healthcare resource utilization. Poisson regression was used to evaluate eteplirsen effects on healthcare resource utilization outcomes. Results: Eteplirsen was associated with statistically significant reductions in rates of hospital encounters (31%), emergency room visits (31%), need for pulmonary management (33%), cardiac management (21%), tracheostomy (86%), and assisted ventilation (39%) versus the control group. Other assessed outcomes favored eteplirsen numerically but did not all reach statistical significance. Conclusion: Eteplirsen-treated patients had reduced rates of multiple healthcare resource utilization measures versus matched controls.

本数据集为发表于《比较疗效研究杂志》的论文《美国杜氏肌营养不良症患者在eteplirsen治疗中的真实世界疗效》的同行评审补充数据。补充表1：泊松回归分析结果；补充表2：匹配治疗组和SoC患者的敏感性分析泊松回归。研究目的：评估eteplirsen在杜氏肌营养不良症患者中的治疗效果。研究方法：利用真实世界的索赔数据和电子病历数据，本项回顾性比较分析评估了按年龄、疾病进展状态和基线期医疗资源利用情况匹配的eteplirsen治疗组与对照组。采用泊松回归模型对eteplirsen对医疗资源利用结果的影响进行评估。研究结果：与对照组相比，eteplirsen治疗与医院就诊率（31%）、急诊室访问率（31%）、需要肺管理（33%）、心脏管理（21%）、气管切开术（86%）和辅助通气（39%）的显著降低相关。其他评估结果虽在数值上倾向于eteplirsen，但并非所有结果均达到统计学意义。研究结论：与匹配的对照组相比，eteplirsen治疗组在多个医疗资源利用指标方面有所降低。