Supplementary tables: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients

These are peer-reviewed supplementary tables for the article 'Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients' published in the Journal of Comparative Effectiveness Research. Supplementary Table 1. Baseline demographics and disease characteristics for Study 019 patients with nmDMD who received ataluren (40 mg/kg/day) plus SoC in at least Study 019 (N = 59) and for propensity-score matched patients with DMD receiving SoC alone in the CINRG DNHS (N = 59), using age at diagnosis for both datasets as the fourth covariate for matching, for the sensitivity analysis of age at LoA. Supplementary Table 2. Baseline demographics and disease characteristics for non-ambulatory Study 019 patients with nmDMD who received ataluren 40 mg/kg/day plus SoC in at least this study (N = 45) and propensity-score matched patients with DMD who received SoC alone in the CINRG DNHS (N = 45), using age at diagnosis for both datasets as the fourth covariate for matching, for the sensitivity analysis of age at decline in respiratory function. Supplementary Table 3. Treatment-emergent adverse events† experienced by patients in the as-treated population of Study 019 (N = 94). Supplementary Table 4. Lipid profile of patients in the as-treated population of Study 019 (N = 94) at baseline and Week 240. Supplementary Table 5. Hypertensive status of patients in the as-treated population of Study 019 (N = 94) from baseline to Week 240. Supplementary Table 6. ECG results of patients of patients in the as-treated population of Study 019 (N = 94) from screening to post-treatment. Supplementary Table 7. Hepatic and renal abnormalities in patients in the as-treated population of Study 019 (N = 94) from screening to post-treatment. Summary: Aim: We investigated the effect of ataluren plus standard of care (SoC) on age at loss of ambulation (LoA) and respiratory decline in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) versus patients with DMD on SoC alone. Patients & methods: Study 019 was a long-term Phase III study of ataluren safety in nmDMD patients with a history of ataluren exposure. Propensity score matching identified Study 019 and CINRG DNHS patients similar in disease progression predictors. Results & conclusion: Ataluren plus SoC was associated with a 2.2-year delay in age at LoA (p = 0.0006), and a 3.0-year delay in decline of predicted forced vital capacity to

本数据集为发表于《比较疗效研究杂志》的论文《Ataluren延缓无义突变杜氏肌营养不良症患者的步行能力丧失和呼吸功能下降》的同行评审补充表格。补充表1：对于019研究中的nmDMD患者，其接受ataluren（40 mg/kg/天）联合标准治疗方案（SoC），以及CINRG DNHS中仅接受SoC的DMD患者（N = 59），以诊断时的年龄作为两个数据集匹配的第四个协变量，进行年龄在失去步行能力时间点的敏感性分析。补充表2：对于019研究中无法行走的nmDMD患者，其接受ataluren 40 mg/kg/天联合SoC，以及CINRG DNHS中仅接受SoC的DMD患者（N = 45），以诊断时的年龄作为两个数据集匹配的第四个协变量，进行呼吸功能下降的敏感性分析。补充表3：019研究中治疗人群（N = 94）所经历的治疗相关不良事件†。补充表4：019研究中治疗人群（N = 94）在基线和第240周时的血脂谱。补充表5：019研究中治疗人群（N = 94）从基线到第240周的血压状况。补充表6：019研究中治疗人群（N = 94）从筛选到治疗后期的心电图结果。补充表7：019研究中治疗人群（N = 94）从筛选到治疗后期的肝肾功能异常。摘要：目的：本研究旨在探讨ataluren联合标准治疗方案（SoC）对无义突变杜氏肌营养不良症（nmDMD）患者的步行能力丧失时间（LoA）和呼吸功能下降时间与仅接受SoC治疗的DMD患者相比的影响。研究方法：019研究是一项关于ataluren在nmDMD患者中安全性的长期III期研究，研究对象有ataluren暴露史。通过倾向性评分匹配确定了019研究和CINRG DNHS中疾病进展预测因子相似的受试者。结果与结论：ataluren联合SoC与LoA时间延迟2.2年（p = 0.0006）和预测肺活量下降时间延迟3.0年相关。