Supplementary materials: Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

These are peer-reviewed supplementary materials for the article 'The impact of willingness-to-pay threshold on price reduction recommendations for oncology drugs: a review of assessments conducted by the Canadian Agency for Drugs and Technologies in Health' published in the Journal of Comparative Effectiveness Research.Supplemental Table 1: Overview of the patients from the placebo arms of DMD clinical trialsSupplemental Table 2: Baseline characteristics for the pooled placebo arms of the DMD clinical trials who received daily corticosteroids, by clinical trial corticosteroid typeSupplemental Table 3: Baseline characteristics for the pooled placebo arms of the DMD clinical trials who received daily corticosteroids, by baseline age groups and corticosteroid typeSupplemental Table 4: Baseline characteristics for the pooled placebo arms of the DMD clinical trials who received daily corticosteroids, by baseline corticosteroid duration and corticosteroid typeSupplemental Table 5: Baseline characteristics for the pooled placebo arms of the DMD clinical trials who received daily corticosteroids, by baseline timed rise from supine and corticosteroid typeSupplemental Table 6: Unadjusted change in motor function outcomes from baseline to 12, 24, 36, and 48 weeks among the daily deflazacort and prednisone groups of the DMD clinical trial placebo armsSupplemental Table 7: Comparison of change in motor function outcomes from baseline to week 48 between the daily deflazacort and prednisone groups in subgroups of the pooled DMD clinical trial placebo armsAim: To examine benefits of corticosteroids for Duchenne muscular dystrophy (DMD) by age and disease progression. Methods: Data from daily steroid users (placebo-treated) were pooled from four phase 2b/3 trials in DMD. Outcomes assessed overall and among subgroups included changes from baseline to 48 weeks in six-minute walk distance (6MWD), timed function tests and North Star Ambulatory Assessment total score. Results: Among 231 patients receiving deflazacort (n = 127) or prednisone (n = 104), observed differences in 6MWD favoring deflazacort over prednisone were significant for patients with relatively older age (≥8-years-old), greater disease progression (baseline timed stand from supine ≥5 s), or longer corticosteroid use (>3 years). Conclusion: Daily deflazacort had greater benefits than daily prednisone particularly among older/more progressed patients.

本数据集为发表于《比较有效性研究杂志》之文章《支付意愿阈值对肿瘤药物降价建议的影响：加拿大药物与医疗技术署评估综述》的同行评审补充材料。补充表1：Duchenne 肌营养不良症（DMD）临床试验安慰剂组的患者概述；补充表2：接受每日皮质类固醇的DMD临床试验安慰剂组的基线特征，按临床试验皮质类固醇类型划分；补充表3：接受每日皮质类固醇的DMD临床试验安慰剂组的基线特征，按基线年龄组和皮质类固醇类型划分；补充表4：接受每日皮质类固醇的DMD临床试验安慰剂组的基线特征，按基线皮质类固醇使用时长和皮质类固醇类型划分；补充表5：接受每日皮质类固醇的DMD临床试验安慰剂组的基线特征，按基线仰卧位至站立位计时提升时长和皮质类固醇类型划分；补充表6：DMD临床试验安慰剂组的每日deflazacort和prednisone组在基线至12、24、36和48周时运动功能结果的无调整变化；补充表7：在DMD临床试验安慰剂组的亚组中，每日deflazacort和prednisone组基线至48周时运动功能结果变化的比较。研究目的：通过年龄和疾病进展考察皮质类固醇对Duchenne肌营养不良症（DMD）的益处。研究方法：从四个DMD 2b/3期临床试验中收集每日皮质类固醇使用者（安慰剂治疗）的数据。评估的总体和亚组结果包括基线至48周六分钟步行距离（6MWD）、计时功能测试和North Star 行走评估总分的变化。研究结果：在231名接受deflazacort（n = 127）或prednisone（n = 104）治疗的受试者中，观察到6MWD方面deflazacort相对于prednisone的差异在年龄相对较大（≥8岁）、疾病进展较大（基线仰卧位至站立位计时≥5秒）或皮质类固醇使用时长较长（>3年）的受试者中具有统计学意义。研究结论：每日deflazacort相较于每日prednisone在老年/疾病进展较重的患者中具有更大的益处。