Departments of Physical Medicine & Rehabilitation and Pediatrics, University of California - Davis, Davis, CA 95616, USA.
Analysis Group, Inc., Boston, MA 02199, USA.
J Comp Eff Res. 2023 Apr;12(4):e220190. doi: 10.57264/cer-2022-0190. Epub 2023 Feb 7.
To examine benefits of corticosteroids for Duchenne muscular dystrophy (DMD) by age and disease progression. 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. 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). Daily deflazacort had greater benefits than daily prednisone particularly among older/more progressed patients.
按年龄和疾病进展来研究皮质类固醇类药物对杜氏肌营养不良症（DMD）的益处。 数据来自四项 DMD 2b/3 期临床试验中每日使用皮质类固醇类药物（安慰剂治疗）的患者。评估的结果包括 6 分钟步行距离（6MWD）、计时功能测试和 North Star 动态评估总分从基线到 48 周的变化。在接受地夫可特（n = 127）或泼尼松龙（n = 104）治疗的 231 名患者中，地夫可特在 6MWD 方面优于泼尼松龙的观察结果差异，在年龄较大（≥8 岁）、疾病进展较快（基线从仰卧位站立时间≥5 秒）或皮质类固醇类药物使用时间较长（>3 年）的患者中更为显著。地夫可特每日用药的获益大于泼尼松龙，特别是在年龄较大/进展较快的患者中。
