Department of Pediatric Neurology, Catholic University, Rome, Italy.
Centro Clinico Nemo, Policlinico Universitario A Gemelli IRCCS, Rome, Italy.
J Comp Eff Res. 2020 Apr;9(5):341-360. doi: 10.2217/cer-2019-0171. Epub 2020 Jan 30.
Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p ≤ 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. NCT02369731. NCT02369731.
STRIDE（注册和国际卓越数据库的策略目标）是一个正在进行的多中心注册处，提供有关非义突变杜氏肌营养不良症（nmDMD）患者使用ataluren 的真实世界证据。我们研究了在注册处中ataluren + 标准治疗（SoC）与单独使用 SoC 在合作国际神经肌肉研究组（CINRG）杜氏肌营养不良自然史研究（DNHS）中的疗效，DMD 基因型-表型/-ataluren 获益相关性和 ataluren 的安全性。使用倾向评分匹配来识别 STRIDE 和 CINRG DNHS 患者，这些患者在既定疾病进展预测因素（注册截止日期，2018 年 7 月 9 日）方面具有可比性。Kaplan-Meier 分析表明，与单独使用 SoC 相比，ataluren + SoC 显著延迟了丧失步行能力的年龄和定时功能测试中表现恶化的年龄（p ≤ 0.05）。没有 DMD 基因型-表型/-ataluren 获益相关性。Ataluren 耐受性良好。这些结果表明，在常规临床实践中，ataluren + SoC 可延缓 nmDMD 患者 DMD 进展的功能里程碑。NCT02369731。NCT02369731。
