依伐布雷定改善射血分数降低心衰患者运动耐量及生活质量
Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.
机构信息
Department of Pediatrics, University of California Davis School of Medicine, Davis, CA, USA.
Dubowitz Neuromuscular Centre & MRC Centre for Neuromuscular Diseases, University College London, Institute of Child Health & Great Ormond Street Hospital for Children Foundation Trust, London, UK.
出版信息
J Comp Eff Res. 2022 Feb;11(3):139-155. doi: 10.2217/cer-2021-0196. Epub 2021 Nov 18.
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. 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. 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 <60% in nonambulatory patients (p = 0.0004), versus SoC. Ataluren plus SoC delays disease progression and benefits ambulatory and nonambulatory patients with nmDMD. : NCT01557400.
我们研究了在标准治疗（SoC）基础上加用ataluren 对具有无义突变的杜氏肌营养不良症（nmDMD）患者与单纯接受 SoC 的 DMD 患者的失能行走年龄（LoA）和呼吸功能下降的影响。研究 019 是一项在有ataluren 暴露史的 nmDMD 患者中进行的 ataluren 安全性的长期 III 期研究。倾向评分匹配确定了在疾病进展预测因素上与 CINRG DNHS 患者相似的 Study 019 患者。ataluren 加 SoC 与 LoA 年龄延迟 2.2 年相关（p=0.0006），与 SoC 相比，非步行患者预测用力肺活量下降至<60%的时间延迟 3.0 年（p=0.0004）。ataluren 加 SoC 可延缓疾病进展，并使具有 nmDMD 的可步行和不可步行患者受益。：NCT01557400。
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