Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: Results from a 12 month, double-blind, randomized placebo-controlled trial

被引：83

作者：

Buyse, Gunnar M. ^{[1
]}

Goemans, Nathalie ^{[1
]}

van den Hauwe, Marleen ^{[1
]}

Thijs, Daisy ^{[1
]}

de Groot, Imelda J. M. ^{[2
]}

Schara, Ulrike ^{[3
]}

Ceulemans, Berten ^{[4
]}

Meier, Thomas ^{[5
]}

Mertens, Luc ^{[1
]}

机构：

[1] Univ Hosp Leuven, B-3000 Louvain, Belgium

[2] Radboud Univ Nijmegen Med Ctr, Nijmegen, Netherlands

[3] Univ Essen Gesamthsch, Essen, Germany

[4] Univ Hosp Antwerp, Antwerp, Belgium

[5] Santhera Pharmaceut, Liestal, Switzerland

来源：

NEUROMUSCULAR DISORDERS | 2011年 / 21卷 / 06期

关键词：

Duchenne; Muscular dystrophy; Treatment; Idebenone; Cardiac; Cardiomyopathy; Respiratory function; MYOCARDIAL DYSFUNCTION; STRAIN-RATE; MUSCLE WEAKNESS; CARDIOMYOPATHY; CHILDREN; PHARMACOKINETICS; FLOW;

D O I：

10.1016/j.nmd.2011.02.016

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Early mortality in Duchenne muscular dystrophy (DMD) is related to cardiac and respiratory complications. A phase ha double-blind randomized placebo-controlled clinical trial was conducted to investigate the tolerability and efficacy of idebenone therapy in children with DMD. Twenty-one DMD patients (aged 8-16 years) were randomly assigned to daily treatment with 450 mg idebenone (Catena (R)) (n = 13) or placebo (n = 8) for 12 months. All subjects completed the study and idebenone was safe and well tolerated. Idebenone treatment resulted in a trend (p = 0.067) to increase peak systolic radial strain in the left ventricular inferolateral wall, the region of the heart that is earliest and most severely affected in DMD. A significant respiratory treatment effect on peak expiratory flow was observed (p = 0.039 for PEF and p = 0.042 for PEF percent predicted). Limitations of this study were the small sample size, and a skewed age distribution between treatment groups. Data from this study provided the basis for the planning of a confirmatory study. (C) 2011 Elsevier B.V. All rights reserved.

引用

页码：396 / 405

页数：10

共 40 条

[1] Occult cardiac contractile dysfunction in dystrophin-deficient children revealed by cardiac magnetic resonance strain imaging [J].