期刊
MUSCLE & NERVE
卷 48, 期 1, 页码 55-67出版社
WILEY
DOI: 10.1002/mus.23808
关键词
adolescent; adult; child; preschool; follow-up studies; health status; humans; locomotion; male; muscular dystrophies; classification; muscular dystrophies; Duchenne; physiopathology; muscular dystrophies; therapy; muscle strength; physiology; phenotype; quality of life; psychology; respiratory function tests
资金
- U.S. Department of Education/NIDRR [H133B031118, H133B090001]
- U.S. Department of Defense [W81XWH-09-1-0592]
- NIH [UL1RR031988, U54HD053177, UL1RR 024992, U54RR026139, 2U54HD053177, G12RR003051, 1R01AR061875, RO1AR062380]
- Parent Project Muscular Dystrophy
Introduction: Glucocorticoid (GC) therapy in Duchenne muscular dystrophy (DMD) has altered disease progression, necessitating contemporary natural history studies. Methods: The Cooperative Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 DMD males, ages 2-28 years. A comprehensive battery of measures was obtained. Results: A novel composite functional milestone scale scale showed clinically meaningful mobility and upper limb abilities were significantly preserved in GC-treated adolescents/young adults. Manual muscle test (MMT)-based calculations of global strength showed that those patients <10 years of age treated with steroids declined by 0.4 +/- 0.39 MMT unit/year, compared with -0.4 +/- 0.39 MMT unit/year in historical steroid-naive subjects. Pulmonary function tests (PFTs) were relatively preserved in steroid-treated adolescents. The linearity and magnitude of decline in measures were affected by maturational changes and functional status. Conclusions: In DMD, long-term use of GCs showed reduced strength loss and preserved functional capabilities and PFTs compared with previous natural history studies performed prior to the widespread use of GC therapy. Muscle Nerve, 2013
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据