Author: Brooke, M.H.; Fenichel, G.M.; Griggs, R.C.; Mendell, J.R.; Moxley, R.; Miller, J.P.; Province, M.A.
Description: A prospective study of 114 patients with DMD provided data for “power” calculations for future therapeutic trials. There was a decline in strength of 0.4 units per year (on a 0-10 scale). Contractures of the iliotibial bands, hip flexors, and heel cords developed before 6 years. Contractures of other joints accompanied the increased use of wheelchairs. All children walked until 8 years with functional “improvement” between 3-6 years. Children of the same age varied widely in their strength, degree of contracture, and functional abilities. Fifteen percent of the patients appear to have a milder variety of the disease and are termed “outliers.” To test a drug which might slow the disease to 25% of its original rate of progression, two groups (placebo and treatment) of 40 patients each would have to be followed for one year.
Subject Headings: Adolescent; Child; Child, Preschool; Clinical Trials as Topic; Contracture/drug therapy; Humans; Infant; Motor Skills/drug effects; Muscle Contraction/drug effects; Muscular Dystrophies/diagnosis/drug therapy; Respiration/drug effects
Keywords: Clinical investigation in Duchenne dystrophy: 2. Determination of the “power” of therapeutic trials based on the natural history
Publication year: 1983
Journal or book title: Muscle & Nerve
Find the full text : https://onlinelibrary.wiley.com/doi/abs/10.1002/mus.880060204
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Type: Journal Article
Serial number: 2712