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Muscle Nerve. 2015 Apr;51(4):522-32. doi: 10.1002/mus.24346. Epub 2015 Feb 11.

Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy.

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  • 1Department of Neurology, Washington University School of Medicine in Saint Louis, St. Louis, Missouri, 63110, USA; Department of Pediatrics, Washington University School of Medicine in Saint Louis, St. Louis, Missouri, USA.

Abstract

INTRODUCTION:

Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial.

METHODS:

Non-ambulatory boys/men with DMD (N = 91; 16.7 ± 4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured.

RESULTS:

Forced vital capacity (FVC), assessed in all subjects, showed a mean of 47.8 ± 22% predicted (ICC 0.98). Brooke Upper Extremity Functional Rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs ranging from 0.93 to 0.99. Manual muscle testing, range of motion, 9-hole peg test, and Jebsen-Taylor Hand Function Test (JHFT) demonstrated varied feasibility (99% to 70%), with ICCs ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for the Brooke scale, percent predicted FVC, and hand and finger strength.

CONCLUSIONS:

Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use.

© 2014 Wiley Periodicals, Inc.

KEYWORDS:

Duchenne muscular dystrophy; corticosteroids; non-ambulatory; pulmonary function; quality of life; strength

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