Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy

• Published in: Muscle & nerve Vol. 51; no. 4; pp. 522 - 532
• Main Authors: Connolly, Anne M., Malkus, Elizabeth C., Mendell, Jerry R., Flanigan, Kevin M., Miller, J. Philip, Schierbecker, Jeanine R., Siener, Catherine A., Golumbek, Paul T., Zaidman, Craig M., Mcdonald, Craig M., Johnson, Linda, Nicorici, Alina, Karachunski, Peter I., Day, John W., Kelecic, Jason M., Lowes, Linda P., Alfano, Lindsay N., Darras, Basil T., Kang, Peter B., Quigley, Janet, Pasternak, Amy E., Florence, Julaine M.
• Format: Journal Article
• Language: English
• Published: United States Blackwell Publishing Ltd 01-04-2015; Wiley Subscription Services, Inc
• Subjects: Adolescent; Adrenal Cortex Hormones - therapeutic use; Adult; Child; corticosteroids; Disability Evaluation; Duchenne muscular dystrophy; Hand - innervation; Hand - physiopathology; Humans; Male; Muscular Dystrophy, Duchenne - diagnosis; Muscular Dystrophy, Duchenne - drug therapy; Muscular Dystrophy, Duchenne - physiopathology; non-ambulatory; pulmonary function; quality of life; Reproducibility of Results; strength; Treatment Outcome; Young Adult; pulmonary function; corticosteroids; strength; quality of life; Duchenne muscular dystrophy; non-ambulatory
• ISSN: 0148-639X; 1097-4598
• DOI: 10.1002/mus.24346

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. Muscle Nerve 51: 522–532, 2015