Outcome reliability in non‐Ambulatory Boys/Men with duchenne muscular dystrophy. Issue 4 (11th February 2015)

• Record Type: Journal Article
• 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.
• Is Part Of: Muscle & nerve. Volume 51:Issue 4(2015:Apr.)
• Page Start: 522
• Page End: 532
• Publication Date: 2015-02-11
• Subjects: corticosteroids -- Duchenne muscular dystrophy -- non‐ambulatory -- pulmonary function -- quality of life -- strength; Neuromuscular diseases -- Periodicals; Muscles -- Periodicals; Nerves -- Periodicals; 616.74
• DOI: 10.1002/mus.24346
• Languages: English
• ISSNs: 0148-639X

Abstract

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