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OUTCOME RELIABILITY IN NON-AMBULATORY BOYS/MEN WITH DUCHENNE MUSCULAR DYSTROPHY MUSCLE & NERVE Connolly, A. M., Malkus, E. C., Mendell, J. R., Flanigan, K. M., Miller, J. P., Schierbecker, J. R., Siener, C. A., Golumbek, P. T., Zaidman, C. M., McDonald, C. M., Johnson, L., Nicorici, A., Karachunski, P. I., Day, J. W., Kelecic, J. M., Lowes, L. P., Alfano, L. N., Darras, B. T., Kang, P. B., Quigley, J., Pasternak, A. E., Florence, J. M. 2015; 51 (4): 522-532

Abstract

Background: 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) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intra-class correlation coefficients (ICCs) between AM and PM tests] were measured. Results: Forced Vital Capacity (FVC, 100% of subjects) showed a mean 47.8 ± 22% predicted (ICC 0.98). Brooke upper extremity functional rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs from 0.93-0.99. Manual muscle testing, range of motion, nine-hole peg, and Jebsen-Taylor Hand function testing (JHFT) had variable feasibility (99% to 70%) with ICC also ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for Brooke Scale, %predicted FVC, and hand and finger strength. Conclusion: Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use. © 2014 Wiley Periodicals, Inc.

View details for DOI 10.1002/mus.24346

View details for Web of Science ID 000351679400009

View details for PubMedID 25056178