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Outcome reliability in non‐Ambulatory Boys/Men with duchenne muscular dystrophy
Author(s) -
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.
Publication year - 2015
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.24346
Subject(s) - duchenne muscular dystrophy , ambulatory , intraclass correlation , medicine , physical therapy , clinical trial , physical medicine and rehabilitation , muscular dystrophy , surgery , psychometrics , clinical psychology
: 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