Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy.
Muscle Nerve
; 51(4): 522-32, 2015 Apr.
Article
em En
| MEDLINE
| ID: mdl-25056178
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.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Corticosteroides
/
Distrofia Muscular de Duchenne
/
Mãos
Tipo de estudo:
Guideline
/
Prognostic_studies
Aspecto:
Patient_preference
Limite:
Adolescent
/
Adult
/
Child
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Humans
/
Male
Idioma:
En
Revista:
Muscle Nerve
Ano de publicação:
2015
Tipo de documento:
Article
País de afiliação:
Estados Unidos