Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history.
Muscle Nerve
; 58(5): 631-638, 2018 11.
Article
em En
| MEDLINE
| ID: mdl-29742798
ABSTRACT
INTRODUCTION:
Tests of ambulatory function are common clinical trial endpoints in Duchenne muscular dystrophy (DMD). Using these tests, the ImagingDMD study has generated a large data set that can describe the contemporary natural history of DMD in 5-12.9-year-olds.METHODS:
Ninety-two corticosteroid-treated boys with DMD and 45 controls participated in this longitudinal study. Participants performed the 6-minute walk test (6MWT) and timed function tests (TFT 10-m walk/run, climbing 4 stairs, supine to stand).RESULTS:
Boys with DMD had impaired functional performance even at 5-6.9 years old. Boys older than 7 had significant declines in function over 1 year for 10-m walk/run and 6MWT. Eighty percent of participants could perform all functional tests at 9 years old. TFTs appear to be slightly more responsive and predictive of disease progression than the 6MWT in 7-12.9 year olds.DISCUSSION:
This study provides insight into the contemporary natural history of key functional endpoints in DMD. Muscle Nerve 58 631-638, 2018.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Imageamento por Ressonância Magnética
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Caminhada
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Avaliação de Resultados em Cuidados de Saúde
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Distrofia Muscular de Duchenne
Tipo de estudo:
Clinical_trials
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Etiology_studies
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Incidence_studies
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Observational_studies
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Prognostic_studies
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Risk_factors_studies
Limite:
Adolescent
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Child
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Child, preschool
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Female
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Humans
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Male
Idioma:
En
Revista:
Muscle Nerve
Ano de publicação:
2018
Tipo de documento:
Article
País de afiliação:
Estados Unidos