Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up.
Muscle Nerve
; 54(4): 681-9, 2016 10.
Article
em En
| MEDLINE
| ID: mdl-26930423
ABSTRACT
INTRODUCTION:
Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established.METHODS:
Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6-month intervals for 2 years. We analyzed all subjects using an intent-to-treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures.RESULTS:
Eight patients (12-33 years old) died during the study. Sixty-six completed 12-month follow-up, and 51 completed 24-month follow-up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years.CONCLUSION:
Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non-ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54 681-689, 2016.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Participação do Paciente
/
Distrofia Muscular de Duchenne
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Limitação da Mobilidade
Tipo de estudo:
Observational_studies
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Prognostic_studies
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Risk_factors_studies
Limite:
Adolescent
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Adult
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Child
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Humans
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Male
Idioma:
En
Revista:
Muscle Nerve
Ano de publicação:
2016
Tipo de documento:
Article
País de afiliação:
Estados Unidos