The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used.
Muscle Nerve
; 48(1): 32-54, 2013 Jul.
Article
en En
| MEDLINE
| ID: mdl-23677550
ABSTRACT
UNLABELLED Contemporary natural history data in Duchenne muscular dystrophy (DMD) is needed to assess care recommendations and aid in planning future trials. METHODS:
The Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 individuals, aged 2-28 years, with DMD in a longitudinal, observational study at 20 centers. Assessments obtained every 3 months for 1 year, at 18 months, and annually thereafter included clinical history; anthropometrics; goniometry; manual muscle testing; quantitative muscle strength; timed function tests; pulmonary function; and patient-reported outcomes/health-related quality-of-life instruments.RESULTS:
Glucocorticoid (GC) use at baseline was 62% present, 14% past, and 24% GC-naive. In those ≥6 years of age, 16% lost ambulation over the first 12 months (mean age 10.8 years).CONCLUSIONS:
Detailed information on the study methodology of the CINRG DMD-NHS lays the groundwork for future analyses of prospective longitudinal natural history data. These data will assist investigators in designing clinical trials of novel therapeutics.
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Proyectos de Investigación
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Distrofia Muscular de Duchenne
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Glucocorticoides
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Cooperación Internacional
Tipo de estudio:
Clinical_trials
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Guideline
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Observational_studies
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Risk_factors_studies
Límite:
Adolescent
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Adult
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Child
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Child, preschool
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Humans
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Male
Idioma:
En
Revista:
Muscle Nerve
Año:
2013
Tipo del documento:
Article
País de afiliación:
Estados Unidos