Analysis of the functional capacity outcome measures for myotonic dystrophy.
Ann Clin Transl Neurol
; 6(8): 1487-1497, 2019 08.
Article
en En
| MEDLINE
| ID: mdl-31402614
ABSTRACT
OBJECTIVES:
Defining clinically relevant outcome measures for myotonic dystrophy type 1 (DM1) that can be valid and feasible for different phenotypes has proven problematic. The Outcome Measures for Myotonic Dystrophy (OMMYD) group proposed a battery of functionaloutcomes:
6-minute walk test, 30 seconds sit and stand test, timed 10 m walk test, timed 10 m walk/run test, and nine-hole peg test. This, however, required a large-scale investigation,METHODS:
A cohort of 213 patients enrolled in the natural history study, PhenoDM1, was analyzed in cross-sectional analysis and subsequently 98 patients were followed for longitudinal analysis. We aimed to assess (1) feasibility and best practice; (2) intra-session reliability; (3) validity; and (4) behavior over time, of these tests.RESULTS:
OMMYD outcomes proved feasible as 96% of the participants completed at least one trial for all tests and more than half (n = 113) performed all three trials of each test. Body mass index and disease severity associate with functional capacity. There was a significant difference between the first and second trials of each test. There was a moderate to strong correlation between these functional outcomes and muscle strength, disease severity and patient-reported outcomes. All outcomes after 1 year detected a change in functional capacity except the nine-hole peg test.CONCLUSIONS:
These tests can be used as a battery of outcomes or independently based on the shown overlapping psychometric features and strong cross-correlations. Due to the large and heterogeneous sample of this study, these results can serve as reference values for future studies.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Fuerza Muscular
/
Prueba de Paso
/
Distrofia Miotónica
Tipo de estudio:
Guideline
/
Observational_studies
/
Prevalence_studies
/
Risk_factors_studies
Aspecto:
Patient_preference
Límite:
Adult
/
Female
/
Humans
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Male
/
Middle aged
Idioma:
En
Revista:
Ann Clin Transl Neurol
Año:
2019
Tipo del documento:
Article
País de afiliación:
Reino Unido