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Journal Article

Citation

Vuillerot C, Payan C, Girardot F, Fermanian J, Iwaz J, Bérard C, Ecochard R. Arch. Phys. Med. Rehabil. 2012; 93(12): 2251-6.e1.

Affiliation

Hospices Civils de Lyon, Hôpital Femme-Mère-Enfant, L'Escale, Service de Médecine Physique et de Réadaptation Pédiatrique, Bron, F-69500, France.

Copyright

(Copyright © 2012, Elsevier Publishing)

DOI

10.1016/j.apmr.2012.05.025

PMID

22705238

Abstract

OBJECTIVES: To study the responsiveness (sensitivity to change) of the Motor Function Measure in detecting change in neuromuscular disease patients with the intent of using this Measure in future clinical trials. DESIGN: Prospective cohort observational study. SETTING: Inpatient and outpatient facilities for follow-up and treatment of neuromuscular diseases. PARTICIPANTS: Patients (152) with various neuromuscular diseases aged 6-60 years old. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURE(S): We used the Motor function Measure Total score and its four subscores on two measurements grossly one year apart. The physicians and the patients (or proxy) were asked to provide their perceived change in functional status since the first Motor Function Measure. These changes were expressed in three outcomes: deterioration, stability, or improvement RESULTS: The overall 12-month-standardized mean change of the total score mean±SD annual total score change was -2.4±5.5 points (p<0.0001), Duchenne patients presenting the most significant change (-5.8±6.3, p<0.0001). The change in patients reporting deterioration (34%) was significantly larger than that of those reporting stability (47%) or improvement (10%) (-4.4±6.4 vs. -2.0±5.6 and +0.9±4.4 points, respectively; p<0.01). The 12-month-standardized total score changes were significantly greater in physician-rated deteriorated (49%) vs. stable patients (51%) with mean differences in scores -5.3±7.6 and -1.2±5.3 and, respectively (p<0.001). CONCLUSIONS: The Motor Function Measure showed a good responsiveness, especially in Duchenne patients, and agreements with patients' and physician's perceived change. Confirming this responsiveness requires larger age-groups of patients with Duchenne and other neuromuscular diseases as well as disease-specific inter-examination delays.


Language: en

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