Responsiveness of the motor function measure in neuromuscular diseases.
OBJECTIVE
To study the responsiveness (sensitivity to change) of the Motor Function Measure (MFM) in detecting change in neuromuscular disease patients with the intent of using this measure in future clinical trials.


METHODS
Prospective cohort observational study.


METHODS
Inpatient and outpatient facilities for follow-up and treatment of neuromuscular diseases.


METHODS
Patients (N=152) with various neuromuscular diseases aged 6 to 60 years.


METHODS
Not applicable.


METHODS
We used the MFM total score and its 3 subscores on 2 measurements grossly 1 year apart. The physicians and the patients (or proxy) were asked to provide their perceived change in functional status since the first MFM. These changes were expressed in 3 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<.001), with patients with Duchenne muscular dystrophy (DMD) presenting the most significant change (-5.8±6.3, P<.001). 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<.01). The 12-month-standardized total score changes were significantly greater in physician-rated deteriorated (49%) versus stable patients (51%), with mean differences in scores being -5.3±7.6 and -1.2±5.3, respectively (P<.001).


CONCLUSIONS
The MFM showed a good responsiveness, especially in patients with DMD and agreements with patients' and physicians' perceived change. Confirming this responsiveness requires larger age groups of patients with DMD and other neuromuscular diseases as well as disease-specific interexamination delays.
Hospices Civils de Lyon, Hôpital Femme-Mère-Enfant, L'Escale, Service de Médecine Physique et de Réadaptation Pédiatrique, Bron, France. carole.vuillerot@chu-lyon.fr
Carole
C
Vuillerot
Christine
C
Payan
Françoise
F
Girardot
Jacques
J
Fermanian
Jean
J
Iwaz
Carole
C
Bérard
René
R
Ecochard
D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D004185:Disability Evaluation; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D051346:Mobility Limitation; D009048:Motor Skills; D020388:Muscular Dystrophy, Duchenne / Q000503:physiopathology / Q000534:rehabilitation; D009468:Neuromuscular Diseases / Q000503:physiopathology* / Q000534:rehabilitation*; D010465:Perception; D026741:Physical Therapy Modalities; D011446:Prospective Studies; D055815:Young Adult