251. Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials.
- Author
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Schreiber, Audrey, Brochard, Sylvain, Rippert, Pascal, Fontaine‐Carbonnel, Stephanie, Payan, Christine, Poirot, Isabelle, Hamroun, Dalil, Vuillerot, Carole, MFM DMD Corticosteroids Group, and Fontaine-Carbonnel, Stephanie
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CORTICOSTEROIDS , *DUCHENNE muscular dystrophy , *SENSITIVITY analysis , *CLINICAL trials , *MOTORS , *HORMONE therapy , *COMPARATIVE studies , *FUNCTIONAL assessment , *LONGITUDINAL method , *RESEARCH methodology , *MEDICAL cooperation , *MOVEMENT disorders , *MUSCLE strength , *NONPARAMETRIC statistics , *RESEARCH , *TIME , *EVALUATION research , *SEVERITY of illness index , *DISEASE complications - Abstract
Aim: To monitor the evolution of the motor function of ambulatory patients with Duchenne muscular dystrophy (DMD) treated by corticosteroids for 2 years in comparison with untreated patients.Method: This observational, multicentre cohort study explores the evolution of the motor function measure (MFM) over a 24-month period for 29 ambulant corticosteroids-treated and 45 ambulant untreated patients with DMD.Results: Significant differences were found between mean MFM scores in corticosteroids-treated and untreated groups for domain 1 of the MFM (standing position and transfers; D1), domain 2 of the MFM (axial and proximal motor function; D2), and domain 3 of the MFM (distal motor function; D3). Subscores were between 0 months and 6 months, and 0 months and 24 months. For the D1 subscore specifically, there was a significant increase in the corticosteroids-treated group (mean±standard deviation [SD] slope of change=12.6±15.5%/y), while a decrease was observed in the untreated group (-17.8±17.7%/y) between 0 months and 6 months (p<0.001). Sensitivity to change as assessed by standardized response means was high between 12 months and 24 months for D1 of both corticosteroids-treated and untreated groups (1.0 and 1.2 respectively), and low for D2 and D3 of both treated and untreated groups.Interpretation: Patients with DMD treated by corticosteroids present a different course of the disease as assessed by MFM, confirming the sensitivity to change of the MFM in this population.What This Paper Adds: Corticosteroids have a quantitative impact on muscle strength 6 to 24 months after starting treatment. Motor function measure is a valid outcome measure in Duchenne muscular dystrophy patients under corticosteroid treatment. [ABSTRACT FROM AUTHOR]- Published
- 2018
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