Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials
Schreiber A, Brochard S, Rippert P, Fontaine-Carbonnel S, Payan C, Poirot I, Hamroun D, Vuillerot C
Dev Med Child Neurol. 2018. 60(2):185-191.
The aim of the study was 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.
Significant differences were found between mean MFM D1, D2 and D3 scores in corticosteroids-treated and untreated groups bewteen 0 and 6 months and between 0 and 24 month. For MFM D1 specifically, there was a significant increase in the corticosteroids-treated group (mean slope of change=12.6±15.5%/y), while a decrease was observed in the untreated group (mean slope of change=-17.8±17.7%/y) between 0 months and 6 months.
Keywords: Duchenne muscular dystrophy, corticosteroids, Motor Function Measure
Articles dans les revues scientifiques
Association Between Health-Related Quality of Life and Motor Function in Ambulant and Nonambulant Duchenne Muscular Dystrophy Patients
Gocheva V, Schmidt S, Orsini AL, Hafner P, Schaedelin S, Rueedi N, Weber P, Fischer D. J Child Neurol. 2019. 34(14), 873-885. This cross-sectional study showed several moderate to high correlations between different aspects of the generic and disease-specific...
Cross-cultural Adaptation and Multi-centric Validation of the Motor Function Measure Chinese Version (MFM-32-CN) for Patients with Neuromuscular Diseases
Huang M, Cao J, Sun J, Li W, Qin L, Li H, Zhai C, Huang W, Gui T, Zhang K, Wang J, Vuillerot C, Wang Y, Dai M. Dev Neurorehabil. 2019. doi: 10.1080/17518423.2019.1642413. Validation study of a Chinese version of the MFM-32, the MFM-32-CN. PubMed link Keywords: Chinese...
Motor Function Performance in Individuals with RYR1-Related Myopathies
Witherspoon JW, Vuillerot C, Vasavada RP, Waite MR, Shelton M, Chrismer IC, Jain MS, Meilleur KG. Muscle Nerve. 2019. 60(1),80-87. Natural history study of 34 individuals with RYR1- related myopathy (RYR1-RM) during 6 months. Motor deficits according to MFM-32 were...
X-linked myotubular myopathy: A prospective international natural history study
Annoussamy M, Lilien C, Gidaro T, Gargaun E, Chê V, Schara U, Gangfuß A, D'Amico A, Dowling JJ, Darras BT, Daron A, Hernandez A, de Lattre C, Arnal JM, Mayer M, Cuisset JM, Vuillerot C, Fontaine S1, Bellance R, Biancalana V, Buj-Bello A, Hogrel JY, Landy H, Servais L....
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Nagy S, Schmidt S, Hafner P, Klein A, Rubino-Nacht D, Gocheva V, Bieri O, Vuillerot C, Bonati U, Fischer D. J Vis Exp. 2019. (143), e58784. Discussion in a video format on outcome measures, including MFM, used in the trial "Treatment with L-citrulline and metformin in...
Analysis of motor and respiratory function in Duchenne muscular dystrophy patients
Luiz LC, Lima Marson FA, Almeida CCB, Contrera Toro AAD, Nucci A, Ribeiro JD. Respir Physiol Neurobiol. 2019. 262:1-11. Assessment of motor and respiratory impairment of 19 Duchenne muscular dystrophy (DMD) patients. Non-ambulatory condition was associated with worse...