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
Facioscapulohumeral dystrophy in children: design of a prospective, observational study on natural history, predictors and clinical impact (iFocus FSHD)
Goselink RJ, Schreuder TH, Mul K, Voermans NC, Pelsma M, de Groot IJ, van Alfen N, Franck B, Theelen T, Lemmers RJ, Mah JK, van der Maarel SM, van Engelen BG, Erasmus CE. BMC Neurol. 2016. 17;16:138. Protocol of a population-based prospective cohort study on...
Muscle MRI Findings in Childhood/Adult Onset Pompe Disease Correlate with Muscle Function
Figueroa-Bonaparte S, Segovia S, Llauger J, Belmonte I, Pedrosa I, Alejaldre A, Mayos M, Suárez-Cuartín G, Gallardo E, Illa I, Díaz-Manera J; Spanish Pompe Study Group. PLoS One. 2016. 6;11(10):e0163493. The main objective of the study was to assess the use of muscle...
The 6-minute walk test, motor function measure and quantitative thigh muscle MRI in Becker muscular dystrophy: A cross-sectional study
Fischer D, Hafner P, Rubino D, Schmid M, Neuhaus C, Jung H, Bieri O, Haas T, Gloor M, Fischmann A, Bonati U. Neuromuscul Disord. 2016. 26(7):414-22. This study investigated the relation between validated functional scores, such as MFM, timed function tests, such as...
Therapy Taping Method: Therapeutic approach in two children with Duchenne Muscular Dystrophy
Iwabe-Marchese C, Morini NJr. Br J Med Med Res. 2016. 15(3): 1-7. The aim of the study is to assess the effect of elastic bandage through the Therapy Taping Method in two children with DMD. The Motor Function Measure-20 scale (MFM-20) was used as an outcome...
Natural history of LGMD2A for delineating outcome measures in clinical trials
Richard I, Hogrel JY, Stockholm D, Payan CA, Fougerousse F; Calpainopathy Study Group, Eymard B, Mignard C, Lopez de Munain A, Fardeau M, Urtizberea JA. Ann Clin Transl Neurol. 2016. 4;3(4):248-65. Observational study of clinical manifestations and disease progression...
Broadening The Imaging Phenotype of Dysferlinopathy at Different Disease Stages
Díaz J, Woudt L, Suazo L, Garrido C, Caviedes P, Cardenas AM, Castiglioni C, Bevilacqua JA. Muscle Nerve. 2016. 54(2):203-10. The study aimed at describes MRI of dysferlinopathy and to correlate it with objective measures of motor dysfunction.MRI infiltration score...