Title :

Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials.

Author(s) :

Schreiber, Audrey [Auteur]
Hôpital Morvan - CHRU de Brest [CHU - BREST ]
Brochard, Sylvain [Auteur]
CHRU Brest - Service de médecine physique et de réadaptation [CHU Brest - Service de médecine physique ]
Rippert, Pascal [Auteur]
Pôle Information Médicale Evaluation Recherche [IMER]
Hôpital Femme Mère Enfant [CHU - HCL] [HFME]
Hospices Civils de Lyon [HCL]
Fontaine-Carbonnel, Stephanie [Auteur]
Hôpital Femme Mère Enfant [CHU - HCL] [HFME]
Payan, Christine [Auteur]
CHU Pitié-Salpêtrière [AP-HP]
Poirot, Isabelle [Auteur]
Hôpital Femme Mère Enfant [CHU - HCL] [HFME]
Hamroun, Dalil [Auteur]
Centre Hospitalier Régional Universitaire [Montpellier] [CHRU Montpellier]
Vuillerot, Carole [Auteur]
Laboratoire de Biométrie et Biologie Evolutive - UMR 5558 [LBBE]
Université Claude Bernard Lyon 1 [UCBL]

Journal title :

Developmental Medicine and Child Neurology

Abbreviated title :

Dev Med Child Neurol

Volume number :

60

Pages :

185-191

Publication date :

2018-02-01

ISSN :

1469-8749

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

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. This observational, multicentre ...
Show more >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. 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. 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. 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. 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.Show less >

Language :

Anglais

Peer reviewed article :

Oui

Audience :

Internationale

Popular science :

Non

Collections :

• Unité de Recherche Pluridisciplinaire Sport, Santé, Société (URePSSS) - ULR 7369

Research team(s) :

Activité Physique, Muscle, Santé (APMS)

Submission date :

2019-09-24T10:01:07Z
2024-02-26T09:24:37Z