2005-2015: Ten years clinical experience in treating DMD patients by corticosteroids in Lyon.

OBJECTIVE: Since 2005, in France, corticosteroid therapy is now widely used in Duchenne muscular dystrophy (DMD). This treatment has changed our practice of pediatric rehabilitation teams. We describe here our 10-year clinical experience in treating DMD patients by CS according to international guidelines i.e. prednisone 0.75mg/kg/day started from the plateau of motor function.

MATERIALS/PATIENTS AND METHODS: We conducted a prospective study. Information was given on the expected effects and side effects. A diet was prescribed. At baseline, a cardiac ultrasound, an ophthalmologic examination, a bone density test, a physical and biological examination to ensure the absence of against indication, check the anti-varicella immunization. The motor function measure were performed each 6months with a medical examination and blood sample to verify the tolerance and adapt the treatment function to weight and side effects.

RESULTS: During this 10-year period, CS was proposed to 51 patients and 48 patients were finally treated up to 10.5 years. The age of onset of CS varies from 6 years to 20 years between 2005 and 2010, from 5 to 8 years after 2010. Premature stops were observed for 14 patients: 9 in the first 2 years, 5 after 2 years for side effects or inefficacy. No acute adrenal insufficiency was observed. During the first 2 years of treatment, MFM D1 score (standing position and transfers) remained stable. Then a slow decrease was observed after 2years to be null at M72. MFM D3 score (distal motor function) is more preserved than D2 (axial and proximal motor function) and remained stable during the follow up for the great majority of patients. Eleven patients started corticosteroids after losing the ability to walk (mean age 9.1±1.6 years), 7/11 had spinal fusion. Thirty-seven ambulant patients started, in this group, the mean age of losing ambulation was 11.3±2.2 years and no patient had spinal surgery.

DISCUSSION/CONCLUSION: Increased appetite, irritability, cushingoid facies are common but well tolerated by children in view of efficacy. Dose adjustments were discussed case by case if accelerated weight gain, sluggish growth in stature and behavioral disorders.