On the basis of a review of the literature, the Filnemus neuromuscular rare diseases health network and the French Paediatric Neurology Society (SFNP), including clinicians from I-Motion, have published recommendations to harmonise practices for prescribing corticosteroid therapy and monitoring associated adverse effects in children with Duchenne muscular dystrophy.
With regard to the long-term use of corticosteroid therapy, the authors based their recommendations on international guidelines.
- In France, two corticosteroid therapies are used: prednisone/prednisolone (0.75 mg/kg/day) and deflazacort (0.9 mg/kg/day), with initiation between 4 and 6 years old.
- The minimum effective dose is 0.33 mg/kg/day for prednisone/prednisolone.
- The daily dose should be adapted to weight (with a maximum of 30-40 mg prednisone/day).
- Treatment may be prescribed after clinical, biological and ophthalmological examinations, bone densitometry, updated vaccinations and family agreement.
- Efficacy should be assessed by means of interviews and functional tests, at least using the Motor Function Measurement (MFM) scale and the 6-minute walk test.
Concerning the management of adverse reactions :
- tolerance should be assessed by regular clinical and biological monitoring, radiological surveillance at least every two years and an annual ophthalmological examination.
- if corticosteroid therapy is to be discontinued because of major adverse effects, the dose should be reduced gradually to prevent the risk of acute adrenal insufficiency.
- recommendations for a number of adverse effects (osteoporosis, growth retardation, overweight and obesity, delayed puberty, cataracts, hypertension, etc.) are also detailed.
As the French recommendations point out, the benefits of corticosteroids justify their use despite the adverse effects. Careful management of this treatment, with maintenance of an optimal dose, is essential.
