Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade
Section snippets
Patients
All 74 boys who met the following criteria were included: between 10 and 18 years of age, diagnosed with DMD, could cooperate for reproducible muscle and pulmonary function testing and were followed in our comprehensive neuromuscular clinic between January 1990 and December 2004. All patients fulfilled the following five diagnostic criteria for DMD: (1) onset of weakness before 5 years of age, (2) male sex, (3) proximal muscle weakness, (4) increased serum creatine kinase, and (5) a muscle
Results
Seventy-four boys met the inclusion criteria, 40 in the deflazacort group (mean age 15.2±2.7 years) and 34 in the not treated group (boys not treated with deflazacort) (mean age 15.2±2.5 years). The mean age of starting deflazacort was 7.7±1.2 years. The mean time on deflazacort was 5.5 years.
Discussion
Until there is a cure for this fatal genetic disorder, the mainstays of treatment include corticosteroids, surgery when needed and comprehensive medical and physical rehabilitation with a focus on cardiorespiratory health. There is general agreement that boys with DMD benefit from corticosteroid treatment. [16], [17], [18], [19], [20]. Daily treatment with prednisone (0.75 mg/kg per day) or deflazacort (0.9 mg/kg per day) [24] seems to offer the most effective treatment when the boys are still
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