Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade

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Abstract

We compare the clinical course of 74 boys 10–18 years of age with Duchenne muscular dystrophy (DMD) treated (40) and not treated (34) with deflazacort. Treated boys were able to rise from supine to standing, climb stairs and walk 10 m without aids, 3–5 years longer than boys not treated. After 10 years of age, treated boys had significantly better pulmonary function than boys not treated and after15 years of age, 8 of 17 boys not treated required nocturnal ventilation compared with none of the 40 treated boys. For boys over 15 years of age, 11 of 17 boys not treated required assistance with feeding compared to none of the treated boys. By 18 years, 30 of 34 boys not treated had a spinal curve greater than 20° compared to 4 of 40 treated boys. By 18 years, 7 of 34 boys not treated had lost 25% or more of their body weight (treated 0 of 40) and 4 of those 7 boys required a gastric feeding tube. By 18 years, 20 of 34 boys not treated had cardiac left ventricular ejection fractions less than 45% compared to 4 of 40 treated boys and 12 of 34 died in their second decade (mean 17.6±1.7 years) primarily of cardiorespiratory complications. Two of 40 boys treated with deflazacort died at 13 and 18 years of age from cardiac failure. The treated boys were significantly shorter, did not have excessive weight gain and 22 of 40 had asymptomatic cataracts. Long bone fractures occurred in 25% of boys in both the treated and not treated groups. This longer-term study demonstrates that deflazacort has a very significant impact on health, quality of life and health care costs for boys with DMD and their families, and is associated with few side effects.

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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