Objectives: To evaluate the efficacy and long-term side effects of steroid therapy in Duchenne
Muscular Dystrophy (DMD).
Methods: From 1997 to 2005, 100 boys with DMD have received steroid therapy: deflazacort,
1mg/kg/day in 80, and prednisolone 0.75mg/kg/day in alternating cycles of 10 days
on and 10 days off (Muntoni & Manzur) in 20.
Results: Seventy-five boys have been treated with deflazacort from 13 months to 8 years (5
abandoned follow-up): among 61 who are older than 10 years (Y), 32 maintained independent
walking and 29 lost it (9 at 10y, 6 at 11y, 4 at 12y, 3 at 13y and 7 at 14y); among
the remaining 14 boys who are younger than 10 years, 3 patients lost independent walking
at 8 y, after one year of treatment (these boys had moderate mental deficiency and
discontinued physiotherapy). Among these 75 patients, one manifested untreatable gastralgia
and depression and deflazacort was withdrawn. Nine boys developed incipient cataracts
that are under periodic evaluation. Growth retardation occurred in 10 boys, osteoporosis
in 3 and weight gain in 10; slight gastralgia, “moon” face, and excessive hair growth
were found in all. Among the 20 patients treated with prednisolone for 12 to 24 months,
3 were severely affected from the onset and lost independent walking (2 at 7 y and
one at 8 y); only 2 developed incipient cataracts after 17 months and other significant
side-effects did not occur.
Conclusion: Most of DMD patients profit from steroid therapy showing muscular strength improvement,
prolonged ability for walking and few significant side-effects.
