A comparison of daily and alternate-day prednisone therapy in the treatment of Duchenne muscular dystrophy
G. M. Fenichel, J. R. Mendell, R. T. Moxley 3rd, R. C. Griggs, M. H. Brooke, J. P. Miller, A. Pestronk, J. Robison, W. King, L. Signore and al. et
Department of Neurology, Vanderbilt University Medical Center, Nashville, TN 37212.
We previously reported the results of a randomized, double-blind 6-month
trial of prednisone therapy in which 102 boys aged 5 to 15 years with
Duchenne muscular dystrophy received daily doses of 1.5 and 0.75 mg/kg per
day and were compared with those receiving placebo. The strength and
function in both prednisone-treated groups improved equally and were
significantly better than in the placebo group. To compare alternate-day
and daily dosing of prednisone with respect to benefits and adverse side
effects, the placebo group was started on alternate-day prednisone therapy,
and the treatment group regimens were changed to equivalent doses of
alternate-day prednisone without breaking the double-blind nature. At the
end of 6 months, the group that was changed from daily to alternate-day
therapy had declined in strength back to levels observed 12 months
previously, at the start of daily therapy. The group in which alternate-day
therapy was started showed a significant improvement in strength at 3
months, similar in magnitude to the response of boys treated with daily
therapy. However, their strength declined significantly in the subsequent 3
months compared with boys who received daily therapy. The frequency of side
effects was not significantly different for alternate-day therapy compared
with daily therapy. We conclude that alternate-day prednisone therapy
effectively increases strength but does not sustain the improvement to the
same extent as daily therapy or mitigate side effects.