For a study, researchers sought to examine the effectiveness and side effects of the three most commonly administered corticosteroid regimens in boys with Duchenne muscular dystrophy. Double-blind, parallel-group randomized clinical study including 196 boys aged 4 to 7 years with Duchenne muscular dystrophy who had not previously been treated with corticosteroids; enrolment took place between January 30, 2013, and September 17, 2016, at 32 clinic locations across five countries. For three years, the boys were evaluated (last participant visit on October 16, 2019). Daily prednisone (0.75 mg/kg) (n=65), daily deflazacort (0.90 mg/kg) (n=65), or intermittent prednisone (0.75 mg/kg for 10 days on and 10 days off) (n = 66) were given to participants. The global primary outcome included three end points: rise from the floor velocity (in rise/seconds), forced vital capacity (in liters), and participant or parent global satisfaction with treatment as measured by the Treatment Satisfaction Questionnaire for Medication (TSQM; score range, 0 to 100), all of which were averaged across all study visits following baseline. A Bonferroni-adjusted significance level of .017 was utilized for pairwise group comparisons.
About 164 (84%) of the 196 boys randomized (mean age, 5.8 [SD, 1.0 years]) completed the experiment. Daily prednisone and daily deflazacort were both more effective than intermittent prednisone for the primary outcome (P=.017 for daily deflazacort vs intermittent prednisone using a global test; P =.001 for daily prednisone vs intermittent prednisone using a global test), and the daily regimens were not significantly different (P=.38 for daily prednisone vs daily deflaza Rise from the floor velocity (0.06 rise/s [98.3% CI, 0.03 to 0.08 rise/s] for daily prednisone vs intermittent prednisone [P=.003]; 0.06 rise/s [98.3% CI, 0.03 to 0.09 rise/s] for daily deflazacort vs intermittent prednisone [P=.017]; and -0.004 rise/s [98.3% CI, −0.03 to 0.02 rise/s] for daily prednisone vs daily deflazacort [P=.75]). The comparisons between forced vital capacity and TSQM global satisfaction subscale score were not statistically significant. The most common side effects were abnormal behavior (22 [34%] in the daily prednisone group, 25 [38%] in the daily deflazacort group, and 24 [36%] in the intermittent prednisone group), upper respiratory tract infection (24 [37%], 19 [29%], and 24 [36%] in the intermittent prednisone group, respectively), and vomiting (19 [29%], 17 [26%], and 15 [23%]).
Treatment with daily prednisone or daily deflazacort, compared to intermittent prednisone alternating 10 days on and 10 days off, resulted in significant improvement over 3 years in a composite outcome consisting of measures of motor function, pulmonary function, and treatment satisfaction in patients with Duchenne muscular dystrophy; there was no significant difference between the two daily corticosteroid regimens. The findings supported the use of a daily corticosteroid regimen as the initial therapy for boys with Duchenne muscular dystrophy rather than the intermittent prednisone regimen examined in the trial.
Reference:jamanetwork.com/journals/jama/article-abstract/2790925
