Abstract
IMPORTANCE: Corticosteroids improve strength and function in boys with Duchenne muscular dystrophy. However, there is uncertainty regarding the optimum regimen and dosage. OBJECTIVE: To compare efficacy and adverse effects of the 3 most frequently prescribed corticosteroid regimens in boys with Duchenne muscular dystrophy. DESIGN, SETTING, AND PARTICIPANTS: Double-blind, parallel-group randomized clinical trial including 196 boys aged 4 to 7 years with Duchenne muscular dystrophy who had not previously been treated with corticosteroids; enrollment occurred between January 30, 2013, and September 17, 2016, at 32 clinic sites in 5 countries. The boys were assessed for 3 years (last participant visit on October 16, 2019). INTERVENTIONS: Participants were randomized to 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 then 10 days off) (n = 66). MAIN OUTCOMES AND MEASURES: The global primary outcome comprised 3 end points: rise from the floor velocity (in rise/seconds), forced vital capacity (in liters), and participant or parent global satisfaction with treatment measured by the Treatment Satisfaction Questionnaire for Medication (TSQM; score range, 0 to 100), each averaged across all study visits after baseline. Pairwise group comparisons used a Bonferroni-adjusted significance level of .017. RESULTS: Among the 196 boys randomized (mean age, 5.8 years [SD, 1.0 years]), 164 (84%) completed the trial. Both daily prednisone and daily deflazacort were more effective than intermittent prednisone for the primary outcome (P < .001 for daily prednisone vs intermittent prednisone using a global test; p =" .017" for daily deflazacort vs intermittent prednisone using a global test) and the daily regimens did not differ significantly (p =" .38" for daily prednisone vs daily deflazacort using a global test). the between-group differences were principally attributable to 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 pairwise comparisons for forced vital capacity and tsqm global satisfaction subscale score were not statistically significant. the most common adverse events 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%], respectively), and vomiting (19 [29%], 17 [26%], and 15 [23%]). conclusions and relevance: among patients with duchenne muscular dystrophy, treatment with daily prednisone or daily deflazacort, compared with intermittent prednisone alternating 10 days on and 10 days off, resulted in significant improvement over 3 years in a composite outcome comprising measures of motor function, pulmonary function, and satisfaction with treatment; there was no significant difference between the 2 daily corticosteroid regimens. the findings support the use of a daily corticosteroid regimen over the intermittent prednisone regimen tested in this study as initial treatment for boys with duchenne muscular dystrophy. trial registration: clinicaltrials.gov identifier: nct01603407.>
