Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy

• DOI: 10.1001/jama.2022.4315
• Publication Date: 2022-04-05T19:30:32Z
• Authors: Michela Guglieri; Kate Bushby; Michael P. McDermott; Kimberly A. Hart; Rabi Tawil; William B. Martens; Barbara E. Herr; Elaine McColl; Chris Speed; Jennifer Wilkinson; Janbernd Kirschner; Wendy M. King; Michelle Eagle; Mary W. Brown; Tracey Willis; Robert C. Griggs; ; Volker Straub; Henriette van Ruiten; Anne-Marie Childs; Emma Ciafaloni; Perry B. Shieh; Stefan Spinty; Lorenzo Maggi; Giovanni Baranello; Russell J. Butter...; I. A. Horrocks; Helen Roper; Zoya Alhaswani; Kevin M. Flanigan; Nancy L. Kuntz; Adnan Manzur; Basil T. Darras; Peter B. Kang; Leslie Morrison; Monika Krzesniak-...; Jean K. Mah; Tiziana E. Mongini; Federica Ricci; Maja von der Hagen; Richard S. Finkel; Kathleen O’Reardon; Matthew Wicklund; Ashutosh Kumar; Craig M. McDonald; Jay J. Han; Nanette Joyce; Erik K. Henricson; Ulrike Schara-Sch...; Andrea Gangfuss; Ekkehard Wilichowski; Richard J. Barohn; Jeffrey M. Statland; Craig Campbell; Giuseppe Vita; Gian Luca Vita; James F. Howard; Imelda Hughes; Hugh J. McMillan; Elena Pegoraro; Luca Bello; W. Bryan Burnette; Mathula Thangarajh; Taeun Chang
• Keywords: Male; Medizin; 610; Duchenne; Muscular Dystrophy, Duchenne; Child; Child, Preschool; Female; Humans; Male; Pregnenediones; Glucocorticoids; Muscular Dystrophy, Duchenne; Prednisone; 03 medical and health sciences; 0302 clinical medicine; Pregnenediones; Child, Preschool; Humans; Prednisone; Female; Muscular Dystrophy; Preschool; Child; Glucocorticoids

Corticosteroids improve strength and function in boys with Duchenne muscular dystrophy. However, there is uncertainty regarding the optimum regimen dosage.To compare efficacy adverse effects of 3 most frequently prescribed corticosteroid regimens dystrophy.Double-blind, parallel-group randomized clinical trial including 196 aged 4 to 7 years dystrophy who had not previously been treated corticosteroids; enrollment occurred between January 30, 2013, September 17, 2016, at 32 clinic sites 5 countries. The were assessed for (last participant visit on October 16, 2019).Participants daily prednisone (0.75 mg/kg) (n = 65), deflazacort (0.90 or intermittent mg/kg 10 days then off) 66).The global primary outcome comprised end points: rise from floor velocity (in rise/seconds), forced vital capacity liters), parent satisfaction treatment measured by Treatment Satisfaction Questionnaire Medication (TSQM; score range, 0 100), each averaged across all study visits after baseline. Pairwise group comparisons used a Bonferroni-adjusted significance level .017.Among (mean age, 5.8 [SD, 1.0 years]), 164 (84%) completed trial. Both more effective than (P < .001 vs using test; P .017 test) did differ significantly .38 test). between-group differences principally attributable (0.06 rise/s [98.3% CI, 0.03 0.08 rise/s] [P .003]; 0.06 0.09 .017]; -0.004 -0.03 0.02 .75]). pairwise TSQM subscale statistically significant. common events abnormal behavior (22 [34%] group, 25 [38%] 24 [36%] group), upper respiratory tract infection (24 [37%], 19 [29%], [36%], respectively), vomiting (19 17 [26%], 15 [23%]).Among patients dystrophy, deflazacort, compared alternating off, resulted significant improvement over composite comprising measures motor function, pulmonary treatment; was no difference 2 regimens. findings support use tested this as initial dystrophy.ClinicalTrials.gov Identifier: NCT01603407.