BACKGROUND: Corticosteroid treatment with prednisone/prednisolone or deflazacort is the standard of care for dystrophinopathies. Improved understanding of real-world switching is needed following deflazacort approval.
OBJECTIVE: To describe reasons for switching from prednisone/prednisolone to deflazacort, and clinical outcomes, among US patients with dystrophinopathies.
METHODS: Chart review conducted by 55 neurologists for patients with dystrophinopathy who switched from prednisone to deflazacort between February 2017 and December 2018.
RESULTS: Charts were reviewed for 102 males in community (49%) and academic (51%) settings. Seventy had Duchenne muscular dystrophy; mean ± SD age 11.6 ± 10.4 years and 80% ambulatory at switch. Thirty-two had Becker’s muscular dystrophy; mean age 21.2 ± 12.5 years and 81% ambulatory at switch. Average treatment duration was 3.3 years for prednisone and 6 months for deflazacort prior to the chart extraction. Physicians ranked “desire to slow disease progression” and “tolerability issues” as primary reasons for switching in 78% and 57%, respectively. Switching was reported as “very” or “somewhat” effective at addressing primary reasons in 95% of patients. Commonly recorded adverse effects during prednisone and deflazacort treatment included weight gain (38% and 15%), Cushingoid appearance (26% and 10%), increased appetite (20% and 6%), central obesity (10% and 3%), and fluid retention (9% and 2%); hirsutism occurred for 2% and 4%. Findings were similar in the subgroup (n=62) with ≥ 3 months deflazacort. Among 34 patients with recorded Global Impression of Illness Severity, 8 (24%) improved, 24 (70%) remained the same, and 2 (6%) worsened after switching.
CONCLUSIONS: In this real-world study, the majority of switching from prednisone to deflazacort aimed to improve benefit-risk. During 6-months average follow-up after switching in this uncontrolled study, physicians reported lower proportions of most adverse effects compared to the pre-switch period. Among the minority of charts with disease severity recorded, most showed stability of progressive disease.