Background: People with Duchenne Muscular Dystrophy (DMD) are living into adulthood with increased options for treatment. The 2018 guidelines for steroid dosing recommend a starting dose of 0.75mg/kg/day of prednisone or 0.9 mg/kg/day of deflazacort.(1) In a 2019 study, only 49% of non-ambulatory teenagers in the Duchenne Registry were on steroids.(2) There is limited evidence or guidelines on optimal care in adults with DMD seen in adult MDA clinics.
Objectives: To describe current steroid practice patterns in adults with DMD at a single-center adult MDA clinic.
Results: A total of 64 adults with DMD were seen from 2010-2024 for at least one visit. Forty-two patients (66%) were on corticosteroids at their first visit. Twenty-five (60%) were on prednisone and seventeen were on deflazacort (40%). Most patients were on daily steroids (32, 76%) and the average daily dose for prednisone was 0.18 mg/kg/day and for deflazacort was 0.27 mg/kg/day. Additional dosing regimens included every other day, five days per week, and two days per week. At a subsequent encounter, three individuals increased their steroid dose, three decreased, and one stopped. No individuals who were off steroids at their initial visit started them at a future visit.
Conclusions: Two-thirds of adults with DMD were on corticosteroids at their first visit. All patients not on steroids or underdosed were offered appropriate treatment doses of steroids, but only three adults chose to increase their dose. Daily dosing was the most common dosing regimen. The average prednisone dose of 0.18 mg/kg/day and deflazacort dose of 0.27 mg/kg/day were below the recommended dosing. This cohort highlights the variability and technical sub-optimal dosing of steroids in adults with DMD. It remains unclear, though, what the true optimal dosing in adults should be and this requires further investigation.
1 Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management [published correction appears in Lancet Neurol. 2018 Jun;17(6):495. doi: 10.1016/S1474-4422(18)30125-X]. Lancet Neurol. 2018;17(3):251-267. doi:10.1016/S1474-4422(18)30024-3
2 Cowen L, Mancini M, Martin A, Lucas A, Donovan JM. Variability and trends in corticosteroid use by male United States participants with Duchenne muscular dystrophy