Deflazacort (Emflaza®) delays the progression of multiple ambulatory milestones in Duchenne muscular dystrophy (DMD) patients when compared to prednisone, confirmed a new study presented at the 2022 Muscular Dystrophy Association Clinical & Scientific Conference.

The study compared the ages of patients who were treated with daily deflazacort or prednisone at disease progression milestones.

“This study adds to the evidence comparing outcomes across DMD patients on different steroid types using a longitudinal and geographically-diverse dataset,” the researchers wrote. 


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The study compared 288 DMD patients treated with deflazacort and 175 treated with prednisone. The dose and duration of the treatment and the functional status of patients at baseline were similar.

Read more about the treatment of DMD.

The researchers reported that deflazacort was associated with significant delays in median age at all progression milestones. In terms of the timed rising from floor test, patients treated with deflazacort had a delay of 10 seconds or more 0.88 years later and a delay of 5 seconds or more 0.94 years later, compared to patients treated with prednisone. 

The time it took for patients treated with deflazacort to not be able to rise from the floor any longer was 1.61 years longer and to not be able to complete a 4-stair climb was 1.87 years longer than patients treated with prednisone.

Patients treated with deflazacort also completely lost ambulation when they were older compared to those who were treated with prednisone (15.92 years vs 14.89 years).

Corticosteroids are the standard of care for the treatment of patients with DMD. They do not address the underlying cause of the disease but can be prescribed to delay the loss of ambulation. They work by strengthening the membrane of muscle cells and reducing inflammation. However, their side effects can be severe, especially if used for prolonged periods , and include weight gain, osteoporosis, hypertension, and glaucoma. 

Reference

McDonald C, Santos CL, Able R, et al. Association between daily deflazacort or prednisone and ages at disease progression milestones among patients with Duchenne muscular dystrophy (DMD). Poster presented at: 2022 Muscular Dystrophy Association Clinical & Scientific Conference; March 13-16, 2022; Nashville, TN. Poster 110.