OBJECTIVES:

This study aims to understand the holistic disease burden and treatment experiences for Duchenne muscular dystrophy (DMD) patients despite the use of pharmacotherapy, with a focus on corticosteroid (CS) use.

METHODS:

An IRB-approved, retrospective chart audit was conducted between October-November 2022. Forty-six neurologists, cardiologists, and endocrinologists who manage at least 10 DMD patients completed a study instrument and provided a total of 103 charts for DMD patients who have recent CS experience. Patient demographics, comorbidities, treatment history, symptoms, and healthcare resource utilization (HCRU) were abstracted.

RESULTS:

Of the charts, 97% of patients were currently taking CSs (53% prednisone; 44% deflazacort). Most patients (72% of prednisone users; 54% of deflazacort users) experienced side effects. Stunted growth and behavioral problems were common side effects (≥15%) experienced by patients on both prednisone and deflazacort. Regarding CS dosing, 33% of prednisone users and 46% of deflazacort users were prescribed lower/less frequent doses than indicated. Physicians reported the top reason for this was to reduce side effects/improve tolerability (62% and 64%, respectively) which reportedly impacted efficacy for >80% of patients that received the lower dose. Physicians reported the leading reason to stop prednisone was the availability of a more effective treatment (30%), while the leading reason to stop deflazacort was efficacy not outweighing safety/tolerability issues (33%). Regarding patient management, physicians reported 17% of their DMD patients are CS naïve. Nearly half of physicians (44%) reported their CS naïve patients refused CSs with the most common reason given as fear of side effects (92%).

CONCLUSIONS:

DMD patients currently being treated by CSs experience side effects and are often prescribed sub-therapeutic doses, which can drive HCRU and suggests some patients may not experience the intended therapeutic benefit. This study highlights the need for a therapy with comparable efficacy that can deliver an improved safety/tolerability profile.