Long-Term Corticosteroids Benefit Motor, Respiratory Function in Duchenne Patients

A new meta-analysis of longitudinal studies supports the use of corticosteroids as a means to preserve motor and respiratory function in patients with Duchenne muscular dystrophy (DMD). The analysis, however, found the benefits are less clear in terms of cardiac outcomes.

The findings were presented in an abstract at the Muscular Dystrophy Association’s 2025 Clinical and Scientific Conference, held last month in Dallas.

Lead author Mahmoud M. Elsayed, M.D., of the MME Foundation, and colleagues wrote that while corticosteroids are considered central to DMD management, the long-term effects of the therapy on key body systems have been under-explored.

Elsayed and colleagues searched academic databases to identify clinical trials and longitudinal studies involving the use of corticosteroids in DMD. They found a total of 18 studies that collectively included 1,245 participants.

When they analyzed the data, they found corticosteroid therapy delayed patients’ loss of ambulation by 2.1 years. The data further showed that patients who used corticosteroid therapy had better scores on the North Star Ambulatory Assessment, with the most benefit seen in patients who began corticosteroid therapy before age 7. The North Star Ambulatory Assessment is a rating scale used to measure motor function in ambulant children with Duchenne muscular dystrophy.

Elsayed and colleagues reported similar findings in terms of pulmonary function, with participants on corticosteroids reporting an annualized reduction in forced vital capacity (FVC) of 0.8, an improvement over participants who were not on corticosteroids.

The investigators said the data on cardiac outcomes were mixed, although at least one corticosteroid appeared to have an effect.

“Deflazacort (Calcort) may provide cardioprotective benefits, but more research is needed,” they wrote.

They said quality of life was also improved as disease progression was slowed, although they said that benefit could be limited by side effects of corticosteroid use.

Elsayed and colleagues’ report was the second meta-analysis in recent weeks to examine corticosteroids.

An earlier report, published in the Annals of Medicine & Surgery, looked at whether dosing regimens affected the efficacy of corticosteroids in people with DMD. The report noted that corticosteroid regimens have been linked with weight gain, behavioral changes, and growth delays, leading some patients and clinicians to opt for intermittent dosing regimens.

Pratik Bhattarai, MBBS

Given the trade-offs involved in corticosteroid use in patients with DMD, corresponding author Pratik Bhattarai, MBBS, of the Manipal College of Medical Sciences, in Nepal, and colleagues conducted a literature review to find studies that compared daily with intermittent prednisone use in DMD.

The results showed that for most parameters, there was no significant difference between the two dosing strategies. FVC, blood pressure, loss of ambulation, weight, bone fractures, behavioral changes, and bone density were all statistically similar regardless of whether patients took prednisone daily or intermittently.

The only areas where a gap existed were in cushingoid appearance, hair growth, and hypertension, all of which were more prevalent among patients receiving intermittent prednisone. The largest gap was an increased risk ratio (RR) for hypertension among patients with intermittent prednisone.

Bhattarai and colleagues said their findings support “the use of either daily or intermittent prednisone based on individual patient needs and side effect profiles. This flexibility in treatment options can help optimize outcomes and patient experiences.”

Still, they added that their work also exposes the need for more research into the effects of corticosteroids in DMD. They argued future studies should examine how corticosteroids may fit with newer therapies, including gene therapy.