The effects of glucocorticoids on cardiac function of patients with Duchenne muscular dystrophy: benefit or not?

Duchenne muscular dystrophy (DMD) is a progressive, incurable X-linked neuromuscular disease caused by mutations in the dystrophin gene, resulting in functional dystrophin deficiency. Currently, cardiovascular complications are the leading cause of death in patients with DMD. Glucocorticoids are considered the gold standard treatment for children with DMD. Long-term glucocorticoid therapy can delay the loss of independent ambulation, improve lung function, and extend lifespan. However, the effects of glucocorticoids on cardiac function in patients with DMD remain controversial. This scoping review aims to summarize and analyze published clinical studies investigating the effects of glucocorticoids on cardiac function in children with DMD. A comprehensive search was conducted using PubMed, Web of Science, and Embase databases with relevant search terms. Abstracts and full texts of retrieved studies were reviewed. The studies were categorized into four themes: glucocorticoid use, Types of glucocorticoids, administration methods, and timing of glucocorticoid initiation. A total of 21 studies were included. Of these, 18 studies investigated the effects of glucocorticoids on cardiac function in patients with DMD, and the study of Koeks et al. reported both effective and non-effective outcomes of glucocorticoids on cardiac function stratified by age group, respectively. One study examined the impact of different glucocorticoid types, one study assessed the effects of glucocorticoid administration methods and one study evaluated the timing of glucocorticoid initiation. Among the 21 studies, 13 studies (n = 1814 patients) indicated that glucocorticoids could delay the progression of cardiac dysfunction in patients with DMD. Six studies (n = 6294 patients) reported no significant effects of glucocorticoids on cardiac function, while one study (n = 111 patients) suggested that early glucocorticoid therapy increased the risk of cardiomyopathy.

conclusionIt has been suggested that corticoids may delay the deterioration of cardiac function in patients with DMD. However, limited data exist on the long-term effects of early glucocorticoid therapy on cardiac function, leading to inconclusive findings. Prospective longitudinal studies are needed to determine the optimal timing, dose regimen, and long-term impact of glucocorticoid therapy in patients with DMD. WHAT IS KNOWN: • The effects of glucocorticoids on cardiac function in patients with DMD remain controversial. WHAT IS NEW: • Glucocorticoids can delay the deterioration of cardiac function in DMD patients. However, prospective longitudinal studies are still needed to determine the optimal timing, dose regimen, and long-term effect of glucocorticoid therapy in DMD patients.