Duchenne muscular dystrophy: updates on corticosteroid treatment

*Correspondence: [e-mail].

Abstract

Background: Corticosteroids are the current standard of care for patients with Duchenne Muscular Dystrophy (DMD), an X-linked recessive dystrophinopathy that causes muscle weakness, atrophy and premature death from cardiorespiratory complications. Corticosteroids are the only medication that so far has effectively altered the natural history of the disease and those that have evolved both in terms of safety and efficacy are the focus of this review.

Objective: To review the advances related to corticosteroid treatment and its effects on patients with DMD and to describe the different types of corticosteroids and compare them in terms of their effectiveness.

Methods: A systematic review was carried out in the Pubmed, Scielo, BVS and Web of Science databases with the descriptors duchenne, treatment and corticosteroids. Exclusion criteria: samples with non-humans, focus on treatments other than corticosteroid therapy, duplicates, not written in English or Portuguese and articles that have not been published in the last 5 years. Of 993 studies, 28 were included.

Results: The described options for corticosteroids were Prednisone, Deflazacort and Valmorolone. The benefits include increased survival, later loss of ambulation, maintenance of upper limb function, reduced incidence of severe scoliosis, and improved cardiopulmonary function. Prednisone was the first disease-modifying agent found to improve motor function. Adverse effects observed were bone loss, behavioral changes, weight gain, upper respiratory tract infections, and vomiting. Deflazacort was better tolerated due to being associated with fewer adverse effects of weight gain than Prednisone, facilitating ambulation. However, it presents growth deceleration, development of non-severe cataracts and reduction in serum levels of collagen I. Valmorolone has shown reduced side effects, such as improved bone profile without a decrease in bone turnover markers. However, it was observed that all three medications lead to increased adrenal insufficiency. There is evidence of positive effects in corticosteroid therapy. Adverse effects have been described, such as bone loss.

Conclusion: New medications such as Valmolorone present greater benefits by reducing adverse effects. The mechanisms of action for different dosage regimens are beginning to be understood in preclinical models, so further studies are necessary to expand the treatment of DMD.

Publication History

Article published online:
12 May 2025