Adrenal insufficiency in individuals with Duchenne muscular dystrophy treated with glucocorticoids: Insights from the past, current challenges, and future directions.

Duchenne muscular dystrophy (DMD) is an X-linked recessive dystrophinopathy characterized by progressive loss of muscle strength, respiratory insufficiency, cardiac dysfunction, and premature death. Despite a rapidly evolving therapeutic landscape that now includes multiple disease modifying treatments including exon skipping medications, microdystrophin gene therapy, and givinostat, high dose glucocorticoids (GCs) initiated at a young age remain central to the standard of care for DMD. Individuals treated with GCs are at high risk of adrenal insufficiency, an iatrogenic and potentially life-threatening condition that can arise from adrenal suppression caused by prolonged exposure to supraphysiologic doses of GCs. The objective of this manuscript is to provide an up-to-date review of the best practices for the management of adrenal insufficiency in individuals with DMD treated with GCs. The approach described in this document also applies to individuals with related dystrophinopathies including Becker muscular dystrophy and female manifesting carriers treated with GCs, recognizing that GCs are not standard of care for these conditions and are prescribed on an individualized basis at some centers to severely affected individuals. Key themes discussed include best practices for individual and caregiver education about adrenal suppression, creation and implementation of stress steroid plans, safe transition between GC treatment regimens, and discontinuation of GC therapy.