Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid Vamorolone
Adrenal suppression is an iatrogenic form of adrenal insufficiency that occurs secondary to exogenous glucocorticoids (GCs) and is a documented cause of premature mortality among individuals with Duchenne muscular dystrophy (DMD). Adrenal suppression in DMD necessitates awareness and careful management, given that GCs are currently the mainstay of therapy for individuals living with DMD. Vamorolone, a novel GC that has recently been approved in some regions worldwide for the treatment of DMD, has also been reported to place individuals at high risk of adrenal suppression in a dose-dependent fashion, requiring health care professional awareness. Vamorolone is a mineralocorticoid receptor antagonist, which differentiates it from classic GCs, and this characteristic impacts the approach to adrenal suppression management. This contemporary perspective provides insights into the mechanisms underlying adrenal suppression due to both classic GCs and novel vamorolone therapy, followed by an overview of adrenal suppression management with a particular focus on the unique aspects of providing care for individuals treated with vamorolone. It also emphasizes the importance of educating the DMD community and health care providers about the recognition and management of adrenal suppression and outlines critical concepts for clinicians managing adrenal suppression risk, tapering GCs, and transitioning from classic GC therapy to vamorolone. The key principles of managing adrenal suppression due to classic GCs and novel vamorolone therapy highlighted in this perspective are expected to enhance clinical practice, mitigate mortality, and optimize health outcomes for individuals with DMD.
Authors
- Anne Marie Sbrocchi (ORCID: https://orcid.org/0009-0005-3682-8293)
- Kathi Kinnett (ORCID: https://orcid.org/0000-0002-2704-7349)
- Laura C. McAdam (ORCID: https://orcid.org/0000-0002-4650-7232)
- Diana Xerxes Bharucha-Goebel (ORCID: https://orcid.org/0000-0002-7437-0982)
- Kim Phung (ORCID: https://orcid.org/0000-0003-2546-2853)
- Nat Nasomyont (ORCID: https://orcid.org/0000-0001-8996-8760)
- Mena T. Scavina (ORCID: https://orcid.org/0000-0002-1371-9765)
- Claire Louise Wood (ORCID: https://orcid.org/0000-0002-6084-5174)
- Jaclyn G. Tamaroff (ORCID: https://orcid.org/0000-0002-3630-9937)
- Meilan Marianne Rutter (ORCID: https://orcid.org/0000-0003-4142-9527)
- Shipra Bansal (ORCID: https://orcid.org/0000-0002-0133-784X)
- Stefan Nicolau (ORCID: https://orcid.org/0000-0001-5631-0559)
- Sze Choong Wong (ORCID: https://orcid.org/0000-0003-2845-6555)
- K. Selby
- Cuixia Tian (ORCID: https://orcid.org/0009-0003-7673-7295)
- Carol Lam (ORCID: https://orcid.org/0000-0002-9736-3347)
- Aravindhan Veerapandiyan (ORCID: https://orcid.org/0000-0002-3065-3956)
- Sonum Bharill (ORCID: https://orcid.org/0000-0003-2933-5579)
- Susan D. Apkon (ORCID: https://orcid.org/0009-0008-9873-7970)
- David R. Weber (ORCID: https://orcid.org/0000-0001-6895-2372)
- Prasanth Surampudi
- Leanne Marie Ward (ORCID: https://orcid.org/0000-0003-1557-9185)
- Marı́a Teresa Molina (ORCID: https://orcid.org/0000-0003-0241-0196)
- Rana Halloun (ORCID: https://orcid.org/0000-0002-8446-7737)
- Nadia Merchant (ORCID: https://orcid.org/0000-0001-8775-6481)
- Hugh J. McMillan (ORCID: https://orcid.org/0000-0001-8927-2018)
- Alexandra H. Ahmet (ORCID: https://orcid.org/0000-0002-3226-8322)
- Paula R. Clemens (ORCID: https://orcid.org/0000-0002-0652-6703)
- Mélissa Fiscaletti (ORCID: https://orcid.org/0000-0003-2092-0691)
- Maria-Elena Lautatzis (ORCID: https://orcid.org/0009-0000-2415-8062)
Institutions
- Cincinnati Children's Hospital Medical Center (US)
- Arkansas Children's Hospital (US)
- Nationwide Children's Hospital (US)
- Children's Hospital of Philadelphia (US)
- Johns Hopkins University (US)
- University of Ottawa (CA)
- Pontificia Universidad Católica de Chile (CL)
- University of Pittsburgh (US)
- Children's Hospital of Eastern Ontario (CA)
- Johns Hopkins Medicine (US)
- Veterans Health Administration (US)
- Montreal Children's Hospital (CA)
- Royal Hospital for Children (GB)
- Royal Victoria Infirmary (GB)
- Hospital for Sick Children (CA)
- Holland Bloorview Kids Rehabilitation Hospital (CA)
- Centre Hospitalier Universitaire Sainte-Justine (CA)
- Hospital for Sick Children Pediatric Center (US)
- Children's Hospital Research Institute of Manitoba (CA)
- Children's Hospital Colorado (US)
- Parent Project Muscular Dystrophy (US)
- Nemours Children's Health System (US)
- University of Pittsburgh Medical Center (US)
- Department of Veterans Affairs (AU)
- BC Children's Hospital (CA)
- University of California Davis Medical Center (US)
- The Ohio State University (US)
- Université de Montréal (CA)
- University of Glasgow (GB)
- University of Pennsylvania (US)
- University of Arkansas for Medical Sciences (US)
- The University of Texas Southwestern Medical Center (US)
- Vanderbilt University Medical Center (US)
- University of Cincinnati Medical Center (US)
- University of Colorado Denver (US)
- Ottawa University (US)
Publication Details
- Journal
- Journal of the Endocrine Society
- Published
- 2025-11-26
- DOI
- https://doi.org/10.1210/jendso/bvaf181
- Primary Topic
- Adrenal Hormones and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Parent Project Muscular Dystrophy