Evidence map›Paper›PMID 41509125›Full record

ArticleJournal of the Endocrine Society2026

Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid Vamorolone.

Anne Marie Sbrocchi, Kathi Kinnett, Maria-Elena Lautatzis, Hugh J McMillan, Kathryn A Selby, Aravind Veerapandiyan, David R Weber, Susan Apkon, Diana X Bharucha-Goebel, Sonum Bharill and 21 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of the Endocrine Society, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

31 authors.

Anne Marie SbrocchiDivision of Pediatric Endocrinology and Metabolism, Montreal Children's Hospital, McGill University, Montreal, QC H4A 3J1, Canada.ORCID https://orcid.org/0009-0005-3682-8293
Kathi KinnettParent Project Muscular Dystrophy (PPMD), Washington, DC 20005, USA.
Maria-Elena LautatzisDivision of Pediatric Endocrinology, Department of Pediatrics and Child Health, Manitoba Children's Hospital, University of Manitoba, Winnipeg, MB R3B 0L8, Canada.
Hugh J McMillanDivision of Neurology, Department of Pediatrics, Children's Hospital of Eastern Ontario (CHEO), University of Ottawa, Ottawa, ON K1H 8L1, Canada.
Kathryn A SelbyDivision of Neurology, Department of Pediatrics, BC Children's Hospital, University of British Columbia, Vancouver, BC V6H 3V4, Canada.
Aravind VeerapandiyanDepartment of Pediatrics, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR 72202, USA.
David R WeberDivision of Endocrinology and Diabetes, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Susan ApkonDepartment of Physical Medicine and Rehabilitation, Children's Hospital Colorado and University of Colorado School of Medicine, Aurora, CO 80262, USA.
Diana X Bharucha-GoebelCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Sonum BharillDivision of Endocrinology, Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, MD 21211, USA.
Shipra BansalDepartment of Pediatrics, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR 72202, USA.
Paula R ClemensDepartment of Neurology, University of Pittsburgh School of Medicine, Pittsburgh, PA 15213, USA.
Melissa FiscalettiDepartment of Pediatrics, Sainte Justine University Hospital, Université de Montréal, Montreal, QC H3T 1J4, Canada.
Rana HallounThe Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, University of Ottawa, Ottawa, ON K1H 8L1, Canada.
Carol LamDivision of Endocrinology, Department of Pediatrics, the Hospital for Sick Children, University of Toronto, Toronto, ON M5G 1X8, Canada.ORCID https://orcid.org/0000-0002-9736-3347
Nadia MerchantDivision of Pediatric Endocrinology, Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Laura McAdamDepartment of Pediatrics, Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Toronto, ON M4G 1R8, Canada.
Nat NasomyontDivision of Diabetes and Endocrinology, Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH 45219, USA.ORCID https://orcid.org/0000-0001-8996-8760
Stefan NicolauCenter for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA.
Maria F Ochoa MolinaEndocrinology Unit, Division of Pediatrics, School of Medicine, Pontifical Catholic University of Chile, Marcoleta, Santiago 340, Chile.
Kim PhungDivision of Endocrinology, Children's Hospital of Eastern Ontario (CHEO), Ottawa, ON K1H 8L1, Canada.ORCID https://orcid.org/0000-0003-2546-2853
Meilan M RutterDivision of Diabetes and Endocrinology, Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH 45219, USA.
Mena ScavinaDivision of Neurology, Nemours Children's Hospital, Delaware, Parent Project Muscular Dystrophy, Wilmington, DE 19803, USA.
Prasanth N SurampudiDepartment of Internal Medicine, Division of Endocrinology, University of California, Davis Medical Center, Sacramento, CA 95817, USA.
Jaclyn TamaroffDivision of Endocrinology and Diabetes, Department of Pediatrics, Vanderbilt University Medical Center, Nashville, TN 37232, USA.ORCID https://orcid.org/0000-0002-3630-9937
Cuixia TianDivision of Neurology, Cincinnati Children's Hospital Medical Center, College of Medicine, University of Cincinnati, Cincinnati, OH 45229, USA.
Leanne M WardDivision of Endocrinology, Children's Hospital of Eastern Ontario (CHEO), Ottawa, ON K1H 8L1, Canada.
Claire L WoodDepartment of Paediatric Endocrinology, Royal Victoria Infirmary, Faculty of Medical Sciences, Newcastle University, Newcastle Upon Tyne NE1 7RU, UK.
Sze Choong WongDepartment of Pediatric Endocrinology, Royal Hospital for Children, Glasgow, Scotland G51 4TF, UK.
Alex AhmetDivision of Endocrinology, Children's Hospital of Eastern Ontario (CHEO), Ottawa, ON K1H 8L1, Canada.
OPTIMIZE DMD Consortium

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

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.

Indexed as

adrenal insufficiencyadrenal suppressiondisease managementDuchenneglucocorticoidsvamorolone

Identifiers

PMID41509125
PMCPMC12776010

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.