Evidence map›Paper›PMID 42770351›Full record

ReviewJournal of neuromuscular diseases2026

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

David R Weber, Alexandra Ahmet, Kathi Kinnett, Maria-Elena Lautatzis, Hugh J McMillan, Susan Apkon, Shipra Bansal, Robert W Benjamin, Paula R Clemens, Janet L Crane and 31 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Journal of neuromuscular diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

41 authors.

David R WeberDivision of Pediatric Endocrinology and Diabetes, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.ORCID 0000-0001-6895-2372
Alexandra AhmetDivision of Endocrinology and Metabolism, Department of Pediatrics, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.
Kathi KinnettParent Project Muscular Dystrophy (PPMD), Washington, DC, USA.
Maria-Elena LautatzisDepartment of Pediatrics and Child Health, Manitoba Children's Hospital, University of Manitoba, Winnipeg, MB, Canada.
Hugh J McMillanDivision of Neurology, Department of Pediatrics, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.ORCID 0000-0001-8927-2018
Susan ApkonDepartment of Physical Medicine and Rehabilitation, Children's Hospital Colorado and Anschutz University of Colorado School of Medicine, Aurora, CO, USA.
Shipra BansalSection of Pediatric Endocrinology and Diabetes, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR, USA.
Robert W BenjaminDivision of Pediatric Endocrinology and Diabetes, Duke University Medical Center, Durham, NC, USA.
Paula R ClemensDepartment of Neurology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.
Janet L CraneDivision of Pediatric Endocrinology, Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Melissa FiscalettiDepartment of Pediatrics, Sainte Justine University Hospital, Université de Montréal, Montreal, QC, Canada.
Carol LamDivision of Endocrinology, Department of Pediatrics, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
Laura McAdamDepartment of Pediatrics, Holland Bloorview Kids Rehabilitation Hospital, University of Toronto, Toronto, ON, Canada.
Meilan M RutterDivision of Diabetes and Endocrinology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Nadia MerchantDivision of Pediatric Endocrinology, Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Nat NasomyontDivision of Diabetes and Endocrinology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Stefan NicolauCenter for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, USA.
Kim PhungDivision of Endocrinology and Metabolism, Department of Pediatrics, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.
Nora RenthalDepartment of Pediatrics, Division of Endocrinology, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA.ORCID 0000-0002-8186-4462
Mena ScavinaParent Project Muscular Dystrophy (PPMD), Washington, DC, USA.
Julia SorbaraDivision of Endocrinology, Department of Pediatrics, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
Jaclyn TamaroffDivision of Endocrinology and Diabetes, Department of Pediatrics, Vanderbilt University Medical Center, Nashville, TN, USA.
Cuixia TianDivision of Neurology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Claire WoodDepartment of Paediatric Endocrinology, Great North Childrenc's Hospital, Newcastle upon Tyne, UK.
Tommaso AversaDepartment of Human Pathology of Adulthood and Childhood, Pediatric Unit, "G. Martino" University Hospital, University of Messina, Messina, Italy.
Luca BelloDepartment of Neurosciences, DNS Clinica Neurologica, Azienda Ospedale University of Padova, Padua, Italy.
Sabrina CorbettaBone Metabolic Diseases and Diabetes Unit, IRCCS Istituto Auxologico Italiano, Milan, Italy.ORCID 0000-0001-8140-3175
Francesca CumboUnit of Muscular and Neurodegenerative Diseases, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
Luisa De SanctisDepartment of Pediatric Endocrinology, Regina Margherita Children's Hospital, Turin, Italy.
Natascia Di IorgiDepartment of Pediatrics, Pediatric Endocrinology Unit, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
Alberto FerlinUnit of Andrology and Reproductive Medicine, Department of Medicine, University of Padova, Padova, Italy.
Danilo FintiniEndocrinology and Diabetology Unit, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
Eugenio Maria MercuriPediatric Neurology, Catholic University of the Sacred Heart, Rome, Italy.
Chiara PanicucciCenter of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
Gianluca TorneseDepartment of Medical Surgical and Health Sciences, University of Trieste, Trieste, Italy.
Rachel SchraderParent Project Muscular Dystrophy (PPMD), Washington, DC, USA.
Kathryn A SelbyDivision of Neurology, Department of Pediatrics, British Columbia Children's Hospital, University of British Columbia, Vancouver, BC, Canada.
Aravindhan VeerapandiyanDivision of Neurology, Department of Pediatrics, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR, USA.
Sze Choong WongBone, Endocrine, Nutrition Research Group in Glasgow, Human Nutrition, University of Glasgow, Glasgow, UK.
Leanne M WardDivision of Endocrinology and Metabolism, Department of Pediatrics, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.ORCID 0000-0003-1557-9185
Anne Marie SbrocchiDivision of Pediatric Endocrinology and Metabolism, Department of Pediatrics, Montreal Children's Hospital, McGill University Health Center, Montreal, QC, Canada.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

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.

Indexed as

adrenal insufficiencyDuchenne muscular dystrophyglucocorticoidvamorolone

Identifiers

PMID42770351
PMCPMC13597954

What OpenQuestion holds

Textmetadata
Read underepoch 390

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.