Evidence map›Paper›PMID 42787106›Full record

ReviewFrontiers in neurology2026

A structured approach to address variability in the global management of adrenal insufficiency in Duchenne muscular dystrophy: the 2025 PJ Nicholoff steroid protocol.

Anne Marie Sbrocchi, Alexandra Ahmet, Kathi Kinnett, Maria-Elena Lautatzis, Hugh J McMillan, Susan Apkon, Meilan M Rutter, Leanne M Ward, Sasigarn A Bowden, Anne M Connolly and 6 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Frontiers in neurology, 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

16 authors.

Anne Marie SbrocchiDivision of Pediatrics and Metabolism, Montreal Children's Hospital, McGill University Health Center, Montreal, QC, Canada.
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, United States.
Maria-Elena LautatzisDepartment of Pediatrics and Child Health, Children's Hospital of Manitoba, University of Manitoba, Winnipeg, MB, Canada.
Hugh J McMillanDivision of Endocrinology and Metabolism, Department of Pediatrics, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.
Susan ApkonDepartment of Physical Medicine and Rehabilitation, Children's Hospital Colorado and Anschutz University of Colorado School of Medicine, Aurora, CO, United States.
Meilan M RutterDivision of Diabetes and Endocrinology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, United States.
Leanne M WardDivision of Endocrinology and Metabolism, Department of Pediatrics, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.
Sasigarn A BowdenDepartment of Pediatrics, Nationwide Children's Hospital, Ohio State University, Columbus, OH, United States.
Anne M ConnollyDepartment of Pediatrics, Nationwide Children's Hospital, Ohio State University, Columbus, OH, United States.
Garey NoritzDepartment of Pediatrics, Nationwide Children's Hospital, Ohio State University, Columbus, OH, United States.
Rachel SchraderParent Project Muscular Dystrophy (PPMD), Washington, DC, United States.
Kathryn SelbyDivision of Neurology, Department of Pediatrics, British Columbia Children's Hospital, University of British Columbia, Vancouver, BC, Canada.
Aravindhan VeerapandiyanDepartment of Pediatrics, Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR, United States.
Sze Choong WongBone, Endocrine, Nutrition Research Group in Glasgow, University of Glasgow, Glasgow, United Kingdom.
David R WeberDivision of Pediatric Endocrinology and Diabetes, The Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, United States.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Adrenal suppression is a potentially life-threatening consequence of chronic glucocorticoid (GC) therapy in Duchenne muscular dystrophy (DMD), placing individuals at risk for adrenal insufficiency and adrenal crisis during increased physiological stress, missed GC doses, abrupt discontinuation, or transitions between GC regimens. Management is increasingly complex because DMD treatment now includes multiple GC agents, including prednisone/prednisolone, deflazacort, and vamorolone, as well as daily, intermittent, and transition regimens that differ across countries and clinical settings. Although general principles of adrenal insufficiency prevention are well established, implementation remains challenging because of global variability in drug availability, institutional stress-steroid protocols, clinician experience, and caregiver education. This article discusses key sources of variability in adrenal suppression management for individuals with DMD and presents the 2025 PJ Nicholoff Steroid Protocol as an example of a practical, DMD-specific framework. The updated protocol incorporates newer therapies such as vamorolone and emphasizes anticipatory education, written stress-steroid plans (including a weight-based approach), safe GC tapering, confirmation of hypothalamic-pituitary-adrenal axis recovery, and careful management of transitions between GC agents and regimens. This structured approach aims to support safer and more consistent adrenal suppression management for individuals with DMD receiving chronic GC therapy through six critical concepts.

Indexed as

Adrenal InsufficiencyGlucocorticoidsMuscular Dystrophy, DuchenneHumansPregnenedionesGlucocorticoidsPregnenedionesadrenal insufficiencycorticosteriodsduchenneglucocorticoidvamorolone

Identifiers

PMID42787106
PMCPMC13600995

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.