Evidence map›Paper›PMID 41920460›Full record

ArticleEndocrine2026

Survey of Adrenal Insufficiency Management for Duchenne muscular dystrophy in Italy.

Gianluca Tornese, Sze Choong Wong, Anne Marie Sbrocchi, Fernanda De Angelis, Ilaria Zito, David R Weber, Tommaso Aversa

Abstract read
In one paragraph

Article in Endocrine, 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

7 authors.

Gianluca TorneseDepartment of Medicine, Surgery and Health Sciences, University of Trieste, Trieste, Italy. gianluca.tornese@burlo.trieste.it.
Sze Choong WongDepartment of Paediatric Endocrinology, Royal Hospital for Children, Glasgow, UK.
Anne Marie SbrocchiDivision of Pediatric Endocrinology and Metabolism, Montreal Children's Hospital, McGill University Health Centre, Montreal, Canada.
Fernanda De AngelisParent Project aps, Rome, Italy.
Ilaria ZitoParent Project aps, Rome, Italy.
David R WeberDivision of Endocrinology and Diabetes, The Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, USA.
Tommaso AversaDepartment of Human Pathology of Adulthood and Childhood, University of Messina, Messina, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeThe aim of this study was to evaluate current real-world practices for the prevention and management of adrenal insufficiency (AI) among Italian physicians caring for children and adolescents with Duchenne muscular dystrophy (DMD) receiving chronic glucocorticoid (GC) therapy, a treatment known to improve motor and cardiopulmonary outcomes but to suppress the hypothalamic–pituitary–adrenal axis and increase the risk of adrenal crisis.

methodsA cross-sectional online survey was distributed via national networks to Italian pediatric endocrinologists and neuromuscular specialists. The questionnaire explored clinician characteristics, patient caseloads, glucocorticoid regimens, stress-dose recommendations for mild, moderate, and severe illness, and proactive measures such as family education, emergency kits, written plans, medical alert identification, and electronic hospital alerts. Participation was anonymous.

resultsThirty-five clinicians responded (57% pediatric endocrinologists, 43% neuromuscular specialists), most following 11–50 patients. Deflazacort was the predominant treatment (86%). For mild stress, 80% advised no additional GC. For moderate stress, 43% recommended no change to management, 31% provided oral hydrocortisone (HC), 17% advised extra daily GC, and 9% prescribed intramuscular HC. For severe stress, although 77% prescribed parenteral HC, 14% advised no change. Proactive measures were inconsistently implemented: 82% provided family education, 43% gave intramuscular HC prescriptions and training, 71% had written emergency plans, and 31% recommended medical alert identification.

conclusionsThis national survey reveals substantial variability and gaps in AI prevention and management for children and adolescents with DMD on chronic GC therapy in Italy. Harmonized national protocols, multidisciplinary coordination, and strengthened family education are needed to improve adrenal crisis prevention.

Indexed as

Adrenal InsufficiencyGlucocorticoidsMuscular Dystrophy, DuchennePractice Patterns, Physicians'AdolescentChildCross-Sectional StudiesFemaleHumansHydrocortisoneItalyMaleSurveys and QuestionnairesGlucocorticoidsHydrocortisoneadrenal insufficiencyDuchenne muscular dystrophyeducationItalymanagementsteroid treatment

Identifiers

PMID41920460
PMCPMC13043509

What OpenQuestion holds

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LicenceCC BY
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Registered trials

None linked

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.