Survey of Adrenal Insufficiency Management for Duchenne muscular dystrophy in Italy

Purpose The aim of this study was to evaluate current real-world practices for the prevention and management of adrenalinsufficiency (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 tosuppress the hypothalamic–pituitary–adrenal axis and increase the risk of adrenal crisis.Methods A cross-sectional online survey was distributed via national networks to Italian pediatric endocrinologists andneuromuscular 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, emer-gency kits, written plans, medical alert identification, and electronic hospital alerts. Participation was anonymous.Results Thirty-five clinicians responded (57% pediatric endocrinologists, 43% neuromuscular specialists), most follow-ing 11–50 patients. Deflazacort was the predominant treatment (86%). For mild stress, 80% advised no additional GC. Formoderate stress, 43% recommended no change to management, 31% provided oral hydrocortisone (HC), 17% advised extradaily GC, and 9% prescribed intramuscular HC. For severe stress, although 77% prescribed parenteral HC, 14% advised nochange. Proactive measures were inconsistently implemented: 82% provided family education, 43% gave intramuscular HCprescriptions and training, 71% had written emergency plans, and 31% recommended medical alert identification.Conclusions This national survey reveals substantial variability and gaps in AI prevention and management for children andadolescents with DMD on chronic GC therapy in Italy. Harmonized national protocols, multidisciplinary coordination, andstrengthened family education are needed to improve adrenal crisis prevention.