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Adrenal (Excluding Mineralocorticoids)

SUN-460 - Real-World Data Analysis of the Management of Children with Congenital Adrenal Hyperplasia in the United Kingdom and Ireland

Sunday, June 14, 2026
9:00 AM - 4:00 PM CT
Location: ENDOExpo; Poster Floor
Disclosure(s):
Nils P. Krone, MD FRCPCH: Crinetics Pharmaceuticals: Consultant (); Neurocrine Biosciences: Consultant (), Grant Recipient (), Honorarium Recipient ()
Background
To characterise current clinical practice in the management of congenital adrenal hyperplasia (CAH) in children, a five-year observational project with longitudinal real-world data was initiated in March 2022. The study involves the annual collection of longitudinal data for patients under 18 years of age with 21-hydroxylase deficiency (21OHD) receiving care in centres across the United Kingdom and Ireland.

Methods
Data were obtained from the I-CAH on the SDMregistries platform in April 2025 on a total of 1,291 clinical visits from 182 patients managed across 15 centres since 1 January 2016. Participation per centre varied between 1 and 34, with a median of 8 patients/centre. Regression analyses were conducted using R to describe the variation with age and sex of treatment regimens, biochemical markers, and World Health Organization height standard deviation scores (SDS). Glucocorticoid (GC) doses were standardised and expressed as hydrocortisone (HC) equivalents per body surface area.

Results
The majority of patients (95%) were treated with hydrocortisone administered three or four times daily, while eight patients received prednisolone (age range 7-18 years). The mean daily GC dose was 12.2 ± 4.0 mg HC-equivalent/m²/day, with mean individual doses ranging from 4.6 ± 1.2 to 22.3 ± 0.4 mg/m²/day. Substantial inter-centre variability was observed, with mean GC doses per centre ranging from 9.8 to 16.7 mg/m²/day. GC dose increased with age by 0.2 mg/m²/day per year, with centre-specific differences, and was significantly influenced by sex, with doses in males being on average 0.8 mg/m²/day lower than in females. Considerable variation was also noted in GC dosing schedules, with six centres administering late evening doses between 22:00 and 24:00. Total daily fludrocortisone relative doses ranged from 18 to 869 µg /m2/day and varied significantly between centres (R²=0.02, p< 0.01). Biochemical control was heterogeneous: 17-hydroxyprogesterone levels were elevated in 42% of visits and suppressed in 14%, while androstenedione levels were elevated in 25% and suppressed in 20% of visits. Mean height SDS varied across childhood, increasing from −0.5 in infancy to a peak of 1.2 at 10 years of age, followed by a decline to −1.0 by 17.5 years.

Conclusions
The findings demonstrate marked variability in therapeutic approaches to paediatric CAH management across centres. Further analyses examining associations between treatment strategies and clinical outcomes are warranted to inform best practice, support benchmarking of care, and guide improvements in CAH management across the United Kingdom and Ireland.

*Unless otherwise noted, all abstracts presented at ENDO must not be released to the press or the public until the date and time of presentation. For oral presentations, the abstracts are embargoed until the session begins. The Endocrine Society reserves the right to lift the embargo on specific abstracts that are selected for promotion prior to or during ENDO.*