ECEESPE2025 Poster Presentations Adrenal and Cardiovascular Endocrinology (169 abstracts)
Insights into the health provision for children with congenital adrenal hyperplasia in the UK and implementation of longitudinal collection and analysis of real-world data
Irina A Bacila 1,2 , Neil Lawrence 1 , Chamila Balagamage 3 , Jillian Bryce 4 , Salma Rashid Ali 4,5 , Malika Alimussina 4 , Minglu Chen 4 , Assunta Albanese 6 , Nadia Amin 7 , Justin Davies 8,9 , Gabriella Gazdagh 10 , Evelien Gevers 11 , Marta Korbonits 11 , Ruth Krone 3 , Harriet Miles 12 , Anuja Natarajan 13 , Avanti Patwardhan 11 , Tabitha Randell 14 , Fiona Ryan 15 , Savitha Shenoy 16 , Mars Skae 17 , Ajay Thankamony 18 , S Faisal Ahmed 4,5 & Nils Krone 1,2
1The University of Sheffield, School of Medicine and Population Health, Division of Clinical Medicine, Sheffield, UK; 2The Sheffield Children’s Hospital, Endocrinology, Sheffield, UK; 3Birmingham Women’s & Children’s Hospital, Birmingham, UK; 4Royal Hospital for Children & Queen Elizabeth University Hospital, Glasgow, UK; 5University of Glasgow, Developmental Endocrinology Research Group, Glasgow, UK; 6St George’s University Hospital, London, UK; 7Leeds General Infirmary, Leeds, UK; 8University Hospital Southampton, Southampton, UK; 9University of Southampton, Southampton, UK; 10Southampton General Hospital, Southampton, UK; 11William Harvey Research Institute, Queen Mary University London, Centre for Endocrinology, London, UK; 12Royal Hospital for Children and Young People, Edinburgh, UK; 13Doncaster and Bassetlaw Hospitals NHS Trust, Doncaster, UK; 14Nottingham Children’s Hospital, Nottingham, UK; 15Oxford University Hospitals NHS Foundation Trust, Oxford, UK; 16Leicester Royal Infirmary, Leicester, UK; 17Royal Manchester Children’s Hospital, Manchester, UK; 18University of Cambridge, Cambridge, UK
JOINT2341
Background: Congenital adrenal hyperplasia (CAH) is one of the commonest forms of primary adrenal insufficiency, around 70 children being diagnosed every year in the UK. Our national survey indicated variations among clinicians in the management of children and young persons with CAH. We wanted to use the SDMregistries to gain further insight regarding the current practice of CAH clinical management in children in the UK.
Methodology: We launched a 5-year-project in 03/2022, collecting annually longitudinal data from the SDMregistries platform on UK patients under 18 with 21-hydroxylase deficiency, the most common form of CAH.
Results: The first data extraction in 09/2022 provided data from 44 eligible patients (5 centres), with a median of 8 patients per centre (range 3–15). The second data extraction in 03/2024 showed an increase in participation, including 96 patients from 15 centres, with a median of 4 patients per centre (range 1–20). Of these, only 68 (70%) had sufficient data to allow an analysis of the replacement medication. We analysed information related to medication and height standard deviation scores (SDS) for age and sex. The mean daily glucocorticoid (GC) dose was 12.0 (±3.8 S.D.) mg/m2 per day hydrocortisone-equivalent, with broad variations between centres ranging from 7.7 (±1.2) to 19.1 (±7.7) mg/m2 per day. The dose decreased with age by 0.2 mg/m2 per day per year, and depended upon centre; it also varied with sex, doses used in girls being lower by 1.1 mg/m2 per day compared to boys. We identified different GC administration regimes, with variation in the time of administration of the first daily hydrocortisone dose between 04:00 and 09:00, and six centres using midnight doses. Total daily fludrocortisone doses ranged between 50 and 300 μg/day, with significant variation between centres (R2=0.44, P<0.01). Generalised additive model fit showed height SDS fluctuated with age, starting from −0.6 in infancy increasing to 0.7 at 10 years and then decreasing to −1.0 at 17.5 years.
Conclusions: These preliminary findings warrant further investigation to establish potential different approaches and analyse outcomes. Thus, we are implementing a strategy to increase recruitment by actively engaging UK centres involved in the management of CAH patients under 18 into recording their data in I-CAH. Data will be collected and analysed annually, to assess the current level of care provision and inform the development of national CAH standards. This work will help to develop a standardised approach to hormone replacement in CAH and create a framework for benchmarking management at national and international level.
Volume 110
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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