BSPED2024 Poster Presentations Adrenal 2 (8 abstracts)
Analysis of real-world data on the care provision of children with congenital adrenal hyperplasia (CAH) in the united kingdom
Irina A Bacila 1,2 , Neil R. Lawrence 1,2 , Chamila Balagamage 3 , Jillian Bryce 4 , Salma R. Ali 4,5 , Malika Alimussina 4 , Minglu Chen 4 , Assunta Albanese 6 , Nadia Amin 7 , Justin H. Davies 8,9 , Gabriella Gazdagh 10 , Evelien Gevers 11 , Marta Korbonits 12 , Ruth E. Krone 3 , Harriet Miles 13 , Anuja Natarajan 14 , Tabitha Randell 15 , Fiona J. Ryan 16 , Savitha Shenoy 17 , Mars Skae 18 , Ajay Thankamony 19 , S Faisal. Ahmed 4,5 & Nils P. Krone 1,2
1The University of Sheffield, Sheffield, United Kingdom; 2Sheffield Children’s Hospital, Sheffield, United Kingdom; 3Birmingham Women’s & Children’s Hospital, Birmingham, United Kingdom; 4Office for Rare Conditions, Royal Hospital for Children & Queen Elizabeth University Hospital, Glasgow, United Kingdom; 5Developmental Endocrinology Research Group, University of Glasgow, Glasgow, United Kingdom; 6Paediatric Endocrinology, St George’s University Hospital, London, United Kingdom; 7Leeds General Infirmary, Leeds, United Kingdom; 8University Hospital Southampton, Southampton, United Kingdom; 9University of Southampton, Southampton, United Kingdom; 10Clinical Genetics, Southampton General Hospital, Southampton, United Kingdom; 11Centre for Endocrinology, William Harvey Research Institute, Queen Mary University London, London and Barts Health NHS Trust - The Royal London Hospital, London, United Kingdom; 12Centre for Endocrinology, William Harvey Research Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, United Kingdom; 13Royal Hospital for Children and Young People, Edinburgh, United Kingdom; 14Department of Paediatrics, Doncaster and Bassetlaw Hospitals NHS Trust, Doncaster, United Kingdom; 15Nottingham Children’s Hospital, Nottingham, United Kingdom; 16Oxford Children’s Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, United Kingdom; 17Department of Paediatric Endocrinology, Leicester Royal Infirmary, UHL NHS Trust, Leicester, United Kingdom; 18Department of Paediatric Endocrinology, Royal Manchester Children’s Hospital, Manchester, United Kingdom; 19Department of Paediatrics, University of Cambridge, Cambridge, United Kingdom
Background: Following a national survey indicating variations across the United Kingdom in the management of children and young persons with CAH, we aimed to explore the current practice of CAH clinical management.
Methods: As part of an ongoing project, we collected data on 96 UK patients under 18 with 21-hydroxylase deficiency (48 females), 649 clinic visits from 15 centres, recorded in the I-CAH registry since 01/01/2016. We analysed information related to medication, biochemical markers and height standard deviation scores (SDS) for age and sex (WHO reference) in RStudio. Glucocorticoid (GC) doses were expressed as hydrocortisone (HC) equivalent per body surface area. To explore variations in GC doses between centres, controlling for age and sex, we used a mixed effects random intercept model
Results: Most patients (98%) were treated with hydrocortisone three or four times daily, prednisolone being used in 2 patients. The mean daily GC dose was 12.07 (±3.8 SD) HC-equivalent mg/m2/day, with broad variations between centres ranging from 7.7 (±1.2) to 19.1 (±7.7) mg/m2/day. Modelling showed dose in centres decreased with age by 0.18 mg/m2/day per year and varied with sex. This equated to 10-year-old boys on an average dose of 12.4 mg/m2/day ranging between 10.3-19.3 mg/m2/day dependent upon centre. In girls, doses were 1.1 mg/m2/day lower than boys. Fludrocortisone was used in 76% of cases, total daily doses ranging between 50 and 300 mµ/day, with significant variation between centres (R2=0.44, P < 0.01). The most used biomarkers for monitoring GC replacement were 17-hydroxyprogesterone and androstenedione, recorded in 32% and 25% visits, respectively. 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.
Conclusion: Our findings indicate marked differences between UK centres in the relative doses and times of administration of GC replacement in children with CAH. Moreover, there was variation in the growth trajectory in patients with CAH compared to WHO standards which warrants further investigation. Improved data collection and robust analysis of different treatment approaches at a national level will better inform optimal management strategies in CAH.
Volume 103
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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