ESPE Abstracts (2019) 92 P1-10

Global Practice of Glucocorticoid and Mineralocorticoid Treatment in Children and Adults with Congenital Adrenal Hyperplasia – Insights from the I-CAH Registry

Irina-Alexandra Bacila1, Oliver Blankenstein2, Uta Neumann2, Hedi L Claahsen van der Grinten3, Ruth Krone4, Carlo Acerini5, Tania SS Bachega6, Mirella C Miranda6, Berenice Mendonca6, Niels H Birkebaek7, Martine Cools8, Tatjana Milenkovic9, Walter Bonfig10,11, Jeremy Tomlinson12, Heba Elsedfy13, Antonio Balsamo14, Sabine Hannema15,16, Claire Higham17, Navoda Atapattu18, Corina Lichiardopol19, Tulay Guran20, Zehra Abali20, Klaus Mohnike21, Martijn JJ Finken22, Ana Vieites23, Feyza Darendeliler24, Ayla Guven25, Marta Korbonits26, Liat de Vrie27,28, Eduardo Costa29, Silvia Einaudi 30, Hetty van der Kamp 31, Violeta Iotova32, Richard Ross1, S Faisal Ahmed33, Nils P Krone1


1Department of Oncology and Metabolism, University of Sheffield, Sheffield, United Kingdom. 2Institute for Experimental Pediatric Endocrinology and Center for Chronically Sick Children, Charite-Universitätsmedizin Berlin, Berlin, Germany. 3Department of Pediatric Endocrinology, Radboud University Medical Centre, Nijmegen, Netherlands. 4Department of Endocrinology and Diabetes, Birmingham Women's and Children's Hospital, Birmingham, United Kingdom. 5Department of Pediatrics, University of Cambridge and Addenbrooke's Hospital, Cambridge, United Kingdom. 6Department of Internal Medicine, University of Sao Paulo, Sao Paulo, Brazil. 7Department of Pediatrics, Aarhus University Hospital, Aarhus, Denmark. 8Pediatric Endocrinology, Pediatric and Genetics Research Unit, University Hospital Ghent, Ghent University, Ghent, Belgium. 9Department of Endocrinology, Institute for Mother and Child Healthcare of Serbia "Dr Vukan Čupić" Belgrade, Belgrade, Serbia. 10Department of Pediatrics, Technical University Munich, Munich, Germany. 11Department of Pediatrics, Klinikum Wels-Grieskirchen, Wels, Austria. 12Oxford Centre for Diabetes, Endocrinology & Metabolism, Churchill Hospital, Oxford, United Kingdom. 13Pediatrics Department, Ain Shams University, Cairo, Egypt. 14Department of Medical and Surgical Sciences, Pediatric Unit, Center for Rare Endocrine Diseases, S.Orsola-Malpighi University Hospital, Bologna, Italy. 15Department of Pediatric Endocrinology, Sophia Children's Hospital, Erasmus Medical Centre, Rotterdam, Netherlands. 16Department of Paediatrics, Leiden University Medical Centre, Leiden, Netherlands. 17Department of Endocrinology, Christie Hospital NHS Foundation Trust, Manchester, United Kingdom. 18Pediatric Endocrinology, Lady Ridgeway Hospital, Colombo, Sri Lanka. 19Department of Endocrinology, University of Medicine and Pharmacy Craiova, Craiova, United Kingdom. 20Pediatric Endocrinology and Diabetes, Marmara University, Istanbul, Turkey. 21Department of Pediatrics, Otto-von-Guericke University, Magdeburg, Germany. 22Department of Paediatric Endocrinology, Emma Children's Hospital, Amsterdam UMC, Vrije Universiteit Amsterdam, Amsterdam, Netherlands. 23Centro de Investigaciones Endocrinológicas (CEDIE-CONICET), Hospital de Niños Ricardo Gutiérrez, Buenos Aires, Argentina. 24Paediatric Endocrinology Unit, Istanbul University, Istanbul Faculty of Medicine, Istanbul, Turkey. 25Saglik Bilimleri University, Medical Faculty Zeynep Kamil Maternity and Children Hospital, Pediatric Endocrinology Clinic, Istanbul, Turkey. 26Centre for Endocrinology, Queen Mary University of London Barts and The London School of Medicine and Dentistry, London, United Kingdom. 27Institute for Diabetes and Endocrinology, Schneider's Children Medical Center of Israel, Petah-Tikva, Israel. 28Sackler School of Medicine, Tel-Aviv University, Tel-Aviv, Israel. 29Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil. 30Department of Paediatric Endocrinology, Regina Margherita Children's Hospital, University of Torino, Torino, Italy. 31Pediatric Endocrinology Wilhelmina Childrens Hospital, University Medical Centre Utrecht, Utrecht, Netherlands. 32Department of Paediatrics, Medical University of Varna, Varna, Bulgaria. 33Royal Hospital for Children, University of Glasgow, Glasgow, United Kingdom


Introduction: Despite existing guidelines there is no unified approach to glucocorticoid and mineralocorticoid replacement in congenital adrenal hyperplasia (CAH). Consequently, treatment varies in adults and children as well as across countries.

Objective: We used data from the I-CAH Registry to identify geographical and temporal variations in the treatment with glucocorticoids and mineralocorticoids of children and adults with CAH.

Methods: Data extraction was conducted in January 2019. We analysed 4866 patient visits (31 centres from 16 countries) between 1982 and 2018 with regards to the type, dose and timing of glucocorticoid and mineralocorticoid replacement. Hydrocortisone dose equivalents were calculated as 20 mg hydrocortisone = 4 mg prednisolone = 750 mg dexamethasone = 25 mg cortisone acetate.

Results: Data from 618 patients (350 females, 268 males) were analysed. Information on the glucocorticoid treatment was recorded in 4831 visits for 598 patients. The most frequently used glucocorticoid was hydrocortisone in children (88%), prednisolone (51%) and dexamethasone (28%) in adults. Most children received three glucocorticoid doses per day (74%); adults frequently received one (49%) or two (34%) daily doses. Glucocorticoid doses varied across age groups, with the hydrocortisone-equivalent in mg/m2/day (median with interquartile range) of 13.4 (10.3–17.8) in 0-1 years, 12.0 (10.0–14.4) in 1-8 years, 12.9 (10.6–15.4) in 8-12 years, 11.8 (6.0-15.1) in 12-18 years, 5.6 (3.5-12.4) in 18-30 years and 9.4 (5.8-14.3) in over 30 year-old patients. 500 patients (80.9%) had mineralocorticoid replacement (4474 visits). Most patients (63.5% of children, 67% of adults) received fludrocortisone once daily. Relative mineralocorticoid doses were significantly different between age groups, with a fludrocortisone dose (mg/m2/day, median with interquartile range) of 312 (212-476) in 0-1 years, 140 (94-205) in 1-8 years, 54 (41-91) in 8-12years, 51 (34-76) in 12-18 years, 41 (31-76) in 18-30 years and 85 (51-107) in over 30 years old patients. A significant reduction in the glucocorticoid doses for children of 0-1 years was noted after 2010: 15.0 (11.6–20.3) mg/m2/day to 12.2 (10.0–15.9) mg/m2/day (P<0.001); however, this was not found in other age groups. There was huge variation among different countries and centres regarding type, dose and timing of glucocorticoid and mineralocorticoid treatment.

Conclusion: Data available through the I-CAH Registry suggests international variations in hormone replacement therapy, with a tendency for high doses in younger patients. Further evidence regarding the impact of different treatment regimens on health outcomes will help improve the medical management of patients with CAH.

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