ISSN 1470-3947 (print) | ISSN 1479-6848 (online)

Endocrine Abstracts (2019) 66 OC1.1 | DOI: 10.1530/endoabs.66.OC1.1

Exploring trends in the glucocorticoid and mineralocorticoid treatment of congenital adrenal hyperplasia by analysing data from the I-CAH registry

Irina-Alexandra Bacila1, Oliver Blankenstein2, Uta Neumann2, Heidi L Claahsen-van der Grinten3, Ruth Krone4, Tania SS Bachega5, Mirela C Miranda5, Berenice Mendonca5, Niels H Birkebaek6, Martine Cools7, Tatjana Milenkovic8, Walter Bonfig9,10, Jeremy W Tomlinson11, Heba Elsedfy12, Antonio Balsamo13, Rita Ortolano13, Sabine Hannema14,15, Claire Higham16, Navoda Atapattu17, Corina Lichiardopol18, Tulay Guran19, Zehra Abali19, Klaus Mohnike20, Martijn JJ Finken21, Ana Vieites22, Feyza Darendeliler23, Ayla Guven24, Marta Korbonits25, Liat de Vries26,27, Eduardo Costa28, Silvia Einaudi29, Hetty van der Kamp30, Violeta Iotova31, Richard Ross1, S Faisal Ahmed32 & Nils Krone1


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


Introduction: There is no unified approach in clinical practice regarding the medical management of congenital adrenal hyperplasia (CAH), despite existent international guidance. We aimed to explore geographical and temporal variations in the treatment with glucocorticoids and mineralocorticoids of patients with CAH.

Methods: We collected data recorded by 33 centres from 16 countries in the I-CAH Registry. We analysed patient visits between 1982 and 2018, exploring the type, dose and timing of glucocorticoid and mineralocorticoid replacement. We used the conversion rate: 20 mg hydrocortisone = 4 mg prednisolone = 0.25 mg dexamethasone = 25 mg cortisone acetate.

Results: 4934 patient visits from 601 patients with CAH (56% females) were analysed. Glucocorticoid replacement consisted primarily of hydrocortisone in children (88.8%) most frequently given in three daily doses (75%) and of prednisolone in adults (50.1%) usually given as one daily dose (67%). Glucocorticoid doses expressed as hydrocortisone-equivalent in mg/m2 per day (median with interquartile range) were 13.5 (10.3–17.8) in the 0–1 years, 11.9 (9.9–14.4) in 1–8 years, 13.0 (10.7–15.5) in 8–12 years, 14.0 (11.6–17.5) in 12–18 years, 13.5 (11.1–19.2) in 18–30 years and 12.9 (8.9–16.8) in the over 30 year-old patient subgroup. Glucocorticoid doses were significantly reduced after 2010 in patients 0–1 years (P<0.001) and 1–8 years (P<0.001), increased in patients 18–30 years (P=0.014) and statistically similar in the other age subgroups. Mineralocorticoid replacement was used for 81.9% patients, relative doses varying across age groups, with a fludrocortisone dose (μg/m2/day, median with interquartile range) of 312 (208–476) in the 0–1 years, 139 (94–205) in 1–8 years, 54 (42–91) in 8–12 years, 51 (34–76) in 12–18 years, 41 (31–74) in 18–30 years and 85 (51–107) in the over 30 year-old patients. There was wide variation among different countries and centres regarding type, dose and timing of glucocorticoid and mineralocorticoid treatment.

Conclusion: Our findings suggest international variations in hormone replacement therapy, with a tendency for higher doses in younger patients. Further evidence regarding the impact of different treatment regimens on health outcomes is needed to explore the benefits of a more uniform approach in the management of CAH.

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