ESPE Abstracts (2019) 92 P1-167

Development of an International Benchmark for Sick Day Episodes as a Core Clinical Outcome in People with Congenital Adrenal Hyperplasia

Salma Ali1, Eleni Daniel2, Jillian Bryce1, Adalia Ikiroma1, James Lewsey3, Richard Ross2, Ruth Krone4, Carlo Acerini5, Nils Krone6, Urmi Das7, Jeremy Tomlinson8, Marta Korbonits9, Claire Higham10, Feyza Darendeliler11, Tulay Guran12, Ayla Guven13, Navoda Attapatu14, Tatjana Milenkovic15, Corina Raducanu-Lichiardopol16, Sabine Hannema17, Hedi Claahsen18, Martijn Finken19, Federico Baronio20, Antonio Balsamo20, Silvia Einaudi21, Liat de Vries22, Andrea Luczay23, Uta Neumann24, Oliver Blankenstein24, Klaus Mohnike25, Walter Bonfig26, Heba Elsedfy27, Niels Birkebaek28, Violeta Iotova29, Tania Bachega30, Berenice Mendonca30, Martine Cools31, Eduardo Correa Costa32, Guilherme Guaragna-Filho33, Rodolfo Rey34, S. Faisal Ahmed1


1Developmental Endocrinology Research Group, School of Medicine, Dentistry & Nursing, University of Glasgow, Glasgow, United Kingdom. 2Department of Oncology and Metabolism, University of Sheffield,, Sheffield, United Kingdom. 3Health Economics and Health Technology Assessment, Institute of Health and Wellbeing, University of Glasgow, Glasgow, United Kingdom. 4Institute of Metabolism and Systems Research, University of Birmingham, Birmingham, United Kingdom. 5Department of Paediatrics, University of Cambridge, Cambridge, United Kingdom. 6University of Sheffield, Sheffield, United Kingdom. 7Alder Hey Childrens Hospital, Liverpool, United Kingdom. 8Department of Endocrinology, Oxford Centre for Diabetes, Endocrinology and Metabolism (OCDEM) and NIHR Oxford Biomedical Research Centre, Churchill Hospital, University of Oxford, Oxford, United Kingdom. 9Centre for Endocrinology, William Harvey Research Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, United Kingdom. 10Department of Endocrinology, Christie Hospital Nhs Foundation Trust, Manchester, University Of Manchester, Manchester Academic Health Science Centre, Manchester, United Kingdom. 11Istanbul Faculty of Medicine, Department of Pediatrics, Pediatric Endocrinology Unit, Istanbul University, Istanbul, Turkey. 12Marmara University, Department of Pediatric Endocrinology and Diabetes, Istanbul, Turkey. 13Istanbul Medeniyet University, Goztepe Education and Research Hospital, Istanbul, Turkey. 14The Central Hospital, Columbo, Sri Lanka. 15University of Belgrade, Belgrade, Serbia. 16University of Medicine and Pharmacy, Craiova, Romania. 17Leiden University Medical Centre, Leiden, Netherlands. 18Radboudumc Amalia Children's Hospital, Nijmegen, Netherlands. 19VU University Medical Center, Department of Pediatrics, Subdivision of Endocrinology, Amsterdam, Netherlands. 20Department of Woman, Child and Urologic Diseases, S. Orsola-Malpighi Hospital, University of Bologna, Bologna, Italy. 21Torino - Regina Margherita Children's Hospital, Torino, Italy. 22The Jesse Z and Sara Lea Shafer Institute for Endocrinology And Diabetes, National Center For Childhood Diabetes, Schneider Children's Medical Center Of Israel, Petah Tikva, Israel. 23Ist Department of Paediatrics, Semmelweis University, Budapest, Hungary. 24Charité Universitätsmedizin, Berlin, Germany. 25Otto-von-Guericke Universität Magdeburg, Magdeburg, Germany. 26Department of Pediatrics, Klinikum Wels-Grieskirchen, Wels, Austria; Department of Pediatrics, Technical University München, Munich, Germany. 27Ain Shams University, Cairo, Egypt. 28Dept of Pediatrics, Aarhus University Hospital, Aarhus, Denmark. 29; Clinic Of Paediatric Endocrinology - Umhat 'St. Marina', Medical University of Varna, Varna, Bulgaria. 30University of Sao Paulo, Sao Paulo, Brazil. 31Department of Paediatric Endocrinology, Ghent University Hospital, Department of Internal Medicine and Paediatrics, Ghent, Belgium. 32Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil. 33Catholic University, Porto Alegre, Brazil. 34Centro de Investigaciones Endocrinológicas "Dr. César Bergadá" (CEDIE) CONICET – FEI – División de Endocrinología, Hospital de Niños Ricardo Gutiérrez Gallo 1330, Buenos Aires, Argentina


Background: Congenital adrenal hyperplasia (CAH) is a rare condition characterised by adrenal insufficiency and a life-long risk of adrenal crises. There is a paucity of information on the epidemiology of acute adverse events in this population.

Objective: To investigate the frequency, aetiology and consequences of acute adverse events attributed to adrenal insufficiency in CAH.

Methods: A longitudinal analysis of patients with CAH in the International Congenital Adrenal Hyperplasia Registry (I-CAH registry, www.i-cah.org) which collects information on acute adverse events including sick day episodes and adrenal crises.

Results: 509 patients (n= 478, 21-OHD) from 31 centres in 16 countries and a total of 3880 visits were evaluated. 261 patients (n=255, 21-OHD) had one or more sick day episodes (684 visits); of these, 215 (82%) were less than 18 years of age. 1034 sick day episodes were recorded in total, with 920 (89%) episodes recorded in those less than 18 years of age. The overall median number of sick day episodes for all centres per patient year was 3.0 for children (IQR 1.7-4.7) and 3.9 for adults (IQR 1.8-10.2) (P=0.26). The median duration of sick day episodes was 3 days (IQR 2.0-5.0) and 2 days (IQR 1.0-3.0) in children and adults respectively (P<0.05). During childhood, younger age and low hydrocortisone dose (mg/m2/day) were associated with a greater number of sick day episodes (P<0.01). Female sex was associated with higher rates of admission amongst both children and adults (P<0.01). Infectious illness was the most frequent event causing illness episodes and adrenal crises in both children (66%) and adults (23%). An adrenal crisis was reported in 37 (4%, 37/920) and 34 (30%, 34/114) sick day episodes amongst children and adults, respectively (P<0.05) and all adults required hospital admission.

Conclusions: The real world data within the I-CAH registry are a valuable resource for studying a core clinical outcome that can be used as a benchmark for improving clinical care. Further work needs to be undertaken to understand the determinants of the observed variations in the occurrence of sick day episodes.

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