BSPED2024 Oral Communications Endocrine Oral Communications 1 (9 abstracts)
Glucocorticoid replacement therapy in congenital adrenal hyperplasia and its associations with growth outcomes - real world data analysis from an international cohort of 1500 patients
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 , Ved Bhushan. Arya 6 , Navoda Atapattu 7 , Tânia Bachega 8 , Pascal Barat 9 , Federico Baronio 10 , Silvano Bertelloni 11 , Niels Birkebæk 12 , Walter Bonfig 13,14 , Christiaan de. Bruin 15 , Hedi Claahsen. – van der Grinten 16,17 , Martine Cools 18 , Eduardo Costa 19 , Elizabeth Crowne 20 , Justin H. Davies 21 , Miguel Debono 1,22 , Liat de. Vries 23 , Katja Dumic. Kubat 24 , Heba Elsedfy 25 , Olcay Evliyaoglu 26 , Christa Flüeck 27 , Gabriella Gazdagh 28 , Alina German 29 , Evelien F. Gevers 30 , Evgenia Globa 31 , Tulay Guran 32 , Ayla Güven 33 , Laura Guazzarotti 34 , Sabine Hannema 35,36 , Nihal Hatipoglu 37 , Dominika Janus 38 , Hetty van. de. Kamp 39 , Zacharoula Karabouta 40 , Ruth E. Krone 3 , Sofia Leka-Emiri 41 , Nina Lenherr. Taube 42 , Corina Lichiardopol 43 , Mona Mamdouh. Hassan 44 , Otilia Marginean 45 , Renata Markosyan 46 , Inas Mazen 47 , Harriet Miles 48 , Anna L. Mitchell 49 , Klaus Mohnike 50 , Uta Neumann 51 , Marek Niedziela 52 , Anna Nordenstrom 53 , Susan M. O’Connell 54 , Franziska Phan-Hug 55 , Sukran Poyrazoglu 56 , Ursina Probst-Scheidegger 57 , Rodolfo Rey 58 , Gianni Russo 59 , Mariacarolina Salerno 60 , Luisa de. Sanctis 61 , Sumudu N. Seneviratne 62 , Anat Segev-Becker 63 , Savitha Shenoy 64 , Mars Skae 65 , Ajay Thankamony 66 , Ahmet Ucar 67 , Agustini Utari 68 , Ana Vieites 58 , Malgorzata Wasniewska 69 , 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 and Diabetes, King’s College Hospital, London, United Kingdom; 7Lady Ridgeway Hospital Colombo, Colombo, Sri Lanka; 8Endocrinology Department, School of Medicine, São Paulo University, Sao Paulo, Brazil; 9Pedatric Endocrinology, centre of reference for rare adrenal diseases, University Hospital of Bordeaux, Bordeaux, France; 10Department Hospital of Woman and Child, Pediatric Unit, Endo-ERN Center for Rare Endocrine Diseases, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy; 11Paediatric and Adolescent Endocrinology, Division of Pediatrics, Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy; 12Department of Pediatrics, Aarhus University Hospital, Aarhus, Denmark; 13Department of Pediatrics, Technical University Munich, Munich, Germany; 14Department of Pediatrics, Klinikum Wels-Grieskirchen, Wels, Austria; 15Division of Endocrinology, Department of Pediatrics, Willem-Alexander Children’s Hospital, Leiden University Medical Center, Leiden, Netherlands; 16Department of Pediatric Endocrinology, Radboud University Medical Centre, Nijmegen, Netherlands; 17Amalia Children’s Hospital, Radboud University Medical Centre, Nijmegen, Netherlands; 18Pediatric Endocrinology, Ghent University and Ghent University Hospital, Ghent, Belgium; 19Pediatric Surgery Service, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil; 20Bristol Royal Hospital for Children, University Hospitals Bristol & Weston Foundation Trust, Bristol, United Kingdom; 21University Hospital Southampton, Southampton, United Kingdom; 22Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, United Kingdom; 23Institute for Diabetes and Endocrinology, Schneider’s Children Medical Center of Israel, Petah-Tikvah; Faculty of Medical & Health Sciences, Tel-Aviv University, Tel-Aviv, Israel; 24University Hospital Centre Zagreb, Zagreb, Croatia; 25Department of Pediatrics, Ain Shams University, Cairo, Egypt; 26İstanbul University-Cerrahpaşa, Cerrahpaşa Faculty of Medicine, Department of Pediatric Endocrinology, Istanbul, Turkey; 27Paediatric Endocrinology, Diabetes and Metabolic medicine, Medizinische Universitätskinderklink, Bern, Switzerland; 28Clinical Genetics, Southampton General Hospital, Southampton, United Kingdom; 29Pediatric Endocrinology and Diabetes Unit, Haemek Medical Center, Afula, Israel; 30Centre for Endocrinology, William Harvey Research Institute, Queen Mary University London, London and Barts Health NHS Trust - The Royal London Hospital, London, United Kingdom; 31Ukrainian Scientific and Practical Center of Endocrine Surgery, Transplantation of Endocrine Organs and Tissues of the Ministry of Health of Ukraine, Kyiv, Ukraine; 32Department of Pediatric Endocrinology and Diabetes, Marmara University, Istanbul, Turkey; 33Istanbul Baskent University Hospital, Istanbul, Turkey; 34Pediatric Endocrinology Unit University of Padova, Padova, Italy; 35Department of Paediatric Endocrinology, Erasmus MC, Sophia Children’s Hospital, University Medical Center Rotterdam, Rotterdam, Netherlands; 36Leiden University Medical Centre, Department of Paediatrics, Leiden, Netherlands; 37Division of Pediatric Endocrinoloji, Faculty of Medicine, Erciyes University, Kayseri, Turkey; 38University Children Hospital in Krakow, Jagiellonian University Medical College, Department of Pediatric and Adolescent Endocrinology, Krakow, Poland; 39Pediatric Endocrinology Wilhelmina Children’s Hospital, University Medical Centre Utrecht, Utrecht, Netherlands; 40Paediatric Endocrinology Department, St George’s University Hospital, London, United Kingdom; 41"P.& A. KYRIAKOU" Children’s Hospital, Athens, Greece; 42Division of Pediatric Endocrinology and Diabetology and Children’s Research Center, University Children’s Hospital, Zurich, Switzerland; 43Department of Endocrinology, University of Medicine and Pharmacy Craiova, Craiova, Romania; 44The Diabetes Endocrine and Metabolism Pediatric Unit, Children’s Hospital, Cairo University, Cairo, Egypt; 45Victor Babes University of Medicine and Pharmacy of Timisoara, Timişoara, Disturbances of Growth and Development on children BELIVE; University of Medicine and Pharmacology Timisoara, Timisoara, Romania; 46Wigmore Womens and Children Hospital, Head of Pediatric Endocrinology Unit, Yerevan State Medical University, Endocrinology Department, Yerevan, Armenia; 47Clinical Genetics Department, Human Genetics and Genome Research Institute, National Research Centre, Cairo, Egypt; 48Royal Hospital for Children and Young People, Edinburgh, United Kingdom; 49Department of Endocrinology, Royal Victoria Infirmary, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle, United Kingdom; 50Otto-von-Guericke University, Department of Pediatric, Magdeburg, Germany; 51Clinic for paediatric endocrinology and diabetology, Center for chronik sich children, Charité Universitätsmedizin Berlin, Berlin, Germany; 52Department of Pediatric Endocrinology and Rheumatology, Institute of Pediatrics, Karol Jonscher’s Clinical Hospital, Poznan University of Medical Sciences, Poznan, Poland; 53Department of Women’s and Children’s Health, Karolinska Institutet, Stockholm, Sweden; 54Department of Diabetes and Endocrinology, Children’s Health Ireland, Dublin, Ireland; 55Department of Service of Endocrinology, Diabetes, and Metabolism, Faculty of Biology and Medicine, University of Lausanne, Lausanne University Hospital, Lausanne, Switzerland; 56Paediatric Endocrinology Unit, Istanbul University, Istanbul Faculty of Medicine, Istanbul, Turkey; 57Pediatric Department, Kantonsspital Winterthur, Winterthur, Switzerland; 58Centro de Investigaciones Endocrinológicas “Dr César Bergadá” (CEDIE), CONICET-FEI-División de Endocrinología, Hospital de Niños Ricardo Gutiérrez, Buenos Aires, Argentina; 59Department of Paediatrics, Endocrine Unit, Scientific Institute San Raffaele, Milan, Italy; 60Pediatric Endocrine Unit, Department of Translational Medical Sciences, University of Naples, Naples, Italy; 61Paediatric Endocrinology, Regina Margherita Children’s Hospital, Torino, Italy Department of Public Sciences and Pediatrics, University of Torino, Torino, Italy; 62Faculty of Medicine, University of Colombo, Colombo, Sri Lanka; 63Pediatric Endocrinology and Diabetes Unit, Dana-Dwek Children’s Hospital, Tel Aviv Sourasky Medical Center, Tel-Aviv, Israel; 64Department of Paediatric Endocrinology, Leicester Royal Infirmary, UHL NHS Trust, Leicester, United Kingdom; 65Department of Paediatric Endocrinology, Royal Manchester Children’s Hospital, Manchester, United Kingdom; 66Department of Paediatrics, Biomedical Campus, University of Cambridge, Cambridge, United Kingdom; 67University of Health Sciences, Şişli Hamidiye Etfal Health Practices and Research Centre, Department of Pediatric Endocrinology and Diabetes, Istanbul, Turkey; 68Division of Pediatric Endocrinology, Department of Pediatrics, Faculty of Medicine, Diponegoro University, Semarang, Indonesia; 69Department of Human Pathology in Adulthood and Childhood, University of Messina, Messina, Italy
Background and Aim: Previous research using data from the I-CAH registry showed wide variation between countries in the provision of glucocorticoid (GC) replacement in congenital adrenal hyperplasia (CAH). In this study, we aimed to establish the impact of different GC doses on height and weight in children and young people with CAH.
Methods: Data from children with CAH recorded in the I-CAH registry since 2003 was collected, providing a cohort of 1522 patients (770 females) from 22 countries (60 centres). We analysed information from 12,401 clinic visits, to study the relationship between GC doses (hydrocortisone (HC) equivalent/m2/day) and height and weight standard deviation scores (SDS) calculated for age and sex, using the WHO normative data
Results: We found wide variability in the relative daily GC dose used in different countries, ranging between a mean of 5.0 (±2.1) to 19.6 (±7) HC-equivalent/m2/day. The country where patients were treated was found to influence significantly the GC dose used, as shown by regression analysis (R2=0.17, P < 0.01). Height-SDS’ were low (under 0) during infancy and increased during early childhood up until the age of 9 years, then decreased again, following an “inverted U-shape” trend with age. In patients under 9 years of age, height-SDS increased with the GC dose (R2=0.01, P < 0.01), while in older patients the relationship was inverse (R2=0.01, P < 0.01). Multivariable regression showed that weight, relative GC dose and the country of residence accounted for up to 70% of the variance in height-SDS in both patients younger than 9 years (boys: R2=0.73, P < 0.01; girls: R2=0.67, P < 0.01) and those over 9 years (boys: R2=0.51, P < 0.01; girls: R2=0.62, P < 0.01). Weight-SDS increased weakly with the GC dose (R2=0.002, P < 0.01), however, a stronger relationship was found with birth weight and patients’ country of origin (boys: R2=0.19, P < 0.01; girls: R2=0.20, P < 0.01).
Conclusions: We believe that our findings indicate wide variations in clinical practice for GC replacement in children with CAH. Importantly, the relative GC doses used appear to have a significant impact on patients’ growth. The low height-SDS during infancy may indicate growth suppression by GC overexposure in early life, highlighting the need to optimise steroid replacement.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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