ESPE2024 Free Communications Adrenals and HPA Axis 1 (6 abstracts)
Real world data analysis of contemporary therapy and its association to anthropometric outcomes in 1500 patients with congenital adrenal hyperplasia (CAH).
Irina A Bacila 1,2 , Neil R Lawrence 1 , 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 A Bachega 8 , Pascal Barat 9 , Federico Baronio 10 , Silvano Bertelloni 11 , Niels Birkebæk 12 , Walter Bonfig 13,14 , Christiaan de Bruin 15,16 , Hedi Claahsen – van der Grinten 17,18 , Martine Cools 19 , Eduardo Costa 20 , Elizabeth Crowne 21 , Justin H Davies 22 , Miguel Debono 1,23 , Liat de Vries 24,25 , Katja Dumic Kubat 26 , Heba Elsedfy 27 , Olcay Evliyaoglu 28 , Christa Flüeck 29 , Gabriella Gazdagh 30 , Alina German 31 , Evelien F Gevers 32,33 , Evgenia Globa 34 , Tulay Guran 35 , Ayla Güven 36 , Laura Guazzarotti 37 , Sabine Hannema 38,39 , Nihal Hatipoglu 40 , Dominika Janus 41 , Hetty van der Kamp 42 , Zacharoula Karabouta 43 , Ruth Krone 3 , Nina Lenherr Taube 44 , Sofia Leka-Emiri 45 , Corina Lichiardopol 46 , Mona Mamdouh Hassan 47 , Otilia Marginean 48 , Renata Markosyan 49 , Inas Mazen 50 , Harriet Miles 51 , Anna L Mitchell 52 , Klaus Mohnike 53 , Uta Neumann 54 , Marek Niedziela 55 , Anna Nordenstrom 56 , Susan M O’Connell 57 , Franziska Phan-Hug 58,59 , Sukran Poyrazoglu 60 , Ursina Probst-Scheidegger 61 , Rodolfo Rey 62 , Gianni Russo 63 , Mariacarolina Salerno 64 , Luisa de Sanctis 65,66 , Sumudu N Seneviratne 67 , Anat Segev-Becker 68 , Savitha Shenoy 69 , Mars Skae 70 , Ajay Thankamony 71 , Ahmet Ucar 72 , Agustini Utari 73 , Ana Vieites 62 , Malgorzata Wasniewska 74 , S. Faisal Ahmed 4,5 & Nils P Krone 1,2
1University 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 EndocrinologKing’s College Hospital, London, United Kingdom. 7Lady Ridgeway Hospital Colombo, Colombo, Sri Lanka. 8School of Medicine, São Paulo University, São Paulo, Brazil. 9University Hospital of Bordeaux, Bordeaux, France. 10Endo-ERN Center for Rare Endocrine Diseases, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy. 11University of Pisa, Pisa, Italy. 12Aarhus University Hospital, Aarhus, Denmark. 13Technical University Munich, Munich, Germany. 14Klinikum Wels-Grieskirchen, Wels, Austria. 15Willem-Alexander Children's Hospital, Leiden, Netherlands. 16Leiden University Medical Center, Leiden, Netherlands. 17Radboud University Medical Centre, Nijmegen, Netherlands. 18Amalia Children’s Hospital, Nijmegen, Netherlands. 19Ghent University and Ghent University Hospital, Ghent, Belgium. 20Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil. 21Bristol Royal Hospital for Children, University Hospitals Bristol & Weston Foundation Trust, Bristol, United Kingdom. 22University Hospital Southampton, Southampton, United Kingdom. 23Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, United Kingdom. 24Schneider's Children Medical Center of Israel, Petah-Tikvah, Israel. 25Faculty of Medical & Health Sciences, Tel-Aviv University, Tel-Aviv, Israel. 26University Hospital Centre Zagreb, Zagreb, Croatia. 27Ain Shams University, Cairo, Egypt. 28İstanbul University-Cerrahpaşa, Istanbul, Turkey. 29Medizinische Universitätskinderklink, Bern, Bern, Switzerland. 30Southampton General Hospital, Southampton, United Kingdom. 31Pediatric Endocrinology and Diabetes Unit, Haemek Medical Center, Afula, Israel. 32William Harvey Research Institute, Queen Mary University London, London, United Kingdom. 33The Royal London Hospital, London, United Kingdom. 34Ukrainian Scientific and Practical Center of Endocrine Surgery, Transplantation of Endocrine Organs and Tissues of the Ministry of Health of Ukraine, Kyiv, Ukraine. 35Marmara University, Istanbul, Turkey. 36Istanbul Baskent University Hospital, Istanbul, Turkey. 37University of Padova, Padova, Italy. 38Sophia Children's Hospital, University Medical Center Rotterdam, Rotterdam, Netherlands. 39Leiden University Medical Centre, Leiden, Netherlands. 40Erciyes University, Faculty of Medicine, Kayseri, Turkey. 41University Children Hospital in Krakow, Krakow, Poland. 42Pediatric Endocrinology Wilhelmina Children’s Hospital, University Medical Centre Utrecht, Utrecht, Netherlands. 43, St George’s University Hospital, London, United Kingdom. 44University Children's Hospital, Zurich, Zurich, Switzerland. 45"P.& A. KYRIAKOU" Children's Hospital, Athens, Greece. 46University of Medicine and Pharmacy Craiova, Craiova, Romania. 47Children's Hospital, Cairo, Cairo, Egypt. 48Victor Babes University of Medicine and Pharmacy of Timisoara, Timisoara, Romania. 49Wigmore Womens and Children Hospital, Yerevan State Medical University, Yerevan, Armenia. 50Human Genetics and Genome Research Institute, National Research Centre, Cairo, Egypt. 51Royal Hospital for Children and Young People, Edinburgh, United Kingdom. 52Royal Victoria Infirmary, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle, United Kingdom. 53Otto-von-Guericke University, Magdeburg, Germany. 54Center for chronik sich children, Charité Universitätsmedizin Berlin, Berlin, Germany. 55Karol Jonscher's Clinical Hospital, Poznan University of Medical Sciences, Poznan, Poland. 56Karolinska Institutet, Stockholm, Sweden. 57Children’s Health Ireland, Dublin, Ireland. 58EHC-Hôpital de Morges, Morges, Switzerland. 59Hôpital du Valais-Sion, Sion, Switzerland. 60Istanbul University, Istanbul, Turkey. 61Kantonsspital Winterthur, Winterthur, Switzerland. 62Centro de Investigaciones Endocrinológicas “Dr César Bergadá” (CEDIE), CONICET-FEI-División de Endocrinología, Hospital de Niños Ricardo Gutiérrez, Buenos Aires, Argentina. 63Scientific Institute San Raffaele, Milan, Italy. 64University of Naples Federico II, Naples, Italy. 65Regina Margherita Children's Hospital, Torino, Italy. 66University of Torino, Torino, Italy. 67University of Colombo, Colombo, Sri Lanka. 68Dana-Dwek Children's Hospital, Tel Aviv Sourasky Medical Center, Tel-Aviv, Israel. 69Leicester Royal Infirmary, Leicester, United Kingdom. 70Royal Manchester Children's Hospital, Manchester, United Kingdom. 71University of Cambridge, Cambridge, United Kingdom. 72University of Health Sciences, Şişli Hamidiye Etfal Health Practices and Research Centre, Istanbul, Turkey. 73Faculty of Medicine, Diponegoro University, Semarang, Indonesia. 74University of Messina, Messina, Italy
Introduction and Objective: Previous evidence from the I-CAH registry showed wide variation of glucocorticoid (GC) replacement between different countries and centres. We wanted to explore the impact of different GC doses on height and weight in children and young people with CAH.
Methods: We analysed data from patients under 18 years with 21-hydroxylase deficiency recorded in the I-CAH registry from assessments since 2003. We explored the relationship between GC doses expressed as hydrocortisone (HC) equivalent/m2/day and height and weight standard deviation scores (SDS) for age and sex, calculated using WHO as reference. The hydrocortisone dose equivalent ratios used were 20 : 4 : 0.25 : 25 (mg) for hydrocortisone : prednisolone : dexamethasone : cortisone acetate, respectively. Analysis consisted of descriptive statistics and linear multivariable regression in R.
Results: We analysed data from 1522 patients (770 females), 12,401 clinic visits, from 22 countries (60 centres). There was large variability in the GC dose per body surface area (BSA) used in different countries, ranging from a mean of 5.0 (±2.1) to 19.6 (±7) HC-equivalent/m2/day. Regression analysis with GC dose as dependent variable showed that it was influenced significantly by the country where patients were treated (R2=0.17, P <0.01). Patients’ height-SDS had an inverted U-shaped relationship with age, with low height SDS (below 0) during infancy, increased height-SDS during early childhood up to 9 years, and final decrease afterwards, in adolescence. Height-SDS increased with GC dose in children under 9 years of age (R2=0.01, P <0.01) and decreased with dose in older children (R2=0.01, P <0.01). Multivariable regression showed up to 70% of the variance in height-SDS was related to weight-SDS, GC dose per BSA and the country in which patients were treated 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 with HC dose per BSA; however, the association was weak (R2=0.002, P <0.01). The variables most strongly associated with weight-SDS were birth weight and patients’ country of origin (boys: R2=0.19, P <0.01; girls: R2=0.20, P <0.01).
Conclusions: There is wide variation among countries in the clinician’s practice of GC replacement in children with CAH. The relationship between height-SDS, GC doses and country of treatment indicate that different doses of GC replacement impact on patients’ growth. The low height-SDS during infancy highlights the pressing need to optimise replacement that avoids GC overexposure from early life.
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