ECEESPE2025 Poster Presentations Adrenal and Cardiovascular Endocrinology (169 abstracts)
Growth-promoting therapy in congenital adrenal hyperplasia due to 21-hydroxylase deficiency: Real-world data from the I-CAH registry
Chamila Balagamage 1 , Irina Bacila 2 , Jan Idkowiak 1,3 , Neil R Lawrence 2 , Jillian Bryce 4,5 , Faisal Ahmed 4,5 , Malika Alimussina 4,5 , Nermine Amr 6 , Ved Bhushan Arya 7 , Navoda Atapattu 8 , Tânia Bachega 9 , Federico Baronio 10 , Walter Bonfig 11,12 , Christiaan de Bruin 13 , Ariadna Campos 14 , Maria Clemente 14 , Liat de Vries 15,16 , Yana Deyanova 17 , Heba Elsedfy 6 , Christa E Flück 18 , Evgenia Globa 19 , Tulay Guran 20 , Ayla Güven 21 , Sabine Hannema 22 , Dominika Janus 23 , Sofia Leka 24 , Violeta Lotova 17 , Renata Markosyan 25 , Inas Mazen 26 , Mirela Miranda 9 , Klaus Mohnike 27 , Jessica Munarin 28,29 , Uta Neumann 30 , Marek Niedziela 31 , Anna Nordenstrom 32 , Susan O Connell 33 , Rumeysa Ozsaray’s 20 , Sukran Poyrazoglu 34 , Rodolfo Rey 35,36 , Gianni Russo 37 , Luisa de Sanctis 28,29 , Valerie Schwitzgebel 38 , Anat Segev-Becker 39 , Sumudu Seneviratne 40 , Savitha Shenoy 41 , Marianna Stancampiano 37 , Jerzy Starzyk 23 , Ahmet Ucar 42 , Agustini Utari 43 , Ana Vieites 35,36 , Nils Krone 2 & Ruth Krone 1
1Birmingham Women’s & Children’s NHS Foundation Trust, Department of Endocrinology, Birmingham, UK; 2University of Sheffield, Division of Clinical Medicine, Sheffield, UK; 3University of Birmingham, Birmingham, UK; 4University of Glasgow, Office for Rare Conditions, Glasgow, UK; 5Royal Hospital for Children, Developmental Endocrinology Research Group, Glasgow, UK; 6Ain Shams University, Department of Pediatrics, Cairo, Egypt; 7King’s College Hospital, Paediatric Endocrinology and Diabetes, London, UK; 8Lady Ridgeway Hospital for Children, Department of Endocrinology, Colombo, Sri Lanka; 9Universidade de Sao Paulo, Laboratorio de Hormonios e Genetica Molecular LIM 42, Disciplina de Endocrinologia e Metabologia, Faculdade de Medicina, Sao Paulo, Brazil; 10Endo-ERN Center for Rare Endocrine Diseases, S.Orsola-Malpighi University Hospital, Department of Medical and Surgical Sciences, Bologna, Italy; 11Technical University Munich, Department of Pediatrics, Munich, Germany; 12Klinikum Wels-Grieskirchen, Department of Pediatrics, Wels, Austria; 13Willem-Alexander Children’s Hospital, Leiden University Medical Center, Division of Endocrinology, Department of Pediatrics, Leiden, Netherlands; 14Hospital Vall d’Hebron, Barcelona, Spain; 15Schneider’s Children Medical Center of Israel, Institute for Diabetes and Endocrinology, Petah-Tikvah, Israel; 16Tel Aviv University, Faculty of Medical & Health Sciences, Tel Aviv, Israel; 17UMHAT Sveta Marina, Varna, Bulgaria; 18Inselspital University Children’s Hospital, University of Bern, Pediatric Endocrinology, Diabetology and Metabolism, Bern, Switzerland; 19Ukrainian Scientific and Practical Center of Endocrine Surgery, Transplantation of Endocrine Organs and Tissues of the Ministry of Health of Ukraine, Kyiv, Ukraine; 20Marmara University, Department of Pediatric Endocrinology and Diabetes, Istanbul, Türkiye; 21University of Health Sciences, Faculty of Medicine, Istanbul, Türkiye; 22Amsterdam UMC location Vrije Universiteit, Department of Paediatric Endocrinology, Amsterdam, Netherlands; 23University Children’s Hospital, Krakow, Poland; 24”P.& A. KYRIAKOU” Children’s Hospital, Department of Endocrinology- Growth and Development, Athens, Greece; 25Yerevan State Medical University, Wigmore Children’s and Women’s Hospital, Yerevan, Armenia; 26Human Genetics and Genome Research Institute, National Research Centre, Clinical Genetics Department, Cairo, Egypt; 27Otto-von-Guericke University, Department of Pediatrics, Magdeburg, Germany; 28Regina Margherita Children’s Hospital, Department of Endocrinology, Torino, Italy; 29University of Torino, Department of Public Health and Pediatric Sciences, Torino, Italy; 30Universitätsmedizin, Berlin, Clinic for paediatric endocrinology and diabetology and Center for Chronically Sick Children, Charite, Berlin, Germany; 31Karol Jonscher’s Clinical Hospital, Poznan University of Medical Sciences, Department of Pediatric Endocrinology and Rheumatology, Poznan, Poland; 32Karolinska Institutet, Department of Women’s and Children’s Health, Stockholm, Sweden; 33Children’s Health Ireland at Crumlin, Department of Diabetes and Endocrinology, Dublin, Ireland; 34Faculty of Medicine, Istanbul University, Paediatric Endocrinology Unit, Istanbul, Türkiye; 35Hospital de Niños Ricardo Gutiérrez, Department of Endocrinology, Buenos Aires, Argentina; 36Faculty of medicine, University of Buenos Aires, Department of Cellular Biology, Histology, Embryology and Genetics, Buenos Aires, Argentina; 37Scientific Institute San Raffaele, Department of Paediatrics, Endocrine Unit, Milan, Italy; 38Geneva University Hospital, Geneva, Switzerland; 39Dana-Dwek Children’s Hospital, Tel Aviv Sourasky Medical Center, The Institute of Pediatric Endocrinology, Diabetes and Metabolism, Tel Aviv, Israel; 40University of Colombo, Faculty of Medicine, Colombo, Sri Lanka; 41Leicester Royal Infirmary NHS Trust, Department of Paediatric Endocrinology, Leicester, UK; 42Istanbul School of Medicine, Istanbul University, Growth-Development and Pediatric Endocrine Unit, Istanbul, Türkiye; 43Faculty of Medicine, Diponegoro University, Division of Pediatric Endocrinology, Semarang, Indonesia
JOINT1863
Introduction: Individuals with congenital adrenal hyperplasia (CAH) often experience reduced final adult height due to androgen excess (AE). AE induces early puberty, accelerates bone age, and leads to premature epiphyseal fusion. Growth-promoting therapies aim to mitigate sex-steroid effects and improve height outcomes. We aim to evaluate the use of growth-promoting therapies in CAH patients and their impact on height outcomes using real-world data from the I-CAH registry.
Methods: A retrospective analysis was conducted on 162 CAH patients from I-CAH registry from 33 centres in 19 countries, who were on growth-promoting therapies. Data on types of treatment agents, dose, and age at treatment initiation were analysed. Height outcomes were assessed using change of height standard deviations (S.D.s) corrected for bone age (BA), and height deviation from the mid-parental height (MPH) after treatment.
Results: The cohort included 162 patients (93 males, 57%; 69 females,43%) diagnosed at a median age of 0.22 years (IQR: 0.04–3.55), with growth-promoting therapy initiated at a median age of 7.75 years (IQR: 5.73–9.14). Median average hydrocortisone equivalent glucocorticoid (GC) dose before treatment was 13.56 mg/m2/day (IQR: 11.41–16.4), with 113 patients (70%) receiving fludrocortisone in combination with GC. Adherence issues with GC therapy were reported in 40%. Growth-promoting therapies included GnRH analogues (n=104, median initiation age: 8.01 years), aromatase inhibitors (AIs) (n=54, 7.68 years), growth hormone (GH) (n=34, 7.45 years), cyproterone acetate (n=38, 6.83 years), and spironolactone (n=8, 8.83 years), administered alone or in combination. Seventeen different drug combinations were used. Letrozole was the most used AI (69%, n=36) at a dose of 2.5 mg (97%, n=35). GH was administered at a median dose of 40 mcg/kg/day (range: 25–50), while median doses for cyproterone acetate and spironolactone were 50 mg/day (range: 25–200) and 100 mg/day (range: 25–150), respectively. After a mean treatment duration of 3.83 years (S.D. 2.14), significant improvements in height outcomes were observed. Mean height S.D. adjusted for BA increased significantly from −2.36 pre-treatment to −1.44 post-treatment (Δ height S.D.: 0.92, P<0.00001). Similarly, height S.D.s deviation from MPH improved significantly from −1.64 pre-treatment to −0.65 post-treatment (Δ height deviation: 0.65, P<0.00001).
Conclusion: This real-world study demonstrates the diverse use of growth-promoting therapies in CAH management and their potential to improve height outcomes by reducing the height deficit relative to BA and MPH. However, lack of a unifying approach emphasizes the need for standardized treatment strategies to improve patient outcomes through establishing evidence-based guidelines.
Volume 110
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Endocrine Abstracts
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