BSPED2025 Oral Communications Endocrine Oral Communications 1 (8 abstracts)
Practice patterns and treatment outcomes of growth-promoting therapies in CAH: insights from real-world data
Chamila Balagamage 1 , Irina Bacila 2 , Neil R. Lawrence 2 , Jan Idkowiak 1,3 , Jillian Bryce 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 22,23 , Dominika Janus 24 , Sofia Leka 25 , Violeta Lotova 17 , Renata Markosyan 26 , Inas Mazen 27 , Mirela Costa Miranda 9 , Klaus Mohnike 28 , Jessica Munarin 29 , Uta Neumann 30 , Marek Niedziela 31 , Anna Nordenstrom 32 , Susan O Connell 33 , Rumeysa Ozsaray 20 , Katja Palm 28 , Sukran Poyrazoglu 34 , Rodolfo Rey 35,36 , Gianni Russo 37 , Luisa de Sanctis 29 , Valerie Schwitzgebel 38 , Anat Segev-Becker 39 , Sumudu Seneviratne 40 , Savitha Shenoy 41 , Marianna Stancampiano 37 , Jerzy Starzyk 24 , Ahmet Ucar 42 , Agustini Utari 43 , Ana Vieites 35,36 , Faisal Ahmed 4,5 , Nils Krone 2 & Ruth Krone 1
1Department of Endocrinology, Birmingham Women’s & Children’s NHS Foundation Trust, Birmingham, United Kingdom; 2Division of Clinical Medicine, University of Sheffield, Sheffield, United Kingdom; 3Department of Metabolism and Systems Science, University of Birmingham, Birmingham, United Kingdom; 4Office for Rare Conditions, University of Glasgow, Glasgow, United Kingdom; 5Developmental Endocrinology Research Group, Royal Hospital for Children, Glasgow, United Kingdom; 6Department of Pediatrics, Ain Shams University, Cairo, Egypt; 7Paediatric Endocrinology and Diabetes, King’s College Hospital, London, United Kingdom; 8Lady Ridgeway Hospital for Children, Colombo, Sri Lanka; 9Laboratorio de Hormonios e Genetica Molecular LIM 42, Disciplina de Endocrinologia e Metabologia, Faculdade de Medicina, Universidade de Sao Paulo, Sao Paulo, Brazil; 10Department of Medical and Surgical Sciences, Pediatric Unit, Endo-ERN Center for Rare Endocrine Diseases, S.Orsola-Malpighi University Hospital, Bologna, Italy; 11Department of Pediatrics, Technical University Munich, Munich, Germany; 12Department of Pediatrics, Klinikum Wels-Grieskirchen, Wels, Austria; 13Division of Endocrinology, Department of Pediatrics, Willem-Alexander Children’s Hospital, Leiden University Medical Center, Leiden, Netherlands; 14Hospital Vall d’Hebron, Barcelona, Spain; 15Institute for Diabetes and Endocrinology, Schneider’s Children Medical Center of Israel, Petah-Tikvah, Israel; 16Faculty of Medical & Health Sciences, Tel Aviv University, Tel Aviv, Israel; 17UMHAT Sveta Marina, Varna, Bulgaria; 18Pediatric Endocrinology, Diabetology and Metabolism, Department of Pediatrics, Inselspital University Children’s Hospital, University of Bern, 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; 20Department of Pediatric Endocrinology and Diabetes, Marmara University, Istanbul, Turkey; 21Faculty of Medicine, University of Health Sciences, Istanbul, Turkey; 22Department of Paediatric Endocrinology, Amsterdam UMC location Vrije Universiteit Amsterdam, Amsterdam, Netherlands; 23Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, Netherlands; 24University Children’s Hospital, Krakow, Poland; 25Department of Endocrinology- Growth and Development, "P.& A. KYRIAKOU" Children’s Hospital, Athens, Greece; 26Department of Endocrinology, Yerevan State Medical University, Wigmore Children`s and Women`s Hospital, Yerevan, Armenia; 27Clinical Genetics Department, Human Genetics and Genome Research Institute, National Research Centre, Cairo, Egypt; 28Otto-von-Guericke University, Department of Pediatrics, Magdeburg, Germany; 29Paediatric Endocrinology, Regina Margherita Children’s Hospital - Department of Public Health and Pediatric Sciences, University of Torino, Torino, Italy; 30Clinic for paediatric endocrinology and diabetology and Center for Chronically Sick Children, Charite - Universitätsmedizin, Berlin, Germany; 31Department of Pediatric Endocrinology and Rheumatology, Institute of Pediatrics, Karol Jonscher’s Clinical Hospital, Poznan University of Medical Sciences, Poznan, Poland; 32Department of Women’s and Children’s Health, Karolinska Institutet, Stockholm, Sweden; 33Department of Diabetes and Endocrinology, Children’s Health Ireland at Crumlin, Dublin, Ireland; 34Paediatric Endocrinology Unit, Istanbul University, Istanbul Faculty of Medicine, Istanbul, Turkey; 35Department of Endocrinology, Hospital de Niños Ricardo Gutiérrez, Buenos Aires, Argentina; 36Department of Cellular Biology, Histology, Embryology and Genetics, Faculty of medicine, University of Buenos Aires, Buenos Aires, Argentina; 37Department of Paediatrics, Endocrine Unit, Scientific Institute San Raffaele, Milan, Italy; 38Geneva University Hospital, Geneva, Switzerland; 39The Institute of Pediatric Endocrinology, Diabetes and Metabolism, Dana-Dwek Children’s Hospital, Tel Aviv Sourasky Medical Center, Tel Aviv, Israel; 40Faculty of Medicine, University of Colombo, Colombo, Sri Lanka; 41Department of Paediatric Endocrinology, Leicester Royal Infirmary, Leicester, United Kingdom; 42Growth-Development and Pediatric Endocrine Unit, Istanbul School of Medicine, Istanbul University, Istanbul, Turkey; 43Division of Pediatric Endocrinology, Department of Pediatrics, Faculty of Medicine, Diponegoro University, Semarang, Indonesia
Background: Children with CAH are at risk of reduced adult height due to early bone maturation. Adjunct Growth promoting therapies aim to optimize height outcomes by delaying skeletal maturation and pubertal progression. This study evaluates real-world practice patterns and the impact of such therapies on growth trajectories in children with CAH.
Methods: We conducted a retrospective analysis of children with CAH due to 21 hydroxylase deficiency, who received adjunct growth-promoting therapy. Data from 19 countries was collected through the I-CAH registry.
Results: Of 162 children (92 males, 69 females), median age at diagnosis was 0.20 years (range 0.04–3.55). Aromatase inhibitors (AIs), GnRH analogues, growth hormone (GH), cyproterone acetate, and spironolactone were used in diverse combinations Letrozole was the most commonly used AI (68%,n = 36), predominantly at a dose of 2.5 mg daily (97.2%), with a median treatment duration of 2.98 years (IQR 1.84–4.6). Letrozole monotherapy significantly improved both bone age (BA)-adjusted height SDS (Δ 0.81, P < 0.0001) and height SDS relative to mid-parental height (MPH) (Δ 0.85, P = 0.0002). Combined use of Letrozole with GnRH analogues also demonstrated significant gains (BA-adjusted height SDS Δ 1.27, P = 0.0078; vs MPH Δ 1.27, P = 0.0312). The positive effect of AIs correlated with duration of therapy in monotherapy use (r = 0.70, P = 0.0006). No significant changes in glucocorticoid dose or 17-OHP levels were observed with AI therapy. Cyproterone acetate (n = 37) was initiated at a median age of 6.4 years, with a median dose of 50 mg/day (IQR 50-75)over 3.56 years. GH(n = 34) and GnRH analogues showed variable results; GnRH monotherapy led to significant improvement in height SDS (Δ 0.576, P = 0.0036). GH, whether alone or in combination, showed limited efficacy. Notably, a combination of cyproterone, GH, and GnRH resulted in a significant improvement (Δ height SDS 2.23, P = 0.037).
Conclusion: There is marked heterogeneity in global clinical practice regarding growth-promoting therapies in CAH. AIs and GnRH analogues, particularly in combination, appear most effective. These findings underscore the need for standardized international guidelines and long-term safety evaluation.
Volume 111
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