BSPED2021 Poster Presentations Adrenal (7 abstracts)
Analysis of therapy monitoring in the International Congenital Adrenal Hyperplasia Registry
Neil Lawrence 1,2 , Irina Bacila 1 , Jeremy Dawson 3 , Jillian Bryce 4 , Erica van den Akker 5 , Tânia Aparecida Sartori Sanchez Bachega 6 , Federico Baronio 7 , Niels Holtum Birkebæk 8 , Walter Bonfig 9 , Hedi Claahsen 10 , Eduardo Correa Costa 11 , Liat Devries 12,13 , Heba Elsedfy 14 , Ayla Guven 15 , Sabine Hannema 16 , Violeta Iotova 17 , Hetty J van der Kamp 18 , Maria Clemente Leon 19 , Corina Raducanu Lichiardopol 20 , Tatjana Milenkovic 21 , Uta Neumann 22 , Ana Nordenström 23,24 , Sukran Poyrazoglu 25 , Ursina Probst-Scheidegger 26 , Luisa De Sanctis 27 , Ajay Thankamony 28 , Ana Vieites 29 , Zehra Yavas 30 , Faisal Ahmed 4 & Nils Krone 1,2
1University of Sheffield, Sheffield, United Kingdom; 2Sheffield Children’s Hospital NHS Foundation Trust, Sheffield, United Kingdom; 3Sheffield University Management School, Sheffield, United Kingdom; 4University of Glasgow, Glasgow, United Kingdom; 5Sophia Children’s Hospital, Rotterdam, Netherlands; 6University of Sao Paulo, Sao Paulo, Brazil; 7S.Orsola-Malpighi University Hospital, Bologna, Italy; 8Aarhus University Hospital, Aarhus, Denmark; 9Technical University Munich, Munich, Germany; 10Radboud University Medical Centre, Nijmegen, Netherlands; 11Hospital de Clínicas de Porto Alegre, Porto alegre, Brazil; 12Schneider’s Children Medical Center of Israel, Petah-Tikvah, Israel; 13Tel-Aviv University, Tel-Aviv, Israel; 14Ain Shams University, Cairo, Egypt; 15Baskent University İstanbul Hospital, Istanbul, Turkey; 16Leiden University Medical Centre, Leiden, Netherlands; 17Medical University of Varna, Varna, Bulgaria; 18University Medical Centre Utrecht, Utrecht, Netherlands; 19Hospital Universitario Vall d’Hebron, Barcelona, Spain; 20University of Medicine and Pharmacy Craiova, Craiova, Romania; 21Institute for Mother and Child Healthcare of Serbia “Dr Vukan Čupić”, Belgrade, Serbia; 22Charite - Universitätsmedizin, Berlin, Germany; 23Karolinska Institutet, Stockholm, Sweden; 24Karolinska University Hospital, Stockholm, Sweden; 25Istanbul University, Istanbul, Turkey; 26Kantonsspital Winterthur, Winterthur, Switzerland; 27Regina Margherita Children’s Hospital, University of Torino, Italy; 28University of Cambridge, Cambridge, United Kingdom; 29Hospital de Niños Ricardo Gutiérrez, Buenos Aires, Argentina; 30Marmara University, Istanbul, Turkey.
Background: Congenital Adrenal Hyperplasia (CAH) requires exogenous steroid replacement and can be monitored with 17-OH Progesterone and Androstenedione. We reviewed real world data to evaluate these markers in relation to hydrocortisone dose in patients treated in 21 centres throughout 14 countries.
Method: Retrospective cohort study using pseudonymised data from patients with 21α-Hydroxylase Deficiency recorded in the International Congenital Adrenal Hyperplasia Registry. Assessments between January 2000 and October 2020 in patients prescribed hydrocortisone were reviewed. Recent biomarkers and doses between patients were summarised, and longitudinal assessment of measures within patients was carried out using linear mixed effects models (LMEM).
Results: Recent biomarkers were from a cohort of 345 patients, 52.2% female, median age 4.3 years (Interquartile Range (IQR) 3.1 to 9.2), with median weight z-score of 0.3 (-1.1 to 1.7) taking a median 11.3 mg/m2/day (IQR 8.6 to 14.4) of hydrocortisone. Median 17OH-Progesterone (17OHP) was 35.7nmol/l (IQR 3.0 to 104), 15.9% within a target of 12-36nmol/l and 50.0% above this range. Median Androstenedione (D4) under 12 years was 0nmol/l (IQR 0 to 2)) and those 12 years and over median 10.5nmol/l (IQR 3.9 to 21.0). There were significant differences in biomarkers between centres. Multiple regression showed strongest correlation of D4 with 17OHP when age was a covariate (P < 0.001, R2=0.29). In Longitudinal assessment, 17OHP did not change with age, whereas D4 increased significantly with age (D4=0.86+0.56xAge, P < 0.001, R2=0.08). Multivariate LMEM showed dose per body surface area (BSA) decreasing by 1.0 mg/m2/day for every 1 point increase in weight z-score. Neither 17OHP nor D4 were statistically significant when added to this model (P>0.05).
Discussion: I-CAH Registry data show large variability in 17OHP and D4, 17OHP commonly above target range and D4 concentrations increasing with age, with significant variability between treatment centres. Hydrocortisone dose per BSA decreases with weight gain and in the absence of poor control, the practice of increasing absolute dose to maintain a specific dose for BSA needs reconsideration.
Volume 78
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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