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Contemporary global management of 21-hydroxylase deficiency congenital adrenal hyperplasia in early infancy:a multi-national registry study

Title: Contemporary global management of 21-hydroxylase deficiency congenital adrenal hyperplasia in early infancy:a multi-national registry study
Authors: Lim, David B.N.; Bryce, Jillian; Ali, Salma R.; Tseretopoulou, Xanthippi; Birkebaek, Niels H.; Hannema, Sabine E.; Campos-Martorell, Ariadna; Clemente, Maria; Neumann, Uta; Flück, Christa E.; Metzger, Sara; Krone, Ruth E.; German, Alina; Baronio, Federico; Barat, Pascal; Delagrange, Marine; Vautier, Vanessa; Vieites, Ana; Rey, Rodolfo; Elsedfy, Heba; Atapattu, Navoda; Seneviratne, Sumudu N.; Cools, Martine; Guran, Tulay; Yavas Abali, Zehra; Fu, Antony; Janus, Dominika; Shenoy, Savitha; Wasniewska, Malgorzata; Coco, Roberto; Russo, Gianni; Stancampiano, Marianna Rita; Bonfig, Walter; Salerno, Mariacarolina; Claahsen-Van der Grinten, Hedi L.; Adriaansen, Bas P.H.; Mozzato, Chiara; Guazzarotti, Laura; Niedziela, Marek; Banaszak-Ziemska, Magdalena; Van Eck, Judith; Bachega, Tania; Miranda, Mirela C.; Marginean, Otilia; Munarin, Jessica; De Sanctis, Luisa; Probst-Scheidegger, Ursina; Lenherr-Taube, Nina; Konrad, Daniel; O’Connell, Michele; Gawlik-Starzyk, Aneta; Sandberg, David E.; Shnorhavorian, Margarett; Krone, Nils; Ahmed, S. Faisal; Davies, Justin H.
Source: Lim, D B N, Bryce, J, Ali, S R, Tseretopoulou, X, Birkebaek, N H, Hannema, S E, Campos-Martorell, A, Clemente, M, Neumann, U, Flück, C E, Metzger, S, Krone, R E, German, A, Baronio, F, Barat, P, Delagrange, M, Vautier, V, Vieites, A, Rey, R, Elsedfy, H, Atapattu, N, Seneviratne, S N, Cools, M, Guran, T, Yavas Abali, Z, Fu, A, Janus, D, Shenoy, S, Wasniewska, M, Coco, R, Russo, G, Stancampiano, M R, Bonfig, W, Salerno, M, Claahsen-Van der Grinten, H L, Adriaansen, B P H, Mozzato, C, Guazzarotti, L, Niedziela, M, Banaszak-Ziemska, M, Van Eck, J, Bachega, ....
Publication Year: 2026
Subject Terms: /dk/atira/pure/sustainabledevelopmentgoals/good_health_and_well_being; name=SDG 3 - Good Health and Well-being
Description: Objective: Management of 21-hydroxylase deficiency (21-OHD) congenital adrenal hyperplasia (CAH) in early infancy is challenging, with extent of variation in management unclear. Design and Methods: Using the I-CAH Registry, we retrospectively reviewed management over the first 90 days of life of 154 infants with 21-OHD born in 2018-2023, across 33 centers in 18 countries. Results: Of 154 infants (92 female, 62 male), 136 were diagnosed postnatally, with median (10th centile, 90th centile) presentation age of Day 4 (0, 20.8). At initial hospital discharge, median doses of hydrocortisone (HC), fludrocortisone (FC), and salt were 17 (11.4, 39.6) mg/m 2 /day, 100 (50, 200) mcg/day and 3.5 (1.6, 8.7) mmol/kg/day, and at Day 90 (D90) 14.5 (8.7, 24.1) mg/m 2 /day, 100 (50, 200) mcg/day, and 2.1 (1.0, 5.2) mmol/kg/day, respectively. Hyponatremia, hyperkalemia, and hypoglycemia were reported in 70.0%, 71.9%, and 13.0% of infants, respectively. At D90, hyponatremia and hyperkalemia were reported in 7.4% and 28.6%, respectively. At D90, BP measurements were recorded in 30.5%, amongst whom 31.9% had hypertension reported. Median total hospitalization duration over 90 days was 9 days (2, 24). Adrenal crises were associated with 40. 6% of hospitalization episodes. Percentages (males:females) of cases seen by a pediatric endocrinologist, psychologist, pediatric endocrine nurse specialist, and surgeon by D90 were 95.9% (58:84), 33.3% (9:35), 42.1% (20:36), and 23.8% (0:35), respectively. Conclusions: Contemporary management of CAH in early infancy varies considerably. Hypertension and hyperkalemia are frequently reported. Our data may help inform development of quality indicators for benchmarking CAH care in infancy.
Document Type: article in journal/newspaper
File Description: application/pdf
Language: English
ISSN: 0804-4643; 1479-683X
Relation: info:eu-repo/semantics/altIdentifier/pmid/41531283; info:eu-repo/semantics/altIdentifier/pissn/0804-4643; info:eu-repo/semantics/altIdentifier/eissn/1479-683X
DOI: 10.1093/ejendo/lvag004
Availability: https://pure.eur.nl/en/publications/6585f2c3-0edd-45c5-9c96-4c363147feaf; https://doi.org/10.1093/ejendo/lvag004; https://pure.eur.nl/ws/files/225686023/Contemporary_global_management_of_21-hydroxylase_deficiency_congenital_adrenal_hyperplasia_in_early_infancy.pdf; https://www.scopus.com/pages/publications/105029373297
Rights: info:eu-repo/semantics/openAccess ; http://creativecommons.org/licenses/by/4.0/
Accession Number: edsbas.482776A1
Database: BASE