Article ; Online: Analysis of therapy monitoring in the International Congenital Adrenal Hyperplasia Registry.
2022 Volume 97, Issue 5, Page(s) 551–561
Abstract: Objective: Congenital adrenal hyperplasia (CAH) requires exogenous steroid replacement. Treatment is commonly monitored by measuring 17-OH progesterone (17OHP) and androstenedione (D4).: Design: Retrospective cohort study using real-world data to ... ...
| Abstract | Objective: Congenital adrenal hyperplasia (CAH) requires exogenous steroid replacement. Treatment is commonly monitored by measuring 17-OH progesterone (17OHP) and androstenedione (D4). Design: Retrospective cohort study using real-world data to evaluate 17OHP and D4 in relation to hydrocortisone (HC) dose in CAH patients treated in 14 countries. Patients: Pseudonymized data from children with 21-hydroxylase deficiency (21OHD) recorded in the International CAH Registry. Measurements: Assessments between January 2000 and October 2020 in patients prescribed HC were reviewed to summarise biomarkers 17OHP and D4 and HC dose. Longitudinal assessment of measures was carried out using linear mixed-effects models (LMEM). Results: Cohort of 345 patients, 52.2% female, median age 4.3 years (interquartile range: 3.1-9.2) were taking a median 11.3 mg/m Discussion: Registry data show large variability in 17OHP and D4 between centres. 17OHP correlates with D4 well when accounting for age. Prescribed HC dose per body surface area decreased with weight gain. |
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| MeSH term(s) | 17-alpha-Hydroxyprogesterone ; Adrenal Hyperplasia, Congenital/drug therapy ; Androstenedione ; Child ; Child, Preschool ; Female ; Humans ; Hydrocortisone/therapeutic use ; Male ; Progesterone ; Registries ; Retrospective Studies | |||||
| Chemical Substances | Androstenedione (409J2J96VR) ; Progesterone (4G7DS2Q64Y) ; 17-alpha-Hydroxyprogesterone (68-96-2) ; Hydrocortisone (WI4X0X7BPJ) | |||||
| Language | English | |||||
| Publishing date | 2022-07-11 | |||||
| Publishing country | England | |||||
| Document type | Journal Article ; Research Support, Non-U.S. Gov't | |||||
| ZDB-ID | 121745-8 | |||||
| ISSN | 1365-2265 ; 0300-0664 | |||||
| ISSN (online) | 1365-2265 | |||||
| ISSN | 0300-0664 | |||||
| DOI | 10.1111/cen.14796 | |||||
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| Database | MEDical Literature Analysis and Retrieval System OnLINE |
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