Analysis of therapy monitoring in the International Congenital Adrenal Hyperplasia Registry.

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,

SUBMITTER: Lawrence N 

PROVIDER: S-EPMC9796837 | biostudies-literature | 2022 Nov

REPOSITORIES: biostudies-literature