Background: Recommendations for initial and maintenance dose of HC in CAH differ widely. However, treatment of CAH in young children is complicated by the lack of a suitable low-dose HC preparations.
Methods: The German Society for Paediatric Endocrinology and Diabetology (DGKED) initiated a registry for patients with classical CAH (German CAH registry). Anonymized data are transferred for central analysis, including a validation step and a benchmarking report, twice yearly. Until March 2018, the DGKED-CAH database included 26818 patient visits from 1480 patients (654 males). 43 centres from Germany and 3 centres from Austria contributed longitudinal data. Parameters were selected based on current treatment guidelines. A custom-made electronic health record software is used at participating centres for standardized documentation and anonymous transfer of patient records. Linear regression model were implemented with SAS 9.4.
Results: Daily hydrocortisone (HC) dose per m2 body surface area (BSA) was 20.03±0.35 mg (mean ±SE) for patients younger than 3 months of age (n=309), 15.10±0.3 mg/m2 for the age-group 412 months (n=442), 14.31±0.24 for patients 15 years (n=707) and 14.54±0.20 for patients 618 years (n=969). Overall, there was no significant gender difference regarding dose. In patients on fludrocortisone (presumable salt-losing CAH), HC dose was lower in the age-group 03 months (19.6 versus 24.4 mg/m2, P<0.05) and in the age-group 15 years (14.1 versus 15.1 mg/m2, P<0.05), while in patients older than 6 years a higher HC dose was recorded in patients on fludrocortisone (15.1 versus 13.5 mg/m2, P<0.0001). ANOVA regression modeling revealed a significant interaction between age-group and fludrocortisone use, as well as between gender and fludrocortisone use (both P<0.001).
On average, the largest HC dose was administered in the morning. Median absolute doses morning midday evening were 2.0 [Q1-Q3: 2.02.5] 1 [1.01.4] 1.0 [1.01.5] mg for children <3 months of age, and 2.5 [2.03.0] 1.1 [1.01.8] 1.3 [1.02.0] in the age-group 412 months. For children 15 years of age, the respective median doses were 4.2 [3.05.3], 2.2 [1.72.6] and 2.3 [1.82.9] mg.
Conclusion: This large, multicentre database provides comprehensive information on prescribed hydrocortisone substitution doses in children with CAH. Low absolute doses required in children younger than 6 years of age are difficult to achieve, considering that currently only a 10 mg tablet formulation is available in Austria and Germany.
27 - 29 Sep 2018
European Society for Paediatric Endocrinology