Introduction: Despite existing guidelines there is no unified approach to glucocorticoid and mineralocorticoid replacement in congenital adrenal hyperplasia (CAH). Consequently, treatment varies in adults and children as well as across countries.
Objective: We used data from the I-CAH Registry to identify geographical and temporal variations in the treatment with glucocorticoids and mineralocorticoids of children and adults with CAH.
Methods: Data extraction was conducted in January 2019. We analysed 4866 patient visits (31 centres from 16 countries) between 1982 and 2018 with regards to the type, dose and timing of glucocorticoid and mineralocorticoid replacement. Hydrocortisone dose equivalents were calculated as 20 mg hydrocortisone = 4 mg prednisolone = 750 mg dexamethasone = 25 mg cortisone acetate.
Results: Data from 618 patients (350 females, 268 males) were analysed. Information on the glucocorticoid treatment was recorded in 4831 visits for 598 patients. The most frequently used glucocorticoid was hydrocortisone in children (88%), prednisolone (51%) and dexamethasone (28%) in adults. Most children received three glucocorticoid doses per day (74%); adults frequently received one (49%) or two (34%) daily doses. Glucocorticoid doses varied across age groups, with the hydrocortisone-equivalent in mg/m2/day (median with interquartile range) of 13.4 (10.317.8) in 0-1 years, 12.0 (10.014.4) in 1-8 years, 12.9 (10.615.4) in 8-12 years, 11.8 (6.0-15.1) in 12-18 years, 5.6 (3.5-12.4) in 18-30 years and 9.4 (5.8-14.3) in over 30 year-old patients. 500 patients (80.9%) had mineralocorticoid replacement (4474 visits). Most patients (63.5% of children, 67% of adults) received fludrocortisone once daily. Relative mineralocorticoid doses were significantly different between age groups, with a fludrocortisone dose (mg/m2/day, median with interquartile range) of 312 (212-476) in 0-1 years, 140 (94-205) in 1-8 years, 54 (41-91) in 8-12years, 51 (34-76) in 12-18 years, 41 (31-76) in 18-30 years and 85 (51-107) in over 30 years old patients. A significant reduction in the glucocorticoid doses for children of 0-1 years was noted after 2010: 15.0 (11.620.3) mg/m2/day to 12.2 (10.015.9) mg/m2/day (P<0.001); however, this was not found in other age groups. There was huge variation among different countries and centres regarding type, dose and timing of glucocorticoid and mineralocorticoid treatment.
Conclusion: Data available through the I-CAH Registry suggests international variations in hormone replacement therapy, with a tendency for high doses in younger patients. Further evidence regarding the impact of different treatment regimens on health outcomes will help improve the medical management of patients with CAH.
19 - 21 Sep 2019
European Society for Paediatric Endocrinology