ESPE Abstracts

Context: Children with congenital adrenal hyperplasia (CAH) require hydrocortisone replacement from birth. The highest ACTH driven endogenous production of androgens happens in the early morning. To achieve a good therapy control, immediate release hydrocortisone is given early in the morning or late at night. In year 2021 the hydrocortisone modified-release hard capsule (Efmody®) was approved by the European medicine agency and from September 2021 it is licenced in Germany for therapy in children aged 12 years and older with adrenal insufficieny. Due to the modified-release of hydrocortisone, a peak concentration of Cortisol is achieved in the early morning by taking the highest daily dose of the medication at bedtime.

Objective: To investigate growth, pubertal development, safety and long-term disease control in children and youth with CAH treated with hydrocortisone modified-release hard capsules and monitored through 17-OHP saliva profiles.

Methods: A retrospective and descriptive analysis of CAH-patients treated in a one-centre outpatient clinic. Analysis of weight, length, blood pressure, dose of hydrocortisone before and after start of treatment with hydrocortisone modified-release hard capsules, as well as therapy control by 17-OHP saliva sampling and incidence of adrenal crisis.

Results: Forty-five children with CAH (20 females) are treated with the modified-release hard capsules since September 2021. Median age of treated children is 12.2 years (12,24 +/-4,73 years). Mean dose relation of immediate release hydrocortisone/hydrocortisone modified-release hard capsule at changeover to Efmody® was 0.96 +/- 0.11. Height was analysed according parental target height (z-scores). Further, weight, blood pressure, pubertal development and skeletal age was analysed. No increase of adrenal crisis was observed despite a lot of illnesses calling for stress dosing. This was done with immediate release hydrocortisone or a combination of Efmody® and immediate release hydrocortisone.

Conclusion: Hydrocortisone modified-release hard capsules, approved for the therapy of adrenal insufficiency in children older than 12 years of age is a safe therapy in children. There was no increase in adrenal crisis observed.