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Research Article: Modified-release hydrocortisone (Efmody ® ) in children with congenital adrenal hyperplasia: a retrospective registry study

Date Published: 2026-07-07

Abstract:
Replacement therapy for congenital adrenal hyperplasia (CAH) in childhood includes hydrocortisone and, in salt-wasting forms, addition of fludrocortisone. Immediate-release hydrocortisone often fails to adequately suppress early morning 17-hydroxyprogesterone (17OHP) levels. Hydrocortisone modified-release capsules (HMRC, Efmody ® ) were approved by the EMA in 2021 for the treatment of patients aged ?12 years with CAH. This study evaluates the efficacy and safety of HMRC in children and adolescents based on real-world data collected from the I-CAH registry. A single-center retrospective analysis of children with CAH was conducted. Linear mixed models (LMMs) were used to calculate pre–post comparisons of 17OHP saliva profiles, growth rate, bone age, body mass index, hydrocortisone and fludrocortisone dosage, blood pressure, and safety parameters before and during HMRC therapy (study registration, https://drks.de/search/de , DRKS00038319). The median age of the 36 children with CAH at the time of treatment switching to HMRC was 10 years (interquartile range [IQR]: 8–14 years). Of these, 15 were prepubertal and 21 were (post)pubertal. HMRC therapy was associated with a deceleration of previously increased growth velocity (pre-switch: 0.1 SDS/year, 95% CI: 0.0 to 0.2; post-switch: -0.1 SDS/year, 95% CI: –0.2 to 0.1) and with a reduction of median difference of bone age and chronological age by 0.50 years (IQR -1.17–0.27, range -2.2–2.00). Mean hydrocortisone dose increased by 2.4 mg/m²/day during HMRC treatment. Mean morning 17OHP concentrations significantly decreased from 337 ng/L (95% CI: 240–437) to 214 ng/L (95% CI: 155–294) after switching to HMRC treatment. No adrenal crisis was observed. Twice-daily HMRC therapy can also improve CAH therapy in children and adolescents in terms of hormonal control and growth.

Introduction:
Replacement therapy for congenital adrenal hyperplasia (CAH) in childhood includes hydrocortisone and, in salt-wasting forms, addition of fludrocortisone. Immediate-release hydrocortisone often fails to adequately suppress early morning 17-hydroxyprogesterone (17OHP) levels. Hydrocortisone modified-release capsules (HMRC, Efmody ® ) were approved by the EMA in 2021 for the treatment of patients aged ?12 years with CAH. This study evaluates the efficacy and safety of HMRC in children and adolescents based on…

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