Modified-release hydrocortisone (Efmody®) in children with congenital adrenal hyperplasia: a retrospective registry study - Takeaways - MDSpire
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Efficacy and Safety of Modified-Release Hydrocortisone (Efmody®) in Pediatric Patients with Congenital Adrenal Hyperplasia: A Retrospective Analysis from a Registry

  • By

  • Erwin Lankes

  • Levin Wiebelt

  • Kathrin Bettina Helge

  • Dirk Schnabel

  • Peter Kühnen

  • Oliver Blankenstein

  • Uta Neumann

  • July 7, 2026

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  • 1

    The study evaluated the efficacy and safety of modified-release hydrocortisone (HMRC) in pediatric patients with congenital adrenal hyperplasia (CAH).

  • 2

    A total of 36 children with CAH were analyzed, with a median age of 10 years at the time of switching to HMRC therapy.

  • 3

    HMRC therapy led to a significant decrease in morning 17-hydroxyprogesterone levels from 337 ng/L to 214 ng/L after treatment switch.

  • 4

    The mean hydrocortisone dose increased by 2.4 mg/m²/day during HMRC treatment, indicating a change in dosage requirements.

  • 5

    No adrenal crisis was observed during the study, suggesting a favorable safety profile for HMRC in this pediatric population.

Original Source(s)

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

  • by Erwin Lankes, Levin Wiebelt, Kathrin Bettina Helge, Dirk Schnabel, Peter Kühnen, Oliver Blankenstein, Uta Neumann

  • July 7, 2026

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