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

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

  • 0 min

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Objective:

To evaluate the efficacy and safety of hydrocortisone modified-release capsules (HMRC, Efmody®) in children and adolescents with congenital adrenal hyperplasia (CAH) based on real-world data.

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Key Findings:
  • Median age of participants at treatment switch was 10 years (IQR: 8–14 years).
  • HMRC therapy led to a deceleration in growth velocity (pre-switch: 0.1 SDS/year; post-switch: -0.1 SDS/year).
  • 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.
  • No adrenal crisis was observed during the study.
Interpretation:

Twice-daily HMRC therapy can improve hormonal control and growth in children and adolescents with CAH.

Limitations:
  • Study is based on a single-center retrospective analysis.
  • Data collected from a registry may have inherent biases.
Conclusion:

The study suggests that HMRC is effective in managing CAH in pediatric patients.

Sources:

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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