Real-world outcomes with modified-release hydrocortisone in classic congenital adrenal hyperplasia
Authors' conclusion
MR-HC enabled stable biochemical control with reduced GC exposure and was associated with improvements in insulin sensitivity, blood pressure, and menstrual regularity, supporting its role as a safe and physiological treatment option in adults with CAH.
Study context
- Population
- Adults with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (25 male; 42 salt-wasting), followed ≥12 months (median 23.5 months)
- Participants
- 62 adults
- Exposure
- Switch from conventional glucocorticoids (GCs) to modified-release hydrocortisone (MR-HC)
- MR-HCn = 62
- Outcome
- Normalization of elevated A4
- Follow-up
- Last available visit
Methodological detailssingle tertiary endocrine center, real-world, Visit 4 not yet completed in 6/62
- Single tertiary endocrine center
- Real-world
- Visit 4 not yet completed in 6/62
Main finding
As reported in the abstract
Patients with elevated A4 at baseline who normalized
11 of 13
A4 at baseline: within reference range / elevated / suppressed
42/62; 13/62; 7/62
Observational data: associations, not effects.
Further findings
Salivary 17-hydroxyprogesterone
- Decreased significantly, early morning
- P = 0.025
- Decreased significantly, 12 Pm
- P = 0.036
- Decreased significantly, 4 Pm
- P = 0.042
Hydrocortisone-equivalent dose
- Median dose declined from 30 mg (IQR 25-37.5) to 25 mg (IQR 20-30)
- P < 0.001
HOMA-IR
- HOMA-IR improved (2.15-1.67)
- P = 0.023
- Other metabolic parameters remained stable
Daytime systolic and diastolic BP
- Decreased by ∼5 mmHg (systolic; diastolic)
- P = 0.007 and P = .003
Fludrocortisone reduction in salt-wasting patients
- Fludrocortisone reduced
- 12/41 (29.3%)
Menstrual patterns
- Women with menstrual disturbances
- 12/42
- Normalized, among women with disturbances
- 6/12
- Conceived, among women with disturbances
- 1/12
- Developed irregular cycles or amenorrhea, among women with regular cycles
- 3/12
Safety
- Adrenal crisis rate
- 5.0 per 100 patient-years
Original publication
Real-world outcomes with modified-release hydrocortisone in classic congenital adrenal hyperplasia. Eur J Endocrinol. Published 19 May 2026. doi:10.
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