Endocrine Abstracts

Introduction: In classic congenital adrenal hyperplasia (CAH), maintaining adequate glucocorticoid replacement to suppress androgen excess while avoiding treatment-related metabolic complications remains a significant clinical challenge. There is limited real world data on the effectiveness of modified release hydrocortisone (Efmody) in CAH in achieving these goals.

Aim: To evaluate biochemical and metabolic outcomes before and after transition to Efmody in adults with classic CAH

Methodology: Retrospective review was conducted from the CAH database of Sheffield Teaching Hospitals NHS Foundation Trust. Data was collected on demographics, anthropometric measurements, pre and post Efmody biochemical parameters including androgens, HbA1c, and lipid profile.

Results: Data of 18 adults were analysed; 83.3% (n = 14) had a salt losing phenotype and 10(52.6%) were men. Mean age 47.2 years(20-68 years). Duration of follow up after switching to Efmody ranged from 7-529 weeks(mean 226.8). Before Efmody, 10 patients were taking hydrocortisone and 8 were on prednisolone, with a mean hydrocortisone-equivalent dose of 24.1 mg/day. After transition, mean Efmody dose was 23.1mg/day (SD±5.2mg; P = 0.68). Mean fludrocortisone dose 125microgramsg/day (SD±87.2micrograms). Among 15 patients with paired biochemical results, significant reductions were observed in 17-hydroxyprogesterone (pre Efmody median 88.1nmol/l (IQR 26.8-209.0nmol/l) vs postEfmody median 51.9nmol/l, (IQR 10.1-81.8nmol/l); P = 0.03) and androstenedione (pre Efmody median 14.6nmol/l (IQR 6.4-23.5nmol/l); vs post Efmody median 6.2 nmol/l (IQR 3.1-9.2nmol/l); P = 0.018). Weight showed a non significant rise by 2 kg (P = 0.68) and HbA1c demonstrated a trend of improvement by 4.5mmol/mol following Efmody (P = 0.46). The parameters in the Lipid profile (Total cholesterol, non HDL cholesterol, HDL, Triglycerides) remained largely similar following initiation of Efmody.

Conclusion: Overall, Efmody resulted in significant improvement of disease control particularly for 17 OHP and androstenedione in CAH with stable metabolic parameters. The small sample size is a limitation and further studies are needed to evaluate the effect on metabolic health.