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Introduction <p>Congenital adrenal hyperplasia (CAH) is an autosomal recessive disorder characterized by impaired cortisol secretion and excess androgen production. The primary treatment for CAH is glucocorticoid (GC) replacement, which is essential to prevent adrenal crises and manage androgen excess. Dual-release hydrocortisone (DR-HC) is a once-daily modified-release formulation of hydrocortisone approved for adrenal insufficiency, which showed to improve the metabolic profile and reduce cardiovascular risk compared to conventional GCs. However, data on its use in CAH patients are scant.</p> Aim <p>To retrospectively evaluate clinical, metabolic, and hormonal characteristics of CAH patients switched from conventional GC treatment to DR-HC.</p> Patients and Method <p>This retrospective study involved 17 patients (10 males and 7 females) with classic CAH due to 21-OH-hydroxilase deficiency, who were switched to DR-HC. Clinical, metabolic, and hormonal data were assessed at baseline, at six months, and at the last available visit.</p> Results <p>Patients showed a tendency to a worse hormonal control, despite an overall increase in daily hydrocortisone equivalent dose; in fact, we observed a trend of increase in androstenedione and 17-OHP levels and a significant deterioration in androstenedione to total testosterone ratio in male (1.37 vs 2.10, p = 0.04); additionally, there was a decrease in the proportion of patients with good disease control [5/17 (29%) vs 1/17 (6%) at last available visit, p = 0.07]. Notably, compared to prior conventional GC regimens total and LDL-c cholesterol levels significantly improved (respectively 178 mg/dL vs 156 mg/dL, p = 0.015 and 101 mg/dL vs 83 mg/dL, p = 0.027), while there were no significant changes in glucose profile. In addition, we did not record any adrenal crises during follow-up; however, unsatisfactory disease control was the primary reason for discontinuation in 11/17 patients after a median duration of 26 months.</p> Conclusions <p>While DR-HC can replicate the circadian rhythm of cortisol secretion, it offers suboptimal hormone control in CAH patients despite increasing HC equivalent doses. Nonetheless, DR-HC is a safe treatment that could improve the metabolic profile compared to conventional GCs. However, DR-HC may be unsuitable in cases requiring tight regulation of androgen excess.</p>

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Impact of dual-release hydrocortisone on disease control and metabolism in congenital adrenal hyperplasia: a retrospective cohort study

  • 期刊论文

  • 发表日期：

    2025年7月3日

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  • Pierluigi Mazzeo,
  • Irene Tizianel,
  • Chiara Sabbadin,
  • Giacomo Voltan,
  • Giorgia Antonelli,
  • Filippo Ceccato,
  • Mattia Barbot

摘要

Introduction

Congenital adrenal hyperplasia (CAH) is an autosomal recessive disorder characterized by impaired cortisol secretion and excess androgen production. The primary treatment for CAH is glucocorticoid (GC) replacement, which is essential to prevent adrenal crises and manage androgen excess. Dual-release hydrocortisone (DR-HC) is a once-daily modified-release formulation of hydrocortisone approved for adrenal insufficiency, which showed to improve the metabolic profile and reduce cardiovascular risk compared to conventional GCs. However, data on its use in CAH patients are scant.

Aim

To retrospectively evaluate clinical, metabolic, and hormonal characteristics of CAH patients switched from conventional GC treatment to DR-HC.

Patients and Method

This retrospective study involved 17 patients (10 males and 7 females) with classic CAH due to 21-OH-hydroxilase deficiency, who were switched to DR-HC. Clinical, metabolic, and hormonal data were assessed at baseline, at six months, and at the last available visit.

Results

Patients showed a tendency to a worse hormonal control, despite an overall increase in daily hydrocortisone equivalent dose; in fact, we observed a trend of increase in androstenedione and 17-OHP levels and a significant deterioration in androstenedione to total testosterone ratio in male (1.37 vs 2.10, p = 0.04); additionally, there was a decrease in the proportion of patients with good disease control [5/17 (29%) vs 1/17 (6%) at last available visit, p = 0.07]. Notably, compared to prior conventional GC regimens total and LDL-c cholesterol levels significantly improved (respectively 178 mg/dL vs 156 mg/dL, p = 0.015 and 101 mg/dL vs 83 mg/dL, p = 0.027), while there were no significant changes in glucose profile. In addition, we did not record any adrenal crises during follow-up; however, unsatisfactory disease control was the primary reason for discontinuation in 11/17 patients after a median duration of 26 months.

Conclusions

While DR-HC can replicate the circadian rhythm of cortisol secretion, it offers suboptimal hormone control in CAH patients despite increasing HC equivalent doses. Nonetheless, DR-HC is a safe treatment that could improve the metabolic profile compared to conventional GCs. However, DR-HC may be unsuitable in cases requiring tight regulation of androgen excess.