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Purpose <p>Puberty poses challenges for the management of congenital adrenal hyperplasia (CAH) due to changes in the endocrine milieu and non-compliance with treatment. Uncontrolled hyperandrogenemia is a significant problem, especially for CAH patients in this age group. Along with the attainment of final height, a therapeutic window emerges for employing more potent glucocorticoids (GCs) to improve disease management in the pediatric age group. Dexamethasone (Dex) is a long-acting and more potent GC, and offers a convenient dosing schedule for patients. In this study, we aimed to evaluate the efficacy of Dex treatment on biochemical and clinical hyperandrogenemia in patients with inadequately controlled CAH.</p> Methods <p>Children with CAH who had persistent biochemical/clinical hyperandrogenemia despite recommended hydrocortisone dosing were included in the study at a single institution within the past 20 years. Participants were switched to Dex therapy at or near final height. Clinical and laboratory data and treatment outcomes for all participants were retrospectively evaluated.</p> Results <p>Thirty-two patients (karyotype 46,XX in 18/32, 46,XY in 14/32) with a mean age of 15.5 ± 2 years were included in the study. 3 of 46,XX cases were male-reared (M<sup>R</sup>), and so a total of 17/32 (53%) of the patients were M<sup>R</sup>, and 15/32 (47%) were female-reared (F<sup>R</sup>). Following the transition to Dex therapy, both 17-hydroxyprogesterone (17OHP) (<i>p</i> &lt; 0.01) and 1,4-delta-androstenedione (A4) (<i>p</i> &lt; 0.01) levels showed a significant decrease after 6–12 months in all patients. In F<sup>R</sup> patients, puberty progressed in 5/5, menstrual regularity improved in 3/5, and hirsutism improved in 3/7. In M<sup>R</sup> patients, A4/testosterone ratio decreased in 10/10, testis volumes increased in 7/8. While there was no change in BMI z-score during treatment in M<sup>R</sup> patients, an increase in BMI z-score was observed only during the first six months in F<sup>R</sup> patients (<i>p</i> = 0.014).</p> Conclusion <p>Dex effectively suppresses adrenal androgens and can be used to restore the hypothalamo-pituitary-gonad axis in children with CAH who have uncontrolled hyperandrogenemia and have reached final height. To minimize weight gain and metabolic side effects, treatment should be initiated at a low dose and androgen levels should be monitored without delay.</p>

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Dexamethasone therapy in adolescents with inadequately controlled congenital adrenal hyperplasia: effects on hormonal Suppression, Puberty, and gonadal outcomes

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年3月10日

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  • N. Berna Çelik Ertaş,
  • Burcu Şenkalfa,
  • Doğuş Vurallı,
  • Z. Alev Özön,
  • E. Nazlı Gönç

摘要

Purpose

Puberty poses challenges for the management of congenital adrenal hyperplasia (CAH) due to changes in the endocrine milieu and non-compliance with treatment. Uncontrolled hyperandrogenemia is a significant problem, especially for CAH patients in this age group. Along with the attainment of final height, a therapeutic window emerges for employing more potent glucocorticoids (GCs) to improve disease management in the pediatric age group. Dexamethasone (Dex) is a long-acting and more potent GC, and offers a convenient dosing schedule for patients. In this study, we aimed to evaluate the efficacy of Dex treatment on biochemical and clinical hyperandrogenemia in patients with inadequately controlled CAH.

Methods

Children with CAH who had persistent biochemical/clinical hyperandrogenemia despite recommended hydrocortisone dosing were included in the study at a single institution within the past 20 years. Participants were switched to Dex therapy at or near final height. Clinical and laboratory data and treatment outcomes for all participants were retrospectively evaluated.

Results

Thirty-two patients (karyotype 46,XX in 18/32, 46,XY in 14/32) with a mean age of 15.5 ± 2 years were included in the study. 3 of 46,XX cases were male-reared (MR), and so a total of 17/32 (53%) of the patients were MR, and 15/32 (47%) were female-reared (FR). Following the transition to Dex therapy, both 17-hydroxyprogesterone (17OHP) (p < 0.01) and 1,4-delta-androstenedione (A4) (p < 0.01) levels showed a significant decrease after 6–12 months in all patients. In FR patients, puberty progressed in 5/5, menstrual regularity improved in 3/5, and hirsutism improved in 3/7. In MR patients, A4/testosterone ratio decreased in 10/10, testis volumes increased in 7/8. While there was no change in BMI z-score during treatment in MR patients, an increase in BMI z-score was observed only during the first six months in FR patients (p = 0.014).

Conclusion

Dex effectively suppresses adrenal androgens and can be used to restore the hypothalamo-pituitary-gonad axis in children with CAH who have uncontrolled hyperandrogenemia and have reached final height. To minimize weight gain and metabolic side effects, treatment should be initiated at a low dose and androgen levels should be monitored without delay.