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A Prospective Study of Children Aged 0-8 Years with CAH and Adrenal Insufficiency Treated with Hydrocortisone

Uta Neumann1, Katarina Braune1, Martin J Whitaker2

  • 1Charité Universitaetsmedizin Berlin, Berlin, Germany.

The Journal of Clinical Endocrinology and Metabolism
|September 5, 2020
PubMed

Insights

Accurate hydrocortisone dosing for children with adrenal insufficiency (AI) and congenital adrenal hyperplasia (CAH) using saliva profiles led to normal growth and no adrenal crises. This study highlights the benefits of precise monitoring from birth.

Area of Science:

  • Pediatric Endocrinology
  • Pharmacology
  • Genetics

Background:

  • Children with congenital adrenal hyperplasia (CAH) and adrenal insufficiency (AI) require precise daily hydrocortisone replacement therapy.
  • Accurate dosing is crucial for managing these conditions and preventing complications.

Purpose of the Study:

  • To evaluate the efficacy and safety of hydrocortisone granules in pediatric patients with AI and CAH.
  • To assess the utility of 17-hydroxyprogesterone (17-OHP) saliva profiles for monitoring hydrocortisone therapy.

Main Methods:

  • A prospective study involving 18 children (birth to 6 years) with AI or CAH.
  • Hydrocortisone medication was switched to granules, with therapy adjusted every 3 months using 17-OHP saliva profiles.
  • Parameters monitored included hydrocortisone dose, growth, pubertal status, adverse events, and adrenal crises.

Main Results:

  • No significant trends in accelerated or reduced growth were observed over the 2-year study period.
  • No adrenal crises occurred, despite 193 treatment-emergent adverse events, primarily common childhood illnesses.
  • Hydrocortisone doses were adjusted based on salivary measurements, resulting in doses at the lower end of the recommended range.

Conclusions:

  • This study demonstrates that precise hydrocortisone dosing and monitoring from birth in children with AI and CAH leads to normal growth.
  • The use of 17-OHP saliva profiles facilitates accurate dose adjustments, preventing adrenal crises.
  • Hydrocortisone granules are an effective and safe treatment option for pediatric AI and CAH.
Abstract

Keywords:
adrenal insufficiencychildrencongenital adrenal hyperplasiahydrocortisonesaliva

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