Aromatase inhibitors in pediatrics.

Area of Science:

• Endocrinology
• Pediatric Endocrinology
• Pharmacology

Background:

• Aromatase, encoded by CYP19A1, is crucial for androgen to estrogen conversion.
• Aromatase's role in epiphyseal plate closure is recognized, driving interest in its inhibitors for height enhancement.
• Selective aromatase inhibitors (e.g., letrozole, anastrozole) are approved for breast cancer and used off-label in pediatrics.

Purpose of the Study:

• To review the efficacy and safety of off-label aromatase inhibitor use in pediatric conditions.
• To evaluate treatment outcomes for hyperestrogenism, hyperandrogenism, pubertal gynecomastia, and short stature/pubertal delay.

Main Methods:

• Review of current data and clinical features related to aromatase inhibitor use in pediatric patients.
• Analysis of treatment effectiveness across various endocrine and pubertal disorders.

Main Results:

• Aromatase inhibitors are likely effective for aromatase excess syndrome and testotoxicosis.
• Partial effectiveness is noted in Peutz-Jeghers and McCune-Albright syndromes; likely ineffective for gynecomastia.
• Data is insufficient for congenital adrenal hyperplasia and ovarian cysts. Height increase observed in boys with short stature/pubertal delay, but with safety concerns.

Conclusions:

• Aromatase inhibitors offer potential therapeutic benefits for specific pediatric endocrine conditions.
• Efficacy varies significantly by condition, with notable successes in aromatase excess and testotoxicosis.
• Safety monitoring is essential due to potential adverse effects, particularly when used for height enhancement.