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Bicalutamide and third-generation aromatase inhibitors in testotoxicosis

Anne M Lenz1, Dorothy Shulman, Erica A Eugster

  • 1Department of Pediatric Endocrinology, Diabetes, and Metabolism, University of South Florida, All Children's Hospital, 501 Sixth Ave S, Box 6900, St Petersburg, FL 33701, USA. alenz@health.usf.edu

Pediatrics
|August 18, 2010
PubMed

Insights

Testotoxicosis treatment with antiandrogen and aromatase inhibitor combination therapy is well tolerated in boys. Longer-term results show slowed bone-age advancement and prevented virilization, supporting continued linear growth.

Area of Science:

  • Pediatric Endocrinology
  • Reproductive Endocrinology
  • Molecular Endocrinology

Background:

  • Testotoxicosis is gonadotropin-independent precocious puberty caused by luteinizing hormone receptor mutations in Leydig cells.
  • This condition leads to early virilization, advanced bone age, and potential short stature in affected males.

Observation:

  • A combination therapy using bicalutamide (antiandrogen) and anastrozole or letrozole (aromatase inhibitor) was previously reported with positive short-term outcomes.
  • This study evaluated the longer-term efficacy and tolerability of this combination therapy in two pediatric patients.

Findings:

  • The combination therapy was well tolerated over 4.5 and 5 years of treatment.
  • It effectively slowed bone-age advancement while allowing for continued linear growth.
  • The treatment also successfully prevented the progression of virilization.

Implications:

  • Longer-term use of antiandrogen and aromatase inhibitor combination therapy is a viable strategy for managing testotoxicosis.
  • This therapeutic approach offers a promising method to mitigate the adverse effects of precocious puberty on final adult height.
  • Further research may explore optimal treatment durations and long-term outcomes in a larger cohort.

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