Treatment of girls and boys with McCune-Albright syndrome with precocious puberty - Update 2017

Anna Neyman, Erica Eugster

Research output: Contribution to journalReview article

4 Scopus citations

Abstract

The most common endocrinopathy associated with McCune-Albright Syndrome (MAS) is peripheral precocious puberty (PP) which occurs far more often in girls than in boys. We will discuss the latest advancements in the treatment of precocious puberty in MAS that have been achieved during the past 10 years. However, due to the rarity of the condition and the heterogeneity of the disease, research in this field is limited particularly in regards to treatment in boys. In girls, a period of watchful waiting is recommended prior to initiating therapy due to extreme variability in the clinical course. This article will review in detail current pharmacologic treatment in girls, which typically consists of either inhibiting estrogen production or blocking estrogen action at the level of the end-organ. The two treatments with the most evidence at this time are Tamoxifen (which is an estrogen receptor modulator) and Letrozole (which is a 3rd generation aromatase inhibitor). This article will also review the current treatment strategies in boys which typically include using an androgen receptor blocker and an aromatase inhibitor. Due to the rarity of the condition, large multicenter collaborative studies are needed to further investigate efficacy and safety with the goal of establishing the gold standard for treatment of PP in children with MAS.

Original languageEnglish (US)
Pages (from-to)136-141
Number of pages6
JournalPediatric Endocrinology Reviews
Volume15
Issue number2
DOIs
StatePublished - Dec 1 2017

Keywords

  • Bicalutamide
  • Letrozole
  • McCune-Albright Syndrome
  • Precocious Puberty
  • Tamoxifen

ASJC Scopus subject areas

  • Medicine(all)

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