What is the role of medications in the treatment of McCune-Albright syndrome (MAS)?

Updated: Jan 17, 2019
  • Author: Gabriel I Uwaifo, MD; Chief Editor: George T Griffing, MD  more...
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Answer

At present, no therapy addresses the underlying molecular problem in McCune-Albright syndrome (MAS) (ie, inappropriate activation of the G protein subunit Gs alpha). Various medications may be administered to correct various endocrine and metabolic derangements, including aromatase inhibitors, hormones, steroids, somatostatin analogues, dopamine agonists, bisphosphonates, estrogen receptor antagonists, antithyroid agents, and metabolic agents.

In a 2014 study, alendronate therapy induced improvement in aBMD and decreased the level of the bone resorption marker NTX-telopeptides but did not effect pain or functional parameters or serum osteocalcin. [65]

Continuous positive effect with long-term safety data was found for zoledronic acid therapy for MAS with severe bone destruction. [66]

Combined therapy with cyproterone acetate, ketoconazole, and leuprolide depot in a boy with concomitant atypical MAS increased predicted adult height. [67]


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