Cowden Disease (Multiple Hamartoma Syndrome) Medication

Updated: May 14, 2018
  • Author: Katherine H Fiala, MD; Chief Editor: William D James, MD  more...
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Medication

Medication Summary

Systemic therapy with retinoids may temporarily control some of the cutaneous lesions of Cowden disease (multiple hamartoma syndrome). Topical treatment usually is unsatisfactory.

Rapamycin has shown great promise in both regression of cutaneous lesions and prevention of the development of manifestations of Cowden disease (multiple hamartoma syndrome) in a mouse model with a PTEN deletion. In the mouse model, rapamycin inhibited a key downstream target, mammalian target of rapamycin (mTOR), which caused rapid regression of Cowden disease (multiple hamartoma syndrome)–like lesions. [34] At this time, however, use of rapamycin, should be limited to clinical trials. A clinical trial is currently underway that is studying the effects of rapamycin on syndromes with PTEN mutations.

 

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Retinoids

Class Summary

These agents are partially effective in treating cutaneous lesions.

Acitretin (Soriatane)

Acitretin is a retinoic acid analog similar to etretinate and isotretinoin. Acitretin is the main metabolite of etretinate and has demonstrated clinical effects close to those seen with etretinate. Its mechanism of action is suspected to be through its ability to cause increased differentiation of cells.

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