Document Type


Publication Date



Nursing, Maine Medical Center Research Institute

MeSH Headings

Sirolimus, Fibromatosis, Aggressive



• Desmoid tumor represents an intermediate grade neoplasm with a striking predilection for locally invasive growth and recurrence following resection

• More effective, well-tolerated non-surgical treatment options are needed

• Current approaches

• If feasible, watchful waiting is the preferred approach

• 20-30% spontaneous regression

• In situations where treatment is indicated, the following approaches are utilized

• Surgery is the primary approach if minimal morbidity is anticipated

• Medical therapies

• Cytotoxic drugs

• Tyrosine kinase inhibitors

• Hydroxyurea

• Gamma secretase inhibitors

• mTOR Inhibitor Rationale

• Desmoid tumor is well-known to be associated with deregulation of the APC/β-catenin pathway

• Deregulation of the mTOR cell proliferation/survival pathway may play an important role in tumor biology when the APC/βcatenin pathway is disrupted

• The mTOR inhibitor sirolimus is attractive as a potential targeted therapy for desmoid tumor

• Well-tolerated in children and young adults • Can be given orally in tablet or liquid formulation


Lambrew Research Retreat

Acknowledgements: Thank you to the Desmoid Tumor Research Foundation for funding, Pfizer for drug supply, and all of the participating sites, and patients.



To view the content in your browser, please download Adobe Reader or, alternately,
you may Download the file to your hard drive.

NOTE: The latest versions of Adobe Reader do not support viewing PDF files within Firefox on Mac OS and if you are using a modern (Intel) Mac, there is no official plugin for viewing PDF files within the browser window.