Document Type

Poster

Publication Date

5-1-2019

Institution/Department

Nursing; Maine Medical Center Research Institute

MeSH Headings

Sirolimus, Fibromatosis, Aggressive

Abstract

Background:

• 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

Comments

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.

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