Sorafenib for desmoid tumors – pro

Test biopsy c on microscope for cancer cells


Desmoid tumors, also called aggressive fibromatosis, deep musculoaponeurotic fibromatosis, and fibrosarcoma grade I of the desmoid type, do not generally metastasize but can cause pain and compromise organs. Few treatments for this condition are well established, although many novel agents are being studied. Among them is Nexavar (sorafenib). The basis for the interest in this drug is that it appears to inhibit MPNST cell growth in vitro.

A recent study of sorafenib in children with neurofabromatosis was not able to establish the minmally tolerated dose. A series from Sloan Kettering used sorafenib as first-line therapy in 11/26 patients and the remaining 15/26 had received a median of 2 prior lines of therapy. Twenty-three of 26 patients had shown evidence of progressive disease by imaging, whereas 3 patients had achieved maximum benefit or toxicity with chemotherapy. Sixteen of 22 (∼70%) patients reported significant improvement of symptoms. At a median of 6 months (2-29) of treatment, the best response evaluation criteria in solid tumors (RECIST) 1.1 response included 6/24 (25%) patients with partial response (PR), 17/24 (70%) with stable disease, and 1 with progression and death. NCCN lists sorafenib on p. SARC-E and references the Goundar paper as the basis for its recommendation.

See also here for a more general discussion of chemotherapy for desmoid tumors.

Other options, see here and here and here

For Lay version see here


39 year-old woman who had been treated with Doxil and experimental therapies of fibromatosis adn nwo Nexvar is being proposed.

23.Ambrosini G, Cheema HS, Seelman S, et al. Sorafenib inhibits growth and mitogen-activated protein kinase signaling in malignant peripheral nerve sheath cells. Mol Cancer Ther. Apr 2008;7(4):890-6.

 Kim A, Dombi E, Tepas K, Fox E, Martin S, Wolters P, Balis FM,Phase I trial and pharmacokinetic study of sorafenib in children with neurofibromatosis type I and plexiform neurofibromas.Pediatr Blood Cancer. 2012 Sep 7.

Current Perspectives on Desmoid Tumors: The Mayo Clinic ApproachCancers 2011, 3, 3143-3155

Gounder MM, Lefkowitz RA, Keohan ML, et al. Activity of Sorafenib against desmoid tumor/deep fibromatosis. Clin Cancer Res 2011; 17:4082.

nccn, Soft TIssue Sarcoma, 2012

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