Activity of Sorafenib Against Desmoid Tumor/Deep Fibromatosis

The document summarizes a study on the efficacy of sorafenib, a tyrosine kinase inhibitor, in treating patients with Desmoid Tumor/Deep Fibromatosis. The study involved 26 patients, with 15 having received prior systemic therapy and 11 treated with sorafenib as first-line therapy. Sorafenib was administered at a median dose of 200 mg daily, with dose adjustments for toxicities. The results indicated that approximately 70% of patients experienced symptom improvement, with 6 out of 24 patients (25%) showing a partial response and 17 (70%) having stable disease. MRI evaluations revealed a greater than 30% decrease in T2 signal intensity in 12 out of 13 patients, suggesting increased fibrosis and loss of cellularity. The study concluded that sorafenib is active against Desmoid Tumor/Deep Fibromatosis and recommended further research to validate MRI T2 signal intensity as a response marker. The median follow-up was 6 months, and 25 out of 26 patients were still alive with disease at the time of the study. The study was supported by National Cancer Institute grants and other sources, and potential conflicts of interest were disclosed by one of the authors.