Efficacy of sorafenib in patients with desmoid-type fibromatosis.

Abstract

11065Background: Desmoid tumors (DT) are rare fibroblastic neoplasms with a variable course. Observation, surgery, radiation and systemic agents are known therapies with variable outcomes. We previously reported on the efficacy of sorafenib (SO). Here we review a larger cohort with long-term follow up (FU). Methods: Patients (pts) with DT treated with SO were retrospectively identified. Baseline/treatment characteristics were analyzed using descriptive statistics. Response rates (RR) were calculated according to RECIST 1.1 and WHO criteria. Kaplan-Meier curves were estimated for survival and variables correlated with treatment outcomes were investigated. Results: We treated 79 pts with progressive or symptomatic DT with SO; median age was 37 years (range 17-81), 67% were female. Primary sites included extremities (n = 22; 28%), abdomen (n = 19; 24%), chest wall (n = 15; 19%), abdominal wall (n = 13; 16%) and other (n = 10; 13%). SO was the first systemic treatment in 49 pts (62%), most frequently at 400mg...