Sorafenib for Advanced and Refractory Desmoid Tumors.

ABSTRACT:

Background

Desmoid tumors (also referred to as aggressive fibromatosis) are connective tissue neoplasms that can arise in any anatomical location and infiltrate the mesentery, neurovascular structures, and visceral organs. There is no standard of care.

Methods

In this double-blind, phase 3 trial, we randomly assigned 87 patients with progressive, symptomatic, or recurrent desmoid tumors to receive either sorafenib (400-mg tablet once daily) or matching placebo. Crossover to the sorafenib group was permitted for patients in the placebo group who had disease progression. The primary end point was investigator-assessed progression-free survival; rates of objective response and adverse events were also evaluated.

Results

With a median follow-up of 27.2 months, the 2-year

SUBMITTER: Gounder MM 

PROVIDER: S-EPMC6447029 | biostudies-literature | 2018 Dec

REPOSITORIES: biostudies-literature