Toulmonde M, et al. - In this non-comparative, randomized, open-label, phase 2 trial, researchers evaluated antitumor activity as well as the safety of targeted therapy or combination chemotherapy in progressive desmoid tumors. Using randomization (2:1), they allocated patients with progressive desmoid tumors to receive oral pazopanib 800 mg per day for up to 1 year or an intravenous regimen combining vinblastine (5 mg/m² per dose) and methotrexate (30 mg/m² per dose), given weekly for 6 months and then every other week for 6 months. In the first 43 patients who received one complete or two incomplete cycles of pazopanib, the proportion of patients who had not progressed at 6 months was assessed as the primary endpoint; in this study, 83.7% of patients in the pazopanib group had not progressed at 6 months, while in the methotrexate–vinblastine it was 45.0%. Findings revealed the clinical activity of pazopanib in patients with progressive desmoid tumors. In this rare and disabling condition, pazopanib may be a valid treatment option.