Single-center phase 3 trial of pegylated liposomal doxorubicin for patients with advanced or refractory desmoid tumors

A phase 3 trial at Sun Yat-sen University Cancer Center (Guangzhou, China) evaluated efficacy and safety of pegylated liposomal doxorubicin (PLD) for advanced or refractory desmoid tumors (DTs). Patients were randomized 2:1 to receive either PLD (50 mg/m² intravenously; n=49) or placebo (n=24) every 4 weeks for 6 cycles. Crossover from placebo to PLD was permitted upon disease progression. The primary endpoint was progression-free survival (PFS). With a median follow-up of 16.1 months, the PFS was not reached in the PLD group versus 4.3 months in the placebo group (HR 0.05; p<0.001). The two-year PFS rates were 90.4% versus 19.6%. PFS benefit favored PLD across most prespecified subgroups. The confirmed objective response rate was 40.4% in the PLD group and 4.3% in the placebo group (p=0.002). Common grade 3 or higher adverse events with PLD included decreased neutrophil count (10.6% of patients), oral mucositis (6.4%), and decreased leukocyte count (4.3%). The authors conclude that PLD significantly prolonged PFS and induced durable responses, with a favorable safety profile, among patients with advanced or refractory DTs.