Pegylated Liposomal Doxorubicin Significantly Extends Progression-Free Survival in Advanced Desmoid Tumors

Key Clinical Takeaways:

• Study Design: This investigator-initiated, double-blind, phase 3 trial compared pegylated liposomal doxorubicin with placebo in patients with advanced or refractory desmoid tumors, allowing crossover upon disease progression.
• Efficacy: Pegylated liposomal doxorubicin significantly improved progression-free survival (PFS) and objective response rate compared with placebo, with a 2-year PFS rate exceeding 90%.
• Safety: Treatment with PLD was associated with manageable toxicity, with low rates of grade ≥ 3 or higher hematologic and mucosal adverse events.

According to results from an investigator-initiated, phase 3 trial, pegylated liposomal doxorubicin significantly prolonged progression-free survival compared with placebo among patients with advanced or refractory desmoid tumors. 

In this double-blind study, 73 patients were randomized 2:1 to receive either 50 mg/m² of intravenous pegylated liposomal doxorubicin (n = 49) or placebo (n = 24) once every 4 weeks for up to 6 cycles. Patients were permitted to crossover from placebo upon disease progression. The primary end point was progression-free survival (PFS). Key secondary end points including objective response and safety.

At a median follow-up of 16.1 months, median PFS was not reached in the pegylated liposomal doxorubicin arm and 4.3 months in the placebo arm (hazard ratio [HR], 0.05; 95% confidence interval [CI], 0.01 to 0.17; P < .001). The 2-year PFS rates were 90.4% and 19.6%, respectively. Confirmed objective response was observed in 19 patients in the pegylated liposomal doxorubicin arm and 1 patient in the placebo arm (P = .002). The most frequently reported grade ≥ 3 adverse events included neutrophil count decrease (10.6%), oral mucositis (6.4%), and white cell decrease (4.3%).

As study authors concluded, “[pegylated liposomal doxorubicin] significantly prolonged PFS and induced [more] durable responses than placebo, with a favorable safety profile, for patients with progressive or symptomatic desmoid tumors.”

Source:

Xu H, Hu J, Zhang Y, et al. Phase 3 trial of pegylated liposomal doxorubicin for patients with advanced and refractory desmoid tumors. Clin Cancer Res. Published online: January 8, 2026. doi: 10.1158/1078-0432.CCR-25-3128