Pegylated Liposomal Doxorubicin Improves Progression-Free Survival in Patients With Progressive Desmoid Tumors
Clinical Summary:
- Design/Population: This randomized, double-blind, phase 3 trial evaluated pegylated liposomal doxorubicin in patients with progressive or symptomatic advanced/refractory desmoid tumors.
- Key Outcomes: Pegylated liposomal doxorubicin significantly improved progression-free survival and objective response compared with placebo while demonstrating a manageable safety profile.
- Clinical Relevance: These findings support pegylated liposomal doxorubicin as an effective systemic treatment option for patients with progressive or symptomatic desmoid tumors.
Results from a phase 3 study demonstrated that pegylated liposomal doxorubicin significantly improved progression-free survival (PFS) and objective response rate (ORR) compared with placebo among patients with progressive or symptomatic desmoid tumors.
“Although not malignant, [desmoid tumors] are often locally aggressive and may present with severe functional impairments, disfigurement, and pain, resulting in a substantial clinical burden,” stated Huaiyuan Xu, MD, Sun Yat-Sen University Cancer Center, Guangzhou, China, and coauthors. “Even after complete surgical resection, the risk of recurrence remains high, presenting a considerable challenge for disease management.”
In this double-blind, placebo-controlled trial, 70 patients with symptomatic, unresectable, or potentially disabling disease who experienced radiographic progression within 6 months of trial enrollment were randomized 2:1 to receive either 50 mg/m² of pegylated liposomal doxorubicin (n = 49) or placebo (n = 24) every 4 weeks for up to 6 cycles. Patients were permitted to crossover from the placebo arm upon disease progression. The primary end point was investigator-assessed PFS. Key secondary end points included ORR and safety.
At a median follow-up of 16.1 months, median PFS was not estimable in the pegylated liposomal doxorubicin arm and was 4.3 months in the placebo arm (hazard ratio [HR], 0.05; 95% confidence interval [CI], 0.01 to 0.17; P <.001). Estimated 1-year PFS rates were 95.7% and 19.6%, with estimated 2-year PFS rates of 90.4% and 19.6%, respectively. The confirmed ORR was was 40.4% in the pegylated liposomal doxorubicin arm and 4.3% in the placebo arm (P = .002). Most responders in the pegylated liposomal doxorubicin group continued to have an ongoing response at data cutoff.
Any-grade adverse events were reported in 91.5% of patients in the pegylated liposomal doxorubicin arm and 36.4% of patients in the placebo arm, with grade ≥3 events reported in 14.9% of patients in the pegylated liposomal doxorubicin arm. The most frequently reported grade ≥ 3 adverse events included neutrophil count decrease (10.6%), oral mucositis (6.4%), and white cell count decrease (4.3%). Two patients required dose reductions in the pegylated liposomal doxorubicin arm due to adverse events. No adverse events led to treatment discontinuation in either group.
“Given its efficacy, safety, and accessibility, [pegylated liposomal doxorubicin] could be considered a preferred treatment option with a favorable benefit/risk balance for patients with advanced [desmoid tumors],” concluded Dr Xu and coauthors.
Source:
Xu H, Hu J, Zhang Y, et al. Phase III trial of pegylated liposomal doxorubicin for patients with advanced and refractory desmoid tumors. Clin Cancer Res. Published online June 1, 2026. doi:10.1158/1078-0432.CCR-25-3128
