Pegylated Liposomal Doxorubicin Improves Progression-Free Survival in Desmoid Tumors
Clinical Summary:
Design/Population: This phase 3 trial evaluated pegylated liposomal doxorubicin in 73 patients with advanced or refractory desmoid tumors. Patients were randomized to receive PLD or placebo, with crossover allowed upon progression.
Key Outcomes: PLD significantly improved progression-free survival and response rates compared with placebo. Durable disease control was observed across subgroups.
Clinical Relevance: PLD represents an effective treatment option for patients with progressive or symptomatic desmoid tumors. These findings support its use in a setting with limited therapeutic options.
Results from a phase 3 trial demonstrated that pegylated liposomal doxorubicin (PLD) significantly improves progression-free survival (PFS) compared with placebo among patients with advanced or refractory desmoid tumors.
“The clinical course of [desmoid tumors] is highly variable and often unpredictable, which has led to considerable controversy about its optimal management strategy, especially for patients with progressive or highly symptomatic disease,” stated Huaiyuan Xu, MD, Sun Yat-Sen University Cancer Center, Guangzhou, China, and coauthors. “[PLD] is a modified anthracycline that [has been shown to] reduce cardiotoxicity while preserving antitumor efficacy.”
In this double-blind trial, 73 patients with advanced or refractory desmoid tumors were randomized 2:1 to receive either 50 mg/m2 of PLD (n = 49) or placebo (n = 24) once every 4 weeks for up to 6 cycles. Crossover from the placebo arm was permitted upon disease progression. The primary end point was PFS. Key secondary end points included objective response and safety.
At a median follow-up of 16.1 months, median PFS was not reached in the PLD 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). The 2-year PFS rates were 90.4% and 19.6%, respectively. Confirmed objective response was observed in 19 patients in the PLD arm and 1 patient in the placebo arm (P = .002).
The most common grade ≥3 adverse events with PLD included neutrophil count decrease (10.6%), oral mucositis (6.4%), and white cell decrease (4.3%). Significant between-group differences in certain patient-reported toxicities were observed (P <.05).
“Results demonstrated significant improvement in PFS with PLD over placebo,” concluded Dr Xu et al. “PLD could be added to the available options to be discussed with patients with progressive or symptomatic DTs according to their single and unique situation.”
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 Can Res. Published online: January 23, 2026. doi:10.1158/1078-0432.CCR-25-3128
