日本におけるマスターキープロジェクトにおける、高分化型肉腫を含む進行性または転移性肉腫患者のリアルワールド特性評価

Efficacy of available treatments for liposarcoma (LPS), including dedifferentiated liposarcoma (DDLPS) is limited, and detailed real-world clinical data for patients with LPS treated in Japan are scarce. We used the MASTER KEY Registry Database to evaluate the clinical characteristics and outcomes of patients with advanced/metastatic LPS treated with systemic antineoplastic therapy in Japan.

This non-interventional/observational cohort study included patients with LPS, including DDLPS, and well-differentiated LPS (WDLPS), and a history of systemic antineoplastic therapy, enrolled in the MASTER KEY Project Registry Database from 1 May 2017 to 31 December 2022. Outcomes were analysed by type of treatment. Primary outcomes: progression-free survival (PFS); overall survival (OS). Secondary outcomes: overall response rate; disease control rate. Sensitivity analyses (prospective registry subgroup) were performed to minimize immortal time bias.

Since 2017, 100 patients with LPS were prospectively enrolled in the database; of these, 62 had DDLPS and 11 had WDLPS. In patients with LPS, median PFS was 11.1 months after first-line (1 L) pharmacotherapy and 6.9 months after 1 L doxorubicin. In patients with DDLPS, median PFS was 6.9 months after 1 L pharmacotherapy and 4.4 months after 1 L doxorubicin. Median OS after 1 L pharmacotherapy was 45.8 months in patients with LPS, and 40.7 months in those with DDLPS. Median OS in the sensitivity analysis was not reached for patients with LPS and was 13.9 months for the DDLPS subgroup.

These data investigating DDLPS in Japanese patients highlight the poor outcomes and lack of effective treatment options in the real-world clinical practice setting.