Trabectedin in the treatment of soft tissue sarcoma: Real-world data on effectiveness, safety, and financial implications from a European comprehensive cancer center

Abstract

Soft-tissue sarcomas (STS) comprise over 150 histological subtypes, with advanced cases showing poor prognosis (5-year survival <10%). Trabectedin, a synthetic alkaloid, is frequently used after anthracycline-based chemotherapy failure. Despite the withdrawal of reimbursement in France in 2016 due to debated efficacy and safety, it remains in clinical use, imposing financial strain on hospitals. This retrospective single-center study evaluated trabectedin's efficacy, safety, and cost in 68 patients treated between 2019 and 2023. L-sarcomas accounted for 78% of cases, including uterine leiomyosarcomas (n = 16), soft-tissue leiomyosarcomas (n = 17), and myxoid liposarcomas (n = 8). Non-L-sarcomas (22%) included mostly synovial sarcomas. The overall disease control rate was 71%, with a median progression-free survival (PFS) of 4.1 months, and an overall survival of 12.3 months. Subtype-specific median PFS was 6.8 months for liposarcomas (11.3 for myxoid vs. 4.5 for other subtypes), 3.1 months for leiomyosarcomas (3.4 months for uterine vs. 3.1 for soft-tissue), and 2.4 months for non-L-sarcomas. Patients received a median of 5 cycles (range: 1-38), with an average total dose of 16 mg [2-81], and an average hospital cost of €9900. Adverse events occurred in 91%, mainly hematological; cardiac toxicity was seen in 9%. This retrospective single-center study in a limited cohort provides real-world insights but should be interpreted with caution. Despite limited reimbursement, trabectedin may retain clinical utility, particularly in L-sarcoma management.

Keywords: drug‐related side effects and adverse events; health care economics and organizations; sarcoma; trabectedin; treatment outcome.