A Multicenter, Prospective, Observational Study to Assess the Clinical Activity and Impact on Symptom Burden and Patients’ Quality of Life in Patients with Advanced Soft Tissue Sarcomas Treated with Trabectedin in a Real-World Setting in Greece

SIMPLE SUMMARY: Soft tissue sarcomas (STS) constitute a group of heterogeneous tumors. For patients with advanced or metastatic disease, prognosis is poor and only a few treatments are available, including trabectedin. The aim of our prospective multicenter study was to evaluate the real-world activity of trabectedin, and its impact on symptom burden and quality of life in patients with advanced STS treated in routine clinical settings in Greece. Between 21 December 2015 and 6 June 2018, 64 eligible patients from 13 Greek centers were evaluated. Our study provides real-world evidence on the effectiveness, tolerability and safety of trabectedin in a population of patients with advanced STS of multiple histological subgroups who have either experienced a relapse or disease progression after standard-of-care front-line therapy, or were unsuited to receive front-line agents. ABSTRACT: This non-interventional, multicenter, prospective study aimed to evaluate the real-world activity of trabectedin, and its impact on symptom burden and quality of life in patients with advanced soft tissue sarcoma (aSTS) treated in routine clinical settings in Greece. Patients with histologically confirmed aSTS newly initiated on trabectedin were enrolled. The primary endpoint was progression-free survival (PFS) rate at 6 months. Secondary endpoints included PFS rate at 3 months, median PFS, objective response rate (ORR), disease control rate (DCR), overall survival (OS), and an assessment of the impact of treatment on health-related quality of life (HRQoL), cancer-related symptom burden and symptom interference with function, as well as all-cause treatment discontinuation rate. A total of 64 eligible patients from 13 Greek centers were evaluated. Patients received a median of three trabectedin cycles per patient (interquartile range [IQR]: 2.0–6.0). Median PFS was 6.6 months with 67.9% and 51.2% of patients free from progression at 3 and 6 months, respectively. ORR was 7.8% and DCR 21.9%. Median OS was 13.1 months. No significant changes from enrolment were noted in HRQoL scores. In total, 30 patients (46.9%) had at least one trabectedin-related adverse drug reaction (ADR) and 9 (14.1%) at least one serious ADR. The treatment discontinuation rate due to toxicity was 9.4%. These results suggest that trabectedin is an active treatment with clinically meaningful benefits in patients with aSTS with no new safety signals.