Open Access

Viktor Grünwald, Daniel Pink, Gerlinde Egerer, Enrico Schalk, Marinela Augustin, Christoph K. W. Deinzer, Viola Kob, Dietmar Reichert, Maxim Kebenko, Stephan Brandl, Dennis Hahn, Lars H. Lindner, Mathias Hoiczyk, Uta Ringsdorf, Lars C. Hanker, Dirk Hempel, Beatriz De Rivas, Tobias Wismann, Philipp Ivanyi

• Simple Summary Active therapeutic options in advanced soft tissue sarcoma (STS), able to induce durable objective responses, are limited beyond first-line chemotherapy. Although results obtained in clinical trials suggest there is a high probability for patients with STS to benefit from treatment with trabectedin (Yondelis®), there is still a paucity of robust real-life data in more diverse patient populations. The prospective, non-interventional phase IV YON-SAR trial (NCT02367924) was designed to evaluate treatment effects of trabectedin in patients with advanced STS in real-life clinical practice across Germany. The efficacy results of this trial, conducted in 128 patients from 19 sites across Germany, further support trabectedin as a standard of care for a second- or further-line treatment of patients with advanced STS in routine clinical practice (median progression-free survival: 5.2 months; median overall survival: 15.2 months). The safety profile of trabectedin was manageable and in line with those observed in previous studies. Abstract This non-interventional, prospective phase IV trial evaluated trabectedin in patients with soft tissue sarcoma (STS) in real-life clinical practice across Germany. The primary endpoints were progression-free survival (PFS) rates at 3 and 6 months, as defined by investigators. Overall, 128 patients from 19 German sites were evaluated for efficacy and 130 for safety. Median age was 58.5 years (range: 23–84) and leiomyosarcoma was the most frequent histotype (n = 45; 35.2%). Trabectedin was mostly used as second/third-line treatment (n = 91; 71.1%). Median PFS was 5.2 months (95% CI: 3.3–6.7), with 60.7% and 44.5% of patients free from progression at 3 and 6 months, respectively. Median overall survival was 15.2 months (95% CI: 9.6–21.4). One patient achieved a complete and 14 patients a partial response, conferring an objective response rate of 11.7%. Decreases in white blood cells (27.0% of patients), platelets (16.2%) and neutrophils (13.1%) and increased alanine aminotransferase (10.8%) were the most common trabectedin-related grade 3/4 adverse drug reactions. Two deaths due to pneumonia and sepsis were considered trabectedin-related. Trabectedin confers clinically meaningful activity in patients with multiple STS histotypes, comparable to that previously observed in clinical trials and other non-interventional studies, and with a manageable safety profile.