Abstract

Soft tissue sarcoma (STS) comprises a large variety of rare malignant tumors. Development of distant metastasis is frequent, even in patients undergoing initial curative surgery. Trabectedin, a tetrahydroisoquinoline alkaloid isolated from the Caribbean marine tunicate Ecteinascidia turbinata, was approved in 2007 for patients with advanced STS after failure of anthracyclines and ifosfamide, or for patients unsuited to receive these agents. In this study, we retrospectively analyzed 25 patients who had been treated with trabectedin at our institution between 2007 and 2010. The majority (72%) had been heavily pre-treated with ≥2 previous lines of chemotherapy. Response assessed by conventional RECIST criteria was low, with only one patient achieving a partial remission (PR) and 10 stable disease (SD) after three cycles of treatment. However, median progression-free survival (PFS) and overall survival (OS) were significantly prolonged in this population compared to non-responders, with 7.7 months versus 2.1 months (p < 0.0001; HR 15.37, 95% CI 4.3 to 54.5) and 12.13 months versus 5.54 months (p = 0.0137; HR 3.7, 95% CI 1.3 to 10.5), respectively. PFS for all patients was 58% at three months and 37% at six months. Side effects, including neutropenia, elevation of liver transaminases/liver function tests, and nausea/vomiting, were usually mild and manageable. However, dose reductions due to side effects were necessary in five patients. We conclude that trabectedin is an effective and generally well tolerated treatment for STS even in a heavily pre-treated patient population.

Highlights

  • Soft tissue sarcoma (STS) are a heterogeneous group of rare malignant tumors, predominantly arising from the embryonic mesoderm

  • Development of distant metastases remains the major problem in up to 50% of all STS patients [2]. Prognosis for those individuals with advanced and unresectable disease is grim with a median survival of eight to 13 months from the start of first-line chemotherapy, usually doxorubicin and/or ifosfamid based regimens [3]

  • With STS were treated at our institution

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Summary

Introduction

Soft tissue sarcoma (STS) are a heterogeneous group of rare malignant tumors, predominantly arising from the embryonic mesoderm. 3,680 deaths due to STS in the United States [1]. STS require a multidisciplinary approach in a specialized center involving oncologic surgeons, sarcoma pathologists, radiation therapists and medical oncologists. Development of distant metastases remains the major problem in up to 50% of all STS patients [2]. Prognosis for those individuals with advanced and unresectable disease is grim with a median survival of eight to 13 months from the start of first-line chemotherapy, usually doxorubicin and/or ifosfamid based regimens [3].

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