Abstrakt

Targeted Treatment of Soft-Tissue Sarcoma

Anne Iren Riskjell1, Vivi-Nelli Mäkinin1, Birgitte Sandfeld Paulsen2, Ninna Aggerholm-Pedersen

Background: Soft-Tissue Sarcoma (STS) is a heterogeneous group of sarcomas with a low incidence. The treatment of advanced disease is poor, and the mortality is high. In other types of cancer, targeted treatment has shown promising results. Therefore, we aimed to generate an overview of the clinical experiences with targeted treatments based on a pre-specified target in patients with STS.

Methods: A systematic literature search was conducted in PubMed and Embase databases. The programs ENDNOTE and COVIDENCE were used for data management. The literature was screened to assess the article’s eligibility for inclusion. A total of 31 articles were included in this review.

Results: Twenty-eight targeted agents were used in the treatment of 80 patients with advanced STS and a known pre-specified genetic alteration. MDM2-inhibitors were the most studied drug (n=19), followed by crizotinib (n=9), ceritinib (n=8) and 90Y-OTSA (n=8). All patients treated with MDM2 inhibitor achieved a treatment response of Stable Disease (SD) or better with treatment duration of 4 to 83 months. For the remaining drugs, a more mixed response was observed. The evidence is low since most studies were case reports or cohort studies, where only a few STS patients were included.

Conclusion: Many targeted agents are available that can precisely target specific genetic alterations in advanced STS. The MDM2 inhibitor has shown promising results and must be considered in patients with MDM2 amplification; however, further investigation is needed to identify the potential survival effect of targeted treatment in sarcoma.