Efficacy and safety of apatinib in advanced sarcoma: an open-label, nonrandomized, single-center study of 45 patients.

Sarcoma is a rare tumor with more than 50 histologic subtypes. Patients with advanced sarcoma have a poor prognosis. The aim of this study was to evaluate the efficacy and safety of apatinib, an oral vascular endothelial growth factor receptor-2 inhibitor, as salvage treatment for advanced bone and soft tissue sarcomas. From May 2017 to July 2018, a prospective, open-label, nonrandomized, clinical trial of apatinib was carried out in selected patients with advanced sarcoma. After apatinib dosing, progression-free survival (PFS), overall survival (OS), objective response rate, disease control rate, and treatment-related adverse events (AEs) were reviewed and evaluated. Patients were administered apatinib for at least 1 month. Median follow-up time was 6.00 months (1-13 months). The median PFS was 7.88 months, with the longest PFS of 13 months observed in a patient with epithelial sarcoma. The 3-month PFS rate was 66.44%. The median OS was 11.64 months with significant differences observed based on disease subtypes. Four patients achieved a partial response, and 36 patients achieved stable disease. The objective response rate was 8.88% (4/45), and the disease control rate was 88.89% (40/45). The most common grade 3/4 treatment-related AEs were hypertension (12.50%), hand-foot syndrome (6.67%), diarrhea (12.50%), fatigue (6.25%), and proteinuria (14.29%). One drug-related severe AE of thrombocytopenia (21×10/l) occurred 2 months after therapy. Apatinib treatment in our study exhibited objective efficacy in PFS, OS, and manageable toxicity in patients with advanced sarcoma. This result supports future randomized controlled trials to further define apatinib activity in stage IV sarcomas.