Abstract

Background: Sarcoma constitutes a heterogeneous group of rare solid tumors and has no standard second-line treatment. Anti-angiogenesis tyrosine kinase inhibitors have shown promising efficacy for advanced sarcoma after failure of first-line chemotherapy. Apatinib is a highly selective vascular endothelial growth factor receptor 2 (VEGFR-2) antagonist from China. In the present study, we retrospectively assessed apatinib for safety and activity in advanced sarcoma patients from our cancer center. Methods: Patients with advanced sarcomas treated with apatinib 250-500mg daily between August 2015 and February 2018 were retrospectively analyzed. Objective response was determined according to RECIST 1.1 criteria, progression-free survival (PFS) was estimated by the Kaplan-Meier curves and safety profile was analyzed. Results: In total, 52 patients were treated with apatinib and 37 patients (23 males and 14 females) were included for analysis, including 7 alveolar soft part sarcoma (ASPS), 5 PNET/Ewing’s sarcoma, 4 osteosarcoma, 4 chondrosarcoma, 3 synovial sarcoma, 4 undifferentiated pleomorphic sarcoma, 4 leiomyosarcoma, 2 angiosarcoma,1 rhabdomyosarcoma, 1 chordoma, 1dedifferentiated liposarcoma and 1 clear cell sarcoma. 5 (13.5%) patients received apatinib as first-line treatment, 17 (45.9%) and 15 (40.5%) were treated with apatinib as second line or later therapy respectively.18 (48.6%) achieved tumor regression, 1 patient with ASPS had complete response, 8 (21.6%) experienced partial response, and disease was stable in 21 (56.8%) patients. The disease control rate was 100% in 5 patients with ASPS. The median PFS for all patients was 12 months, and median PFS of patients except ASPS was 5.9 m. The most frequent treatment-related adverse events included hypothyroidism [11 (29.7%)], Proteinuria [8 (21.6%)], hypertension [11 (29.7%)), hand-foot syndrome [19 (51.4%)], diarrhea [9 (24.3%)], fatigue [5 (13.5%)], hemorrhage [4 (10.8%)], anorexia [5 (13.5%)], oral ulcer [3 (5.4%)], rash [2 (8.1%)], bleaching hair [2 (5.4%)] and aerothorax [2 (5.4%)]. Conclusions: Apatinib may be effective and tolerable in advanced sarcoma, especially in ASPS. Legal entity responsible for the study: Union Hospital, Tongji Medical College, Huazhong University of Science and Technology. Funding: Clinical Research Physician Program of Tongji Medical College, HUST. Disclosure: All authors have declared no conflicts of interest.

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