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.
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.
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%)].
Apatinib may be effective and tolerable in advanced sarcoma, especially in ASPS.
Clinical trial identification
Legal entity responsible for the study
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology.
Clinical Research Physician Program of Tongji Medical College, HUST.
All authors have declared no conflicts of interest.