A phase II trial of Endostar combined with gemcitabine and cisplatin chemotherapy in patients with metastatic nasopharyngeal carcinoma (NCT01612286).

Despite the efficacy of gemcitabine-cisplatin (GC) regimens, the outcome of patients with metastatic nasopharyngeal carcinoma (M NPC) is poor. We conducted a phase II trial to determine the safety and efficacy of Endostar, an endogenous inhibitor of angiogenesis, in combination with GC chemotherapy. A total of 30 patients with M NPC were enrolled. The treatment regimen was a combination of gemcitabine (1,000 mg/m(2)) on days 1 and 8, cisplatin (80 mg/m(2)) on day 1, and Endostar (15 mg/day) from day 1 to day 14 of a 21-day cycle for a maximum of four cycles. The primary endpoint was progression-free survival (PFS). The median follow-up was 13.1 months (range: 2.9-20.7 months). A total of 28 patients were evaluated. The median PFS was 19.4 months (95% CI, 13.6-25.1 months). The 1-year PFS rate was 69.8%. The confirmed objective response rate was 85.7% (95% CI, 66.4-95.3%), including complete response in 14 patients (50%). The 1-year overall survival rate was 90.2%. The most common grade 3/4 adverse events were neutropenia (46.4%) and thrombocytopenia (14.3%). Our results suggest that a combination of Endostar with GC chemotherapy can lead to effective tumor regression, control disease progression, and improve prognosis in M NPC. Therefore, a combined Endostar and GC regimen should be considered as a potential treatment for patients with M NPC.