Efficacy and safety of apatinib in recurrent/metastatic nasopharyngeal carcinoma: A pilot study.

Abstract Background There is no standard-of-care for recurrent, metastatic nasopharyngeal carcinoma (rmNPC) after first-line chemotherapy. Here, we report the efficacy and safety data of apatinib in rmNPC patients. Methods Thirty-five biopsy-proven rmNPC patients received apatinib at 500 mg/day under a compassionate access programme. Primary end-point was objective response rate (ORR; RECIST v1.1). Kaplan-meier method was used to estimate progression-free survival (PFS) and overall survival (OS). Toxicity was assessed by CTCAE v4.0. Results 82.9% (29 of 35) of patients had poly-metastatic rmNPC. All patients, except five, were platinum-refractory; 37.1% (13 of 35) received ≥ 2 lines. Median number of apatinib cycles was 4.0 (IQR: 2.0–8.0). ORR was 31.4% (11 of 35 [95% CI: 16.9–49.3]) and disease control rate was 74.3% (26 of 35 [95% CI: 56.7–87.5]); 11 (31.4%) and 4 (11.4%) patients demonstrated response for ≥ 6 and ≥ 12 months, respectively. Median PFS and OS was 3.9 (95% CI: 3.1–5.5) months and 5.8 (95% CI: 4.5–8.0) months, respectively. Among the ≥ 12-month responders, all patients had pre-apatinib EBV DNA titer of  Conclusion Our data suggests potential efficacy of apatinib in rmNPC patients. Although incidence of severe toxicities was low, dose modification was required in 42.9% of patients.