Background Apatinib is a small tyrosine kinase inhibitor targeting the vascular endogenous growth receptor 2 (VEGFR-2) that shows potent anti-tumor activities in various advanced cancers via the inhibition of neo-angiogenesis. The effect of apatinib in recurrent or metastatic head and neck cancers is not fully demonstrated. Methods Patients with recurrent or metastatic head and neck cancers consecutively treated in our institute by apatinib from January 2015 to June 2022 were enrolled. Daily 250 mg or 500 mg apatinib was given with or without chemotherapy according to patients' tolerance. Disease response, treatment-related side effects and patient survival were analyzed. Kaplan-Meier analysis was used to estimate patients' overall survival. The R software (version 4.1.3) were used for statistical analysis. Results A total of 85 patients (median [range] age, 53 [23-79] years; 68 male [80.0%]) were included for analysis. The median follow-up time was 30.4 (95% CI: 23.95- 40.70) months. By the end of the last follow-up, 43 (50.6%) patients died of disease progression. The median OS was 29.6 (95% CI: 12.39- 46.75) months, and the median PFS was 10.4 (95% CI: 6.11- 14.63) months. The ORR was 21.2%, and the DCR was 70.6%. The side effects were manageable and no treatment-associated death occurred. Multivariate analysis showed that OS was significantly associated with cancer pathology (squamous cell carcinoma vs. adenoid cystic carcinoma, HR=6.42, 95%CI:1.50-27.39, p = 0.0121; other types (adenocarcinoma, sarcoma, melanoma) vs. adenoid cystic carcinoma, HR=3.58, 95%CI:1.02-12.52, p = 0.0459). Conclusion Apatinib showed promising anti-tumor activities in recurrent or metastatic head and neck cancers. The side effects were manageable. The effect of apatinib in recurrent or metastatic head and neck cancers warrants further verifications in larger-scale randomized studies.
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