An open, multicenter, exploratory study of apatinib mesylate maintenance therapy for recurrent/metastatic head and neck squamous cell carcinoma (ChiCTR1800019375)

Background: This study evaluated the efficacy of apatinib in maintenance therapy in patients with recurrent/metastatic head and neck squamous cell carcinoma (R/M HNSCC).Methods: Twenty-six patients from three centers were enrolled from November 2018 to September 2021. These patients received 2 weeks apatinib, administered at 250 mg qd. Then apatinib dose may be administered to 500 mg qd continuous in 4 weeks cycle if no patients experienced adverse reaction. Enrolled patients can receive a combination of radiotherapy or chemotherapy. The primary endpoints were progression-free survival (PFS), and secondary endpoints included overall survival (OS), disease control rate (DCR), objective response rate (ORR), quality of life (QOL) score, and adverse drug reactions.Results: Median PFS of all patients was 3.2 months (95% CI: 2.06-4.33). Median OS of all patients was 7.3 months (95% CI: 2.14-12.46). The DCR was 92.3%. The ORR was 30.8%. In univariate analysis, the results showed that ECOG score 0-1 (HR = 0.31, p = 0.006) and treated with apatinib for more than 60 days (HR = 0.31, p = 0.003) were independent prognostic indicators affecting PFS, and ECOG score 0-1 (HR = 0.40, p = 0.027) and moderately differentiated or highly differentiated (HR = 0.38, p = 0.048) were independent prognostic indicators of OS. The most common adverse events among treated subjects included hypertension (46.1%), fatigue (42.3%), and hand-foot syndrome (23.1%). There were only two cases (7.7%) of Grade III or above adverse reactions.Conclusions: Maintenance therapy with apatinib is an effective and well-tolerated regimen in patients with R/M HNSCC.