Efficacy and safety of vascular-targeting agents plus chemotherapy versus chemotherapy alone in recurrent or metastatic nasopharyngeal carcinoma patients：A multicenter observational clinical trial

Abstract Background: Platinum-based chemotherapy is important for the treatment for recurrent or metastatic nasopharyngeal carcinomas (RM-NPCs). However, the prognosis of RM-NPC patients remains poor. This study evaluated the efficacy and safety of vascular-targeting agentsplus chemotherapy for RM-NPC treatment. Methods: This observational clinical trial, conducted at seven hospitals in Guangxi between January 2017 and December 2021, included 198 patients (158 males [79.8%]; median age, 51 years [IQR, 45–57years]). Chemotherapy was administered with and without vascular-targeting agents in 66 and 132 patients, respectively. The primary endpoint was progression-free survival (PFS), assessed by a blinded independent review committee according to RECIST v.1.1. The secondary end points were overall survival (OS) and safety. Results: After a median follow-up of 20 months, 31 (47.0%) and 82 (62.1%) deaths occurred in the combination therapy and chemotherapy alone groups, respectively. Median PFS was significantly higher in the combination group (7.0 months [95% CI, 5.7–8.3 months]) than in the chemotherapy alone group (5.0 months [95% CI, 4.1–5.9 months], P = 0.034), with a hazard ratio of 0.73 (95% CI, 0.54–0.98). The combination group exhibited a higher median OS compared to the chemotherapy alone group (37 months [95% CI, 21.3–52.7] vs. 20 months [95% CI, 13.0–27.0months], P = 0.047), representing a hazard ratio of 0.66 (95% CI, 0.45–0.97). In the combination therapy group, the most common adverse events were hypertension (n = 10/66 [15.1%]). Conclusion: The addition of vascular-targeting agents to chemotherapy for RM-NPC patients provided superior efficacy compared to chemotherapy alone, along with a manageable safety profile.