之前的研究表明，口服酪氨酸激酶抑制剂阿帕替尼单药治疗复发性或转移性鼻咽癌（RM-NPC）患者的疗效有希望。本研究旨在评估阿帕替尼联合卡培他滨作为第二线或以上治疗失败一线铂类化疗的RM-NPC患者的疗效和安全性。在这个单臂Ⅱ期研究中，我们招募了RM-NPC患者，根据实体瘤反应评估标准（RECIST v1.1），这些患者至少有一个可测量的病灶。样本量采用Simon的二阶段设计确定。所有患者在每个21天的周期的第1-14天，口服阿帕替尼500mg一次，卡培他滨1000mg/m2两次。主要终点是客观缓解率（ORR），次要终点包括疾病控制率（DCR）、缓解持续时间（DoR）、无进展生存期（PFS）、总生存期（OS）和安全性。我们从2018年9月到2020年8月招募了64名患者。ORR和DCR分别为39.1％（95％ CI，27.1-52.1）和85.9％（95％CI，75.0-93.4）。中位DoR为14.4个月（95％CI，7.8-21.0）。截至2021年4月20日，中位随访时间为12.0个月。中位PFS为7.5个月（95％CI，5.0-10.0），中位OS为15.7个月（95％CI，11.3-20.1）。任何等级的最常见毒副作用是贫血（75.0％），手足综合征（65.6％）和蛋白尿（64.0％）。36名（56.3％）患者观察到3-4级的毒副作用，其中最常见的是高血压（14.1％），口腔炎（12.4％）和疲劳（10.9％）。阿帕替尼加卡培他滨在预处理铂类耐药性RM-NPC患者中作为第二线治疗选择具有良好疗效。考虑到毒副作用，需要进一步研究该联合用药的剂量选择。Chi-CTR1800017229.©2023。作者

Previous studies have shown that monotherapy with apatinib, an oral tyrosine kinase inhibitor, has promising efficacy for treating recurrent or metastatic (RM) nasopharyngeal carcinoma (NPC) patients. In this study, we aimed to assess the efficacy and safety of apatinib combined with capecitabine as a second-line therapy or beyond for treating RM-NPC patients who failed the first-line platinum-based chemotherapy.In this single-arm, phase II study, we enrolled RM-NPC patients who had at least one measurable lesion according to the Response Evaluation Criteria in Solid Tumors (RECIST v1.1). The sample size was determined using Simon's two-stage design. All patients were administered with apatinib 500 mg once daily and capecitabine 1000 mg/m2 twice per day on days 1-14 of each 21-day cycle. The primary endpoint was the objective response rate (ORR), and the secondary endpoints comprised disease control rate (DCR), duration of response (DoR), progression-free survival (PFS), overall survival (OS), and safety.We enrolled 64 patients from September 2018 to August 2020. The ORR and DCR were 39.1% (95% CI, 27.1-52.1) and 85.9% (95% CI, 75.0-93.4), respectively. The median DoR was 14.4 months (95% CI, 7.8-21.0). As of April 20, 2021, the median follow-up duration was 12.0 months. The median PFS was 7.5 months (95% CI, 5.0-10.0) and the median OS was 15.7 months (95% CI, 11.3-20.1). The most common toxicities of any grade were anemia (75.0%), hand-foot syndrome (65.6%), and proteinuria (64.0%). Grade 3-4 toxicities were observed in 36 (56.3%) patients, with hypertension (14.1%), mucositis (12.4%), and fatigue (10.9%) most commonly observed.Apatinib plus capecitabine shows promising efficacy as a second-line treatment option in pretreated platinum-refractory RM-NPC patients. Dose selection of this combination needs further investigation considering the toxicity.Chi-CTR1800017229.© 2023. The Author(s).