LAUSR

e17502 Background: To evaluate the efficacy and safety of apatinib in treating patients with platinum-resistant or platinum-refractory recurrent or metastatic nasopharyngeal carcinoma. Methods: In this phase 2, single-arm, prospective study, we recruited patients aged 18–65 years with platinum-resistant or platinum-refractory recurrent /metastatic nasopharyngeal carcinoma. Patients were treated with apatinib at an initial dose of 500 mg once daily and continued until disease progression, patient withdrawal, or unacceptable toxic effects. The primary endpoint was clinical benefit rate (CBR) and toxicity. Secondary endpoints included progression-free survival (PFS) at 3 months and overall survival (OS). We used Simon’s two-stage design, and analysed efficacy and toxicity in the intention-to-treat and per-protocol populations. This study is registered with ClinicalTrials.gov, number NCT03213587. Results: Between Aug 5,2017 and Oct 13,2018, we enrolled 16 patients. Until the final follow-up (Jan 14, 2019), the CBR (complete response + partial response+ stable disease) was 69.2% (9/13) in the per-protocol population. Median PFS and 3-month PFS rate were 3.70 (95% CI, 0-8.771) months and 67.1%, respectively. Median OS and 1-year OS rate were 12.9 (95% CI 5.56-20.23) months and 47.9%, respectively. The most common grade 3-4 adverse events were neutropenia (1[6.25%]), hand-foot syndrome (2[12.5%]), albuminuria (2[12.5%]), hypertension (1[6.25%]), hyponatremia (1[6.25%]), artery dissection (1[6.25%]) and nasopharyngeal hemorrhage (2[12.5%]) in the intention-to-treat population. Serious adverse event was reported in one patient who died of nasopharyngeal hemorrhage. Conclusions: Apatinib achieved excellent disease control in platinum-resistant or platinum-refractory recurrent or metastatic nasopharyngeal carcinoma. More attention needs to be paid to toxicity management. Clinical trial information: NCT03213587.