Toripalimab plus anlotinib in patients with recurrent or metastatic nasopharyngeal carcinoma: A multicenter, single-arm phase 2 trial (TORAL)
Yuchen Zhang, Qihua Zou, Baitian Zhao, Ning Su, Zhihua Li, Xicheng Wang, Panpan Liu, Xiaopeng Tian, Xiaojie Fang, Jun Cai, Lirong Li, Yingxian Liu, Yi Xia, Qingqing Cai
Abstract
Treatment options for patients with recurrent or metastatic nasopharyngeal carcinoma (RM-NPC) after failure of platinum-based therapy are limited. In this phase 2 trial, 40 patients with RM-NPC who failed platinum-based chemotherapy receive toripalimab plus anlotinib regimen. The objective response rate is 37.5%, and the disease control rate is 85.0%. With a median follow-up of 17.4 months, the median progression-free survival (PFS) is 9.5 months and 1-year overall survival rate is 73.3%. The most common treatment-related grade 3–4 adverse events are hand-foot syndrome (22.5%) and oral mucositis (17.5%). Analyses of plasma circulating tumor DNA (ctDNA) demonstrate that the blood tumor mutation burden at cycle 1/2 is associated with response and PFS, and disease progression indicated by ctDNA precedes radiological progression by a median of 2.3 months. In conclusion, toripalimab plus anlotinib is well tolerated and shows promising efficacy in patients with RM-NPC, and ctDNA could be a potential predictive biomarker. The trial is registered at ClinicalTrials.gov (NCT04996758). • Toripalimab plus anlotinib shows promising efficacy in RM-NPC • The regimen is well tolerated with an acceptable safety profile • Plasma ctDNA is associated with treatment response and survival outcome • Dynamic change of ctDNA may be a potential predictive marker for disease progression Zhang et al. report the promising efficacy and acceptable safety profile of toripalimab plus anlotinib for patients with recurrent or metastatic nasopharyngeal carcinoma and demonstrate the potential predictive role of plasma circulating tumor DNA.