Phase 2 trial of neoadjuvant toripalimab plus neoadjuvant chemoradiotherapy for locally advanced esophageal squamous cell carcinoma

The standard treatment for locally advanced esophageal squamous cell carcinoma (LA ESCC) is neoadjuvant chemoradiotherapy (nCRT) followed by esophagectomy; however, 40% of patients experience disease recurrence or death within 5 years of this therapy. The phase 2 NEOCRTEC1901 trial at Sun Yat-sen University (Guangzhou, China) evaluated the neoadjuvant combination of toripalimab (anti-PD-1) and nCRT for LA ESCC. 50% of patients achieved pCR and 5% were ypT0N+. The R0 resection rate was 98%. 20% of patients had grade 3 or 4 adverse events. One patient (2%) died postoperatively due to tracheal fistula. The 2-year OS rate was 81.5%. Compared to a control cohort receiving nCRT only at the same institution, the pCR rate of the study group was higher but without significant difference (50% versus 36%; p=0.19). Nevertheless, the authors conclude that considering the signs of efficacy and acceptable safety, evalation of neoadjuvant toripalimab plus nCRT in a phase 3 randomized trial might be warranted.