Neoadjuvant Nivolumab or Nivolumab Plus Ipilimumab in Untreated Oral Cavity Squamous Cell Carcinoma

作者：Jonathan Daniel Schoenfeld, Glenn J. Hanna, Vickie Y. Jo, Bhupendra Rawal, Yu‐Hui Chen, Paul Catalano, Ana Lako, Zoe B. Ciantra, Jason L. Weirather, Shana Criscitiello, Adrienne Luoma, Nicole Grace Chau, Jochen H. Lorch, Jason I. Kass, Donald J. Annino, Laura A. Goguen, Anupam M. Desai, Brendan Ross, Hina Shah, Heather A. Jacene, Danielle Nina Margalit, Roy B. Tishler, Kai W. Wucherpfennig, Scott J. Rodig, Ravindra Uppaluri, Robert I. Haddad · 发表于：JAMA Oncology · 年份：2020 · DOI：10.1001/jamaoncol.2020.2955 · 被引用次数：357 · 研究领域：Cancer Immunotherapy and Biomarkers、Head and Neck Cancer Studies、Colorectal and Anal Carcinomas

Importance: Novel approaches are needed to improve outcomes in patients with squamous cell carcinoma of the oral cavity. Neoadjuvant immunotherapy given prior to surgery and combining programmed cell death protein 1 (PD-1) and cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) immune checkpoint inhibitors are 2 strategies to enhance antitumor immune responses that could be of benefit. Design, Setting, and Participants: In this randomized phase 2 clinical trial conducted at 1 academic center, 29 patients with untreated squamous cell carcinoma of the oral cavity (≥T2, or clinically node positive) were enrolled between 2016 to 2019. Interventions: Treatment was administered with nivolumab, 3 mg/kg, weeks 1 and 3, or nivolumab and ipilimumab (ipilimumab, 1 mg/kg, given week 1 only). Patients had surgery 3 to 7 days following cycle 2. Main Outcomes and Measures: Safety and volumetric response determined using bidirectional measurements. Secondary end points included pathologic and objective response, progression-free survival (PFS), and overall survival. Multiplex immunofluorescence was used to evaluate primary tumor immune markers. Results: Fourteen patients were randomized to nivolumab (N) and 15 patients to nivolumab/ipilimumab (N+I) (mean [SD] age, 62 [12] years; 18 men [62%] and 11 women [38%]). The most common subsite was oral tongue (n = 16). Baseline clinical staging included patients with T2 (n = 20) or greater (n = 9) T stage and 17 patients (59%) with node-positive di...