Single-center prospective study on the efficacy of nivolumab against platinum-sensitive recurrent or metastatic head and neck squamous cell carcinoma

Abstract

Nivolumab, an immune checkpoint inhibitor, is beneficial to patients with platinum-refractory recurrent or metastatic head and neck squamous cell carcinoma (R/M-HNSCC). However, platinum-sensitive R/M-HNSCC has not yet been studied. Hence, in this prospective study, we evaluated the efficacy and safety of nivolumab in patients with platinum-sensitive R/M-HNSCC. This prospective single-arm study was conducted in a single institution in Japan. Patients with platinum-sensitive R/M-HNSCC (defined as head and neck cancer that recurred or metastasized at least 6 months after platinum-based chemotherapy or chemoradiotherapy) were enrolled. The primary endpoint was overall survival (OS). The secondary endpoints were progression-free survival (PFS), overall response rate (ORR), immune-related adverse events (irAEs), and quality of life (QOL). This study was registered at the University Hospital Medical Information Network Clinical Trials Registry (UMIN000031324). Twenty-two patients with platinum-sensitive R/M-HNSCC were enrolled. The median OS was 17.4 months, and the 1-year OS rate was 73%. The median PFS was 9.6 months, 1-year PFS rate was 48%, and ORR was 36%. Sixteen irAEs were recorded in 12 patients; however, no grade 4 or 5 irAEs were observed. The QOL assessments revealed that nivolumab did not decrease the QOL of patients. Nivolumab is effective against platinum-sensitive R/M-HNSCC with acceptable safety.

Figure 1

Kaplan–Meier curves of overall survival and progression-free survival. Kaplan–Meier curves of (A) overall survival and (B) progression-free survival. Vertical lines show censored events, and the shaded region represents a 95% confidence interval.

Figure 2

Responses of patients with platinum-sensitive R/M-HNSCC to nivolumab based on the RECIST guidelines (version 1.1). (A) Change in the sum of target lesions over time from baseline to PD. The upper dashed line represents the threshold for PD, and the lower dashed line shows the threshold for PR. (B) Best reduction in the target lesions from baseline. The upper dashed line represents the threshold for PD (20% increase in the sum of the longest diameter of the target lesions). The lower dashed line shows the threshold for PR (30% decrease in the sum of the longest diameter of the target lesions). (C) Time to response and duration of survival. Each bar represents a single patient, with the length of the bar corresponding to overall survival.

Figure 3

Quality of life assessments. (A) Functional scales (physical, role, emotional, cognitive, and social activities) and (B) global health status were assessed using the EORTC QLQ-C30. (C) Domain scales (pain, swallowing, sense problems, speech problems, trouble with social eating, trouble with social contact, and reduced sexuality) were assessed using the QLQ-H&N35. All scales ranged from 0 to 100, and score changes of at least 10 points (dashed line) were considered clinically significant. Higher values for functional and domain scales indicate poor functioning, whereas higher values for global health status indicate better functioning. The I bar indicates a 95% confidence interval.