Safety and effectiveness of nivolumab in patients with locally advanced or metastatic non-small cell lung cancer in China: a nationwide electronic health record dataset analysis

Background: While the efficacy and safety of nivolumab have been demonstrated in clinical trials involving Chinese patients with non-small cell lung cancer (NSCLC), real-world evidence on treatment patterns, safety, and clinical outcomes remains limited. This study aimed to investigate the safety profile and effectiveness of nivolumab in Chinese NSCLC patients. Methods: This retrospective observational cohort included patients diagnosed with advanced NSCLC who received at least one dose of nivolumab between June 1, 2018, and August 31, 2022, as identified from the National Anti-Tumor Drug Surveillance System (NATDSS). Longitudinal electronic health record (EHR) data for the identified patients were used to assess safety outcomes, treatment patterns, overall survival (OS), and real-world progression-free survival (rwPFS). Safety outcomes and treatment patterns were analyzed descriptively. Time-to-event outcomes, including OS and rwPFS, were evaluated using the Kaplan-Meier (KM) method and reported as median time to event with 95% confidence intervals (CIs). Subgroup analyses of safety and effectiveness were conducted according to line of therapy, treatment regimen (monotherapy or combination), and tumor histology. Results: A total of 2,649 eligible patients with locally advanced or metastatic NSCLC were identified across 224 hospitals in China. The median age was 63.0 years, and the median time from diagnosis to nivolumab initiation was 90.0 days [interquartile range (IQR): 12–327 days]. Nivolumab was administered as first-line therapy in 52.9% (n=1,400), second-line in 31.8% (n=842), and third-line or beyond in 23.1% (n=613) of patients. The median follow-up was 12.9 months (95% CI: 12.4–13.7). Immune-related adverse events (irAEs) occurred in 14.8% (n=391) of the patients, with grade 3–4 irAEs in 2.5% (n=67). The incidence of grade 3–4 irAEs was slightly higher in the combination therapy group than in the monotherapy group (2.5% vs. 1.9%). Serious adverse events (SAEs) were reported in 4.3% (n=114) of patients. No new safety signals were identified in older patients (≥75 years), or those with baseline renal or hepatic impairment. The median OS was 21.2 months (95% CI: 18.7–24.5). Conclusions: This large-scale, real-world study provides further evidence of manageable safety profile and favorable effectiveness of nivolumab in Chinese patients with advanced NSCLC. These findings support the continued use of nivolumab in appropriate patients and provide reassurance that real-world outcomes are consistent with those observed in clinical trials.