Background: Immunotherapy has emerged as a breakthrough for the treatment of advanced non-small-cell lung cancer (NSCLC), significantly improving patients’ progression-free survival (PFS) and overall survival (OS). However, the medical burden of response assessment has worsened for long-term maintenance therapy. It remains unclear whether a specific response assessment interval could provide both survival benefits and cost savings. Methods: We retrospectively included patients with advanced NSCLC who underwent immunotherapy and achieved PFS > 12 months. We utilized propensity score matching (PSM) to reduce the selection bias. The survival outcomes were evaluated using the log-rank test and Cox proportional hazard models, while the economic impact was assessed through the performance of a cost minimization analysis (CMA). A medical expenditure extrapolation model was developed based on epidemiological statistics and data from clinical trials. Results: After PSM, a total of 376 patients were included. The survival difference was not significant [hazard ratio (HR) = 0.78, 95% confidence intervals (CIs) = 0.53–1.14;p= 0.200] between the 2-month response assessment group (n= 188) and the 3-month response assessment group (n= 188). Patients receiving immunotherapy alone and those with a positive PD-L1 expression experienced a significant survival benefit. Our extrapolation model projects that, annually, there will be approximately 7026 new long-term responders to immunotherapy in the United States. Adopting a 3-month assessment strategy could reduce annual healthcare expenditure by nearly USD 6 million. Conclusions: This study presented the first statistical evidence supporting a refined response assessment strategy for long-term responders to immunotherapy with advanced NSCLC. These findings support the adoption of a less frequent, yet equally effective, monitoring approach to make tumor surveillance more precise and cost-effective.
背景：免疫治疗已成为晚期非小细胞肺癌（NSCLC）治疗的突破性进展，显著改善了患者的无进展生存期（PFS）和总生存期（OS）。然而，长期维持治疗中的疗效评估给医疗系统带来了日益沉重的负担。目前尚不明确是否存在一种特定的疗效评估间隔，既能保证生存获益又能节约医疗成本。方法：本研究回顾性纳入接受免疫治疗且PFS超过12个月的晚期NSCLC患者。采用倾向性评分匹配（PSM）方法以减少选择偏倚。通过时序检验和Cox比例风险模型评估生存结局，并运用成本最小化分析（CMA）评估经济影响。基于流行病学统计数据和临床试验数据，构建了医疗支出外推模型。结果：经PSM匹配后共纳入376例患者。2个月疗效评估组（n=188）与3个月疗效评估组（n=188）的生存差异无统计学意义[风险比（HR）=0.78，95%置信区间（CI）=0.53-1.14；p=0.200]。接受单药免疫治疗及PD-L1阳性表达患者显示出显著的生存获益。根据外推模型预测，美国每年将新增约7026例免疫治疗长期应答者。采用3个月评估策略可使年度医疗支出减少近600万美元。结论：本研究首次为晚期NSCLC免疫治疗长期应答者提供了优化疗效评估策略的统计学证据。研究结果支持采用频率更低但同等有效的监测方案，使肿瘤监测更精准且更具成本效益。
肿瘤(癌症)患者之家