基于真实世界晚期消化道恶性肿瘤中西医结合治疗研究的统计方法探索

Novel statistical methods for evaluating the treatment effect of integrated Chinese and Western medicine based on real-world data from patients with advanced digestive tract malignant tumors

目的基于新的统计分析方法,利用真实世界数据探究尽早接受中西医结合治疗是否可以延长晚期消化道恶性肿瘤患者的生存时间。方法基于左截断数据,通过Copula模型构建IV期诊断时间到中西医结合治疗时间与IV期生存时间之间的联合模型。以Copula相关性参数η的估计值和显著性结果作为IV期诊断时间到治疗时间与IV期生存时间相关性的评价指标。结果数值模拟显示本研究方法在左截断比例为20%时表现较好。实际数据分析中Copula相关性参数的估计值为0.279(P<0.05),表明中西医结合治疗起始时间与IV期生存时间存在显著相关性,性别、美国东部肿瘤协作组评分、既往是否靶向治疗及转移部位1为影响IV期生存时间的显著因素。结论新统计方法可以为中西医结合治疗晚期消化道恶性肿瘤患者的有效性提供有价值的观点和分析工具。

Objective To explore whether early treatment with integrated Chinese and western medicine can prolong the survival time of patients with advanced digestive tract malignant tumors using a new statistical analysis method and real-world data.Methods Based on the left truncated data,the Copula model was used to construct a joint model of the time from stage IV diagnosis to treatment and the time to overall survival.The estimated value and significance of the Copula dependence parameter,η,were obtained to evaluate the dependence strength between the two event times of interest.Results The numerical simulation showed that this method performed well when the left truncation rate was 20%.The estimated Copula dependence parameter,■,in the real data analysis was 0.279(P<0.05),indicating that there was a significant correlation between times to treatment and overall survival.Gender,the Eastern Cooperative Oncology Group score,targeted treatment,and metastatic site 1 were significant factors associated with overall survival.Conclusion The new statistical method provided valuable insights and analytical tools for the effectiveness of integrated traditional Chinese and western medicine in treating patients with advanced digestive tract malignant tumors.