Applying causal inference methods to improve estimation of the real-world benefits and harms of lung cancer screening

应用因果推理方法来改进对肺癌筛查的现实益处和危害的估计

• 批准号: 10737187
• 负责人: Louise Henderson
• 金额: $ 34.86万
• 依托单位: UNIV OF NORTH CAROLINA CHAPEL HILL
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-07-01 至 2028-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10737187
• 关键词: Address; Adherence; Affect; Behavioral; Behavioral Risk Factor Surveillance System; Breast Cancer Detection; Characteristics; Clinical Trials; Communication; Data; Data Set; Data Sources; Decision Aid; Decision Making; Development; Diagnostic Procedure; Early Diagnosis; Effectiveness; Eligibility Determination; Equilibrium; Female; General Population; Individual; Intervention; Left; Lung; Malignant Neoplasms; Malignant neoplasm of lung; Medicare claim; Methodology; Methods; National Cancer Institute; National Health Interview Survey; North Carolina; Observational Study; Outcome; Outcome Assessment; Patients; Pattern; Policy Maker; Population; Population Characteristics; Procedures; Provider; Randomized; Randomized, Controlled Trials; Recommendation; Registries; Research; Research Personnel; Risk Factors; Sampling; Screening for cancer; Selection Bias; Selection for Treatments; Smoking; Subgroup; Surveys; Time; United States Preventative Services Task Force; Update; Work; cancer therapy; clinical practice; colon cancer screening; cost; design; evidence base; follow-up; human old age (65+); improved; intervention effect; low dose computed tomography; lung cancer screening; mortality; novel; practice setting; randomized trial; routine screening; screening; screening guidelines; screening program; shared decision making; tool; trial enrollment; uptake

ABSTRACT Randomized controlled trials have demonstrated that low-dose computed tomography can substantially reduce lung cancer mortality, albeit at the cost of relatively high rates of false positives and complications from downstream procedures. However, systematic differences between trial and general populations eligible for lung cancer screening raise concerns about the relevance of trial findings for guiding the development and dissemination of lung cancer screening programs in clinical practice. Despite clear recommendations from the United States Preventative Services Task Force, lung cancer screening uptake and adherence remain low. Several studies have documented dramatic and selective attrition across the screening continuum – where about 10-20% of eligible individuals undergo lung cancer screening and of those, only about 40-60% are up-to- date with their annual screening at 15 months. When the benefits and harms of an intervention vary across subgroups and there is selective attrition, the balance of population-level benefits and harms is expected to change. As such, there is an urgent need to better characterize the effectiveness of lung cancer screening with low-dose computed tomography when applied to individuals outside of clinical trial settings. The primary objective of this proposal is to generate real-world evidence of the benefits and harms of lung cancer screening with low-dose computed tomography that explicitly considers the characteristics of populations at each step of the screening continuum, from the screening-eligible, to the screened, to the adherent. To address this objective, we will use cutting-edge causal inference methods, including trial transport and target trial emulation using real-world data, which can avoid the potential for time-related biases. To carry-out our proposed analyses, we will draw upon individual-level data from the randomized National Lung Screening Trial, as well as four real-world datasets including the National Health and Interview Survey, the Behavioral Risk Factors Surveillance Survey (Lung Cancer Screening Module), a 20% nationwide sample of Medicare claims, and the North Carolina Lung Screening Registry. Findings from this study will generate information about the effectiveness of lung cancer screening in real-world settings that can be used by patients, providers, and policymakers. This work will enhance the evidence base used by policymakers to update screening recommendations and refine decision aids to support communication with patients about screening net- benefits during shared decision-making. Ultimately, this work will support efforts to improve the delivery of lung cancer screening at the population level.

抽象的 随机对照试验表明，低剂量计算机断层扫描可以大大减少 肺癌死亡率，尽管其代价是假阳性率和并发症率相对较高 下游程序。然而，试验人群和符合资格的一般人群之间存在系统性差异 肺癌筛查引发了人们对试验结果与指导开发的相关性的担忧 在临床实践中传播肺癌筛查项目。尽管委员会提出了明确的建议 美国预防服务工作组表示，肺癌筛查的采用率和依从性仍然很低。 几项研究记录了整个筛查过程中的戏剧性和选择性减员——其中 大约 10-20% 的符合条件的个人接受了肺癌筛查，其中只有大约 40-60% 的人达到了- 并在 15 个月大时进行年度筛查。当干预措施的好处和坏处因人而异时 亚群体，并且存在选择性减员，人口层面的利益和危害的平衡预计会 改变。因此，迫切需要更好地表征肺癌筛查的有效性 应用于临床试验环境之外的个体时的低剂量计算机断层扫描。初级 该提案的目标是生成肺癌筛查的益处和危害的真实证据 使用低剂量计算机断层扫描，明确考虑每一步的人群特征 筛选连续体，从符合筛选资格，到被筛选，再到依从者。为了解决这个问题 为了实现目标，我们将使用最先进的因果推理方法，包括试验运输和目标试验仿真 使用真实世界的数据，这可以避免潜在的与时间相关的偏差。为落实我们的建议 分析中，我们还将利用来自随机国家肺部筛查试验的个人数据 作为四个真实世界的数据集，包括国家健康和访谈调查、行为风险因素 监测调查（肺癌筛查模块），全国 20% 的医疗保险索赔样本，以及 北卡罗来纳州肺部筛查登记处。这项研究的结果将产生有关 现实世界中肺癌筛查的有效性可供患者、提供者和 政策制定者。这项工作将增强政策制定者用于更新筛查的证据基础 建议并完善决策辅助工具，以支持与患者就筛查网络进行沟通 共同决策过程中的利益。最终，这项工作将支持改善肺输送的努力 人口层面的癌症筛查。