Comparative Effectiveness of Surgery vs Stereotactic Radiation Therapy for Stage I Lung Cancer

手术与立体定向放射治疗 I 期肺癌的疗效比较

基本信息

批准号：
10579175
负责人：
Benjamin D Kozower
金额：
$ 62.66万
依托单位：
WASHINGTON UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2022
资助国家：
美国
起止时间：
2022-02-24 至 2027-01-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10579175
关键词：
Address Adverse event Aftercare Benchmarking Cancer Etiology Cancer Patient Caring Characteristics Clinical Cohort Studies Comparative Study Data Databases Disease Disease-Free Survival Disparate Early Diagnosis Equipoise Evidence based treatment Goals Information Systems Institution Investigation Knowledge Light Malignant Neoplasms Malignant neoplasm of lung Measurement Modeling Morbidity - disease rate National Comprehensive Cancer Network Non-Small-Cell Lung Carcinoma Operative Surgical Procedures Outcome Patient Care Patient Outcomes Assessments Patient-Centered Care Patients Population Study Provider Publications Publishing Radiation Oncology Radiation therapy Randomized Randomized, Controlled Trials Recommendation Research Design Retrospective Studies Sample Size Scanning Selection Bias Societies Surgical Oncology Techniques Thoracic Surgical Procedures Treatment outcome Work X-Ray Computed Tomography cohort comorbidity comparative effectiveness comparative effectiveness study compare effectiveness design experience improved individual patient lung cancer screening medical specialties mortality patient advocacy group personalized medicine pragmatic study predictive modeling preference premature prospective recruit systematic review trial comparing tumor web-based tool

项目摘要

Non-small cell lung cancer (NSCLC) is the leading cause of cancer related morbidity and mortality in the U.S. With the increasing use of computed tomography scans and lung cancer screening, tumors are increasingly being detected at stage I disease, conferring a greater than 70% likelihood of cure. Surgery has been the traditional treatment for stage I NSCLC. However, population-based studies demonstrate a rapid increase in stereotactic body radiation therapy (SBRT) over the past decade. Retrospective studies comparing surgery and SBRT, including several publications from our group, have been hampered by relatively small sample sizes, lack of patient reported outcomes, and sparse information on comorbidities and cancer-related outcomes. Consequently, a recent systematic review concluded, “there is a need to compare both treatments in large prospective trials”. Unfortunately, multiple attempted randomized controlled trials comparing surgery to SBRT have failed to accrue and closed prematurely due to specialty bias and perceived lack of equipoise. Furthermore, our previous work has demonstrated that individual patient characteristics are crucial in the choice of therapy. In the absence of prospective comparative studies, treatment allocation is largely directed by institutional experience, retrospective data, and provider opinion. The lack of evidence-based treatment allocation in patients with stage I lung cancer remains a critical unmet need. To address this fundamental gap in knowledge, we will perform a prospective, pragmatic, multi-center cohort study to compare the effectiveness of surgery and SBRT for stage I NSCLC. This pragmatic study design is ideally suited to provide actionable results. Aim 1: To compare the effectiveness of surgery versus stereotactic body radiation therapy (SBRT) on stakeholder selected short and long-term outcomes in patients with clinical stage I lung cancer. Aim 1a: To compare 3-year disease-free survival (DFS) between surgery and SBRT. We will compare long-term DFS using propensity score matched cohorts. We hypothesize that SBRT will lead to fewer and less severe treatment- related complications, while surgery will result in longer DFS. Aim 1b: To compare patient reported outcomes (PRO) between surgery and SBRT. We will compare short and long-term PRO in propensity-matched cohorts using the Patient Reported Outcomes Measurement Information System. We hypothesize that SBRT will result in better short-term PRO with equivalent long-term PRO. Aim 2: To develop and validate prediction models for treatment outcomes for an individual patient with stage I lung cancer. In prospectively assembled cohorts of patients undergoing surgery or SBRT for stage I lung cancer, we will predict long-term DFS and PRO for an individual patient using regression techniques. These models will be validated and presented as a web-based tool for patients with stage I NSCLC. Our proposal will create a benchmark for personalized treatment allocation in lung cancer and provide actionable results to improve care for Stage I NSCLC patients.

非小细胞肺癌 (NSCLC) 是美国癌症相关发病率和死亡率的主要原因。随着计算机断层扫描和肺癌筛查的越来越多地使用，肿瘤越来越多在 I 期疾病中被检测出来，治愈的可能性超过 70%。然而，基于人群的研究表明，I 期非小细胞肺癌的传统治疗方法正在迅速增加。过去十年的立体定向身体放射治疗（SBRT）的回顾性研究比较了手术和治疗。 SBRT，包括我们小组的几篇出版物，一直受到样本量相对较小、缺乏患者报告的结果以及关于合并症和癌症相关结果的信息很少。最近的一项系统审查得出结论，“有必要在大范围内比较这两种治疗方法” 不幸的是，多项随机对照试验尝试将手术与 SBRT 进行比较。由于专业偏见和缺乏平衡感，未能积累并过早关闭。我们之前的工作表明，患者的个体特征对于治疗的选择至关重要。由于缺乏前瞻性比较研究，治疗分配主要由机构指导经验、回顾性数据和提供者意见缺乏基于证据的患者治疗分配。 I 期肺癌仍然是一个未满足的关键需求，为了解决这一基本的知识差距，我们将进行前瞻性、务实、多中心队列研究来比较手术和 SBRT 的有效性这种务实的研究设计非常适合提供可操作的结果。目标 1：比较手术与立体定向放射治疗 (SBRT) 的疗效利益相关者选择了临床 I 期肺癌患者的短期和长期结局目标 1a：为了比较手术和 SBRT 之间的 3 年无病生存率 (DFS)，我们将比较长期 DFS。我们勇敢地使用倾向评分匹配队列，认为 SBRT 会导致越来越少的严重治疗。相关并发症，而手术将导致更长的 DFS 目标 1b：比较患者报告的结果。我们将比较倾向匹配队列中的短期和长期 PRO。使用患者报告结果测量信息系统，我们勇敢地面对 SBRT 的结果。更好的短期 PRO 与同等的长期 PRO。目标 2：开发并验证个体患者治疗结果的预测模型在前瞻性收集的接受 I 期手术或 SBRT 的患者队列中。对于肺癌，我们将使用回归技术预测个体患者的长期 DFS 和 PRO。我们的建议将针对 I 期 NSCLC 患者对模型进行验证并作为基于网络的工具提供。为肺癌的个性化治疗分配创建基准，并提供可行的结果改善对 I 期 NSCLC 患者的护理。