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

• 批准号: 10363336
• 负责人: Benjamin D Kozower
• 金额: $ 73.36万
• 依托单位: WASHINGTON UNIVERSITY
• 依托单位国家: 美国
• 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/10363336
• 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; Equipoise; Evidence based treatment; Goals; Information Systems; 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; Research Design; Retrospective Studies; Sample Size; Scanning; Selection Bias; Societies; Techniques; Thoracic Surgical Procedures; Treatment outcome; Work; X-Ray Computed Tomography; base; cancer surgery; 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; predictive modeling; preference; premature; prospective; recruit; systematic review; trial comparing; tumor; web-based tool

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使用越来越多，肿瘤越来越多 在I期疾病中被检测到，治愈的可能性超过70%。外科手术一直是 I期非小细胞肺癌的传统治疗。然而，基于人群的研究表明， 立体定向全身放射治疗(SBRT)在过去的十年里。比较外科手术和手术治疗的回顾性研究 SBRT，包括我们组的几份出版物，一直受到样本量相对较小的阻碍，缺乏 患者报告的结果，以及关于合并症和癌症相关结果的稀少信息。 因此，最近的一项系统评估得出结论：“有必要对这两种治疗方法进行总体比较 预期试验“。不幸的是，多次尝试将手术与SBRT进行比较的随机对照试验 由于专业偏见和被认为缺乏平衡而未能产生和过早关闭。此外， 我们以前的工作已经证明，个体患者的特征在治疗选择中是至关重要的。在……里面 缺乏前瞻性比较研究，治疗分配在很大程度上是由机构指导的 经验、回顾数据和提供商意见。患者缺乏循证治疗分配 对于I期肺癌，仍然是一个严重的未得到满足的需求。为了解决这一根本的知识差距，我们将 进行前瞻性、务实的多中心队列研究，比较手术和SBRT的效果 用于第一期非小细胞肺癌。这种务实的研究设计非常适合提供可操作的结果。 目的1：比较手术治疗与立体定向全身放射治疗(SBRT)的疗效。 利益相关者选择临床I期肺癌患者的短期和长期结果。目标1a： 比较手术组和SBRT组的3年无瘤生存率。我们将比较长期DFS 使用倾向分数匹配的队列。我们假设SBRT将导致更少和更不严重的治疗- 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 (Pro)在手术和SBRT之间。我们将在倾向匹配的队列中比较短期和长期PRO 使用患者报告结果评估信息系统。我们假设SBRT将导致 在较好的短期利润率与同等的长期利润率。 目标2：开发和验证治疗结果的预测模型 I期肺癌。在接受手术或SBRT的I期患者的前瞻性队列中 对于肺癌，我们将使用回归技术预测单个患者的长期DFS和PRO。这些 模型将作为I期非小细胞肺癌患者的网络工具进行验证和展示。我们的建议将 创建肺癌个性化治疗分配的基准，并提供可操作的结果 改善对I期非小细胞肺癌患者的护理。