Population-Based Assessment of Cancer Trial Generalizability in the Elderly

基于人群的老年人癌症试验普遍性评估

• 批准号: 7886643
• 负责人: ELIZABETH B LAMONT
• 金额: $ 31.23万
• 依托单位: HARVARD MEDICAL SCHOOL
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-09-26 至 2013-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7886643
• 关键词: Age; Area; Benefits and Risks; Board Certification; Breast; Cancer Patient; Cancer and Leukemia Group B; Caring; Chemotherapy-Oncologic Procedure; Clinical; Clinical Trials; Colorectal; Communities; Comorbidity; Conduct Clinical Trials; Credentialing; Cytotoxic agent; Data; Data Set; Data Sources; Decision Making; Diagnosis; Disease; Effectiveness; Elderly; Empirical Research; Enrollment; General Population; Goals; Individual; Intervention; Knowledge; Left; Lung; Malignant Neoplasms; Malignant neoplasm of lung; Malignant neoplasm of pancreas; Measures; Medical; Medicare; Methods; Modeling; Newly Diagnosed; Oncologist; Outcome; Participant; Patient Representative; Patients; Physicians; Policy Maker; Population; Probability; Provider; Race; Regimen; Relative (related person); Reporting; Research; Role; Scoring Method; Site; Staging; Study Subject; Time; United States; United States National Institutes of Health; Work; base; beneficiary; cancer care; cancer therapy; chemotherapy; clinical practice; cohort; data structure; demographics; experience; meetings; novel; older patient; oncology; population based; preference; prognostic; public health relevance; sex; treatment as usual

DESCRIPTION (provided by applicant): Despite the fact that Medicare spends millions of dollars annually on chemotherapy for elderly cancer patients, surprisingly little is known about the extent to which cancer chemotherapies help or harm such elderly patients. This unsettling paradox is the direct result of the well-described under-enrollment of elderly on the clinical trials of chemotherapy. Through this empirical research that integrates both clinical trial and observational data sources and methods, we seek to narrow this critical gap in clinical knowledge by studying the survival outcomes of both unselected elderly Medicare beneficiaries and their carefully chosen clinical trial counterparts. In Aim 1, we describe survival for stage-specific cohorts of elderly Medicare patients with breast, colorectal, lung, and pancreas cancer (n=100,000) following first-line treatment with one of several "standard" chemotherapies. In Aim 2, we describe the survival of patients from Aim 1 relative to untreated, but otherwise matched similar elderly Medicare patients with cancer (n=100,000) using propensity score methods. In Aim 3, we compare attributes of the cohorts of population elderly Medicare patients who were treated in Aim 1 to those of similarly treated clinical trial elderly Medicare patients. To do this, we create a new data set termed the CALGB-CMS data that relies on linkage (at the individual patient level) of Cancer and Leukemia Group B (CALGB) clinical trial data pertaining to elderly trial enrollees (n=4,000), to their contemporaneous observational Medicare data and other extant administrative data sources in a manner that parallels the SEER- Medicare data structure. After appending SEER-Medicare observational data to the CALGB-CMS data, we compare SEER-Medicare observational and CALGB-CMS clinical trial patients according to attributes of patients (e.g., demographics, comorbidity) and providers (e.g., board certification, years in practice). In Aim 4, we compare the survival outcomes of the population-treated elderly SEER-Medicare patients to those of the clinical trial-treated elderly CALGB-CMS patients following receipt of the same standard chemotherapy regimens. We estimate survival following therapy according to patient type and use multi-level approaches to identify and quantify salient patient and provider determinants of any observed differences in survival outcomes between population-treated and clinical trial-treated patients. In Aim 5, we create clinical prediction models for physicians to estimate survival for individual patients under treatment and no treatment scenarios; the models will tailor estimates to additional patient factors like age, sex, race and comorbidity. Through the proposed research, we will better understand the expected survival of population-treated elderly Medicare patients following the standard chemotherapy regimens whose efficacies were established in clinical trial patients. The work will help practicing oncologists in their care of elderly patients and set the stage for further study of the survival benefits of chemotherapy in the elderly. PUBLIC HEALTH RELEVANCE: While more than two thirds of all cancer patients in the United States are diagnosed at or after age 65, less than one third of participants in chemotherapy clinical trials are 65 or older. This well known under-representation of the elderly on chemotherapy trials has created an enormous void in clinical knowledge regarding the risks and benefits of even standard chemotherapy regimens in the elderly; this void may compromise informed decision-making by physicians and their elderly cancer patients and result in inappropriate over and under-treatment of the elderly. This research is focused on studying the survival outcomes of elderly Medicare cancer patients following treatment with what can be considered standard chemotherapy regimens and, thus, seeks to begin to fill the large void in clinical knowledge regarding the effectiveness of chemotherapy in elderly Medicare patients.

描述（由申请人提供）：尽管医疗保险每年花费数百万美元用于老年癌症患者的化疗，但令人惊讶的是，人们对癌症化疗在多大程度上帮助或伤害这些老年患者知之甚少。这种令人不安的悖论是化疗临床试验中老年人入组不足的直接结果。通过这项整合了临床试验和观察数据源和方法的实证研究，我们试图通过研究未经选择的老年医疗保险受益人及其精心挑选的临床试验对象的生存结果来缩小临床知识中的这一关键差距。在目标 1 中，我们描述了患有乳腺癌、结直肠癌、肺癌和胰腺癌的老年医疗保险患者 (n=100,000) 在采用几种“标准”化疗之一进行一线治疗后的特定阶段的生存率。在目标 2 中，我们描述了目标 1 患者相对于未经治疗的患者的生存率，但使用倾向评分方法匹配了类似的老年医疗保险癌症患者 (n=100,000)。在目标 3 中，我们将在目标 1 中接受治疗的老年医疗保险患者群体的属性与接受类似治疗的临床试验老年医疗保险患者的属性进行比较。为此，我们创建了一个名为 CALGB-CMS 数据的新数据集，该数据集依赖于与老年试验参与者 (n=4,000) 相关的癌症和白血病 B 组 (CALGB) 临床试验数据的关联（在个体患者水平），以与 SEER-Medicare 数据结构平行的方式连接到同期的观察性 Medicare 数据和其他现有的管理数据源。将 SEER-Medicare 观察数据附加到 CALGB-CMS 数据后，我们根据患者的属性（例如，人口统计、合并症）和提供者（例如，委员会认证、实践年限）比较 SEER-Medicare 观察和 CALGB-CMS 临床试验患者）。在目标 4 中，我们将接受相同标准化疗方案后接受人群治疗的老年 SEER-Medicare 患者的生存结果与接受临床试验治疗的老年 CALGB-CMS 患者的生存结果进行比较。我们根据患者类型估计治疗后的生存率，并使用多层次方法来识别和量化人群治疗和临床试验治疗的患者之间观察到的生存结果差异的显着患者和提供者决定因素。在目标 5 中，我们为医生创建临床预测模型，以估计接受治疗和无治疗情况下个体患者的生存率；这些模型将根据年龄、性别、种族和合并症等其他患者因素调整估计值。通过拟议的研究，我们将更好地了解接受人群治疗的老年医疗保险患者遵循标准化疗方案的预期生存率，这些方案的疗效已在临床试验患者中得到证实。这项工作将有助于执业肿瘤学家护理老年患者，并为进一步研究化疗对老年人的生存益处奠定基础。公共卫生相关性：虽然美国超过三分之二的癌症患者是在 65 岁或之后被诊断出来的，但化疗临床试验中只有不到三分之一的参与者年龄在 65 岁或以上。众所周知，老年人在化疗试验中的代表性不足，这在关于老年人标准化疗方案的风险和益处的临床知识中造成了巨大的空白。这种空白可能会影响医生及其老年癌症患者做出明智的决策，并导致对老年人的过度治疗和治疗不足。这项研究的重点是研究老年医疗保险癌症患者在接受标准化疗方案治疗后的生存结果，因此旨在填补有关老年医疗保险患者化疗有效性的临床知识的巨大空白。