Cost-Effectiveness of Hormonal Therapy for Clinically Localized Prostate Cancer

临床局限性前列腺癌激素治疗的成本效益

• 批准号: 7821966
• 负责人: Arnold L Potosky
• 金额: $ 50万
• 依托单位: KAISER FOUNDATION RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-30 至 2011-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7821966
• 关键词: Accounting; Address; Adjuvant; Age; Androgen Therapy; Androgens; Area; Arts; Benefits and Risks; Biological Markers; California; Cardiovascular system; Caring; Cause of Death; Cessation of life; Characteristics; Clinical; Clinical Trials; Cohort Studies; Comorbidity; Coronary heart disease; Data; Databases; Decision Making; Diabetes Mellitus; Diagnosis; Disease; Disease Progression; Elderly man; Ensure; Event; Fracture; Gleason Grade for Prostate Cancer; Health; Health Benefit; Health Care Costs; Health Planning; Health Policy; Health system; Healthcare; Incidence; Inpatients; Knowledge; Life; Literature; Localized Disease; Malignant Neoplasms; Malignant neoplasm of prostate; Measures; Medical; Medical Care Costs; Metastatic Neoplasm to the Bone; Metastatic to; Modeling; Morbidity - disease rate; Newly Diagnosed; Observational Study; Operative Surgical Procedures; Outcome; Outpatients; Pain; Palliative Care; Patient observation; Patients; Pharmacy facility; Policies; Population; Population Heterogeneity; Prevalence; Probability; Prognostic Factor; Progression-Free Survivals; Prostate-Specific Antigen; Public Health; Publishing; Quality of life; Quality-Adjusted Life Years; Race; Radiation; Radiation therapy; Radiology Specialty; Randomized Controlled Clinical Trials; Risk; Risk Factors; Selection Bias; Selection for Treatments; Serious Adverse Event; Serum; Source; Staging; Survival Rate; Survivors; System; Techniques; Testing; Treatment Cost; Tumor Markers; Uncertainty; United States; Variant; advanced disease; anticancer research; bone loss; cancer diagnosis; cancer therapy; cohort; comparative effectiveness; computerized; cost; cost effectiveness; deprivation; disease diagnosis; effectiveness measure; effectiveness research; experience; follow-up; health care service utilization; health related quality of life; high risk; hormone therapy; improved; men; men' s group; mortality; novel; older men; palliative; prognostic; socioeconomics; tumor

DESCRIPTION (provided by applicant): This application addresses broad Challenge Area (05) entitled "Comparative Effectiveness Research (CER)". Within that area, we are addressing the specific high priority challenge topic 05-CA-104* Comparative Effectiveness Research on Cancer Treatment. Our application also responds to the specific challenge topic 04-CA-110 Treatment of Prostate Cancer. Androgen Deprivation Therapy (ADT) has become increasingly used as primary monotherapy for older men with newly diagnosed localized disease not receiving other curative treatments (surgery or radiotherapy), despite the fact that there is no proven mortality benefit from clinical trials. Given the increasing number of elderly men, the high incidence and survival rates from prostate cancer, and the use of ADT in one-third of 2 million men newly diagnosed or surviving with prostate cancer, there is a growing need for information on effectiveness and costs to inform policy and treatment decisions. Clinical trials are not ongoing or likely to be conducted to address these issues. To address the limitations of prior observational database studies, we propose a new comparative effectiveness study to provide information on the risks and potential benefits of immediate ADT in men diagnosed with localized prostate cancer. Our three aims include estimating the comparative effectiveness of immediate ADT versus observation in terms of all cause and prostate-cancer specific mortality and progression-free survival, estimating the longitudinal direct medical care costs to capture the impact of ADT, and calculating the cost- effectiveness (cost per life years saved) and cost-utility (quality-adjusted life years) using published patient utilities for multiple prostate cancer health states. We will assess all outcomes according to prognostic risk groups defined by age, stage, serum biomarker values (PSA), and other pathological markers of tumor aggressiveness. We will account for variations in baseline comorbidity and sociodemographic factors, and use state-of-the-art comparative effectiveness techniques to address selection bias. The retrospective observational study will be conducted using a large, diverse population of nearly 10,000 men with localized disease diagnosed from 1995-2007 with a mean follow up of 6 years. There are comprehensive computerized clinical utilization data for this population from 2 large integrated health care plans, including longitudinal information on tumor characteristics, risk factors and outcomes. Key variables will be derived from inpatient, outpatient, pharmacy and radiology data and lab test values. In contrast to prior observational studies, ours will have the combination of size, follow up, and detailed clinical information over the entire disease trajectory needed to significantly improve the precision of estimates of mortality and progression-free survival following ADT in sub-groups of men at varying levels of baseline risk. These strengths, and our multi-disciplinary team experienced in prostate cancer research using large databases, ensure that our results will be useful to improve practice, policy, and health outcomes. This is a multisite study to investigate the Challenge Area of Comparative Effectiveness Research on Cancer Treatment and specifically the Treatment of Prostate Cancer. We propose a new comparative effectiveness study to provide information on the risks and potential benefits of immediate ADT in men diagnosed with localized prostate cancer using data from two integrated health delivery systems with access to comprehensive health, utilization, cost, and socioeconomic data.

描述（由申请人提供）：本申请涉及题为“比较有效性研究（CER）”的广泛挑战领域（05）。在该领域内，我们正在解决特定的高优先级挑战主题 05-CA-104* 癌症治疗的比较有效性研究。我们的应用程序还响应特定挑战主题 04-CA-110 前列腺癌治疗。雄激素剥夺疗法 (ADT) 已越来越多地用作新诊断出局部疾病且未接受其他治疗（手术或放疗）的老年男性的主要单一疗法，尽管临床试验尚未证明死亡率会降低。鉴于老年男性数量不断增加、前列腺癌的高发病率和存活率，以及 200 万新诊断出前列腺癌或存活的前列腺癌男性中有三分之一使用了 ADT，因此越来越需要有关有效性和成本的信息为政策和治疗决策提供信息。尚未进行或不可能进行临床试验来解决这些问题。为了解决先前观察性数据库研究的局限性，我们提出了一项新的比较有效性研究，以提供有关诊断为局限性前列腺癌的男性立即进行 ADT 的风险和潜在益处的信息。我们的三个目标包括评估立即 ADT 与观察在全因死亡率和前列腺癌特异性死亡率和无进展生存率方面的比较效果，估计纵向直接医疗费用以捕捉 ADT 的影响，并计算成本效益（节省的每生命年的成本）和成本效用（质量调整的生命年）使用已发布的多种前列腺癌健康状态的患者效用。我们将根据由年龄、分期、血清生物标志物值（PSA）和肿瘤侵袭性的其他病理标志物定义的预后风险组来评估所有结果。我们将考虑基线合并症和社会人口因素的变化，并使用最先进的比较有效性技术来解决选择偏差。这项回顾性观察研究将使用 1995 年至 2007 年间诊断出的局部疾病的近 10,000 名男性组成的庞大、多样化的人群，平均随访时间为 6 年。来自两个大型综合医疗保健计划的该人群的综合计算机化临床利用数据，包括有关肿瘤特征、危险因素和结果的纵向信息。关键变量将来自住院患者、门诊患者、药房和放射学数据以及实验室测试值。与之前的观察性研究相比，我们的研究将结合整个疾病轨迹的规模、随访和详细临床信息，以显着提高男性亚组 ADT 后死亡率和无进展生存期估计的精确度不同水平的基线风险。这些优势，以及我们在使用大型数据库进行前列腺癌研究方面经验丰富的多学科团队，确保我们的结果将有助于改善实践、政策和健康结果。这是一项多中心研究，旨在调查癌症治疗特别是前列腺癌治疗的比较有效性研究的挑战领域。我们提出了一项新的比较有效性研究，利用来自两个综合医疗服务系统的数据，获取全面的健康、利用率、成本和社会经济数据，提供关于诊断为局限性前列腺癌的男性立即 ADT 的风险和潜在益处的信息。