Comparative Effectiveness of Treatment Regimens in Lung Cancer

肺癌治疗方案的比较疗效

• 批准号: 8668628
• 负责人: ELIZABETH B LAMONT
• 金额: $ 21.19万
• 依托单位: HARVARD MEDICAL SCHOOL
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-05-01 至 2016-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8668628
• 关键词: Accident and Emergency department; Aftercare; Cancer Patient; Carboplatin; Caring; Cessation of life; Chemotherapy-Oncologic Procedure; Cisplatin; Clinical; Clinical Trials; Colorectal Cancer; Combination Drug Therapy; Conduct Clinical Trials; Data; Data Sources; Decision Making; Diagnosis; Diagnostic Neoplasm Staging; Effectiveness; Elderly; Enrollment; Ensure; Etoposide; Extensive Stage; Family; General Population; Guidelines; Health Policy; Healthcare; Hospitalization; Hospitals; Individual; Inferior; Intensive Care; Left; Length of Stay; Life; Link; Malignant Neoplasms; Malignant neoplasm of lung; Medical Oncology; Medical Surveillance; Medicare; Morbidity - disease rate; National Cancer Institute; Newly Diagnosed; Oncologist; Outcome; Patient Representative; Patients; Pharmaceutical Preparations; Phase III Clinical Trials; Physicians; Policy Maker; Population; Provider; Randomized Clinical Trials; Regimen; Research; Risk; Scoring Method; Site; Staging; Therapeutic; Time; Toxic effect; Treatment Effectiveness; Treatment Protocols; VP 16; Visit; Work; base; burden of illness; cancer care; cancer site; care burden; chemotherapy; cohort; comparative effectiveness; effective therapy; effectiveness research; end of life; health care service utilization; improved; innovation; lens; lung small cell carcinoma; malignant breast neoplasm; mortality; older patient; palliation; patient population; public health relevance; standard of care; treatment as usual

DESCRIPTION (provided by applicant): Medicare spent nearly a billion dollars in 2002 on chemotherapy for newly diagnosed elderly breast, colorectal, and lung cancer patients alone, but surprisingly little is known about the extent to which cancer chemotherapies help or harm such patients. This unsettling paradox is the result of the well- described under-enrollment of the elderly on the clinical trials of chemotherapy. In the absence of trials with representative patients, treating oncologists, patients, and policy-makers are left to extrapolate results of clinical trials conducted in younger and comparatively healthier individuals to the general population with cancer, who tend to be older and have greater comorbid disease burdens. Many results of this common extrapolation are unknown. For most cancers, oncologists lack even basic estimates of survival following standard guideline-recommended chemotherapy regimens in elderly patients with cancer. Our broad aim is to compare two standard of care, guideline-recommended, first-line multi-agent chemotherapy regimens (i.e., (1) cisplatin and etoposide (CDDP/VP16) vs. (2) carboplatin and etoposide (carbo/VP16)) in the treatment of ~1,900 elderly Medicare patients with extensive stage small cell lung cancer (ES SCLC) who are drawn from a population-level data source created by the National Cancer Institute (i.e., SEER-Medicare data) according to two outcomes that patients and physicians have affirmatively identified as meaningful. The outcomes are (1) survival time following treatment and (2) invasive hospital-based health care utilization. While CDDP/VP16 has been established through phase III clinical trials as the most efficacious therapy for ES SCLC (i.e., associated with the longest survival), carbo/VP16 has been shown in the same studies to be less toxic and is used more often than CDDP/VP16 for elderly Medicare patients with ES SCLC. Through this work we broaden the lens of comparative effectiveness research (CER) to include outcomes beyond survival alone, outcomes that acknowledge (1) the effectiveness of these treatments in elderly Medicare patients is not yet established and (2) that the avoidance of invasive health care at the end of life may be at least as important to patients as survival time. For example, if CDDP/VP16 and carbo/VP16 are equally effective with respect to survival time following treatment but patients receiving CDDP/VP16 are twice as likely to be admitted to the hospital as patients receiving carbo/VP16, then treatment with carbo/VP16 might arguably be the more "effective" therapy as it maximizes mortality and minimizes morbidity. This particular information may also be of importance to health policy makers concerned about hospitalizations, the single largest component of Medicare spending for elderly cancer patients. Results of this research will begin to fill what is currently an unacceptable clinical information void in medical oncology regarding the benefits and burdens of national guideline-recommended standard first-line chemotherapy treatments when they are applied to elderly Medicare patients with ES SCLC who are treated in the usual care setting.

描述（由申请人提供）：2002 年，医疗保险仅在新诊断​​的老年乳腺癌、结直肠癌和肺癌患者的化疗上就花费了近 10 亿美元，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患者知之甚少。这种令人不安的悖论是由于众所周知的入学率不足而造成的。 老年人进行化疗的临床试验。在缺乏代表性患者的试验的情况下，治疗肿瘤学家、患者和政策制定者只能将在较年轻和相对健康的个体中进行的临床试验结果外推到一般癌症患者身上，而这些癌症患者往往年龄较大，并存疾病负担也更大。这种常见外推法的许多结果都是未知的。对于大多数癌症，肿瘤学家甚至缺乏对老年癌症患者遵循标准指南推荐的化疗方案后的生存率的基本估计。我们的总体目标是比较两种护理标准、指南推荐的一线多药化疗方案（即 (1) 顺铂和依托泊苷 (CDDP/VP16) 与 (2) 卡铂和依托泊苷 (carbo/VP16)）治疗约 1,900 名患有广泛期小细胞肺癌 (ES SCLC) 的老年医疗保险患者，这些患者 根据患者和医生肯定认为有意义的两个结果，从美国国家癌症研究所创建的人口级数据源（即 SEER-Medicare 数据）中提取。结果是（1）治疗后的生存时间和（2）侵入性医院医疗保健利用。虽然 CDDP/VP16 已通过 III 期临床试验确定为 ES SCLC 最有效的治疗方法（即与最长生存期相关），但在相同的研究中，Carbo/VP16 已被证明毒性较小，并且在治疗患有 ES SCLC 的老年医疗保险患者时比 CDDP/VP16 更常使用。通过这项工作，我们扩大了比较有效性研究（CER）的范围，将结果纳入了生存之外的结果，这些结果承认（1）这些治疗对老年医疗保险患者的有效性尚未确定，（2）在生命结束时避免侵入性医疗保健对患者来说可能至少与生存时间一样重要。例如，如果 CDDP/VP16 和 carbo/VP16 在治疗后的生存时间方面同样有效，但接受 CDDP/VP16 的患者入院的可能性是接受 carbo/VP16 的患者的两倍，那么用 carbo/VP16 治疗可能会更“有效”，因为它最大限度地提高了死亡率并最大限度地降低了发病率。这一特定信息对于关心住院治疗的卫生政策制定者也可能很重要，住院治疗是老年癌症患者医疗保险支出的最大组成部分。这项研究的结果将开始填补目前医学肿瘤学中不可接受的临床信息空白，即国家指南推荐的标准一线化疗治疗应用于在常规护理机构接受治疗的老年医疗保险 ES SCLC 患者的益处和负担。