Comparative Effectiveness of Treatment Regimens in Lung Cancer

描述(申请人提供)：2002年，联邦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，人们对癌症化疗对这些患者的帮助或伤害程度知之甚少。这一令人不安的悖论是众所周知的招生不足的结果 老年人正在进行化疗的临床试验。在缺乏有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人中进行的临床试验的结果推断到一般癌症患者，这些人往往较年长，并有更大的并存疾病负担。这种常见外推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采用标准指南推荐的化疗方案后的生存时间的基本估计。我们的广泛目标是比较两种标准护理、指南推荐的一线多药化疗方案(即(1)顺铂加依托泊苷(CDDP/VP16)与(2)卡铂加依托泊苷(Carbo/VP16))治疗约1,900名患有广泛期小细胞肺癌(ES SCLC)的老年Medicare患者，这些患者来自国家癌症研究所创建的人群级数据源(即SEER-Medicare数据)，根据患者和医生确认为有意义的两项结果。结果是(1)治疗后的存活时间和(2)侵入性的基于医院的卫生保健利用。虽然CDDP/VP16已经通过第三阶段临床试验被确定为治疗ES SCLC的最有效的治疗方法(即与最长的生存期相关)，但同一研究表明，Caro/VP16毒性较小，并且比CDDP/VP16更常用于患有ES SCLC的老年医疗保险患者。通过这项工作，我们拓宽了比较有效性研究(CER)的视角，以包括仅限于生存的结果，承认(1)这些治疗对老年联邦医疗保险患者的有效性尚未确定，以及(2)避免在生命末期进行侵入性医疗保健对患者可能至少与生存时间同样重要的结果。例如，如果CDDP/VP16和Carco/VP16在治疗后的存活时间方面同样有效，但接受CDDP/VP16的患者入院治疗的可能性是接受Carco/VP16的患者的两倍，那么Carbo/VP16治疗可能是更有效的治疗方法，因为它最大限度地提高了死亡率，将发病率降至最低。这一特殊信息可能对担心住院的卫生政策制定者也很重要，住院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在医学肿瘤学中关于国家指南推荐的标准一线化疗疗法的益处和负担的不可接受的临床信息空白，当它们应用于在常规护理环境中接受治疗的患有ES SCLC的老年Medicare患者时。

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.