Prospective Comparative Effectiveness Trial for Malignant Bowel Obstruction

恶性肠梗阻的前瞻性比较有效性试验

• 批准号: 8930994
• 负责人: Robert Scott Krouse
• 金额: $ 7.32万
• 依托单位: UNIVERSITY OF ARIZONA
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-09-30 至 2016-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8930994
• 关键词: Prospective; Comparative; Effectiveness; Trial; Malignant

DESCRIPTION (provided by applicant): Patients with advanced cancer face challenges affecting quality of life (QOL). As the National Cancer Policy Board has identified, research addressing the needs of patients with incurable cancers is severely deficient and effectiveness has rarely been displayed by prospective trials. Malignant bowel obstruction (MBO) is a common problem for advanced cancer patients that involves considerable suffering. There are two major treatment approaches for MBO: surgical management and medical management with specific classes of medications. There is little evidence-based information to choose the optimal clinical care for most patients with a MBO. Goals for these patients may include the ability to eat, relief of nausea, pain control, being at home, and along with an increase in survival. Treatments may improve QOL outcomes in many, but there is no consistent measure defining improvement. The aim of this study is to compare the current treatment algorithms of surgical and non-surgical approaches to MBO in terms of minimizing days in hospital and improving QOL. This will be a five-year multi-site prospective comparative effectiveness study of patients who are admitted to the hospital with an MBO. We have devised a mixed-design study of a smaller randomized population and a larger observational cohort to ensure validity of observational results while enabling adequate accrual. To accomplish this goal we will need to accrue 180 patients (50 in randomized sub-study, 130 in observational study). The preponderance of MBO patients will be accrued to be prospectively followed utilizing general modeling strategy along with propensity score techniques to compare treatment approaches. In addition, we will have a smaller subset of patients who will be randomized to surgical versus non-surgical interventions. Our data collection strategies are designed to minimize patient burden by this alternate research approach with clinically meaningful outcome measures as well as limited health-related quality of life surveys. The primary inclusion criteria for the study are whether a surgical option is being considered in the setting of MBO and there is equipoise to outcomes. The interventions to be compared are an initial surgical versus a non-surgical approach for MBO. Data forms will include demographic and disease history, hospital stay, days with NG tubes, TPN and other hydration, ability to eat or drink with dietary histories, complications, recurrence, total hospital days, and survival. In addition, research coordinators will serially acquire subjective QOL data from the MD Anderson Symptom Inventory - Gastrointestinal, which should not take more than 10 minutes to complete. The primary outcome measure will be "good days", defined as being at home (out of the hospital) and alive. The difference we hope to find is two weeks in a 90 day span (20% difference). Secondary endpoints include days with NG tube, days of intravenous hydration, days eating solid foods, days drinking, and amount of intake, recurrence, morbidity, and survival. We will include a cost analysis plan as well. We anticipate results to be clinically useful to practitioners caring for MB patients.

描述（由申请人提供）：晚期癌症患者面临影响生活质量 (QOL) 的挑战。正如国家癌症政策委员会所指出的那样，针对无法治愈的癌症患者的需求的研究严重不足，并且前瞻性试验很少显示其有效性。恶性肠梗阻（MBO）是晚期癌症患者的常见问题，会给患者带来巨大的痛苦。 MBO 有两种主要的治疗方法：手术治疗和使用特定类别药物的内科治疗。几乎没有基于证据的信息来为大多数 MBO 患者选择最佳的临床护理。这些患者的目标可能包括能够进食、缓解恶心、控制疼痛、待在家里以及提高生存率。治疗可能会改善许多人的生活质量，但没有一致的衡量标准来定义改善。本研究的目的是比较当前 MBO 的手术和非手术方法的治疗算法，以最大限度地减少住院天数和改善生活质量。这将是一项针对因 MBO 入院的患者进行的为期五年的多中心前瞻性比较有效性研究。我们设计了一项针对较小随机人群和较大观察队列的混合设计研究，以确保观察结果的有效性，同时实现足够的应计。为了实现这一目标，我们需要招募 180 名患者（50 名患者进行随机子研究，130 名患者进行观察性研究）。将利用一般建模策略和倾向评分技术来前瞻性地跟踪 MBO 患者的优势，以比较治疗方法。此外，我们将有一小部分患者将被随机分配接受手术干预和非手术干预。我们的数据收集策略旨在通过这种替代研究方法、具有临床意义的结果测量以及有限的健康相关生活质量调查来最大程度地减少患者负担。该研究的主要纳入标准是 在 MBO 的情况下是否正在考虑手术选择以及结果是否平衡。所比较的干预措施是 MBO 的初始手术与非手术方法。数据表格将包括人口统计和疾病史、住院时间、NG 管天数、TPN 和其他水合作用、饮食能力和饮食史、并发症、复发、总住院天数和生存率。此外，研究协调员将从 MD 安德森症状清单 - 胃肠道连续获取主观生活质量数据，完成时间不应超过 10 分钟。主要结果衡量标准是“好日子”，定义为在家（出院）并且还活着。我们希望找到 90 天跨度内的两周差异（20% 差异）。次要终点包括使用 NG 管的天数、静脉补液的天数、吃固体食物的天数、饮酒的天数以及摄入量、复发、发病率和生存率。我们还将包括成本分析计划。我们预计结果对治疗 MB 患者的从业者有临床上的帮助。