Prospective Comparative Effectiveness Trial for Malignant Bowel Obstruction

描述(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确定的那样，针对不治之症患者需求的研究严重不足，前瞻性试验很少显示出有效性。恶性肠梗阻(MBO)是晚期癌症患者的常见问题，涉及相当大的痛苦。MBO有两种主要的治疗方法：手术治疗和药物治疗。对于大多数MBO患者来说，几乎没有循证信息来选择最佳的临床护理。这些患者的目标可能包括进食能力、缓解恶心、疼痛控制、在家生活，以及增加存活率。治疗可能会改善许多人的生活质量结果，但没有一致的衡量标准来定义改善。本研究的目的是比较目前手术和非手术治疗MBO的方法在减少住院天数和改善生活质量方面的作用。这将是一项为期五年的多部位前瞻性比较有效性研究，研究对象是因MBO入院的患者。我们设计了一项混合设计的研究，包括较小的随机总体和较大的观察队列，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，我们将需要增加180名患者(50名随机分组研究，130名观察性研究)。MBO患者的优势将被累积起来，利用一般的建模策略和倾向评分技术来比较治疗方法。此外，我们将有一小部分患者被随机分为手术和非手术干预两组。我们的数据收集策略旨在通过这种具有临床意义的结果测量和有限的与健康相关的生活质量调查的替代研究方法将患者负担降至最低。这项研究的主要纳入标准是 在MBO的设置中是否正在考虑手术选择，以及是否存在对结果的平衡。要比较的干预措施是MBO的初始手术方法和非手术方法。数据表格将包括人口统计和疾病史、住院天数、使用NG管、TPN和其他水合作用的天数、有饮食史的饮食能力、并发症、复发、总住院天数和生存率。此外，研究协调员将从MD Anderson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。主要的结果衡量标准将是“好日子”，定义为在家(医院外)和活着。我们希望发现的差异是90天跨度中的两周(差异20%)。次要终点包括使用NG管的天数、静脉补液天数、吃固体食物的天数、饮酒天数、摄入量、复发、发病率和存活率。我们还将包括一份成本分析计划。我们期望研究结果对治疗MB患者的从业者有临床应用价值。

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.