Prospective Comparative Effectiveness Trial for Malignant Bowel Obstruction

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确定的那样，针对无法治愈的癌症患者需求的研究严重不足，前瞻性试验很少显示有效性。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，选择最佳临床护理的循证信息很少。这些患者的目标可能包括进食的能力，缓解恶心，疼痛控制，在家，以及沿着生存率的增加。治疗可以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的手术和非手术方法来MBO的治疗算法在最大限度地减少住院天数和提高生活质量。这将是一项为期5年的多中心前瞻性比较有效性研究，对象是因MBO入院的患者。我们设计了一项混合设计的研究，包括一个较小的随机人群和一个较大的观察队列，以确保观察结果的有效性，同时实现足够的累积。为了实现这一目标，我们需要招募180例患者（随机子研究中50例，观察性研究中130例）。将累积MBO患者的优势，并使用一般建模策略沿着倾向评分技术进行前瞻性随访，以比较治疗方法。此外，我们将有一个较小的患者子集，他们将被随机分配到手术与非手术干预组。我们的数据收集策略旨在通过这种具有临床意义的结局指标以及有限的健康相关生活质量调查的替代研究方法来最大限度地减少患者负担。本研究的主要入选标准为 在MBO的背景下是否考虑手术选择，以及结果是否平衡。要比较的干预措施是MBO的初始手术与非手术方法。数据表将包括人口统计学和病史、住院时间、NG管、TPN和其他水合作用的天数、饮食史、并发症、复发、总住院天数和生存期。此外，研究协调员将从MD安德森症状量表-胃肠道连续获取主观QOL数据，完成时间不应超过10分钟。主要结局指标将是“好日子”，定义为在家（出院）和活着。我们希望找到的差异是90天内的两周（20%的差异）。次要终点包括NG管天数、静脉补液天数、进食固体食物天数、饮酒天数、摄入量、复发率、发病率和生存率。我们还将制定成本分析计划。我们预计结果将在临床上有用的从业人员照顾MB患者。

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.