Risk and Risk Preferences for Surgery and Medical Therapy in Ulcerative Colitis

溃疡性结肠炎手术和药物治疗的风险和风险偏好

• 批准号: 8088163
• 负责人: Meenakshi Bewtra
• 金额: $ 15.26万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-08-20 至 2014-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8088163
• 关键词: Abdominal Abscess; Address; Admission activity; Adverse event; Affect; Age; Algorithms; Anti-Tumor Necrosis Factor Therapy; Anus; Biological Response Modifier Therapy; Calcineurin inhibitor; Cessation of life; Chronic; Clinical; Colectomy; Colon; Comorbidity; Complication; Consult; Counseling; Cox Proportional Hazards Models; Data; Databases; Decision Making; Diagnosis; Disease; Evaluation; Excision; Exposure to; Failure; Feces; Flare; Frequencies; Future; Gender; General Population; Guidelines; Hospitals; Ileal Reservoirs; Ileostomy; Immune system; Immunomodulators; Immunosuppressive Agents; Infection; Inflammatory; Inflammatory disease of the intestine; Intestines; Life; Logistic Regressions; Lymphoma; Malignant Neoplasms; Marketing; Medical; Medicare/Medicaid; Mesalamine; Methodology; Methods; Morbidity - disease rate; Operative Surgical Procedures; Outcome; Patient Preferences; Patients; Pattern; Peritonitis; Pharmaceutical Preparations; Physicians; Pneumonia; Population Study; Positioning Attribute; Postoperative Period; Quality of life; Refractory; Relapse; Relative (related person); Research Personnel; Resort; Rest; Risk; Sample Size; Sepsis; Surgeon; Therapeutic; Time; Toxic effect; Ulcerative Colitis; United Kingdom; United States; Urinary tract infection; Wound Infection; analog; base; disorder control; effective therapy; exhaust; experience; high risk; improved; mortality; natalizumab; preference; prevent; programs; public health relevance; sex; thiopurine; willingness; wound

DESCRIPTION (provided by applicant): Ulcerative colitis (UC) is associated with significant morbidity and mortality. While removal of the colon is curative, most feel surgery to be a treatment of last resort and treatment paradigms for UC have shifted to emphasize an escalation of potent immunosuppressant therapy during disease flares before going to surgery. These medications come [sic] some significant potential risks including serious infections, lymphoma, medication failure, and death. An Increasing number of UC patients with moderate to severe disease have life-long exposure to these medications; and in those in whom medical therapy fails, these patients have this medication exposure prior to surgery. This carries the risk of increased mortality and post-operative surgical morbidity as a result of this exposure. An additional key component of treatment decisions is the preferences of the patients themselves. If faced with an appropriate representation of different treatment options and risks, UC patients may have very different willingness to accept certain risks in exchange for potential therapeutic benefit. Studies evaluating morbidity and mortality issues related to immunosuppressant therapy have suffered from lack of generalizability, lack of control for confounders, and lack of power due to small sample sizes. This study proposes utilizing the Medicaid-Medicare database, which assure generalizability, sample size, and allows evaluation of a number of potential confounders including medication use. Cox proportional hazards models with adjustment will be used to determine relative survival of UC patients treated with medical and surgical therapy. To further control for confounding on the basis of channeling, matching on propensity scores will be employed. Multivariate logistic regression will determine the relationship between medical exposure prior to surgery and post-operative morbidity. Additional analysis will evaluate the impact on the odds of morbidity of time from last exposure of medical therapy. Finally, by using conjoint analysis, the study will perform a systematic examination of UC patients' risk preferences for medical versus surgical therapy for UC. PUBLIC HEALTH RELEVANCE: It is crtical [sic] to determine the mortality and morbidity risk associated with the use of medical therapy in preference of surgery. If delaying or avoiding surgery exposes UC patients to an increased mortality or post-operative morbidity, such information is critical in informed decision-making. In turn, understanding how UC patients perceive and value the risks of medical versus surgical therapy will have far-reaching effects on management and treatment and will set new thresholds for future therapeutic options.

描述（由申请人提供）： 溃疡性结肠炎（UC）与显著的发病率和死亡率相关。虽然切除结肠是治愈性的，但大多数人认为手术是最后的治疗手段，UC的治疗模式已经转向强调在手术前疾病发作期间加强强效免疫抑制剂治疗。这些药物有一些重大的潜在风险，包括严重感染、淋巴瘤、药物治疗失败和死亡。越来越多的中度至重度UC患者终生暴露于这些药物;在药物治疗失败的患者中，这些患者在手术前暴露于这种药物。由于这种暴露，这带来了死亡率和术后手术发病率增加的风险。治疗决策的另一个关键组成部分是患者自己的偏好。如果面对不同治疗选择和风险的适当表示，UC患者可能会有非常不同的意愿接受某些风险，以换取潜在的治疗获益。评估免疫抑制剂治疗相关发病率和死亡率问题的研究缺乏普遍性，缺乏对混杂因素的控制，以及由于样本量小而缺乏效力。本研究建议使用医疗补助-医疗保险数据库，该数据库确保了普遍性、样本量，并允许评估包括药物使用在内的许多潜在混杂因素。将使用校正后的考克斯比例风险模型确定接受内科和外科治疗的UC患者的相对生存期。为了进一步控制基于通道的混淆，将采用倾向评分匹配。多变量逻辑回归将确定手术前医疗暴露与术后发病率之间的关系。其他分析将评价自末次暴露于药物治疗的时间对发病率的影响。最后，通过联合分析，本研究将对UC患者对药物与手术治疗UC的风险偏好进行系统性检查。 公共卫生关系：确定与优先采用药物治疗而非手术治疗相关的死亡率和发病率风险至关重要。如果延迟或避免手术使UC患者的死亡率或术后发病率增加，则此类信息对于知情决策至关重要。反过来，了解UC患者如何看待和评价药物与手术治疗的风险将对管理和治疗产生深远的影响，并将为未来的治疗选择设定新的阈值。