Harms of Hepatocellular Carcinoma Surveillance in Patients With Cirrhosis
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 2,871
- 试验地点
- 1
- 主要终点
- Physical Harms
研究概览
简要总结
This study leverage a multi-center randomized controlled trial assessing screening-related benefits (i.e. early tumor detection, treatment eligibility, and overall survival) among a racially and socioeconomically diverse population of patients with cirrhosis. However, the randomized controlled trial was not budgeted to assess hepatocellular carcinoma screening-related harms. The goal of this study is to quantify physical, financial, and psychosocial harms across three healthcare settings.
详细描述
Leveraging a multi-center randomized controlled trial assessing screening benefits in a socioeconomically and racially diverse population of patients with cirrhosis followed in 3 healthcare settings over a 4-year period, this study aims to:
Aim 1: Assess the effect of hepatocellular carcinoma screening on a) physical harms due to follow-up tests, b) financial harms, and c) overdiagnosis in patients with severe liver dysfunction or comorbid illness, through electronic medical record data, manual chart review, and validated survey measures.
Aim 2: Assess the effect of hepatocellular carcinoma screening on screening-related psychosocial harms, e.g. cancer-specific worry, situational anxiety, mood disturbances, and decisional regret, through longitudinal validated measures and qualitative interviews.
Aim 3: Create and disseminate a balance sheet of benefits and harms to inform patients, providers, healthcare organizations, payers, and policymakers about the value of hepatocellular carcinoma screening in patients with cirrhosis.
Over a 4-year period, electronic medical record data will be used to compare screening-related physical and financial harms between patients undergoing and those not undergoing hepatocellular carcinoma screening. Psychosocial harms, as ascertained through longitudinal measurement of validated survey instruments and qualitative interviews, will be compared between patients with positive or indeterminate screening results and those with negative results or without any screening. Mixed-effect regression analysis will be used to determine if screening harms differed by factors at multiple levels including patient (e.g. degree of liver dysfunction), provider (e.g. subspecialty training), and healthcare organization (e.g. access to liver transplantation). This study will seamlessly complement data from the parent randomized controlled trial. By immediately translating these high quality data about hepatocellular carcinoma screening benefits and harms into a balance sheet, the investigators will facilitate patient-provider discussions, inform payer decisions about reimbursement, and guide policy decisions. These data are also crucial to identify modifiable and high-yield intervention targets and strategies to reduce hepatocellular carcinoma screening harms in the future.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 21 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult patients (≥ 21 years old)
- •Cirrhosis
- •Outpatient visit in year prior to randomization
- •English or Spanish speaking
排除标准
- •History of hepatocellular carcinoma
- •History of liver transplantation
- •Child Pugh C cirrhosis
- •Significant comorbid conditions with life expectancy < 1 year, (e.g., extrahepatic malignancy)
结局指标
主要结局
Physical Harms
时间窗: 4 Years
Physical harms (contrast injury, radiation exposure, and biopsy complications) can result from screening or follow-up testing and extends beyond medical complications to include discomfort. A binary outcome (harm vs. no harm) will be defined for each person and each type of physical harm (contrast injury, radiation exposure, biopsy, and any physical harm). We will report the point estimate and 95% confidence interval for the proportion of patients with each type of harm in each arm, stratified by health system. Using an intention-to-treat principle, we will use Chi squared test to compare the proportion of patients with physical harms between the screening and usual care arms, with a secondary analysis stratified by health system. We will also perform a sensitivity analysis based on test intent, in which we will only include tests (and corresponding harms) performed as a direct result of hepatocellular carcinoma screening.
Financial Harms
时间窗: 4 Years
Financial harms may include anticipated or real costs of hepatocellular carcinoma screening and diagnostic evaluation including, indirect costs such as missed work, and opportunity costs such as distraction from other health-related activities. Financial harms will be summarized for each arm using descriptive analyses as average and range of costs per person. Degree of financial harms will be compared between the hepatocellular carcinoma screening and usual care arms using Student T test, with a secondary analysis stratified by health system. In a secondary analysis, a mixed-effect model approach will be employed to identify patient-, provider- and system-level factors associated with financial harm.
Overdiagnosis
时间窗: 4 Years
Defined as hepatocellular carcinoma diagnoses that are unlikely to have an effect on mortality, specifically among patients with: 1) significant comorbid conditions or 2) severe liver dysfunction, i.e. Child Pugh C cirrhosis, who are not candidates for liver transplantation, at hepatocellular carcinoma diagnosis. For the primary analysis, Chi squared test will be used to compare the proportion of patients with overdiagnosis between the screening arm and usual care arm, stratified by health system.
Psychosocial Harms
时间窗: 4 Years
Patients will be divided into 4 categories: true positives, false positives, true negatives, and no screening. True positives will be defined as those who develop hepatocellular carcinoma within 6 months of the screening test; false positives as those who remain without hepatocellular carcinoma diagnosis during 6 months of follow-up; and true negatives as those with normal screening tests and without hepatocellular carcinoma diagnosis during 6 months of follow-up. Psychosocial harms (cancer-specific worry, situational anxiety, mood disturbances, and decisional regret) will be defined by change in survey scores from baseline and will be calculated at 1 month and 6 months for each patient.
次要结局
- Patient-, Provider-, and System-level Factors Associated with Physical Harms(4 Years)
- Patient-, Provider-, and System-level Factors Associated with Psychosocial Harms(4 Years)
- Physical Harms(12 Months)
研究者
Amit Singal
Associate Professor
University of Texas Southwestern Medical Center