United BioSource Corporation, Bethesda, MD, USA.
Health Qual Life Outcomes. 2012 Aug 7;10:92. doi: 10.1186/1477-7525-10-92.
Chronic hepatitis C (CHC) and its treatment are associated with a variety of patient-reported symptoms and impacts. Some CHC symptoms and impacts may be difficult to evaluate through objective clinical testing, and more easily measured through patient self-report. This literature review identified concepts raised by CHC patients related to symptoms, impacts, and treatment effects, and evaluated integration of these concepts within patient-reported outcome (PRO) measures. The goal of this work was to provide recommendations for incorporation of PRO measurement of concepts that are relevant to the CHC experience into CHC clinical trial design.
A three-tiered literature search was conducted. This included searches on concepts of importance, PRO measures used in clinical trials, and existing PRO measures. The PRO Concept Search focused on reviewing issues raised by CHC patients about CHC symptoms, disease impact, and treatment effects. The CHC Trials with PRO Endpoints Search reviewed clinical trials with PRO endpoints to assess differences between treatments over time. The PRO Measure Search reviewed existing PRO measures associated with the concepts of interest.
This multi-tiered approach identified five key concepts of interest: depression/anxiety, fatigue, flu-like symptoms, cognitive function, insomnia. Comparing these five concepts of interest to the PRO measures in published CHC clinical trials showed that, while treatment of CHC may decrease health-related quality of life in a number of mental and physical domains, the PRO measures that were utilized in published clinical trials inadequately covered the concepts of interest. Further review of 18 existing PRO measures of the concepts of interest showed only four of the 18 were validated in CHC populations.
This review identified several gaps in the literature regarding assessment of symptoms and outcomes reported as important by CHC patients. Further research is needed to ensure that CHC clinical trials evaluate concepts that are important to patients and include measures that have evidence supporting content validity, reliability, construct validity, and responsiveness.
慢性丙型肝炎（CHC）及其治疗与各种患者报告的症状和影响有关。一些 CHC 症状和影响可能难以通过客观的临床测试评估，而通过患者自我报告则更容易测量。本文献综述确定了 CHC 患者提出的与症状、影响和治疗效果相关的概念，并评估了这些概念在患者报告的结果（PRO）测量中的整合情况。这项工作的目的是提供有关将与 CHC 体验相关的 PRO 测量概念纳入 CHC 临床试验设计的建议。
进行了三级文献检索。这包括对重要概念、临床试验中使用的 PRO 测量以及现有的 PRO 测量的搜索。PRO 概念搜索重点审查了 CHC 患者提出的关于 CHC 症状、疾病影响和治疗效果的问题。CHC 与 PRO 终点试验搜索审查了具有 PRO 终点的临床试验，以评估随着时间的推移不同治疗方法之间的差异。PRO 测量搜索审查了与感兴趣概念相关的现有 PRO 测量。
这种多层次方法确定了五个感兴趣的关键概念：抑郁/焦虑、疲劳、流感样症状、认知功能、失眠。将这五个感兴趣的概念与已发表的 CHC 临床试验中的 PRO 测量进行比较表明，虽然 CHC 的治疗可能会降低许多身体和心理健康领域的健康相关生活质量，但已发表临床试验中使用的 PRO 测量方法并不能充分涵盖感兴趣的概念。对 18 种现有 PRO 测量感兴趣概念的进一步审查仅显示其中 4 种在 CHC 人群中得到验证。
本综述确定了文献中关于评估 CHC 患者认为重要的症状和结果的几个空白。需要进一步研究以确保 CHC 临床试验评估对患者重要的概念，并包括具有支持内容有效性、可靠性、结构有效性和响应性证据的测量方法。
