Public Health England, 5 St Philips Place, Birmingham, B3 2PW, UK.
Centre for Patient Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Edgbaston, Birmingham, B15 2TT, UK.
Health Qual Life Outcomes. 2018 Jul 5;16(1):133. doi: 10.1186/s12955-018-0951-6.
Primary Sclerosing Cholangitis (PSC) is a rare chronic, cholestatic liver condition in which patients can experience a range of debilitating symptoms. Patient reported outcome measures (PROMs) could provide a valuable insight into the impact of PSC on patient quality of life and symptoms. A previous review has been conducted on the quality of life instruments used in liver transplant recipients. However, there has been no comprehensive review evaluating PROM use or measurement properties in PSC patients' to-date. The aim of the systematic review was to: (a) To identify and categorise which PROMs are currently being used in research involving the PSC population (b) To investigate the measurement properties of PROMs used in PSC.
A systematic review of Medline, EMBASE and CINAHL, from inception to February 2018, was undertaken. The methodological quality of included studies was assessed using the Consensus-based Standards for selection of health Measurement Instruments (COSMIN) checklist.
Thirty-seven studies were identified, which included 36 different PROMs. Seven PROMs were generic, 10 disease-specific, 17 symptom-specific measures and 2 measures on dietary intake. The most common PROMs were the Short form-36 (SF-36) (n = 15) and Chronic liver disease questionnaire (CLDQ) (n = 6). Only three studies evaluated measurement properties, two studies evaluated the National Institute of Diabetes Digestive and Kidney Diseases Liver Transplant (NIDDK-QA) and one study evaluated the PSC PRO; however, according to the COSMIN guidelines, methodological quality was poor for the NIDDK-QA studies and fair for the PSC PRO study.
A wide variety of PROMs have been used to assess health-related quality of life and symptom burden in patients with PSC; however only two measures (NIDDK-QA and PSC PRO) have been formally validated in this population. The newly developed PSC PRO requires further validation in PSC patients with diverse demographics, comorbidities and at different stages of disease; however this is a promising new measure with which to assess the impact of PSC on patient quality of life and symptoms.
原发性硬化性胆管炎（PSC）是一种罕见的慢性、胆汁淤积性肝脏疾病，患者可能会经历一系列使人虚弱的症状。患者报告的结果测量（PROM）可以提供对 PSC 对患者生活质量和症状影响的有价值的见解。以前已经对用于肝移植受者的生活质量工具进行了审查。然而，迄今为止，尚无关于在 PSC 患者中使用 PROM 或测量特性的综合审查。本系统评价的目的是：（a）确定并分类目前在涉及 PSC 人群的研究中使用的 PROM；（b）调查 PSC 中使用的 PROM 的测量特性。
对 Medline、EMBASE 和 CINAHL 进行了系统评价，从最初到 2018 年 2 月。使用共识基础的健康测量仪器选择标准（COSMIN）清单评估纳入研究的方法学质量。
确定了 37 项研究，其中包括 36 种不同的 PROM。7 种 PROM 为通用型，10 种为疾病特异性，17 种为症状特异性测量，2 种为饮食摄入测量。最常用的 PROM 是短形式 36（SF-36）（n=15）和慢性肝病问卷（CLDQ）（n=6）。只有三项研究评估了测量特性，两项研究评估了国家糖尿病、消化和肾脏疾病研究所肝移植（NIDDK-QA），一项研究评估了 PSC PRO；然而，根据 COSMIN 指南，NIDDK-QA 研究的方法学质量较差，PSC PRO 研究的方法学质量较好。
已经使用了多种 PROM 来评估 PSC 患者的健康相关生活质量和症状负担；然而，只有两种措施（NIDDK-QA 和 PSC PRO）在该人群中得到了正式验证。新开发的 PSC PRO 需要在具有不同人口统计学、合并症和不同疾病阶段的 PSC 患者中进一步验证；然而，这是一种有前途的新措施，可以用来评估 PSC 对患者生活质量和症状的影响。
