a Department of Pharmacology , Poona College of Pharmacy , Pune , India.
Ren Fail. 2018 Nov;40(1):107-118. doi: 10.1080/0886022X.2018.1427110.
Hemolytic uraemic syndrome (HUS) is progressive renal failure disease and determination of their quality of life (QoL) on the basis of patient-reported outcomes (PROs) are becoming increasingly important in the economic evaluations for its treatment with eculizumab (ECU).
To perform the systematic evaluation of QoL in HUS patients treated with ECU on the basis of Evaluating Measures of Patient Reported Outcomes (EMPRO) tool.
A systematic review was conducted in PubMed, EMBASE, the Cochrane Library, CINAHL and Google Scholar till September 2016 by two independent researchers. Each identified instrument was evaluated for its quality of performance by using the EMPRO tool for its overall score and seven attribute specific scores (range 0-100, worst to best).
Five different PROs instruments were identified from 10 articles (n = 112) which showed eculizumab significantly improves health-related quality of life (HRQOL) in atypical HUS (aHUS) patients. Amongst five instruments viz. EuroQol five dimensions questionnaire (EQ-5 D), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), Headache Impact Test-6 (HIT-6), 36-Item Short Form Health Survey (SF-36) and Visual Analogue Scale (VAS), the overall EMPRO score was higher for VAS (73.83) and EQ-5 D (73.81). Whereas, FACIT-F and HIT- 6 were just able to meet the minimal threshold of EMPRO scoring (50.24 and 59.09, respectively).
Evidence from present investigation support that eculizumab significantly improves HRQoL in patients with aHUS furthermore, EQ-5 D and VAS instrument should be recommended for assessing HRQoL in them. However, selection of PRO instrument for determination of QoL in HUS entirely depend upon the study requirements.
溶血尿毒综合征（HUS）是一种进行性肾衰竭疾病，基于患者报告的结果（PROs）来确定其生活质量（QoL）在其依库珠单抗（ECU）治疗的经济评估中变得越来越重要。
使用Evaluating Measures of Patient Reported Outcomes（EMPRO）工具，对依库珠单抗治疗 HUS 患者的 QoL 进行系统评价。
两位独立研究人员于 2016 年 9 月前在 PubMed、EMBASE、Cochrane 图书馆、CINAHL 和 Google Scholar 中进行了系统评价。使用 EMPRO 工具对每个确定的工具进行性能质量评估，包括总体得分和七个属性特定得分（范围 0-100，最差到最好）。
从 10 篇文章（n=112）中确定了 5 种不同的 PROs 工具，结果表明依库珠单抗可显著改善非典型溶血尿毒综合征（aHUS）患者的健康相关生活质量（HRQoL）。在 5 种工具中，EuroQol 五维度问卷（EQ-5D）、慢性疾病治疗功能评估-疲劳（FACIT-F）、头痛影响测试-6（HIT-6）、36 项简短健康调查问卷（SF-36）和视觉模拟量表（VAS），VAS（73.83）和 EQ-5D（73.81）的总体 EMPRO 得分较高。然而，FACIT-F 和 HIT-6 仅能够达到 EMPRO 评分的最低阈值（分别为 50.24 和 59.09）。
目前的研究结果支持依库珠单抗可显著改善 aHUS 患者的 HRQoL，此外，EQ-5D 和 VAS 仪器应推荐用于评估其 HRQoL。然而，HUS 中 QoL 的测定选择 PRO 仪器完全取决于研究要求。
