Trigg Andrew, Chan Eric, Kitchen Helen, Willgoss Tom, Ho Kai Fai, Pierson Renee, Scott Jane
Formerly of DRG Abacus, Manchester, United Kingdom.
Janssen Global Services, LLC, Raritan, NJ, USA.
J Health Econ Outcomes Res. 2019 Feb 18;6(2):1-19. doi: 10.36469/9675. eCollection 2019.
Hepatitis C virus (HCV) infection and its treatments are associated with significant symptoms, side effects and impact on patients functioning. The Hepatitis C Symptom and Impact Questionnaire version 4 (HCV-SIQv4) was developed according to FDA Patient Reported Outcomes (PRO) Guidance, for evaluating chronic HCV infection and its treatment.
This study evaluated the psychometric properties and clinically important change (CIC) thresholds of the measure.
PRO data were pooled from three Phase IIb and III trials evaluating interferon-free simeprevir-containing regimens for treatment of chronic HCV infection. Scale range adequacy, reliability, validity, responsiveness and CIC thresholds were assessed incorporating knowledge of the appropriate measurement model.
Data from 437 patients were analyzed. Stage of liver disease was associated with symptom severity and functioning at baseline. Reliability was acceptable (test-retest ICC ≥0.7) for most scores except the Gastrointestinal and Integumentary domains. Convergent validity was observed between HCV-SIQv4 scores and concurrent measures of conceptual similarity. Greater symptom severity and worse impact scores were associated with liver cirrhosis, depression, severe fatigue and health limitations. Patients who achieved SVR12 had better outcomes than those failing to. HCV-SIQv4 symptom and domain scores were responsive to changes in health state (effect sizes ≥0.5). Exploratory thresholds for change in scores indicating a clinically important improvement and worsening were HCV-SIQv4 Overall Body System Score (BSS), 8 and 8; Constitutional BSS, 10 and 10; Gastrointestinal BSS, 5 and 5; Psychiatric BSS, 8 and 8; Neurocognitive BSS, 8 and 8; and Integumentary BSS, 5 and 5.
The HCV-SIQv4 offers reliable, responsive assessments within HCV clinical development. CIC thresholds are now available to aid score interpretation.
丙型肝炎病毒（HCV）感染及其治疗会引发显著症状、副作用，并对患者的功能产生影响。丙型肝炎症状与影响问卷第4版（HCV - SIQv4）是根据美国食品药品监督管理局（FDA）患者报告结局（PRO）指南制定的，用于评估慢性HCV感染及其治疗情况。
本研究评估了该量表的心理测量特性及临床重要变化（CIC）阈值。
PRO数据来自三项IIb期和III期试验，这些试验评估了不含干扰素且含西米普明的方案用于治疗慢性HCV感染。结合适当测量模型的知识，评估量表范围的充分性、信度、效度、反应度和CIC阈值。
分析了437例患者的数据。肝病阶段与基线时的症状严重程度和功能相关。除胃肠道和皮肤领域外，大多数评分的信度均可接受（重测组内相关系数≥0.7）。在HCV - SIQv4评分与概念相似性的同期测量之间观察到收敛效度。更高的症状严重程度和更差的影响评分与肝硬化、抑郁、严重疲劳和健康受限相关。实现持续病毒学应答12周（SVR12）的患者比未实现的患者结局更好。HCV - SIQv4症状和领域评分对健康状态变化有反应（效应量≥0.5）。表明临床重要改善和恶化的评分变化的探索性阈值为：HCV - SIQv4全身系统评分（BSS），8分和8分；体质BSS，10分和10分；胃肠道BSS，5分和5分；精神BSS，8分和8分；神经认知BSS，8分和8分；以及皮肤BSS，5分和5分。
HCV - SIQv4在HCV临床研发中提供了可靠、有反应的评估。现在有CIC阈值可帮助解释评分。
