Mohindru Bishal, Turner David, Sach Tracey, Bilton Diana, Carr Siobhan, Archangelidi Olga, Bhadhuri Arjun, Whitty Jennifer A
Norwich Medical School, Norwich Research Park, University of East Anglia, Norwich Norfolk, NR4 7TJ, UK.
Imperial College London, Emmanuel Kaye Building, 1B Manresa Road, London, SW3 6LR, UK.
Pharmacoecon Open. 2020 Mar;4(1):13-25. doi: 10.1007/s41669-019-0144-1.
Cystic fibrosis (CF) is a life-limiting, hereditable condition, with the highest prevalence in Europe. CF treatments have led to improvements in clinical symptoms, disease management and decelerated disease progression. However, little is known about the health state utility (HSU) associated with CF disease states, adverse events, and changes in disease severity. Although HSU data have contributed to existing health economic modelling studies, a lack of such data have been highlighted. This systematic review aims to provide a summary of HSU-related research in CF and highlight related research gaps.
Online searches were performed in six databases and studies in any of the following categories were included: (1) estimation of HSUs in CF; (2) mapping studies between patient-reported outcome measures (PROMs) and HSUs; (3) economic evaluations on the management of CF that report primary HSU data; and (4) any CF clinical trial that reported HSU as an outcome.
A total of 17 studies were reviewed, of which 12 provided HSU values for specific CF populations. The remaining five articles provided HSU data that were broken down by CF relevant health states, including lung transplantations, pulmonary exacerbation (PEx) events and forced expiratory volume in 1 s (FEV).
Current HSU data in CF are limited and there is considerable scope for further research, both in providing HSU values for CF and in investigating methods for HSU elicitation/evaluation in CF populations.
囊性纤维化（CF）是一种危及生命的遗传性疾病，在欧洲发病率最高。CF治疗已使临床症状、疾病管理得到改善，并减缓了疾病进展。然而，对于与CF疾病状态、不良事件及疾病严重程度变化相关的健康状态效用（HSU）知之甚少。尽管HSU数据已用于现有的健康经济模型研究，但此类数据的缺乏也已凸显。本系统评价旨在总结CF中与HSU相关的研究，并突出相关研究空白。
在六个数据库中进行了在线检索，纳入以下任何类别的研究：（1）CF中HSU的估计；（2）患者报告结局指标（PROMs）与HSU之间的映射研究；（3）报告主要HSU数据的CF管理的经济评价；以及（4）任何将HSU作为结局报告的CF临床试验。
共审查了17项研究，其中12项为特定CF人群提供了HSU值。其余五篇文章提供了按CF相关健康状态细分的HSU数据，包括肺移植、肺部加重（PEx）事件和一秒用力呼气量（FEV）。
CF中目前的HSU数据有限，在为CF提供HSU值以及研究CF人群中HSU获取/评估方法方面，都有相当大的进一步研究空间。
