Zhou Zheng-Yi, Bensink Mark E, Hazra Nisha C, Xu Chunyi, Hendry Bruce, Sharpe Claire C, Zhou Mo
Analysis Group, Boston, MA, USA.
Travere Therapeutics, Inc., San Diego, CA, USA.
Pharmacoecon Open. 2025 Jan;9(1):83-92. doi: 10.1007/s41669-024-00527-1. Epub 2024 Sep 21.
Immunoglobulin A nephropathy (IgAN) is a rare progressive disease that can lead to kidney failure. The current study aimed to estimate health state utility values for IgAN from a UK societal perspective.
We used the time trade-off (TTO) method to derive utility values for various health states in IgAN, defined based on chronic kidney disease (CKD) stage, proteinuria, dialysis, and nephrotic syndrome (CKD stages 1-4, proteinuria < 1 g/day vs ≥ 1 g/day; CKD stage 5, dialysis vs non-dialysis). We developed health state vignettes to describe typical symptoms and quality-of-life impairments of IgAN. Eligible participants from the UK general public completed a computer-assisted telephone interview. Estimated TTO utility values were reviewed against visual analogue scale (VAS)-derived values.
In total, 200 participants were included in the study (mean age, 48.9 years; female, 59.0%). Mean (standard deviation [SD]) utility values were 0.84 (0.17) and 0.71 (0.23) for CKD stage 1/2 with proteinuria < 1 g/day and with proteinuria ≥ 1 g/day, respectively; 0.68 (0.23) and 0.61 (0.25) for CKD stage 3; and 0.55 (0.26) and 0.49 (0.27) for CKD stage 4. Mean (SD) utility of CKD stage 5 with and without dialysis was 0.38 (0.30) and 0.42 (0.28), respectively. The mean (SD) utility value of nephrotic syndrome was 0.43 (0.33).
Our results indicated that various IgAN health states are associated with impaired health status, with substantial utility decrements related to disease progression, elevated proteinuria, and nephrotic syndrome.
免疫球蛋白A肾病(IgAN)是一种罕见的进行性疾病,可导致肾衰竭。本研究旨在从英国社会角度评估IgAN的健康状态效用值。
我们采用时间权衡(TTO)方法得出IgAN各种健康状态的效用值,这些健康状态根据慢性肾脏病(CKD)分期、蛋白尿、透析和肾病综合征来定义(CKD 1-4期,蛋白尿<1g/天与≥1g/天;CKD 5期,透析与非透析)。我们编写了健康状态 vignettes 来描述IgAN的典型症状和生活质量损害。来自英国普通公众的合格参与者完成了一次计算机辅助电话访谈。将估计的TTO效用值与视觉模拟量表(VAS)得出的值进行比较。
本研究共纳入200名参与者(平均年龄48.9岁;女性占59.0%)。CKD 1/2期蛋白尿<1g/天和蛋白尿≥1g/天的平均(标准差[SD])效用值分别为0.84(0.17)和0.71(0.23);CKD 3期为0.68(0.23)和0.61(0.25);CKD 4期为0.55(0.26)和0.49(0.27)。CKD 5期透析和未透析的平均(SD)效用分别为0.38(0.30)和0.42(0.28)。肾病综合征的平均(SD)效用值为0.43(0.33)。
我们的结果表明,IgAN的各种健康状态与健康状况受损相关,疾病进展、蛋白尿增加和肾病综合征会导致效用大幅下降。