Burke Tom, Shaikh Anum, Ali Talaha M, Li Nanxin, Curtis Randall, Garcia Diego Daniel-Anibal, Recht Michael, Sannie Thomas, Skinner Mark, O'Hara Jamie
HCD Economics, Daresbury, Cheshire, UK.
Faculty of Health and Social Care, University of Chester, Chester, Cheshire, UK.
J Med Econ. 2022 Jan-Dec;25(1):386-392. doi: 10.1080/13696998.2022.2049552.
Gene therapy trials aim to provide a functional cure for patients with haemophilia B (HB), and treatment impact is analyzed by factor IX expression levels (FELs). We investigated the relationship of FELs with health-related quality of life (HRQoL) and costs.
This was a retrospective cross-sectional analysis of the European (CHESS I-II) and US (CHESS-US) CHESS population studies. Physicians recruited consecutive patients and extracted information from the medical records; patients completed questionnaires between 2014 and 2015 (CHESS-I), 2018-2019 (CHESS-II) and 2019 (CHESS US). Patients with inhibitors were excluded. HRQoL was assessed using the EQ-5D-5L. Twelve-month haemophilia-related direct medical costs included office visits and hospitalizations based on country-level unit costs. A Tobit model was used to analyze FELs and HRQoL and generalized linear models for direct medical costs.
A total of 191 men with HB completed the EQ-5D questionnaire; the mean age was 36.8 years, with a mean FEL of 10.1 IU/dL (median, 4.0). Mean EQ-5D was 0.77 (SD, 0.23). The Tobit model adjusting for age, body mass index and blood-borne viruses showed every 1% increase in FEL was associated with +0.006 points in the mean EQ-5D score ( = .003). Mean haemophilia-related direct medical costs excluding factor replacement therapy were €2,028/year (median, €919) in CHESS I-II (EU, = 226), and $7,171/year (median, $586) in CHESS US ( = 181). Adjusted EU and US models showed every 1% increase in FEL was associated with a decrease in haemophilia-related direct medical costs of €108/year and $529/year, respectively.
Direct medical costs were based on physician extraction of encounters from medical records, potentially underestimating costs of care. The voluntary nature of participation may have introduced selection biases.
We observed a significant association of increases in FEL with increased HRQoL and decreased costs in Europe and the United States among men with HB and no inhibitors.
基因治疗试验旨在为B型血友病（HB）患者提供功能性治愈方法，并通过因子IX表达水平（FELs）分析治疗效果。我们研究了FELs与健康相关生活质量（HRQoL）及成本之间的关系。
这是一项对欧洲（CHESS I-II）和美国（CHESS-US）CHESS人群研究的回顾性横断面分析。医生招募连续的患者并从病历中提取信息；患者在2014年至2015年（CHESS-I）、2018年至2019年（CHESS-II）和2019年（CHESS US）期间完成问卷调查。有抑制剂的患者被排除。使用EQ-5D-5L评估HRQoL。12个月的血友病相关直接医疗成本包括基于国家层面单位成本的门诊就诊和住院费用。使用Tobit模型分析FELs和HRQoL，使用广义线性模型分析直接医疗成本。
共有191名HB男性完成了EQ-5D问卷；平均年龄为36.8岁，平均FEL为10.1 IU/dL（中位数为4.0）。平均EQ-5D为0.77（标准差为0.23）。调整年龄、体重指数和血源病毒后的Tobit模型显示，FEL每增加1%，平均EQ-5D得分增加0.006分（P = 0.003）。在CHESS I-II（欧盟，n = 226）中，不包括因子替代疗法的平均血友病相关直接医疗成本为每年2,028欧元（中位数为919欧元），在CHESS US（n = 181）中为每年7,171美元（中位数为586美元）。调整后的欧盟和美国模型显示，FEL每增加1%，分别与血友病相关直接医疗成本每年降低108欧元和529美元相关。
直接医疗成本基于医生从病历中提取的诊疗信息，可能低估了护理成本。参与的自愿性质可能引入了选择偏倚。
我们观察到在欧洲和美国，对于无抑制剂的HB男性，FEL的增加与HRQoL的提高和成本的降低存在显著关联。
