我们的目的是描述临床,经济,囊性纤维化(CF)的社会负担以及CF跨膜传导调节因子(CFTRm)治疗对CF患者的影响,看护者,和医疗保健系统。材料和方法这项回顾性研究使用了来自瑞典国家基于人口的注册和瑞典CF质量注册的相关现实世界数据来评估临床,经济,以及CF中的社会负担和CFTR影响。来自有CF的人和没有CF的十倍对照人群的记录,出生年份,和位置在2019年进行了比较。比较了2018年年龄>6岁开始lumacaftor/ivacaftor(LUM/IVA)的亚组在治疗开始前和治疗后12个月的结果。结果CF患者(n=743)与对照组(n=7406)相比,每年住院和门诊专科医生的就诊次数>10倍。年龄>18岁的人额外缺勤77·7天(95%CI:70·3,85·1),社会成本为11,563欧元(95%CI:10,463,12,662),而年龄<18岁的护理人员又错过了6.1(5.0,7.2)个工作日。用LUM/IVA治疗,CF患者(n=100)的肺功能显着增加(ppFEV1的平均变化[3·8分;95%CI:1·1,6·6]),平均0·5(95%CI:-0·8,-0·2)更少的肺加重和45·2(95%CI:13·3,77·2)更少的抗生素使用天数。年龄<18岁的CF患者的护理人员损失的工作天数减少了5·4天(95%CI:2·9,7·9)。结论在瑞典,CF与较高的临床经济和社会负担有关。在用LUM/IVA治疗的CF患者中观察到的临床状态的改善反映在降低的照顾者和社会负担。
囊性纤维化(CF)是一种由称为CFTR的单个缺陷基因引起的疾病,影响肺部,胰腺,和其他器官。被称为CFTR调节剂的药物有助于改善这种缺陷基因的功能,并已显示出对CF患者的益处。在瑞典,两种这样的药物,lumacaftor和ivacaftor(LUM/IVA),自2018年7月起可用于治疗CF。这项研究着眼于CF对患者的影响,看护者,和医疗保健系统,以及CFTR调节剂的好处。使用瑞典国家医疗保健和社会保险登记处的数据,该研究将2019年的743名CF患者与约7400名没有CF的人进行了比较,与性别相匹配,出生年份,和位置。调查结果显示,患有CF的人的直接医疗成本高出24倍,包括门诊,住院治疗,和CF相关药物,总计23,233欧元。间接成本,例如18岁以上的CF和照顾者缺勤照顾患病儿童的缺勤,是9,629欧元,比一般人口高出五倍。6岁以上接受LUM/IVA治疗的患者肺部健康得到改善,住院人数减少(虽然不明显),需要更少的抗生素。照顾者\'工作缺勤减少,但是CF成年人的缺勤情况没有变化。总的来说,LUM/IVA治疗改善了临床结局,减轻了护理人员和社会的负担.
UNASSIGNED: We aimed to describe the clinical, economic, and societal burdens of cystic fibrosis (CF) and impact of CF transmembrane conductance regulator modulator (CFTRm) treatment on people with CF, caregivers, and healthcare systems.
UNASSIGNED: This retrospective study used linked real-world data from Swedish national population-based registries and the Swedish CF Quality Registry to assess clinical, economic, and societal burden and CFTR impact in CF. Records from people with CF and a ten-fold control population without CF matched by sex, birth year, and location were compared during 2019. Outcomes for a subset aged >6 years initiating lumacaftor/ivacaftor (LUM/IVA) in 2018 were compared 12 months pre- and post-treatment initiation.
UNASSIGNED: People with CF (n = 743) had >10 times more inpatient and outpatient specialist visits annually vs controls (n = 7406). Those aged >18 had an additional 77·7 (95% CI: 70·3, 85·1) days of work absence, at a societal cost of €11,563 (95% CI: 10,463, 12,662), while caregivers of those aged <18 missed an additional 6.1 (5.0, 7.2) workdays. With LUM/IVA treatment, people with CF (n = 100) had significantly increased lung function (mean change in ppFEV1 [3·8 points; 95% CI: 1·1, 6·6]), on average 0·5 (95% CI: -0·8, -0·2) fewer pulmonary exacerbations and 45·2 (95% CI: 13·3, 77·2) fewer days of antibiotics. Days of work lost by caregivers of people with CF aged <18 decreased by 5·4 days (95% CI: 2·9, 7·9).
UNASSIGNED: CF is associated with a high clinical economic and societal burden in Sweden. Improvements in clinical status observed in people with CF treated with LUM/IVA were reflected in reduced caregiver and societal burden.
Cystic fibrosis (CF) is a disease caused by a single faulty gene called CFTR, which affects the lungs, pancreas, and other organs. Medications known as CFTR modulators help improve the function of this faulty gene and have shown benefits for people with CF. In Sweden, two such medicines, lumacaftor and ivacaftor (LUM/IVA), have been available since July 2018 for treating CF. This study looks at the impact of CF on patients, caregivers, and the healthcare system, as well as the benefits of CFTR modulators. Using data from Swedish national healthcare and social insurance registries, the study compared 743 people with CF in 2019 to about 7400 people without CF, matched by sex, birth year, and location. The findings show that people with CF had 24 times higher direct healthcare costs, including outpatient visits, hospitalizations, and CF-related medications, totaling 23,233 Euros. Indirect costs, such as work absences for those over 18 with CF anssd caregivers’ absences to care for sick children, were 9,629 Euros, which is five times higher than the general population. Those over 6 years old treated with LUM/IVA showed improved lung health, reduced hospitalizations (though not significantly), and needed fewer antibiotics. Caregivers’ work absences decreased, but there was no change in work absences for adults with CF. Overall, treatment with LUM/IVA improved clinical outcomes and reduced the burden on caregivers and society.