Groen Henk, Neelis Esther G, Poley Marten J, Olieman Joanne F, Scheenstra René, Krabbe Paul Fm, Dijkstra Gerard, Rings Edmond Hhm
Departments of Epidemiology and.
Departments of Pediatric Gastroenterology and
Am J Clin Nutr. 2017 Feb;105(2):417-425. doi: 10.3945/ajcn.116.135160. Epub 2017 Jan 4.
Children with intestinal failure (IF) depend on parenteral nutrition (PN). The goal in the treatment of IF is to wean children off PN through intestinal rehabilitation (IR). Although the healthcare burden of IF is enormous, to our knowledge there has been no previous cost-effectiveness analysis in pediatric IF including IR.
We sought to determine the cost-effectiveness of IR in terms of costs and life-years.
We simulated the treatment of IF in children in a discrete-event model. Data for this model were derived from patient records, the Dutch Registry of Intestinal Failure and Transplantation, the Intestinal Transplant Registry, and the literature. The time horizon of the model was 40 y. Simulated patients were enrolled at a rate of 40 patients/mo for 10 y. Actual costs were calculated for hospital admissions, surgical interventions, endoscopies, PN, and immunosuppressive medication. We evaluated the cost-effectiveness of IR by comparing 1 scenario with IR with 1 scenario without IR. In the scenario with IR, a proportion of patients who represented those with the ability to wean off PN were assigned to IR. In the scenario without IR, all patients progressed to home PN (HPN). In both scenarios, a proportion of patients receiving HPN were eventually eligible for an intestinal transplantation.
IR prolonged survival; the mean number of life-years per patient was 19.4 in the scenario with IR compared with 18.2 in the scenario without IR. Average total costs per patient were €819,292 in the scenario with IR compared with €1,176,830 in the scenario without IR (equivalent to 1,129,230 US$ and 1,622,025 US$, respectively, in January 2014); costs mainly included hospital admissions and PN.
On the basis of our simulations, we concluded that IR improved the survival of children with IF and was associated with cost savings. Therefore, we consider IR to be a cost-effective treatment for children with IF.
肠衰竭(IF)患儿依赖肠外营养(PN)。IF治疗的目标是通过肠道康复(IR)使患儿脱离PN。尽管IF的医疗负担巨大,但据我们所知,此前尚无针对包括IR在内的儿科IF的成本效益分析。
我们试图从成本和生命年的角度确定IR的成本效益。
我们在一个离散事件模型中模拟了儿童IF的治疗。该模型的数据来自患者记录、荷兰肠衰竭与移植登记处、肠移植登记处以及文献。模型的时间跨度为40年。模拟患者以每月40例的速度入组,共10年。计算了住院、手术干预、内镜检查、PN和免疫抑制药物的实际成本。我们通过比较有IR的一种方案和无IR的一种方案来评估IR的成本效益。在有IR的方案中,将一部分代表有能力脱离PN的患者分配到IR组。在无IR的方案中,所有患者都进展为家庭肠外营养(HPN)。在两种方案中,一部分接受HPN的患者最终符合肠移植条件。
IR延长了生存期;有IR方案中每名患者的平均生命年数为19.4,而无IR方案中为18.2。有IR方案中每名患者的平均总成本为819,292欧元,无IR方案中为1,176,830欧元(分别相当于2014年1月的1,129,230美元和1,622,025美元);成本主要包括住院和PN。
基于我们的模拟,我们得出结论,IR提高了IF患儿的生存率并节省了成本。因此,我们认为IR是治疗IF患儿的一种具有成本效益的方法。