血液恶性肿瘤儿童预防移植物抗宿主病策略的比较药物经济学分析结果:单中心经验

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引用次数: 0

摘要

尽管造血干细胞移植在肿瘤血液学实践中的应用非常广泛,但围绕同种异体造血干细胞移植的成本、患者随访、计算方法和成本结构的讨论仍然是一个未解决的问题。讨论是复杂的使用各种修改治疗方案关于准备手术的病人和预防并发症。本文的目的是比较两种最常用的技术平台- TCRαβ/CD19消耗和高剂量环磷酰胺“体内”消耗的临床和经济效率,并在一个中心的实际实践中研究了这些方法的特点。材料与方法。为了进行评估,选择了两组“配对”的儿童,这些儿童在2013年5月至2021年1月期间在俄罗斯卫生部以德米特里·罗加乔夫(Dmitry Rogachev)命名的国家儿童血液学、肿瘤学和免疫学医学研究中心根据其中一种方法进行了造血移植。根据获得的临床疗效指标(采用非参数Mann-Whitney检验检验差异的显著性),基于马尔可夫模型进行药物经济学建模,确定成本结构,进行药物经济学分析“成本-效果”,增量分析“成本-效果”,并计算5年后考虑折现的手术成本指标。总生存期和无事件生存期,以及无移植物抗宿主病发展和复发的生存期作为终点。费用分为两个阶段:第一阶段-移植后30天,第二阶段-移植后31天至1年。成本分为直接成本和间接成本。对于间接成本,取现有社会经济数据的平均值。研究结果:两个平台的两个阶段的总成本是相当的:TCRa/b/CD19删除- 6702.094万卢布;7322566.1万卢布。各个阶段的成本分布不均匀:TCR平台的第一阶段成本更高,PtCy平台的第二阶段成本更高。在成本效益分析中,每个单位的效率(每1年1%的存活率)将使PtCy平台的成本更高。鉴于世卫组织建议的每年3%的折扣系数,在5年内,TCR方法的年成本应降至5933316万卢布,而PtСy平台的年成本应降至6,48535.5万卢布,不考虑宏观经济指标。TCR每增加一个单位的效率(1%的存活率)将花费:无GVHD的存活率为207 855.67卢布,复发率为51 963.92卢布。结论。与使用高剂量环磷酰胺的“体内”耗竭平台相比,预防性TCR平台在1年治疗期间的药物经济学成本更低,尽管在治疗的第一阶段使用成本更高。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
RESULTS OF A COMPARATIVE PHRMACOECONOMICS ANALYSIS OF STRATEGIES FOR THE PREVENTION OF GRAFT-VERSUS-HOST DISEASE IN CHILDREN WITH HEMATOLOGICAL MALIGNANCES: A SINGLE CENTER EXPERIENCE
The discussion around the cost of allogeneic hematopoietic stem cell transplantation and follow-up of patients, methods of calculation and cost structure, despite the breadth of application of hematopoietic stem cell transplantation in oncohematological practice, remains an unresolved problem. The discussion is complicated using various modifications of therapeutic options regarding the preparation of the patient for surgery and the prevention of complications. The purpose of this article is a comparative assessment of the clinical and economic efficiency of the two most used technological platforms – TCRαβ/CD19 depletion and ‘’in vivo’’ depletion with high doses of cyclophosphamide, having studied the features of each of these methods in the real practice of one center. Materials and Methods. For evaluation, two “match paired” groups of children were selected, who underwent transplantation according to one of the methods for hemoblastosis in the period from May 2013 to January 2021 at the National Medical Research Center for Pediatric Hematology, oncology and immunology named after Dmitry Rogachev" of the Ministry of Health of Russia. Based on the obtained indicators of clinical effectiveness (significance of differences was made using the nonparametric Mann-Whitney test), pharmacoeconomic modeling was carried out on the basis of the Markov model, the cost structure was determined and the pharmacoeconomic analysis "cost-effectiveness" was performed, incremental analysis "cost-effectiveness" ”, as well as the calculation of indicators of the cost of the operation after 5 years, taking into account discounting. The results of overall and event-free survival, as well as survival without the development of graft-versus-host disease and relapse were used as endpoints. The costs are divided into 2 time periods: stage 1 – up to 30 days after HSCT and stage 2 – from 31 days after HSCT and up to 1 year. Costs are divided into direct and indirect. For indirect costs, the average figures of the available socio-economic data are taken. Results of the study: The total costs for the two stages were comparable for both platforms: TCRa/b/CD19 deletions – 6702.094 thousand rubles; PtCy – 7325.661 thousand rubles. The costs are unevenly distributed over the stages: the 1st stage is more expensive for the TCR platform, the second for PtCy. In cost-effectiveness analysis, each unit of efficiency (1% survival per 1 year of curation will cost more for the PtCy platform. Given the WHO recommended discount factor of 3% per year, over 5 years, the annual cost for the TCR method should decrease to 5933.316 thousand rubles, and for the PtСy platform up to 6,485.355 thousand rubles without considering macroeconomic indicators. Each additional unit of efficiency (1% survival rate) of TCR will cost: survival rate of 207 855.67 rubles, for survival without GVHD and recurrence of 51 963.92 rubles. Conclusions. Pharmacoeconomically less costly for a period of 1 year of curation is the preventive TCR platform compared to the depletion platform ''in vivo'' using high doses of cyclophosphamide, despite the higher cost of its use at the 1st stage of curation.
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