Oncology Pharmacy Department, Centre Léon Bérard.
Institute of Pediatric Hematology and Oncology, Hospices Civils de Lyon.
Ther Drug Monit. 2021 Aug 1;43(4):481-489. doi: 10.1097/FTD.0000000000000886.
Cyclosporine A (CsA) is the main drug used to prevent graft-versus-host disease in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). CsA therapeutic drug monitoring (TDM) has been performed for ages, with studies revealing clinical benefits, but failing to examine its economic impact. In this article, the main objective was to evaluate the economic impact of the CsA TDM strategy, based on a Bayesian approach, by assessing costs related to its clinical impact. Furthermore, TDM effectiveness was analyzed for pharmacokinetics and clinical outcomes.
A cost-effective, nonrandomized, retrospective, single-center study compared 2 CsA monitoring and dose adaptation strategies in pediatric patients undergoing HSCT. From 2014 to 2016, CsA TDM was performed using a population pharmacokinetics model-coupled Bayesian approach by a pharmacist ["pharmacist-assisted individualization" (PAI)]. From 2017 to 2018, CsA TDM was performed by the clinician without a Bayesian approach (non-PAI group). HSCT costs were evaluated from the French National Insurance perspective. Economic and clinical outcomes were assessed by measuring incremental cost-effectiveness ratios.
The study included 144 patients: 90 and 54 patients in PAI and non-PAI groups, respectively. Both groups were comparable for sociodemographic and clinical characteristics. The mean total cost per patient was significantly lower (P < 0.01) in the PAI group (€85,947) than in the non-PAI group (€100,435). Multivariate analysis revealed that TDM based on the Bayesian approach was a protective factor (odds ratio = 0.86) for severe acute graft-versus-host disease. We noted that pharmacist-based TDM was the dominant strategy. Bayesian method-based TDM allowed an increase in the percentage of target attainment at any period post-HSCT.
CsA TDM with a Bayesian approach is a cost-effective procedure, and highlighted clinical benefits encourage the development of new TDM strategies for HSCT.
环孢素 A（CsA）是用于预防异基因造血干细胞移植（HSCT）患者移植物抗宿主病的主要药物。CsA 治疗药物监测（TDM）已经存在了很长时间，研究表明其具有临床获益，但未能考察其经济影响。本文主要通过评估与临床影响相关的成本，采用贝叶斯方法评估 CsA TDM 策略的经济影响。此外，还分析了 TDM 对药代动力学和临床结局的有效性。
本成本效益非随机回顾性单中心研究比较了 2014 年至 2016 年期间接受 HSCT 的儿科患者中两种 CsA 监测和剂量调整策略。在此期间，药师采用群体药代动力学模型结合贝叶斯方法进行 CsA TDM（药师辅助个体化，PAI）。2017 年至 2018 年，临床医生未采用贝叶斯方法进行 CsA TDM（非 PAI 组）。HSCT 成本从法国国家保险角度进行评估。通过测量增量成本效益比评估经济和临床结局。
该研究纳入了 144 名患者：PAI 组和非 PAI 组分别为 90 名和 54 名患者。两组在社会人口统计学和临床特征方面具有可比性。PAI 组每名患者的平均总费用显著较低（P<0.01）（€85947），而非 PAI 组为（€100435）。多变量分析显示，基于贝叶斯方法的 TDM 是严重急性移植物抗宿主病的保护因素（优势比=0.86）。我们注意到，基于药师的 TDM 是一种主要策略。基于贝叶斯方法的 TDM 可提高 HSCT 后任何时期的靶目标达标率。
基于贝叶斯方法的 CsA TDM 是一种具有成本效益的方法，其临床获益突出，鼓励为 HSCT 开发新的 TDM 策略。
