PK-driven prophylaxis versus standard prophylaxis: When a tailored treatment may be a real and achievable cost-saving approach in children with severe hemophilia A
PK-driven prophylaxis versus standard prophylaxis: When a tailored treatment may be a real and achievable cost-saving approach in children with severe hemophilia A
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DOI：
10.1016/j.thromres.2017.07.003
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发表时间：
2017-09-01
影响因子：
7.5
通讯作者：
Zanon, Ezio
中科院分区：
文献类型：
--
作者：
Pasca, Samantha;Milan, Marta;Zanon, Ezio
背景：预防性治疗是治疗儿童重度血友病的金标准。在过去的几年中，一种新的方法，以预防为基础的年出血率（ABR），药代动力学（PK）和生活方式的每一个病人已经开始通过在hemophilia treatment.Aim：目的：我们的观察性回顾性研究的目的是评估是否在一组儿童严重血友病A（HA）的定制方法可以用来取代标准治疗，降低成本。使用计算程序（MyPKfit（R）），在我们血友病中心随访的6名血友病儿童中进行PK评价，这些儿童已经接受了重组因子VIII（rFVIII）预防治疗。结果：1例患儿每周输液次数减少，3例患儿每周输液次数略有增加。至于其余儿童，仅改变了剂量。计划的随访显示完全依从治疗，使用PK方案减少出血，生活质量普遍改善。在标准和PK驱动的预防期间的治疗的直接和间接成本之间的比较显示，在定制prophylaxis.Conclusion的情况下，总节省（原文如此）54，797.40（-10.67%）基于PK和每个患者的临床特征的治疗方法可能会改变标准治疗。根据我们的研究结果，量身定制的预防可能是HA儿童降低成本的有效选择。(C)2017爱思唯尔有限公司版权所有。
Background: Prophylaxis is the gold standard for the treatment of children with severe hemophilia. In the last years a new approach to prophylaxis based on annual bleeding rate (ABR), pharmacokinetics (PK) and lifestyle of each patient has begun to be adopted in hemophilia treatment.Aim: Aim of our observational retrospective study was to evaluate whether in a group of children with severe hemophilia A (HA) a tailored approach may be used to replace standard therapy, reducing costs.Methods: PK evaluation was carried out in six hemophiliac children followed at our Hemophilia Center, and already receiving recombinant factor VIII (rFVIII) on prophylaxis, using a computing program(MyPKfit (R)). Bayesian curve was created for each child and tailored prophylaxis was estimated considering a trough level of 1%.Results: The weekly frequency of infusions was reduced in one patient, while it was slightly increased in three children. As to the remaining children, only the dosage was changed. Scheduled follow-up revealed a complete adherence to treatment, a reduction of bleeds using PK-regimen and a general improvement in the quality of life. The comparison between the direct and indirect costs of treatment during standard and PK-driven prophylaxis showed a total saving of (sic) 54,797.40 (-10.67%) in case of tailored prophylaxis.Conclusion: A therapeutic approach based on PK and clinical characteristics of each patient may change standard treatment. Based on our results, tailored prophylaxis could be an effective option for children with HA reducing costs. (C) 2017 Elsevier Ltd. All rights reserved.