在血友病和相关出血性疾病中实施药代动力学指导的因子替代治疗剂量的道路。通过绘制障碍和促进因素来确定知识差距

IF 6.9 2区 医学 Q1 HEMATOLOGY Blood Reviews Pub Date : 2023-09-01 DOI:10.1016/j.blre.2023.101098
Tine M.H.J. Goedhart , A. Janssen , Ron A.A. Mathôt , Marjon H. Cnossen , for the OPTI-CLOT Study Group and SYMPHONY Consortium
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引用次数: 0

摘要

临床指南和专家组建议使用药代动力学(PK)引导剂量的因子替代疗法治疗出血性疾病,特别是血友病患者。虽然钾离子引导给药越来越多地被应用,但它通常不被认为是标准的临床实践。本次范围审查的目的是绘制在临床实践中实施pk指导给药的障碍和促进因素,并确定知识差距。我们进行了文献检索,纳入了110篇描述出血性疾病(主要是A型血友病)患者pk指导给药的文章。我们定义了两个总体主题,疗效和可行性,并在每个主题中讨论了五个主题。对于每个主题,都描述了障碍、促进因素和知识差距。尽管在一些主题上达成了共识,但在其他主题上发现了相互矛盾的报告,特别是在pk指导给药的功效方面。这些矛盾突出了未来研究阐明当前歧义的必要性。
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The road to implementation of pharmacokinetic-guided dosing of factor replacement therapy in hemophilia and allied bleeding disorders. Identifying knowledge gaps by mapping barriers and facilitators

Clinical guidelines and expert groups recommend the use of pharmacokinetic (PK)-guided dosing of factor replacement therapy for the treatment of bleeding disorders, especially for patients with hemophilia. Although PK-guided dosing is increasingly applied, it is generally not considered standard clinical practice. The aim of this scoping review is to map barriers and facilitators for the implementation of PK-guided dosing in clinical practice and to identify knowledge gaps. A literature search was performed and 110 articles were included that describe PK-guided dosing in patients with bleeding disorders, mostly hemophilia A. We defined two overarching themes, efficacy and feasibility, and discuss five topics within each theme. For each topic, barriers, facilitators and knowledge gaps were described. Although consensus was found with regard to some topics, contradicting reports were found for others, especially with respect to the efficacy of PK-guided dosing. These contradictions highlight the need for future research to elucidate current ambiguities.

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来源期刊
Blood Reviews
Blood Reviews 医学-血液学
CiteScore
13.80
自引率
1.40%
发文量
78
期刊介绍: Blood Reviews, a highly regarded international journal, serves as a vital information hub, offering comprehensive evaluations of clinical practices and research insights from esteemed experts. Specially commissioned, peer-reviewed articles authored by leading researchers and practitioners ensure extensive global coverage across all sub-specialties of hematology.
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