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血友病 A 患者的个体化预防:挑战与成就。

Personalized prophylaxis in people with hemophilia A: challenges and achievements.

机构信息

a Cerrahpasa Medical Faculty, Department of Internal Medicine, Division of Hematology , Istanbul University , Istanbul , Turkey.

出版信息

Expert Rev Hematol. 2016 Dec;9(12):1203-1208. doi: 10.1080/17474086.2016.1252670. Epub 2016 Nov 4.

Abstract

Prophylactic factor replacement is the standard of care for all people with severe hemophilia to prevent bleeding and associated complications. Current weight-based fixed dose prophylaxis regimens are effective; however, they lack flexibility and usually fail to meet the individual needs and expectations of the patients. Recent developments in hemophilia treatment provide new opportunities for a more personalized prophylaxis. Areas covered: Rationale and methods of individualizing prophylaxis in hemophilia A on the basis of current evidence are discussed in this review. For this relevant literature in English and German was searched using PubMed database. Expert commentary: Major determinants of personalized prophylaxis include age, bleeding pattern, personal pharmacokinetics, joint health, co-morbidities, venous access and adherence. An ideal prophylaxis programme should take into account all of the aforementioned items and also be able to meet the needs. Extended half-life factor concentrates, new hemostatic molecules and tools using population pharmacokinetics to estimate personal factor requirements will serve individualizing prophylaxis in a more precise manner.

摘要

预防性因子替代治疗是所有严重血友病患者的标准治疗方法,以预防出血和相关并发症。目前基于体重的固定剂量预防方案是有效的;然而,它们缺乏灵活性,通常无法满足患者的个体需求和期望。血友病治疗的最新进展为更个性化的预防提供了新的机会。 涵盖领域:本综述讨论了基于当前证据在血友病 A 中个体化预防的原理和方法。为此,使用 PubMed 数据库搜索了英语和德语的相关文献。 专家评论:个体化预防的主要决定因素包括年龄、出血模式、个体药代动力学、关节健康、合并症、静脉通路和依从性。理想的预防方案应考虑到所有上述项目,并能够满足需求。延长半衰期的因子浓缩物、新型止血分子以及使用群体药代动力学来估计个体因子需求的工具将以更精确的方式服务于个体化预防。

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