甲型血友病治疗的实验室监测：新挑战
Laboratory monitoring of hemophilia A treatments: new challenges.
作者信息
Lenting Peter J
机构信息
Hémostase Inflammation Thrombose, INSERM, Unité Mixte de Recherche Scientifique 1176, Université Paris-Saclay, Le Kremlin-Bicêtre, France.
出版信息
Blood Adv. 2020 May 12;4(9):2111-2118. doi: 10.1182/bloodadvances.2019000849.
Abstract
Monitoring factor VIII (FVIII) activity has traditionally been complicated by discrepancies between assays for the various sorts of FVIII molecules. The advent of novel nonfactor therapies (emicizumab, fitusiran, and anti-tissue factor pathway inhibitor antibodies) in hemophilia A poses a new level of difficulty on the laboratory monitoring of these patients. To use the correct assays and for a proper interpretation of their results, it is pertinent to understand the mode of action of these nonfactor agents. Furthermore, the biochemical consequences for the different types of activity assays (whether it be specific FVIII activity assays or global coagulation assays) should be taken into account as well. In this review, these aspects will be discussed. In addition, the use of various animal models to estimate FVIII-equivalence of the nonfactor therapies will be presented.
摘要
传统上，监测因子VIII（FVIII）活性一直因各类FVIII分子检测方法之间的差异而变得复杂。新型非因子疗法（依美珠单抗、fitusiran和抗组织因子途径抑制剂抗体）在A型血友病治疗中的出现，给这些患者的实验室监测带来了新的难度。为了使用正确的检测方法并正确解读其结果，了解这些非因子药物的作用模式至关重要。此外，还应考虑不同类型活性检测（无论是特定FVIII活性检测还是整体凝血检测）的生化后果。在本综述中，将讨论这些方面。此外，还将介绍使用各种动物模型来评估非因子疗法的FVIII等效性。
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