因子等效的幻影:检查血友病非因子治疗的复杂性。

IF 3 2区 医学 Q2 HEMATOLOGY
Haemophilia Pub Date : 2025-09-16 DOI:10.1111/hae.70131
Yesim Dargaud, Arianna Colombo, Maria Elisa Mancuso
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引用次数: 0

摘要

背景:本文对应用于血友病非因素治疗(nft)的“因素等效”概念进行了批判性分析,强调了其局限性和临床意义。虽然因子等效估计是评估不同疗法促凝作用的比较工具,但由于不同分子和患者特异性因素的作用机制不同,它们可能无法准确代表真正的止血潜力或临床疗效。目的:本文对广泛采用因子等效作为量化nft止血潜力的通用基准提出了挑战。讨论和结论:它提倡对患者和具体情况进行实验室评估,以反映每种疗法独特的药理学特征。这种方法特别适用于加强个性化治疗策略,特别是在手术或严重突破性出血等高风险情况下,从而优化新型止血疗法的使用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The Mirage of Factor Equivalence: Examining the Complexities of Non-Factor Therapies in Haemophilia.

Background: This article provides a critical analysis of the 'factor equivalence' concept as applied to non-factor therapies (NFTs) for haemophilia, highlighting its limitations and clinical implications. Although factor equivalence estimates serve as a comparative tool for evaluating pro-coagulant effects across different therapies, they may fail to accurately represent the true haemostatic potential or clinical efficacy due to the diverse mechanisms of action of different molecules and patient-specific factors.

Aim: The article challenges the widespread adoption of factor equivalence as a universal benchmark to quantify the haemostatic potential of NFTs.

Discussion and conclusion: It advocates for patient- and context-specific laboratory assessments that reflect each therapy's unique pharmacological profile. This approach is particularly relevant to enhance personalised treatment strategies, especially in high-risk situations such as surgery or severe breakthrough bleeding, thereby optimising the use of novel haemostatic therapies.

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来源期刊
Haemophilia
Haemophilia 医学-血液学
CiteScore
6.50
自引率
28.20%
发文量
226
审稿时长
3-6 weeks
期刊介绍: Haemophilia is an international journal dedicated to the exchange of information regarding the comprehensive care of haemophilia. The Journal contains review articles, original scientific papers and case reports related to haemophilia care, with frequent supplements. Subjects covered include: clotting factor deficiencies, both inherited and acquired: haemophilia A, B, von Willebrand''s disease, deficiencies of factor V, VII, X and XI replacement therapy for clotting factor deficiencies component therapy in the developing world transfusion transmitted disease haemophilia care and paediatrics, orthopaedics, gynaecology and obstetrics nursing laboratory diagnosis carrier detection psycho-social concerns economic issues audit inherited platelet disorders.
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