Anti-tissue factor pathway inhibitors for hemophilia: are these treatments the answer to overcoming current treatment limitations?

IF 2.3 4区医学 Q2 HEMATOLOGY

Expert Review of Hematology Pub Date : 2025-07-01 Epub Date: 2025-06-14 DOI:10.1080/17474086.2025.2519002

Johnny Mahlangu

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引用次数: 0

Abstract

Introduction: Non-factor therapies were developed to address the shortcomings of clotting factor concentrates (CFCs) used for hemophilia bleed management. These CFC unmet needs include high treatment burden, immunogenicity, inconsistent hemostatic cover, poor treatment outcomes, and musculoskeletal progression despite adequate prophylactic treatment. Anti-tissue factor pathway inhibitors (anti-TFPIs) that have completed Phase 3 clinical studies are concizumab and marstacimab. The role of these anti-TFPIs in the hemophilia treatment armamentarium remains unclear.

Areas covered: This review critically appraises data published in PubMed, World of Science, and peer-reviewed congress presentations to determine whether anti-TFPIs merely supplement current treatment options or represent a disruptive shift in the treatment paradigm for hemophilia. It underscores the unmet needs of replacement therapies and compares the pharmacokinetic, efficacy, and safety data of anti-TFPIs and selected FVIII and FIX products.

Expert opinion: As hemophilia treatment goals continue to evolve, the role of currently developed anti-TFPIs is still not fully defined. This review comprehensively summarizes the clinical trial data, which shows that anti-TFPIs are not intended to replace the standard of care CFCs but to expand the therapeutic arsenal for patients with hemophilia treated with these therapeutic agents.

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血友病抗组织因子途径抑制剂：这些治疗方法是克服当前治疗局限性的答案吗？

简介：非因子疗法的发展是为了解决凝血因子浓缩物（CFCs）用于血友病出血管理的缺点。这些未满足的CFC需求包括高治疗负担、免疫原性、不一致的止血覆盖、不良的治疗结果以及尽管进行了适当的预防性治疗，但肌肉骨骼仍有进展。已经完成3期临床研究的抗组织因子通路抑制剂（anti- tfpi）是concizumab和marstacimab。这些抗tfpis在血友病治疗领域的作用尚不清楚。涵盖领域：本综述对发表在PubMed、World of Science和同行评议的大会报告上的数据进行了批判性评估，以确定抗tfpis是否仅仅是对当前治疗方案的补充，还是代表了血友病治疗范式的颠覆性转变。它强调了替代疗法的未满足需求，并比较了抗tfpi和选定的FVIII和FIX产品的药代动力学、疗效和安全性数据。专家意见：随着血友病治疗目标的不断发展，目前开发的抗tfpis的作用仍未完全确定。这篇综述全面总结了临床试验数据，表明抗tfpis不是为了取代CFCs的标准治疗，而是为了扩大血友病患者使用这些治疗药物的治疗库。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Expert Review of Hematology HEMATOLOGY-

CiteScore

4.70

自引率

3.60%

发文量

审稿时长

6-12 weeks

期刊介绍： Advanced molecular research techniques have transformed hematology in recent years. With improved understanding of hematologic diseases, we now have the opportunity to research and evaluate new biological therapies, new drugs and drug combinations, new treatment schedules and novel approaches including stem cell transplantation. We can also expect proteomics, molecular genetics and biomarker research to facilitate new diagnostic approaches and the identification of appropriate therapies. Further advances in our knowledge regarding the formation and function of blood cells and blood-forming tissues should ensue, and it will be a major challenge for hematologists to adopt these new paradigms and develop integrated strategies to define the best possible patient care. Expert Review of Hematology (1747-4086) puts these advances in context and explores how they will translate directly into clinical practice.