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将血友病重新分类,将结果和表型的定义纳入新的目标。

Reclassifying hemophilia to include the definition of outcomes and phenotype as new targets.

机构信息

Department of Haematology, Manchester University Hospitals, Oxford Road, Manchester, UK.

Hematology Division, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.

出版信息

J Thromb Haemost. 2023 Jul;21(7):1737-1740. doi: 10.1016/j.jtha.2023.03.016. Epub 2023 Mar 25.

DOI:10.1016/j.jtha.2023.03.016
PMID:36972786
Abstract

Hemophilia is classified into mild, moderate, and severe based on coagulation factor activity levels. Factor replacement and prophylaxis regimens in persons with hemophilia have helped to reduce bleeding and its related complications. With several newer treatments, some already approved and others soon to be, there may be a need to consider health-related quality of life in addition to bleed prevention as the goals of providing comprehensive care to persons with hemophilia. In this article, we discussed the reasons why such an approach may be relevant and call for the International Society of Thrombosis and Haemostasis to revisit the current classification of hemophilia.

摘要

根据凝血因子活性水平,血友病可分为轻度、中度和重度。血友病患者的因子替代和预防治疗方案有助于减少出血及其相关并发症。随着一些新的治疗方法的出现,其中一些已经获得批准,其他的也即将获批,因此,在为血友病患者提供全面护理时,除了预防出血外,可能还需要考虑与健康相关的生活质量。在本文中,我们讨论了这种方法可能相关的原因,并呼吁国际血栓与止血学会重新审视当前的血友病分类。

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Reclassifying hemophilia to include the definition of outcomes and phenotype as new targets.将血友病重新分类,将结果和表型的定义纳入新的目标。
J Thromb Haemost. 2023 Jul;21(7):1737-1740. doi: 10.1016/j.jtha.2023.03.016. Epub 2023 Mar 25.
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Update on treatment regimens: prophylaxis versus on-demand therapy.治疗方案的最新进展:预防治疗与按需治疗
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Concizumab, a Non-Replacement Therapy for Persons with Hemophilia with Inhibitors.康西珠单抗,一种用于血友病伴抑制物患者的非替代疗法。
J Clin Med. 2025 Apr 25;14(9):2961. doi: 10.3390/jcm14092961.
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Mild or moderate hemophilia is not always a mild or moderate bleeding disorder: Back to the clinical phenotype.轻度或中度血友病并不总是一种轻度或中度出血性疾病:回归临床表型。
Hemasphere. 2025 Mar 25;9(3):e70111. doi: 10.1002/hem3.70111. eCollection 2025 Mar.
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Contemporary approaches to treat people with hemophilia: what's new and what's not?当代血友病治疗方法:新在哪里,旧在何处?
Res Pract Thromb Haemost. 2025 Jan 31;9(1):102696. doi: 10.1016/j.rpth.2025.102696. eCollection 2025 Jan.
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Can hemophilia be cured? It depends on the definition.血友病能被治愈吗?这取决于对“治愈”的定义。
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Future needs for continuing innovation in hemophilia: improving outcomes for individuals of all severities, including women and those in resource-constrained regions.血友病持续创新的未来需求:改善所有严重程度患者的治疗效果,包括女性患者以及资源受限地区的患者。
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