Nothing short of a revolution: Novel extended half-life factor VIII replacement products and non-replacement agents reshape the treatment landscape in hemophilia A

IF 6.9 2区 医学 Q1 HEMATOLOGY Blood Reviews Pub Date : 2024-03-01 DOI:10.1016/j.blre.2023.101164
Hussien Ahmed H. Abdelgawad , Rachel Foster , Mario Otto
{"title":"Nothing short of a revolution: Novel extended half-life factor VIII replacement products and non-replacement agents reshape the treatment landscape in hemophilia A","authors":"Hussien Ahmed H. Abdelgawad ,&nbsp;Rachel Foster ,&nbsp;Mario Otto","doi":"10.1016/j.blre.2023.101164","DOIUrl":null,"url":null,"abstract":"<div><p>Hemophilia A, an X-linked genetic disorder, is characterized by a deficiency or dysfunction of clotting Factor VIII. The treatment landscape has substantially changed by introducing novel extended half-life factor VIII (EHL-FVIII) replacement therapies such as efanesoctocog Alfa and non-factor replacement therapy such as emicizumab. These agents signal a shift from treatments requiring multiple weekly infusions to advanced therapies with long half-lives, offering superior protection against bleeding and improving patient adherence and quality of life. While EHL-FVIII treatment might lead to inhibitor development in some patients, non-factor replacement therapy carries thrombotic risks. Therefore, ongoing research and the generation of robust clinical evidence remain vital to guide the selection of optimal and cost-effective first-line therapies for hemophilia A patients.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":6.9000,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X23001340/pdfft?md5=fdc4f9b24ef41d865f0071c204b7d0ec&pid=1-s2.0-S0268960X23001340-main.pdf","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Blood Reviews","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0268960X23001340","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0

Abstract

Hemophilia A, an X-linked genetic disorder, is characterized by a deficiency or dysfunction of clotting Factor VIII. The treatment landscape has substantially changed by introducing novel extended half-life factor VIII (EHL-FVIII) replacement therapies such as efanesoctocog Alfa and non-factor replacement therapy such as emicizumab. These agents signal a shift from treatments requiring multiple weekly infusions to advanced therapies with long half-lives, offering superior protection against bleeding and improving patient adherence and quality of life. While EHL-FVIII treatment might lead to inhibitor development in some patients, non-factor replacement therapy carries thrombotic risks. Therefore, ongoing research and the generation of robust clinical evidence remain vital to guide the selection of optimal and cost-effective first-line therapies for hemophilia A patients.

查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
不折不扣的革命:新型延长半衰期第八因子替代产品和非替代制剂重塑了 A 型血友病的治疗格局
血友病 A 是一种 X 连锁遗传疾病,其特征是凝血因子 VIII 缺乏或功能障碍。通过引入新型延长半衰期因子 VIII(EHL-FVIII)替代疗法(如 efanesoctocog Alfa)和非因子替代疗法(如 emicizumab),治疗格局发生了重大变化。这些药物标志着从需要每周多次输注的治疗方法转变为具有长半衰期的先进疗法,从而提供更好的出血保护,并提高患者的依从性和生活质量。虽然 EHL-FVIII 治疗可能会导致某些患者出现抑制剂,但非因子替代疗法也存在血栓风险。因此,持续的研究和可靠的临床证据对于指导 A 型血友病患者选择最佳且经济有效的一线疗法仍然至关重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 去求助
来源期刊
Blood Reviews
Blood Reviews 医学-血液学
CiteScore
13.80
自引率
1.40%
发文量
78
期刊介绍: Blood Reviews, a highly regarded international journal, serves as a vital information hub, offering comprehensive evaluations of clinical practices and research insights from esteemed experts. Specially commissioned, peer-reviewed articles authored by leading researchers and practitioners ensure extensive global coverage across all sub-specialties of hematology.
期刊最新文献
From MGUS to multiple myeloma: Unraveling the unknown of precursor states. Advancing CAR T-cell therapies: Preclinical insights and clinical translation for hematological malignancies. Corrigendum to "Measurable residual disease (MRD)-testing in haematological cancers: A giant leap forward or sideways?"[BLOOD REVIEWS, 9 August 2024, https://doi.org/10.1016/j.blre.2024.101226]. Clinical applications of circulating tumor DNA in hematological malignancies: From past to the future Tailoring oral anticoagulant treatment in the era of multi-drug therapies for PAH and CTEPH.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1