Factor VIII stimulants and other novel therapies for the treatment of von Willebrand disease: what's new on the horizon?

IF 2.5 3区 医学 Q3 PHARMACOLOGY & PHARMACY Expert Opinion on Pharmacotherapy Pub Date : 2024-08-01 Epub Date: 2024-08-18 DOI:10.1080/14656566.2024.2391526
Katherine Regling, Robert F Sidonio
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Abstract

Introduction: Von Willebrand disease (VWD) is the most common inherited bleeding disorder, affecting about 0.6% to 1.3% of the population, and is characterized primarily by mucocutaneous bleeding secondary to defective platelet adhesion and aggregation. Current therapeutic options for those with severe disease are limited and require frequent intravenous infusions.

Areas covered: This review discusses the current and recently completed clinical trials involving pathways to FVIII augmentation for the treatment of VWD. Clinical trials registered on clinicaltrials.gov and published data via PubMed searches through June 2024 were included.

Expert opinion: Available treatment options to those with VWD are limited in part due to limited clinical trials, the complexity of VWD types, and the pharmacokinetics of current treatment options. The development of therapeutic options that reduce treatment burden is necessary to improve quality of life and reduce bleeding complications and in recent years there has been an increased interest from industry to apply novel therapeutics for VWD. The FVIII mimetic, emicizumab, has demonstrated early success in patients with severe VWD and is a promising treatment option for those who require prophylaxis. Furthermore, products like efanesoctocog alfa (Altuviiio®) and BT200 have achieved enhanced VWF/FVIII half-life extension could expand the current treatment landscape while concurrently minimizing treatment burden.

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治疗冯-威廉氏病的因子 VIII 促效剂和其他新型疗法:地平线上有什么新进展?
导言:冯-威廉氏病(Von Willebrand disease,VWD)是最常见的遗传性出血性疾病,约占总人口的 0.6% 至 1.3%,其主要特征是由于血小板粘附和聚集缺陷导致的粘膜皮肤出血。目前对重症患者的治疗方案有限,而且需要频繁静脉注射:本综述讨论了目前和最近完成的涉及 FVIII 增强疗法治疗 VWD 的临床试验。本综述纳入了在 clinicaltrials.gov 上注册的临床试验以及通过 PubMed 搜索到 2024 年 6 月发表的数据:专家意见:VWD 患者的可用治疗方案有限,部分原因在于临床试验有限、VWD 类型复杂以及当前治疗方案的药代动力学。为了提高生活质量和减少出血并发症,有必要开发可减轻治疗负担的治疗方案,近年来,业界对应用新型疗法治疗 VWD 的兴趣日益浓厚。FVIII 拟效物 emicizumab 已在重度 VWD 患者中取得了早期成功,对于需要预防性治疗的患者来说是一种很有前景的治疗选择。此外,efanesoctocog alfa (Altuviiio®) 和 BT200 等产品已实现了 VWF/FVIII 半衰期的延长,可以扩大目前的治疗范围,同时最大限度地减轻治疗负担。
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来源期刊
CiteScore
5.60
自引率
3.10%
发文量
163
审稿时长
4-8 weeks
期刊介绍: Expert Opinion on Pharmacotherapy is a MEDLINE-indexed, peer-reviewed, international journal publishing review articles and original papers on newly approved/near to launch compounds mainly of chemical/synthetic origin, providing expert opinion on the likely impact of these new agents on existing pharmacotherapy of specific diseases.
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