Emerging drugs for hemophilia A: insights into phase II and III clinical trials.

IF 2.7 3区医学 Q2 PHARMACOLOGY & PHARMACY

Expert Opinion on Emerging Drugs Pub Date : 2021-12-01 Epub Date: 2021-10-11 DOI:10.1080/14728214.2021.1988073

Hande Kizilocak, Guy Young

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引用次数: 2

Abstract

Introduction: Hemophilia is a lifelong, genetic-bleeding disorder, which inadequately treated results in permanent joint damage. It is characterized by spontaneous and trauma-related bleeding episodes. In the last 50 years, treatment has seen dramatic improvements which have improved the quality of life of persons with hemophilia.

Areas covered: This review will provide a summary of current pharmacological approaches for hemophilia A as well as discuss novel agents which are either approved recently or in phase II-III clinical trials, plasma-derived and recombinant factor VIII (FVIII) products, extended half-life FVIII products, bypassing agents and non-replacement therapies.

Expert opinion: Novel therapies are already changing the way that hemophilia A is managed, and as more new therapies get approved, there will be a revolution in the management of this serious condition. Clinicians will have both the opportunities as well as the challenges of incorporating such new technologies into clinical practice.

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治疗A型血友病的新兴药物:II期和III期临床试验的见解。

血友病是一种终生的遗传性出血性疾病，治疗不当会导致永久性关节损伤。它的特点是自发性和创伤性出血发作。在过去50年中，治疗取得了显著进展，改善了血友病患者的生活质量。涵盖领域:本综述将总结当前治疗a型血友病的药理学方法，并讨论最近批准或处于II-III期临床试验的新药、血浆源性和重组因子VIII (FVIII)产品、延长半衰期的FVIII产品、旁路药物和非替代疗法。专家意见:新疗法已经改变了A型血友病的治疗方式，随着更多新疗法获得批准，这一严重疾病的治疗将发生一场革命。临床医生将既有机会，也有挑战，将这些新技术纳入临床实践。

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 Opinion on Emerging Drugs 医学-药学

CiteScore

5.90

自引率

0.00%

发文量

审稿时长

>12 weeks

期刊介绍： Expert Opinion on Emerging Drugs (ISSN 1472-8214 [print], 1744-7623 [electronic]) is a MEDLINE-indexed, peer-reviewed, international journal publishing structured reviews on Phase II and Phase III drugs/drug classes emerging onto the market across all therapy areas, providing expert opinion on their potential impact on the current management of specific diseases.