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转变血友病A的治疗:对新治疗选择的见解

Transforming Hemophilia A Care: Insights into New Therapeutic Options.

作者信息

Iurea Iasmina-Maria, Severin Emilia, Matei Alexandra

机构信息

Genetics Department, Carol Davila University of Medicine and Pharmacy, Dionisie Lupu 37 Street, 020021 Bucharest, Romania.

出版信息

Life (Basel). 2024 Nov 29;14(12):1568. doi: 10.3390/life14121568.

Abstract

Hemophilia A is a hereditary bleeding disorder characterized by a deficiency in clotting factor VIII, leading to significant morbidity and a reduced quality of life. This review provides an updated overview of the current understanding of hemophilia A, highlighting its genetic underpinnings and advancements in treatment strategies. A literature review was conducted using various available databases. Relevant studies on hemophilia A, covering genetics and treatment options, were selected and summarized. Recent developments in gene therapy are discussed, showcasing their potential to offer long-term solutions and reduce the burden of treatment. Additionally, the review addresses global disparities in care and policy implications, emphasizing the need for comprehensive healthcare frameworks to improve outcomes for individuals living with hemophilia A worldwide. By synthesizing recent findings and insights, this review aims to inform clinicians and policymakers about the evolving landscape of hemophilia A management and the necessity for equitable access to care.

摘要

甲型血友病是一种遗传性出血性疾病,其特征是凝血因子VIII缺乏,导致严重的发病率和生活质量下降。本综述提供了对甲型血友病当前认识的最新概述,重点介绍了其遗传基础和治疗策略的进展。使用各种可用数据库进行了文献综述。选择并总结了有关甲型血友病的相关研究,涵盖遗传学和治疗选择。讨论了基因治疗的最新进展,展示了它们提供长期解决方案和减轻治疗负担的潜力。此外,该综述还涉及全球护理差异和政策影响,强调需要全面的医疗保健框架来改善全球甲型血友病患者的治疗结果。通过综合最近的研究结果和见解,本综述旨在让临床医生和政策制定者了解甲型血友病管理的不断变化的情况以及公平获得护理的必要性。

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