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血友病能被治愈吗?这取决于对“治愈”的定义。

Can hemophilia be cured? It depends on the definition.

作者信息

Baas Lieke, van der Graaf Rieke, Meijer Karina

机构信息

Department of Bioethics and Health Humanities, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands.

Department of Hematology, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands.

出版信息

Res Pract Thromb Haemost. 2024 Aug 27;8(6):102559. doi: 10.1016/j.rpth.2024.102559. eCollection 2024 Aug.

Abstract

Over the years, the palette of treatment options for hemophilia has grown extensively, leading to an increased life expectancy and quality of life for people living with hemophilia. Nonetheless, it is frequently emphasized that none of the current treatment modalities provides a "cure." It is therefore hoped that innovative treatments such as gene therapy may bridge this void. However, the precise definition of a "cure" for hemophilia remains unclear. In this review, we show how the concept of cure is currently used in the field of hemophilia. We then relate the discussion on cure to debates surrounding the classification of hemophilia and philosophical debates on the concepts of health and disease.

摘要

多年来,血友病的治疗选择范围已大幅扩展,使得血友病患者的预期寿命和生活质量均有所提高。尽管如此,人们仍经常强调,目前的治疗方式均无法实现“治愈”。因此,人们寄希望于基因疗法等创新疗法来填补这一空白。然而,血友病“治愈”的准确定义仍不明确。在本综述中,我们展示了“治愈”这一概念目前在血友病领域的应用方式。接着,我们将关于治愈的讨论与围绕血友病分类的争论以及关于健康和疾病概念的哲学辩论联系起来。

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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。
Res Pract Thromb Haemost. 2024 Aug 27;8(6):102559. doi: 10.1016/j.rpth.2024.102559. eCollection 2024 Aug.
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本文引用的文献

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Hemophilia Gene Therapy: The End of the Beginning?血友病基因治疗:开始的结束?
Hum Gene Ther. 2023 Sep;34(17-18):782-792. doi: 10.1089/hum.2023.112.
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Towards achieving a haemophilia-free mind.迈向无血友病的心态。
Haemophilia. 2023 Jul;29(4):951-953. doi: 10.1111/hae.14807. Epub 2023 Jun 1.

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