Can hemophilia be cured? It depends on the definition
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 theref...
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Published in | Research and practice in thrombosis and haemostasis Vol. 8; no. 6; p. 102559 |
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Main Authors | , , |
Format | Journal Article |
Language | English |
Published |
United States
Elsevier Inc
01.08.2024
Elsevier |
Subjects | |
Online Access | Get full text |
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Summary: | 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.
•Many expect that gene therapy will cure hemophilia, but it is unclear what is meant by “cure.”•We review the use of “cure” in hemophilia and relate this to the concepts of health and disease.•“Cure” is used ambiguously and can refer to both factor expression and quality of life.•The field should evaluate the need for a single definition and who has a role in articulating it. |
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Bibliography: | ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 ObjectType-Review-3 content type line 23 |
ISSN: | 2475-0379 2475-0379 |
DOI: | 10.1016/j.rpth.2024.102559 |