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...

Full description

Saved in:
Bibliographic Details
Published inResearch and practice in thrombosis and haemostasis Vol. 8; no. 6; p. 102559
Main Authors Baas, Lieke, van der Graaf, Rieke, Meijer, Karina
Format Journal Article
LanguageEnglish
Published United States Elsevier Inc 01.08.2024
Elsevier
Subjects
Online AccessGet full text

Cover

Loading…
More Information
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.
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