Moving towards Normalization of haemostasis and health equity: Evolving treatment goals for haemophilia A

Background Treatment options for people with haemophilia are evolving at a rapid pace and a range of prophylactic treatment options using various technologies are currently available, each with their own distinct safety and efficacy profile. Treatment goals The access to replacement therapy and prop...

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Detalles Bibliográficos
Autores: Holme, Pål Andre, Blatný, Jan, Chowdary, Pratima, Lassila, Riitta, O’Connell, Niamh, Hermans, Cedric, Álvarez Roman, María Teresa, Négrier, Claude, Coppola, Antonio, Oldenburg, Johannes
Tipo de recurso: artículo
Fecha de publicación:2024
País:España
Institución:Universidad Autónoma de Madrid
Repositorio:Biblos-e Archivo. Repositorio Institucional de la UAM
Idioma:inglés
OAI Identifier:oai:dnet:biblosearchi::addfa10212d27f3caf51d8682cfb2d60
Acceso en línea:https://hdl.handle.net/10486/758580
https://dx.doi.org/10.1111/hae.15031
Access Level:acceso abierto
Palabra clave:haemophilia A
haemostasis
health equity
Normalization
quality of life
treatment goals
Medicina
Descripción
Sumario:Background Treatment options for people with haemophilia are evolving at a rapid pace and a range of prophylactic treatment options using various technologies are currently available, each with their own distinct safety and efficacy profile. Treatment goals The access to replacement therapy and prophylaxis has driven a dramatic reduction in mortality and resultant increase in life expectancy. Beyond this, the abolition of bleeds and preservation of joint health represent the expected, but rarely attained, goals of haemophilia treatment and care. These outcomes also do not address the complexity of health-related quality of life impacted by haemophilia and its treatment. Conclusion Capitalizing on the major potential of therapeutic innovations, ‘Normalization’ of haemostasis, as a concept, should include the aspiration of enabling individuals to live as normal a life as possible, free from haemophilia-imposed limitations. To achieve this—being supported by the data reviewed in this manuscript—the concept of haemostatic and life Normalization needs to be explored and debated within the wider multidisciplinary teams and haemophilia community