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

Authors

HOLME Pal Andre BLATNÝ Jan CHOWDARY Pratima LASSILA Riitta O'CONNELL Niamh HERMANS Cedric ROMAN Maria Teresa Alvarez NEGRIER Claude COPPOLA Antonio OLDENBURG Johannes

Year of publication 2024
Type Article in Periodical
Magazine / Source Haemophilia
MU Faculty or unit

Faculty of Medicine

Citation
web https://onlinelibrary.wiley.com/doi/10.1111/hae.15031
Doi http://dx.doi.org/10.1111/hae.15031
Keywords haemophilia A; haemostasis; health equity; Normalization; quality of life; treatment goals
Description BackgroundTreatment 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 goalsThe 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.ConclusionCapitalizing 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.

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