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Optimising prophylaxis in haemophilia A : The ups and downs of treatment

Berntorp, Erik LU ; Hermans, Cédric ; Solms, Alexander ; Poulsen, Lone and Mancuso, Maria Elisa (2021) In Blood Reviews 50.
Abstract

The classical goals of haemophilia A treatment are to prevent bleeds, minimise the risk of long-term complications associated with joint damage, and improve quality of life by maintaining appropriate factor VIII [FVIII] levels. The dose and frequency of FVIII replacement therapies required to reduce bleeds is now known to vary amongst individuals, and may change for the same individual over time, meaning that a standardised dose and regimen may not provide optimal protection to all patients. Here we review the evolving treatment landscape for haemophilia A, and discuss how an increased understanding of the pharmacology and pharmacokinetics underlying FVIII replacement and non-factor replacement therapies could improve patient outcomes.... (More)

The classical goals of haemophilia A treatment are to prevent bleeds, minimise the risk of long-term complications associated with joint damage, and improve quality of life by maintaining appropriate factor VIII [FVIII] levels. The dose and frequency of FVIII replacement therapies required to reduce bleeds is now known to vary amongst individuals, and may change for the same individual over time, meaning that a standardised dose and regimen may not provide optimal protection to all patients. Here we review the evolving treatment landscape for haemophilia A, and discuss how an increased understanding of the pharmacology and pharmacokinetics underlying FVIII replacement and non-factor replacement therapies could improve patient outcomes. We also review the strengths and weaknesses of current treatments and explore the benefits of personalised therapy and review how this may best be achieved with current treatment options. The key points of our review are summarised in this accompanying short video.

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author
; ; ; and
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Extended half-life factor VIII replacement therapy, Haemophilia A, Non-factor replacement therapy, Pharmacokinetics, Prophylaxis
in
Blood Reviews
volume
50
article number
100852
publisher
Churchill Livingstone
external identifiers
  • scopus:85109432141
  • pmid:34243987
ISSN
0268-960X
DOI
10.1016/j.blre.2021.100852
language
English
LU publication?
yes
id
d56420b4-9529-4de1-83e7-6ca5090f6bb1
date added to LUP
2021-08-23 10:48:49
date last changed
2024-03-08 16:06:31
@article{d56420b4-9529-4de1-83e7-6ca5090f6bb1,
  abstract     = {{<p>The classical goals of haemophilia A treatment are to prevent bleeds, minimise the risk of long-term complications associated with joint damage, and improve quality of life by maintaining appropriate factor VIII [FVIII] levels. The dose and frequency of FVIII replacement therapies required to reduce bleeds is now known to vary amongst individuals, and may change for the same individual over time, meaning that a standardised dose and regimen may not provide optimal protection to all patients. Here we review the evolving treatment landscape for haemophilia A, and discuss how an increased understanding of the pharmacology and pharmacokinetics underlying FVIII replacement and non-factor replacement therapies could improve patient outcomes. We also review the strengths and weaknesses of current treatments and explore the benefits of personalised therapy and review how this may best be achieved with current treatment options. The key points of our review are summarised in this accompanying short video.</p>}},
  author       = {{Berntorp, Erik and Hermans, Cédric and Solms, Alexander and Poulsen, Lone and Mancuso, Maria Elisa}},
  issn         = {{0268-960X}},
  keywords     = {{Extended half-life factor VIII replacement therapy; Haemophilia A; Non-factor replacement therapy; Pharmacokinetics; Prophylaxis}},
  language     = {{eng}},
  month        = {{05}},
  publisher    = {{Churchill Livingstone}},
  series       = {{Blood Reviews}},
  title        = {{Optimising prophylaxis in haemophilia A : The ups and downs of treatment}},
  url          = {{http://dx.doi.org/10.1016/j.blre.2021.100852}},
  doi          = {{10.1016/j.blre.2021.100852}},
  volume       = {{50}},
  year         = {{2021}},
}