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

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. 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 the accompanying short video.

Keywords: Extended half-life factor VIII replacement therapy; Haemophilia A; Non-factor replacement therapy; Pharmacokinetics; Prophylaxis.