Efficacy and Cost Effectiveness of Pharmacokinetic Dosing in Haemophilia A
Status:
Completed
Trial end date:
2015-05-01
Target enrollment:
Participant gender:
Summary
Patients with severe Haemophilia A need prophylactic factor VIII to reduce their risk of
joint and soft tissue bleeds and to prevent or reduce joint damage. It is common practice to
give enough factor VIII to maintain the trough level above 1% of normal and this has been
supported in retrospective studies.
The amount of factor VIII required to maintain this trough level varies markedly between
patients because their factor VIII half lives are different. This study will assess the role
of regular pharmacokinetic (PK)monitoring and dose adjusted factor VIII to establish whether
this is a more cost effective way of giving treatment and whether it is feasible in routine
clinical practice. Patients will be treated for 6 months with their standard factor VIII
regimen and followed up to establish their bleed frequency. They will then receive
pharmacokinetic adjusted factor VIII to maintain a trough above 1.5% for a year and their
bleed rate compared to standard treatment. If they have increased break through bleeds their
factor VIII will be increased to maintain a trough of 3%.
Phase:
Phase 4
Details
Lead Sponsor:
Hampshire Hospitals NHS Foundation Trust
Collaborators:
University of Wales Cardiff United Kingdom (UK) Uppsala University