Efficacy and Cost Effectiveness of Pharmacokinetic Dosing in Haemophilia A

• Status: Completed
• Trial end date: 2015-05-01
• Target enrollment: 0
• Participant gender: Male

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
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hampshire Hospitals NHS Foundation Trust
• Collaborators: University of Wales Cardiff United Kingdom (UK); Uppsala University
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

• Severe haemophilia A (baseline factor VIII < 1IU/dL)

• Age 18 years and above

• Patients taking any regular prophylactic regimen (defined as regular factor VIII infusions, at least 3 times a week, with the aim minimising haemarthroses and other clinically significant bleeds).

• Low titre inhibitors, past history of an inhibitor, abnormal liver function, drugs that interfere with haemostasis and low CD4 counts are allowed.

Exclusion Criteria:

• Presence of a target joint on prophylaxis (defined as 3 bleeds into one joint, during a 6 month period,during the last year).

• The occurrence of more than 3 haemarthroses in the last year which required more than 2 infusions to resolve