Senior Biomedical Scientist Department of Coagulation, Sheffield Haemophilia and Thrombosis Centre, Sheffield Sheffield, United Kingdom
Measuring factor activity: Baseline with treatment including gene therapy The measurement of pre, trough or post infusion Factor VIII or factor IX activities (FVIII:C, FIX:C) is used to guide clinical management. Dosage adjustments, indication of inhibitor development or the highlight of issues with compliance all rely on accurate laboratory testing. More than 20 years ago, differences in FVIII:C were reported when measuring some standard half-life recombinant FVIII concentrates with particular laboratory assays. The introduction of bi-specific antibodies, extended and, recently, ultra long half-life recombinant molecules and gene therapy lead to further monitoring complications for both FVIII:C and FIX:C. Laboratories must pay careful attention to the therapy administered and know whether the assay methodology and reagents available in their centre can accurately measure it. Some haemostasis laboratories are able to use alternative assay methods or reagents but many labs cannot so need to define a plan for patient management. This presentation will discuss some of the monitoring requirements for replacement and non-replacement therapies in haemophilia A and B and highlight some unresolved concerns in monitoring newer molecules.
Learning Objectives:
Measurement of baseline, trough and peak factor activities are used to guide dosage and therapy and highlight any potential issues with inhibitor development or compliance. Advances in treatments for haemophilia A and B has somewhat complicated accurate factor measurement. This presention will discuss how EHL, ULHL and novel therapies for haemophilia can be monitored in the laboratory.
