CLINICAL EFFICACY OF RECOMBINANT FACTOR VIII FC FUSION PROTEIN IN HAEMOPHILIA A PATIENT RECEIVING ON DEMAND TREATMENT ONLY

Abstract

Objective: To evaluate the efficacy of recombinant factor VIII FC fusion protein in haemophilia A patient
receiving on demand treatment only.

Study Design: Comparative cross sectional study.

Place and Duration of Study: Department of Hematology, Armed Forces Institute of Pathology and Pakistan
Hemophilia Welfare Society, Rawalpindi, from Jun to Dec 2017.

Methodology: Eighty-nine male patients of Hemophilia A already receiving recombinant factor VIII (20-30 Units/kg) on demand, with no history of inhibitors were included in study. Patients were divided as per age into paediatric and adult group and also on the basis of their basal factor VIII levels into severe, moderate and mild groups. Same patients were switched to recombinant factor VIII FC fusion protein (20-30 Units/kg) and its efficacy was measured and compared with recombinant Factor VIII in terms of dose requirement, injections, bleeds in six month period, presence of inhibitors and side effects.

Results: Eighty nine male patients were studied. There was significant reduction in dose from median value of
5750 units for group I to 4000 units for group II. Number of bleed in six month period were reduced from 5.3 in
group I to 4.5 in group II. Number of injections were reduced on average to 1-2 injection per bleed in group II. No inhibitors were detected in group II.

Conclusion: rFVIII Fc fusion protein has prolong activity and results in reduction of total dose, number of bleed,
dose per bleed and has reduced antigenecity.