Effect of tertiary prophylaxis with low-dose factor VIII in quality of life in adult patients with severe hemophilia A

Abstract

BACKGROUND: Hemophilia A, characterized by deficiency of factor VIII (FVIII), is an X-linked bleeding disorder. It is graded as mild, moderate, or severe based on the FVIII level in plasma. Bleeding-related complications are often associated with severe form of the disease. Prophylaxis has now become the standard of care for severe hemophilia A patients. OBJECTIVES: The study aimed to describe the effect of tertiary prophylaxis with low dose FVIII in quality of life in adult patients with severe hemophilia A. SUBJECTS AND METHODS: A random 20 adult severe hemophilia A male patients aged 18–40 years with joint deformities, were included for this study. Long-acting recombinant FVIII, Fc Fusion protein (ELOCTATE, Biogen) was then given twice weekly at a dose of 15 IU/Kg/dose twice weekly for 3 months. Results were compared with the previous 3 months records of no prophylaxis. Efficacy was determined in terms of annualized bleed rate (ABR ) and absenteeism from work during this study period. Inhibitor screening was done at regular intervals during prophylaxis. RESULTS: There was significant decrease in mean ABR along with absenteeism from work during prophylaxis as compared to on-demand therapy (3.6 bleed/year and 9.4 days/year compared to 37.8 bleed/year and 64.7 days/year, respectively). The mean FVIII requirement during prophylaxis (1560 IU/kg) was also less than the on-demand therapy (2150 IU/Kg/year). Majority (85%) had a FVIII trough level of <1%. Only one patient complained of myalgia while on prophylaxis. No inhibitor development was found in any of the individuals. CONCLUSION: Low-dose recombinant long-acting FVIII prophylaxis can achieve a significantly lower bleeding rate in adult hemophilia A patients along with improved quality of life.

Keywords

• outcome
• recombinant factor viii fc fusion protein
• severe hemophilia a
• tertiary prophylaxis