Emicizumab Prophylaxis Every Four weeks among Hemophilia a with Inhibitors in Iraq, Multicenter Study

Abstract

Introduction:    Hemophilia A is a chronic inherited bleeding disease required recurrent intravenous infusions of FVIII or bypassing agents.Emicizumab  is a novel drug used in hemophilia prophylaxiswith or without inhibitors, is given subcutaneously weekly, every 2weeks or every 4 weeks.

 Aim: assess safety, efficacy, quality of lifeand cost effectiveness of emicizumab prophylaxis treatment every 4 weeks inadult &pediatric patients with hemophilia A with inhibitor. 

Methods: This is an interventional study, as open label in multi center extension in Iraqi hemophilia centers, it had been conducted from April 2021 to October 2021 on 32 hemophilia A with high titer inhibitors treated  with emicizumab prophylaxis, compared with same patients in previous 6 months with episodic recombinant factor VII.

Results :- The age of patients, ranged from 1 to 46 years , 75% of patients <18years, they have statistically significant improvement in all variable factors: rate of bleeding, joint bleeding , number of hospital admission , blood transfusion , school or work absence , FISH score  , EQ-5D-5L , cost effectiveness , Hb% and aPTT   in 6mo of emicizumab  prophylaxis therapy , in comparison to previous 6month of episodic rFVIIa treatment. Injection site reaction was the most common adverse effect, no risk of thrombosis.

Conclusion: emicizumab prophylaxis therapy every 4 weeks, is safe, effective, improve quality of life & cost effective.