PB0651 - Health-Related Quality-of-Lives in Hemophilia A patients on prophylactic therapy of Emicizumab (Hemlibra)

Background: Hemophilia A (HA) is a congenital bleeding disorder. Understanding the impact of the disease is critical, and improvement in the quality of life from treatments that significantly reduce bleeds is of high interest.

Aims: The following study was conducted to determine the outcome of Emicizumab (Hemlibra) prophylaxis on the Health-Related Quality-Of-Lives in HA patients.

Methods: This study was conducted from January to December 2021. Hemlibra dose was 3mg/kg in the first 4 weeks and the maintenance dose was started at week 5 at 6mg/kg/month. Quality of life and disability in these patients was assessed using EQ-5D-5L and FISH questionnaires.

Results: A total of 36 male HA patients with ABR >8 were enrolled in the study, among them 14(38.8%) patients had positive inhibitor. Median age of the patients was 18 years (IQR-18) with history of consanguinity in 30(83.3%) and family history of bleeding in 29(80.5%) patients. Health related quality of life (Figure 1) was assessed at different time points i.e. baseline, at 6 months and at 12 months after prophylactic treatment. The scores marked by the patients after treatment using visual analog scale (VAS) indicated < 60% of health status of 3(8.3%), 60-80% of 28(77.8%) and >80% of 5(13.9%) patients. Frequency of bleeding symptoms; hemarthrosis, gums bleeding and bruising were also reduced during the follow-up (up to 8.3% vs 2.8 vs 2.8%). Moreover, FISH was also assessed to measure the disability (Table 1). Median (IQR) FISH scores noted were 8.0(5) vs 12.0(16) vs 30(2) with significant change (p value=0.000) in functional independence after Hemlibra. The treatment frequency was observed in 78% patients at baseline which was reduced to 22.8% at 6 months and 8.3% at 12 months.

Conclusion(s): Our results suggest that patients on prophylactic treatment with Hemlibra were less restricted and had improved quality of life, especially improved health and social lives