The effect of emicizumab prophylaxis on long- term, self- reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies

Abstract

IntroductionSevere haemophilia A (HA) has a major impact on health- related quality of life (HRQoL).AimAssess the impact of emicizumab on HRQoL in persons with severe HA (PwHA) without factor VIII (FVIII) inhibitors in the phase 3 HAVEN 3 and 4 studies.MethodsThis pooled analysis examines the HRQoL of PwHA aged - ¥ 18 years treated with emicizumab prophylaxis via Haemophilia- Specific Quality of Life Questionnaire for Adults (Haem- A- QoL) and EuroQoL 5- Dimensions 5- levels (EQ- 5D- 5L). In particular, changes from baseline in Haem- A- QoL - Physical Health- (PH) domain and - Total Score- (TS) are evaluated.ResultsAmong 176 evaluable participants, 96 (55%) had received prior episodic treatment and 80 (45%) prophylaxis; 70% had - ¥ 1 target joint and 51% had experienced - ¥ 9 bleeds in the previous 24Â weeks. Mean Haem- A- QoL PH and TS improved after emicizumab initiation. Mean (standard deviation) - 12.0 (21.26)- and - 8.6 (12.57)- point improvements were observed in PH and TS from baseline to Week 73; Week 73 scores were 27.9 (24.54) and 22.0 (14.38), respectively. Fifty- four percent of participants reported a clinically meaningful improvement in PH scores (- ¥ 10 points) by Week 73. Subgroups with poorer HRQoL prior to starting emicizumab (i.e. receiving episodic treatment, - ¥ 9 bleeds, target joints) had the greatest improvements in PH scores, and corresponding reductions in missed workdays; change was not detected among those previously taking prophylaxis. No change over time was detected by the EQ- 5D- 5L questionnaire.ConclusionsEmicizumab prophylaxis in PwHA without FVIII inhibitors resulted in persistent and meaningful improvements in Haem- A- QoL PH and less work disruption than previous treatment.