Emicizumab use in females with moderate or mild hemophilia A without factor VIII inhibitors who warrant prophylaxis.

BACKGROUND

Hemophilia A (HA) is predominantly associated with males due to X-linked inheritance. Males and females with HA have shared unmet medical needs, highlighting the necessity for comprehensive care irrespective of sex.

OBJECTIVES

This analysis investigated the efficacy and safety of emicizumab prophylaxis in 3 females with HA.

METHODS

HAVEN 6 (NCT04158648) is a phase III study of emicizumab in people with non-severe HA without factor (F)VIII inhibitors warranting prophylaxis per investigator assessment, and the study methodology has been reported previously. Female-specific endpoints included menstruation-related quality of life and menstruation heaviness.

RESULTS

HAVEN 6 enrolled 3 females aged ≥18 years and within reproductive age (n = 2 mild HA; n = 1 moderate HA; n = 2 receiving prior FVIII prophylaxis; n = 1 receiving prior episodic FVIII). Participants presented with diverse bleeding phenotypes at baseline: 2 had no bleeds in the 24 weeks prior to enrollment, while 1 had an annualized bleed rate for all bleeds of 208.6. On-study annualized bleed rates for all bleeds were 0, 2.8, and 11.6, respectively. The 2 evaluable participants indicated improved menstruation-related quality of life vs baseline. Two participants experienced 3 grade 1/2 treatment-related adverse events; no new safety signals were identified. All 3 participants preferred emicizumab over their previous treatment and reported a better score for treatment burden and preoccupation domains of the Comprehensive Assessment Tool of Challenges in Hemophilia questionnaire.

CONCLUSION

Overall, results were consistent with those reported in the male population enrolled in the HAVEN 6 study, suggesting efficacy and a favorable safety profile for emicizumab in females with non-severe HA warranting prophylaxis.