Emicizumab use in females with moderate or mild hemophilia A without factor VIII inhibitors who warrant prophylaxis.
(2023) Research and Practice in Thrombosis and Haemostasis — Vol. 7, n° 8, p. 102239 [1-7] (2023)
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- 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.
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Hermans, C., Ventriglia, G., Obaji, S., Beckermann, B. M., Lehle, M., Catalani, O., d’Oiron, R., & Frenzel, L. (2023). Emicizumab use in females with moderate or mild hemophilia A without factor VIII inhibitors who warrant prophylaxis. Research and Practice in Thrombosis and Haemostasis, 7(8), 102239 [1-7]. https://doi.org/10.1016/j.rpth.2023.102239 (Original work published 2023)