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ASH 2022 | HAVEN 7: emicizumab prophylaxis for the treatment of infants with severe hemophilia A

Steven Pipe, MD, University of Michigan, Ann Arbor, MI, discusses the results from an interim analysis of the HAVEN 7 study (NCT04431726), which is evaluating the safety and efficacy of emicizumab in infants with severe hemophilia A without factor VIII inhibitors. Dr Pipe first highlights the challenges with factor VIII replacement therapy and unmet needs in this patient population, and then goes on to discuss the results of this study. Emicizumab demonstrated promising efficacy, with patients achieving high plasma levels, very low annualized bleed rates (ABR), and no intracranial hemorrhage occurring. This interview took place at the 64th ASH Annual Meeting and Exposition congress in New Orleans, LA.

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Disclosures

Sanofi: Consultancy; Sangamo Therapeutics: Consultancy; Takeda: Consultancy; CSL Behring: Consultancy; UniQure: Consultancy; Freeline: Consultancy; Novo Nordisk: Consultancy; Regeneron/Intellia: Consultancy; Siemens: Research Funding; Spark Therapeutics: Consultancy; BioMarin Pharmaceutical Inc.: Consultancy; HEMA Biologics: Consultancy; Apcintex: Consultancy; Pfizer: Consultancy; ASC Therapeutics: Consultancy; Roche/Genentech: Consultancy; Bayer: Consultancy; Yewsavin: Research Funding.