A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors

Author:

Young Guy1,Liesner Ri2,Chang Tiffany3,Sidonio Robert4,Oldenburg Johannes5ORCID,Jiménez-Yuste Victor6ORCID,Mahlangu Johnny7,Kruse-Jarres Rebecca8,Wang Michael9ORCID,Uguen Marianne10,Doral Michelle Y.3,Wright Lilyan Y.3,Schmitt Christophe10,Levy Gallia G.3,Shima Midori11,Mancuso Maria Elisa12

Affiliation:

1. Children’s Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA;

2. Great Ormond Street Hospital, London, United Kingdom;

3. Genentech, Inc, South San Francisco, CA;

4. Children’s Healthcare of Atlanta, Emory University, Atlanta, GA;

5. Institute of Experimental Hematology and Transfusion Medicine, University of Bonn, Bonn, Germany;

6. Hospital Universitario La Paz, Autónoma University, Madrid, Spain;

7. National Health Laboratory Service (NHLS), Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand, Johannesburg, South Africa;

8. Bloodworks Northwest, Seattle, WA;

9. School of Medicine Hemophilia and Thrombosis Center, Anschutz Medical Campus, University of Colorado, Aurora, CO;

10. F. Hoffmann–La Roche Ltd, Basel, Switzerland;

11. Department of Pediatrics, Nara Medical University, Kashihara, Japan; and

12. Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre, Ospedale Maggiore Policlinico, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda, Milan, Italy

Abstract

In a Plenary Paper, Young et al describe impressive favorable outcomes of emicizumab prophylaxis in children with hemophilia A and factor VIII inhibitors, reporting a 99% reduction in annualized bleeding, with 77% of patients having no treated bleeding events.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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