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

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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.

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Journal

Title: Blood

Publisher: American Society of Hematology

ISSN (Print): 0006-4971

ISSN (Electronic): 1528-0020

Publication date Created: December 12 2019

Publication date (Print): December 12 2019

Volume: 134

Issue: 24

Pages: 2127-2138

Affiliations

[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

Article

DOI: 10.1182/blood.2019001869

PMC ID: 6908828

PubMed ID: 31697801

SO-VID: 2563c36e-ac0e-420f-9c27-967a3acffca6

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

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