Literature DB >> 33872458

Management of children with hemophilia A: How emicizumab has changed the landscape.

Guy Young1.   

Abstract

The key to having a good quality of life for an adult with hemophilia rests largely on how they were managed as children. With effective prophylaxis, young men can begin their adult life with excellent joint function and few, if any, other sequelae from their disease. Unfortunately, this outcome is not always (nor often) attained due to the limitations of the mainstay of treatment which is factor replacement therapy. In resource-rich countries with an adequate supply of factor concentrates, the treatment burden and formation of inhibitors limit the potential for an ideal outcome, while in much of the world factor concentrates are too expensive to even be an option. The novel agent, emicizumab, which has become available in numerous countries around the world, is reshaping how one approaches the treatment of children with hemophilia A. This Forum Article, based on a State-of-the-Art lecture given at the 2020 International Society on Thrombosis and Haemostasis Virtual Meeting, presents an approach including clinically applicable algorithms for treating children with hemophilia A in the new era with emicizumab. This article is protected by copyright. All rights reserved.

Entities:  

Keywords:  children; emicizumab; hemophilia; immune tolerance induction; inhibitor

Year:  2021        PMID: 33872458     DOI: 10.1111/jth.15342

Source DB:  PubMed          Journal:  J Thromb Haemost        ISSN: 1538-7836            Impact factor:   5.824


  3 in total

1.  Current Choices and Management of Treatment in Persons with Severe Hemophilia A without Inhibitors: A Mini-Delphi Consensus.

Authors:  Antonio Coppola; Massimo Franchini; Giovanni Pappagallo; Alessandra Borchiellini; Raimondo De Cristofaro; Angelo Claudio Molinari; Rita Carlotta Santoro; Cristina Santoro; Annarita Tagliaferri
Journal:  J Clin Med       Date:  2022-02-02       Impact factor: 4.241

2.  Emicizumab state-of-the-art update.

Authors:  Johnny Mahlangu; Alfonso Iorio; Gili Kenet
Journal:  Haemophilia       Date:  2022-05       Impact factor: 4.263

3.  Building the blueprint: Formulating a community-generated national plan for future research in inherited bleeding disorders.

Authors:  Leonard A Valentino; Michelle L Witkop; Maria E Santaella; Donna DiMichele; Michael Recht
Journal:  Haemophilia       Date:  2022-06-14       Impact factor: 4.263

  3 in total

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