Literature DB >> 31069799

How I treat children with haemophilia and inhibitors.

Guy Young1.   

Abstract

Haemophilia is a serious inherited bleeding disorder resulting from a deficiency of coagulation factor VIII (haemophilia A) or coagulation factor IX (haemophilia B). While effective therapies have been developed to replace the missing factor and restore normal coagulation, they can lead to the development of neutralizing anti-drug antibodies, termed inhibitors, which significantly worsen morbidity and quality of life. While advancements have been made to improve the management of patients, and particularly for this review, children with inhibitors, there remain significant unmet needs including inhibitor prevention, inhibitor eradication and, when those fail, more effective bleed prevention and a reduction in the treatment burden. This review will discuss current treatment options including the recently licensed bispecific antibody, emicizumab, and provide insights into how I treat children with inhibitors.
© 2019 British Society for Haematology and John Wiley & Sons Ltd.

Entities:  

Keywords:  emicizumab; haemophilia; immune tolerance; inhibitors

Mesh:

Year:  2019        PMID: 31069799     DOI: 10.1111/bjh.15942

Source DB:  PubMed          Journal:  Br J Haematol        ISSN: 0007-1048            Impact factor:   6.998


  5 in total

1.  F8 gene mutation spectrum in severe hemophilia A with inhibitors: A large cohort data analysis from a single center in China.

Authors:  Jie Sun; Zekun Li; Kun Huang; Di Ai; Gang Li; Xingjuan Xie; Hao Gu; Guoqing Liu; Yingzi Zhen; Zhenping Chen; Runhui Wu
Journal:  Res Pract Thromb Haemost       Date:  2022-06-08

2.  [How I treat hemophilia with inhibitors].

Authors:  R C Yang
Journal:  Zhonghua Xue Ye Xue Za Zhi       Date:  2019-10-14

Review 3.  Translational Potential of Immune Tolerance Induction by AAV Liver-Directed Factor VIII Gene Therapy for Hemophilia A.

Authors:  Benjamin J Samelson-Jones; Valder R Arruda
Journal:  Front Immunol       Date:  2020-04-28       Impact factor: 7.561

Review 4.  Clinical Evidence and Safety Profile of Emicizumab for the Management of Children with Hemophilia A.

Authors:  Sandra Le Quellec
Journal:  Drug Des Devel Ther       Date:  2020-02-03       Impact factor: 4.162

5.  Origins, Development, Current Challenges and Future Directions with Activated Prothrombin Complex Concentrate for the Treatment of Patients with Congenital Haemophilia with Inhibitors.

Authors:  Hans H Brackmann; Wolfgang Schramm; Johannes Oldenburg; Viridiana Cano; Peter L Turecek; Claude Négrier
Journal:  Hamostaseologie       Date:  2020-07-27       Impact factor: 2.145

  5 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.