| Literature DB >> 31069799 |
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.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