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Introduction
The UK National Haemophilia Database (NHD) collects data from all UK persons with haemophilia A with inhibitors (PwHA‐I). It is well‐placed to investigate patient selection, clinical outcomes, drug safety and other issues not addressed in clinical trials of emicizumab.
Aims
To determine safety, bleeding outcomes and early effects on joint health of emicizumab prophylaxis in a large,...
Introduction
Factor VIII inhibitor development is currently the most serious complication of the treatment of haemophilia A. Differences in manufacturing and the molecular structure of brands of recombinant factor VIII have led to speculation that concentrates may differ in immunogenicity. This has led to a regulatory focus on the immunogenicity of factor VIII concentrates both before and after licensure...
Introduction
Intracranial haemorrhage in children with inherited bleeding disorders is a potentially life‐threatening complication and presents a significant therapeutic challenge.
Aim
To define the characteristics, management and outcomes of intracranial haemorrhage presenting in UK children ≤16 years of age with inherited bleeding disorders from 2003 to 2015.
Method
Retrospective analysis of...
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