Inhibitor Map Study
Research type
Research Study
Full title
High-Throughput Amino Acid Sequence Epitope Mapping of Inhibitory Antibodies in Non-Severe and Acquired Haemophilia A
IRAS ID
244749
Contact name
Sean Platton
Contact email
Sponsor organisation
Queen Mary, University of London
Duration of Study in the UK
1 years, 2 months, 30 days
Research summary
Factor VIII (FVIII) is an important protein involved in the formation of blood clots. A lack of FVIII leads to bleeding. People may have low levels of FVIII by inheriting this from their parents (congenital haemophilia) or by reacting to their own FVIII (acquired haemophilia). How serious a person’s haemophilia is relates to how much FVIII their body makes. This can be severe, where they make no FVIII or non-severe when a small amount of FVIII is made. Patients with non-severe haemophilia need treatment with injections of manufactured FVIII when they have surgery or bleeding. About 1 in 20 patients react to this treatment by their bodies making an antibody to FVIII, which is commonly called an “inhibitor”. Antibodies work by recognising patterns (or epitopes) on a substance to identify this as being foreign. Inhibitors react to replacement treatment and occasionally patient’s own FVIII, similar to the antibodies seen in acquired haemophilia. We do not know why the body reacts against itself, but think this is due to these antibodies recognising similar patterns. We are developing a new way to test how FVIII antibodies work to identify these patterns rapidly at a molecular level. This will help us understand how our immune system reacts to FVIII. We plan to collect a single blood sample from participants with non-severe haemophilia with an inhibitor, acquired haemophilia and healthy volunteers. We will test these samples using standard tests to confirm a FVIII antibody is present and then using a bespoke laboratory array that mimics FVIII, based in The Netherlands (Pepscan). When we know where these antibodies bind we will confirm these findings using another technique. We hope this study will provide additional information for more personalised approaches to management.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
20/NW/0345
Date of REC Opinion
21 Aug 2020
REC opinion
Favourable Opinion