Lived Experience of Glanzmanns Thrombasthenia UK (Glanzmanns 360)
Research type
Research Study
Full title
The Lived Experience of Glanzmann's Thrombasthenia in the UK (Glanzmanns 360)
IRAS ID
308011
Contact name
Simon Fletcher
Contact email
Sponsor organisation
Haemnet Ltd
Duration of Study in the UK
1 years, 0 months, 0 days
Research summary
Platelets are small cells in the blood that are an important part of how blood clots. Glanzmanns Thrombasthenia is a rare inherited condition affecting 1:1,000,000 males and females globally with 69 males and 85 females diagnosed in the UK.
In Glanzmanns Thrombasthenia the platelets have reduced or absent proteins on the platelet surface, these proteins are important in platelet 'stickiness' which is an important part of how blood clots. For affected individuals bleeding is lifelong and potentially life threatening. Children are usually diagnosed as neonates with bruising and bleeding from the soft membranes of the mouth and nose which continue throughout childhood. At puberty girls suffer from very heavy periods.
Treatment is very reliant on hospital delivered care - this usually means going to hospital once bleeding has started to receive platelet transfusions. There are very limited treatment options for use at home, the burden of treatment is related to the unpredictability of bleeding, hospital care including anaemia which results for constant bleeding.
Due to the rarity of this condition little research has been undertaken about the impact of living with it, not only for the affected individual but also the for the family (who may have more than one affected member). This study aims to capture this impact through the use of validated questionnaires and in-depth interviews to asses quality of life. A short (12-24 week) follow up will assess immediate impacts of bleeding through weekly contact with the research team. We have included patient advocates in the steering committee to ensure that we have a full grasp of the issues that matter to affected individuals across the life age-span.
REC name
South Central - Oxford B Research Ethics Committee
REC reference
22/SC/0095
Date of REC Opinion
29 Mar 2022
REC opinion
Further Information Favourable Opinion