Glanzmann Thrombasthenia NHS +
Research type
Research Study
Full title
Glanzmann Thrombasthenia Natural History Study +
IRAS ID
345584
Contact name
S. Sivapalaratnam
Contact email
Sponsor organisation
UMC Utrecht
Duration of Study in the UK
2 years, 0 months, 1 days
Research summary
Glanzmann Thrombasthenia is a rare autosomal recessive platelet disorder characterized by a lack of functional integrins alpha IIb or beta III (glycoproteins IIb/IIIa). The clinical phenotype is dominated by an increased mucocutaneous bleeding tendency. In absence of a primary bleeding prophylaxis, the current treatment of Glanzmann thrombasthenia is mainly focussed on prevention or management of bleeding. However, as potential new therapies emerge, clinicians require long-term safety and efficacy data on both current treatment and new therapies.
The Glanzmann NHS+ study was designed to investigate the genetic phenotype and prevalence of antibodies against human leukocyte antigen (HLA) and human platelet antigen (HPA), the latter two being a potential consequence of the current golden standard treatment: platelet transfusion. The results of this study will be merged with a longitudinal registry with retrospective and prospective data collection of clinical phenotype, bleeding burden and bleeding management. Analysis of the data from the Glanzmann Natural History Study+ and the Glanzmann Natural History Study (registry) will help us to get a better understanding of the clinical variation among patients with Glanzmann's disease. The ultimate goal is to accelerate improvent of the care of patients with Glanzmann's disease.
REC name
London - Bloomsbury Research Ethics Committee
REC reference
24/LO/0890
Date of REC Opinion
17 Jan 2025
REC opinion
Further Information Favourable Opinion