ROTHEM: Return of the Microvesicle - SCD, Microvesicles & Vasculopathy
Research type
Research Study
Full title
Return of the Microvesicle: An observational study to quantify and qualify microvesicles and vasculopathy in patients with sickle cell disorder in the in- and out-patient setting.
IRAS ID
272663
Contact name
Sara Trompeter
Contact email
Sponsor organisation
UCLH
Clinicaltrials.gov Identifier
Z6364106/0000/00/00, UCL Data Protection Registration
Duration of Study in the UK
10 years, 0 months, 1 days
Research summary
Sickle cell disorder (SCD) is now the commonest inherited single gene disorder in the United Kingdom and the commonest cause of childhood stroke. Patients are prone to develop acute crises, necessitating hospital admission and resulting in long-term complications. Such events result in considerable morbidity, disability and mortality with consequential burden on patients, families, the health service and society. SCD is the commonest cause of childhood stroke in the UK, with up to 40% of children having a stroke (clinical or radiological diagnosis) by school age with up to 40% having had a silent infarction by the age of 5.
There is little ability to predict who will get ill and when, hence it is very difficult to known when and how to administer treatments. Furthermore, there is very little in the way of treatments available and the mainstay of prevention of cerebrovascular disease is a chronic blood transfusion programme which is expensive, requires time off work or school and can be fraught with complications, in a population who are frequently educationally and socially disadvantaged at the outset. Recent evidence in SCD points towards the importance of microvesicles (circulating broken pieces of cells) and blood vessel wall damage (vasculopathy) as being important. By comparing levels of microparticles and vasculopathy in patients with those found in healthy volunteers, this study hopes to identify their role. With this information, we aim to be able to improve the management of these patients and potentially find new therapies. Participants will be required to donate a small amount of blood (two teaspoons in small children and five teaspoons in older children/adults), where possible when people are already having a blood test. In the case of patients having exchanges, blood can be used from the waste bag post procedure.
REC name
Wales REC 7
REC reference
22/WA/0075
Date of REC Opinion
16 May 2022
REC opinion
Further Information Favourable Opinion