Do children with ITP have subclinical intracranial bleeds on MRI scan?
Research type
Research Study
Full title
Pilot study of brain Magnetic Resonance Imaging (MRI) scans to assess bleeding risk in children with immune thrombocytopenia (ITP-low platelet count).
IRAS ID
252871
Contact name
Nichola Cooper
Contact email
Sponsor organisation
Imperial College London and Imperial College Healthcare NHS Trust
Duration of Study in the UK
1 years, 0 months, 0 days
Research summary
Children with immune thrombocytopenia (ITP) have low platelet counts (thrombocytopenia) due to an autoimmune process that destroys the platelets and the cells that produce them. Platelets are essential in blood clotting. ITP therefore causes an increased risk of bleeding.
However, serious bleeding is very uncommon in children, and the effects of treatment (such as steroids) have historically been the worst aspect of the disease. Current UK guidelines therefore recommend that children are not treated unless there is obvious bleeding; with less than 20% of children in the UK receiving
treatment. This has not resulted in any increase in life-threatening bleeding, however, we do not know whether small bleeds are still occurring internally. Small bleeds in the brain (intracranial) have been observed in adults with ITP and these may affect cognitive function. It may also be a warning sign for more significant bleeds.
We propose performing a special MRI brain scan in 30 children with ITP to look for
small bleeds. We will also take blood tests to identify risk factors to predict which patients will have significant bleeding. We will also include bleeding scores, cognitive testing and health-related quality of life tests in the children.
If we identify small bleeds in some patients, our data will be used to help design a UK-wide prospective MRI brain scan study in childhood ITP to incorporate the presence of intracranial bleeding into treatment pathways.
Our study will help identify children at risk of bleeding and may alter the treatment
approach from conservative to pro-active (with novel steroid-sparing drugs).
Alternatively, if our study finds no intracranial bleeding in children this adds weight to the evidence for continued conservative treatment and would be reassuring for parents and children.Lay summary of study results:
Immune thrombocytopenia is a rare disease where the immune system destroys your platelets. Platelets help to form blood clots, therefore if there aren’t enough platelets there is a risk of bleeding. Despite low platelet counts, most children with ITP do not have severe bleeding, and it often gets better on its own. Therefore many children do not receive treatment. However, the long-term impact of ITP in children is not known. Given that adults with ITP have cerebral microbleeds (tiny bleeds in the brain-CMBs), this study aimed to investigate if children with ITP have them too, and if there was any impact on patient reported outcome measures (PROMs). PROMs assessed included quality of life (QoL), fatigue scores and cognitive testing.
Fifty children (average age 13.4 years, range 8-18) with ITP (average length of time with ITP 28 months, range 1-129) and at least one platelet count < 30 x 109/L (normal range 150-450 x109/L) were recruited for a study including MRI brain and PROMs.
CMBs were identified in 8 patients (16%) (average 2, range 1-10 CMBs), none were seen in healthy controls. CMBs were significantly related to higher bleeding scores and more days with very low platelets (<10 x109/L).
38% of children were fatigued, 24% had impaired cognition and 50% of children and 78% of parents had impaired QoL. Fatigue was related to older age at diagnosis and higher bleeding scores. Impaired QoL was related to older age at diagnosis and lower platelets. CMBs were not associated with fatigue, impaired QoL or cognitive impairment. In summary, CMBs, impaired QoL, fatigue and significant parental impact are common in paediatric ITP. Further study is required to understand what causes fatigue and impaired cognition, and how we can use brain MRI to tailor advice and treatment to individual patients.REC name
London - Bloomsbury Research Ethics Committee
REC reference
18/LO/1911
Date of REC Opinion
12 Nov 2018
REC opinion
Favourable Opinion