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Paediatric (including perinatal) stroke has an incidence of between 1.3 and 13.0 per 100.000 yearly in Europe. 1/3 of children with neonatal and 50% of post-natal stroke will develop a hemiplegia with upper limb being generally more affected than lower limb and a severe impact on children's participation and quality of life. Opportunities to advance scientific knowledge of the influence of genomic variation on the pattern, presentations and prognosis of paediatric stroke are lacking. Conversely, the discovery could have an enormous potential to drive the rehabilitation that is the major component of the stroke patient's care and to achieve a good functional outcome. The present proposal aims to change the current management of care and intervention of children with hemiplegia due to stroke, by identifying relevant biomarkers coming from four different areas (omics, clinical assessment, neuroimaging, Information and Communication Technologies) in order to stratify the children and to create a novel transdisciplinary patient-centred model to optimize and tailor the rehabilitation treatment. As a diagnostic tool, the new workflow allows the set-up for planning an individualized treatment based on patient specific needs, creating a model for an evidence-based clinical decision-making process that starts from the measurements of specific biomarkers, clinical measurements and effective use of patient's Upper Limb. The feasibility of the planned approach can be applied for deeply analysing and understanding results of previous researches and in new pilot studies on already available rehabilitative treatments. The results will provide an example of how different kinds of integrated assessments can contribute to create a plan for the management of children with hemiplegia due to stroke, thus leading to a better understanding of the correlation between genetic and phenotypic data. Finally, the Health Technology Assessment will provide estimates of its national and regional cost effectiveness.
Full description
Etiological causes of stroke in children are manifold and comprise both genetic and epigenetic factors but there are no reliable predictors for prevention or treatment strategies. Phenotypic variation of clinical features poses several challenges to the application of precision stroke medicine, making a simple genetic risk assessment only partially informative on an individual basis. The high incidence and prevalence, together with the high hetereogeneity of the etiology and phenotypic profiles, candidates the stroke in childhood as one of the main disorders in childhood that needs of a Personalised Medicine (PM) approach. The guiding principles of PM in adult stroke underscore, in fact, the need to identify, value, organise and analyse the multitude of variables obtained from each individual to generate a precise approach to optimise rehabilitation target.
The present proposal aims to change the current management of care and intervention of children with hemiplegia due to stroke, providing a new model of precision transdisciplinary approach. The main goal is to identify relevant biomarkers coming from four different areas (omics, clinical assessment, neuroimaging, kinematic analysis) in order to stratify the children and create a novel children stroke Patient Specific Model (PSM) to optimize and tailor the rehabilitation treatment. This approach is highly requested by the need of tailoring the rehabilitation treatment not only on the base of rough estimates of outcomes for a particular treatment planning and intervention but on the identification and analysis of the multitude of variables obtained from each individual. Moreover, much of comprehensive data are already collected by clinicians, but yet not readily used, during previous focused diagnostic and/or rehabilitative research project in a whole and personalized treatment approach (called theranostic, in PM approach). CATCH-HEMI will contribute to the improvement of quality of care of each child with hemiplegia due to stroke.
The investigators will collect all the data in a sample of at least 60 children with hemiplegia due to stroke.
The four areas that will be assessed will be: GENETIC (just a blood sample will be collected); NEUROIMAGING (just the already available structural brain MRI images will be collected); CLINICAL ASSESSMENT: all the 60 participants will be clinically evaluated at baseline with a comprehensive battery of standardised outcome measures (the evaluation will last around 2 hours covering all the domains underlined by the ICF, i.e. International Classification of Functioning, Disability and Health). Additional time will be required for children and parents to fill in questionnaires investigating the participation and quality of life. These questionnaires could be filled in at home and then send via mail to the clinicians. Moreover, for the KINEMATIC evaluation, participants will be asked to worn wearable smart commercial technological solutions (e.g. bracelets with sensors inside, app for registering daily life) for one month after the clinical evaluation.
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60 participants in 1 patient group
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Giuseppina Sgandurra, MD, PhD
Data sourced from clinicaltrials.gov
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