Clinical Trial
Study Details
Eye-tracking is an objective, non-invasive method and particularly suited to assess neurodevelopmental outcome in infants.
proteomics analyses methylation analyses
The strengths and difficulties questionnaire is a conventional instrument used to measure the presence of psychosocial problems, the child's strengths and the influence of psychosocial problems on daily functioning.
The TNO-AZL Preschool Children Quality of Life is a questionnaire that measures the health-related quality of life of children over the last three months
The Bayley Scale of Infant Development is a conventional instrument used to measure developmental outcome in infants
Evidence indicates that Sickle Cell Disease (SCD) threatens neurodevelopmental outcome. Although children with SCD may be heterogeneously affected, neurocognitive impairment may already be present in toddlers. Neurocognitive functioning is an important determinant of adaptive daily life functioning later in life and is influenced by both the course of the disease and the often suboptimal environment in which afflicted children grow up. Early identification of children at the highest risk of neurocognitive impairment would enable the deployment of early interventions to mitigate the detrimental effects of SCD on the developing brain. In order to develop such interventions, a deeper understanding of the underlying pathophysiological mechanisms is required. Therefore the main aim is to study early neurocognitive functioning and development in children with SCD between the ages of 6 and 24 months old.
Study Overview
Objective: The main aim is to study early neurocognitive functioning and development in very young children with SCD. The secondary aims are to 1) (i) identify demographic, psychosocial and clinical determinants and (ii) identify biomarkers associated with early neurocognitive deficits in young children with SCD. 2) Analyze the association between early neurocognitive functioning and adaptive daily life functioning.
Study design: Prospective observational study with an accelerated longitudinal design. The accelerated longitudinal design allows for the inclusion of children from a broad age range (6 - 24 months). During the total study period of 30 months, children can enroll in the study at the ages of 6, 12, 18 and 24 months. After enrollment, children will visit every 6 months until they reach the age of 24 months. This design spans the age range of interest in a shorter period of time and a smaller load on participants as compared to a conventional longitudinal design.
Methods: Eye-tracking measurements will take place at every study visit. Demographic, psychosocial and clinical characteristics of the SCD group will be collected using prospective and structured clinical registration as part of regular clinical care. Demographic, psychosocial and clinical information of the control group will be collected using a parent questionnaire administered during the first and last study visit. Blood samples of children with SCD will be taken as part of regular blood checks at the age of 12 and/or 24 months (depending on the age of enrollment in the study). The Child Behavior Checklist, TNO AZL Preschool Children Quality of Life and the Bayley Scale of Infant and Child Development will be administered to all children at the age of 24 months.
Main study parameters/endpoints: Neurocognitive functioning, as measured using eye-tracking.
Secondary study parameters/ endpoints: The following determinants will be measured: Demographic, psychosocial and clinical characteristics, as measured as part of regular clinical care (SCD group) or via parent questionnaire (control group) and Biomarkers, as measured in blood (SCD group). Adaptive daily life functioning, as measured by quality of life, behavioural and emotional functioning questionnaire, and general (cognitive) development assessment.
Contacts and Locations
Amsterdam UMC
1105AZ Amsterdam
Netherlands