Abstract Number: PB/LB04
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Studies conducted decades ago in children with hemophilia demonstrated a negative impact of the disease on cognition. Reduction in HIV and hepatitis C burden and improvements in the standard of care may have changed the ways in which hemophilia influences cognitive development; however, this had not been systematically studied.
Aims: The eTHINK study assessed contemporary effects of hemophilia and associated care on cognitive and neurobehavioral development and established a normative data set for the monitoring of cognitive functions and development in children and adolescents receiving the current hemophilia standard of care.
Methods: Males with hemophilia A or B of any severity, with or without inhibitors, aged 1 to 21 years, were eligible for the study, which was approved by local IRBs at each site. All participants provided informed consent/assent and hemophilia and developmental histories. Children underwent neurological examination and neuropsychological assessment, including age-appropriate standardized tests of developmental (Bayley-III) or intellectual (WPPSI-IV/WASI-II) ability, processing speed, attention (Cogstate Computerized Battery), and parent- and self-report ratings of executive function (BRIEF-P/-2/-A), emotional/behavioral adjustment (BASC-3), and adaptive skills (ABAS-3).
Results: 551 males with hemophilia A (n=433) or B (n=101) were enrolled. For the cohort as a whole, performance on tests of overall intelligence, attention, and processing speed was comparable to that of age-referenced US population norms. However, subgroups of participants and their parents reported more difficulty with adaptive skills and executive function in daily life, particularly adolescents/young adults and those with a diagnosis of attention deficit/hyperactivity disorder (ADHD; n=71) (Figure).
Conclusions: Overall, males with hemophilia performed within age expectation on standardized cognitive tests in the structured test setting. However, adolescents/young adults appear to be at risk for difficulties with attention, executive function, and independence skills in their daily lives. Findings lend support to the need for specific behavioral interventions as patients transition to independence.
[Box and whisker plots of z-score distributions for 5 assessment domains for all participants combined and the subgroup with ADHD.]
To cite this abstract in AMA style:
Mrakotsky C, Buranahirun C, Cooper DL, Croteau SE, Greybels M, Hannemann C, Rajpurkar M, Shapiro KA, Wilkening GN, Ventola P, Walsh KS, on behalf of the eTHINK Study Group . The eTHINK Study: Cognitive and Behavioral Outcomes in Children with Hemophilia [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/the-ethink-study-cognitive-and-behavioral-outcomes-in-children-with-hemophilia/. Accessed September 24, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/the-ethink-study-cognitive-and-behavioral-outcomes-in-children-with-hemophilia/