The eTHINK Study: Cognitive and Behavioral Outcomes in Children with Hemophilia

Authors

Christine Mrakotsky, Boston Children's Hospital/Harvard Medical School, Boston, MA. Electronic address: christine.mrakotsky@childrens.harvard.edu.
Karin S. Walsh, Children's National Hospital and The George Washington University School of Medicine, Washington, DC.
Cathy Buranahirun Burns, Keck School of Medicine, University of Southern California/Children's Hospital Los Angeles, Los Angeles, CA.
Stacy E. Croteau, Boston Children's Hospital/Harvard Medical School, Boston, MA.
Anja Markert, Novo Nordisk Health Care AG, Zurich, Switzerland.
Milan Geybels, Novo Nordisk Health Care AG, Zurich, Switzerland.
Cara Hannemann, Indiana Hemophilia and Thrombosis Center, Indianapolis, IN.
Madhvi Rajpurkar, Carman and Ann Adams Department of Pediatrics, Children's Hospital of Michigan/Wayne State University, Detroit, MI.
Kevin A. Shapiro, Cortica Healthcare, San Diego, CA.
Greta N. Wilkening, University of Colorado School of Medicine/Children's Hospital Colorado, Aurora, CO.
Pamela Ventola, Cogstate, New Haven, CT.
David L. Cooper, Novo Nordisk Inc, Plainsboro, NJ.

Document Type

Journal Article

Publication Date

5-9-2024

Journal

The Journal of pediatrics

Volume

272

DOI

10.1016/j.jpeds.2024.114089

Keywords

hemophilia; neurology; neuropsychology

Abstract

OBJECTIVE: To assess cognitive, behavioral, and adaptive functions in children and young adults with hemophilia treated according to contemporary standards of care. STUDY DESIGN: Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence, and Other Cognitive Functions (eTHINK) is a US-based, prospective, cross-sectional, observational study (September 2018 through October 2019). Males (aged 1-21 years) with hemophilia A or B of any severity, with or without inhibitors, were eligible. Participants underwent neurologic examinations and age-appropriate neuropsychological assessments, including standardized tests/ratings scales of early development, cognition, emotional/behavioral adjustment, and adaptive skills. RESULTS: Five hundred and fifty-one males with hemophilia A (n = 433) or B (n = 101) were enrolled. Performance on cognitive tests was largely comparable with that of age-matched US population norms, although participants in certain age groups (4-5 and 10-21 years) performed worse on measures of attention and processing speed. Furthermore, adolescents and young adults and those with comorbid attention-deficit/hyperactivity disorder (ADHD; n = 64) reported more adaptive and executive function problems in daily life. Incidence of ADHD in adolescents (21%) was higher than expected in the general population. CONCLUSIONS: In general, males with hemophilia demonstrated age-appropriate intellectual, behavioral, and adaptive development. However, specific patient/age groups showed poorer attention performance and concerns for executive and adaptive development. This study established a normative data set for monitoring neurodevelopment in individuals with hemophilia and highlight the importance of screening and intervention for challenges with cognitive and adaptive skills in this population. CLINICAL TRIAL REGISTRATION: Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence, and Other Cognitive Functions (eTHINK); NCT03660774; https://clinicaltrials.gov/ct2/show/NCT03660774.

APA Citation

Mrakotsky, Christine; Walsh, Karin S.; Buranahirun Burns, Cathy; Croteau, Stacy E.; Markert, Anja; Geybels, Milan; Hannemann, Cara; Rajpurkar, Madhvi; Shapiro, Kevin A.; Wilkening, Greta N.; Ventola, Pamela; and Cooper, David L., "The eTHINK Study: Cognitive and Behavioral Outcomes in Children with Hemophilia" (2024). GW Authored Works. Paper 4961.
https://hsrc.himmelfarb.gwu.edu/gwhpubs/4961

Department

Pediatrics