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

Area of Science:

• Pediatric Hematology
• Neurodevelopmental Pediatrics
• Clinical Psychology

Background:

• Contemporary care for hemophilia has evolved significantly, prompting an assessment of its impact on neurodevelopment.
• The Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence, and Other Cognitive Functions (eTHINK) study (NCT03660774) was initiated to investigate these effects.
• Understanding the long-term cognitive and adaptive outcomes in individuals with hemophilia is crucial for comprehensive care.

Purpose of the Study:

• To evaluate cognitive, behavioral, and adaptive functions in children and young adults with hemophilia.
• To establish normative data for neurodevelopmental monitoring in this population.
• To identify specific age or patient groups that may require targeted interventions.

Main Methods:

• A prospective, cross-sectional, observational study involving 551 males aged 1-21 years with hemophilia A or B.
• Participants underwent neurological examinations and standardized neuropsychological assessments.
• Evaluations covered early development, cognition, emotional/behavioral adjustment, and adaptive skills.

Main Results:

• Cognitive test performance was largely comparable to US population norms.
• Specific age groups (4-5 and 10-21 years) showed deficits in attention and processing speed.
• Adolescents and young adults, particularly those with attention-deficit/hyperactivity disorder (ADHD), reported more daily life challenges with adaptive and executive functions. The incidence of ADHD in adolescents was 21%.

Conclusions:

• Males with hemophilia generally exhibit age-appropriate intellectual, behavioral, and adaptive development.
• Poorer attention and concerns regarding executive and adaptive development were noted in specific subgroups.
• The study provides valuable normative data and underscores the importance of screening for and intervening in cognitive and adaptive skill challenges in individuals with hemophilia.