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Updated: Mar 16, 2026

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
A pilot study of parent education intervention improves early childhood development among toddlers with sickle cell
Melanie E Fields1, Catherine Hoyt-Drazen2, Regina Abel2
1Division of Pediatric Hematology/Oncology, Department of Pediatrics, Washington University School of Medicine, St. Louis, Missouri. king_a@wustl.edu.
Insights
Early home visitation for children with sickle cell disease (SCD) improved cognitive and expressive language development. A supportive home environment was key to cognitive gains in this vulnerable population.
Area of Science:
- Pediatric Health
- Developmental Psychology
- Hematology
Background:
- Young children with sickle cell disease (SCD) face risks for cognitive delay.
- Both biologic and environmental factors contribute to cognitive dysfunction in children with SCD.
Purpose of the Study:
- To evaluate the impact of a home visitation model on the development of young children with SCD.
- To describe the role of the home environment and socioeconomic status in early childhood SCD.
- To assess the effectiveness of the Parents as Teachers (PAT) program on cognitive outcomes.
Main Methods:
- A single-arm, prospective cohort study followed 35 children with SCD (3-36 months) and caregivers.
- Data collected from October 2010 to December 2013.
- Primary outcomes included the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) and the Home Observation for Measurement of the Environment (HOME).
Main Results:
- Significant improvements were observed in cognitive (P = 0.016) and expressive language (P = 0.002) domains of the BSID-III.
- Mean scores across all five BSID-III subtests improved from enrollment to exit.
- The HOME score was significantly associated with cognitive development outcomes.
Conclusions:
- This study reports the first longitudinal results of a home visitation program for early childhood SCD.
- The program demonstrated significant improvements in cognitive and expressive language development.
- Home environment emerged as a significant predictor of cognitive development, suggesting a need for targeted interventions.
Background:
Young children with sickle cell disease (SCD) are at risk for cognitive delay. In addition to biologic risk factors associated with SCD, environmental factors contribute to cognitive dysfunction within this cohort.
Methods:
We completed a single-arm, prospective cohort study. Children with SCD between the ages of 3 and 36 months and their caregivers were followed between October 2010 and December 2013. The aim was to describe the role of a home visitation model, the home environment, and socioeconomic status in the development of young children with SCD. Primary outcome measures were the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) and the Home Observation for Measurement of the Environment (HOME). We hypothesized that the home visitation model, Parents as Teachers® (PAT), would encourage positive parent-child interactions and improve cognitive outcomes.
Results:
Thirty-five participants had at least two PAT visits and BSID-III assessments. Mean scores within all five subtests of the BSID-III improved between enrollment and exit, with significant changes within cognitive (P = 0.016) and expressive language (EL) domains (P = 0.002). Multivariate modeling found the HOME score associated with the exit results of the cognitive domain.
Conclusion:
We report longitudinal results of the first home visitation program within the early childhood SCD population and show significant improvement in cognitive and EL development. Additionally, home environment was a significant predictor of cognitive development. Randomized controlled trials to test the impact of interventions targeting the home environment are warranted for this vulnerable population.
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