JMIR Publications

ABSTRACT

Background:

Sickle cell disease (SCD) is the most common monogenic disorder in humans and occurs predominantly among individuals who identify as Black or African American in the United States. In earlier work, we found that developmental delays were present in more than 50% of children with SCD before the age of three, yet none had been diagnosed or referred to intervention services. Children whose caregivers participated in a home-based caregiver education program demonstrated improved scores on standardized developmental measures. When developmental delays go unidentified, children miss a critical opportunity for intervention during a period of rapid neurological change. Yet few, if any, studies have described the incidence and severity of developmental delays among children with SCD compared to controls.

Objective:

The purpose of this study is to determine the incidence and severity of developmental delays in children with SCD under three years of age (Aim 1), test a 12-month home-based Sickle Cell Collaboration for Child Development (SCCCD) intervention (Aim 2), and conduct a mixed methods study to identify contextual determinants to prepare for future scaling of the SCCCD intervention across healthcare systems (Aim 3).

Methods:

Consistent with American Academy of Pediatrics guidelines, children with SCD will be evaluated at 9, 18, and 30 months using the Bayley Scales of Infant Development-4 (Bayley-4) to determine the incidence of developmental delay over the first 3 years of life compared to demographically matched peers (n=100, Aim 1). The SCCCD intervention, adapted from a pilot study, combines skilled occupational therapy, the Parents as Teachers® curriculum, and SCD-specific caregiver education, delivered over 12 monthly home visits (n=25, Aim 2). Interviews with caregivers who participated in and those who declined the intervention will identify contextual determinants (i.e., facilitators and barriers) to inform future testing and broader implementation of the SCCCD (Aim 3).

Results:

This project has been approved by the Institutional Review Board at Washington University School of Medicine (#202104034, 202407080). As of 5/21/26, a total of 50 participants (26 children with SCD, 24 typically developing children for the comparison cohort) have participated for Aim 1. Aim 2 recruitment began in July 2025 and five caregiver/child dyads have participated so far. The study is expected to be completed by 2028.

Conclusions:

These findings will provide the first prospective characterization of developmental trajectories in children with SCD across the first three years of life and establish preliminary evidence for a disease-specific, home-based intervention to improve developmental outcomes in this underserved population. Results will directly inform a future randomized controlled trial of the SCCCD intervention.