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Updated: Oct 24, 2025
A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
Developmental screening of three-year-old children with sickle cell disease compared to controls
Winfred Wang1, Molly Freeman1, Latacha Hamilton1
1Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Insights
Three-year-olds with sickle cell disease (SCD) show higher rates of neurodevelopmental deficits compared to controls. Lower household income and larger household size were linked to these deficits in children with SCD.
Area of Science:
- Pediatrics
- Neuroscience
- Hematology
Background:
- Previous research indicated common neurodevelopmental deficits in young children with sickle cell disease (SCD).
- The clinical significance of these deficits remained uncertain due to the lack of a control group in prior studies.
Purpose of the Study:
- To prospectively compare neurodevelopment in three-year-old children with SCD against an age-appropriate control group.
- To identify factors associated with neurodevelopmental outcomes in children with SCD.
Main Methods:
- A prospective comparative study involving 245 three-year-olds.
- Participants included children with SCD (n=111), community controls (n=114), and SCD patients on hydroxycarbamide treatment (n=20).
- Neurodevelopment was assessed using the Brigance Preschool Screen II, a 15-20 minute screening examination.
Main Results:
- A higher rate of "below cut-off" scores on the Brigance test was observed in children with SCD (73%) compared to controls (61%; P=0.04).
- Both SCD patients and matched controls exhibited high rates of "failing" the Brigance test.
- In multivariate analysis of SCD patients, lower household income and a greater number of people living in the home independently predicted "below cut-off" scores.
Conclusions:
- Three-year-olds with SCD demonstrate elevated rates of neurodevelopmental deficits compared to community controls.
- High rates of neurodevelopmental concerns were noted in both SCD patients and controls, suggesting broader societal factors.
- In untreated children with SCD, low household income and increased household size were associated with poorer neurodevelopmental screening results.
Abstract:
We previously found that neurodevelopmental deficits commonly occurred in three-year-olds with sickle cell disease (SCD), but clinical significance was uncertain because a comparison group was lacking. Our objective in the current study was to prospectively compare neurodevelopment in three-year-old children with SCD to an age-appropriate control group. The Brigance Preschool Screen II is a neurodevelopmental screening examination which can be administered in 15-20 min. SCD patients (Group 1) were compared with community controls of similar age and ethnicity enrolled in daycare/preschool (Group 2). SCD patients who were receiving hydroxycarbamide treatment were also compared (Group 3). Two hundred forty-five three-year-olds were evaluated: Group 1, 111; Group 2, 114; and Group 3, 20. The below cut-off rate on the Brigance test was higher in Group 1 (73%) than in Group 2 (61%; P = 0·04). In multivariate analysis of Group 1 patients, only lower household income and more persons living in the home were independent predictors of this. Patients with SCD and matched controls had high rates of 'failing' the Brigance test. The below cut-off rate in untreated children with SCD was associated with low household income and increased number of persons living in the home.
