Abstract 718: Cerebral Small Vessel Disease In Children With Sickle Cell Disease

Introduction: The neurological complications of sickle cell disease (SCD) are particularly devastating and range from “overt stroke” to cerebral small vessel disease (CSVD) phenotypes such as silent cerebral infarction (SCI) and cognitive deficit in the absence of magnetic resonance imaging (MR)-detectable evidence of cerebral injury, suggesting the presence of covert mechanism such as dilated perivascular spaces (dPVS). Methods: We quantified the presence of dPVS in children with SCD who were screened for or enrolled in the SIT trial, which was a multicenter, randomized clinical trial that evaluated the effect of monthly chronic transfusions to prevent the development of new SCI in children with SCD. dPVS were evaluated on T2-weighted MRI from 491 children with SCD. We used both the Wardlaw (part of the international consensus criteria for evaluating CSVD) and Rotterdam criteria to quantify dPVS burden in 3 or 4 brain regions respectively. Results: The median age was 8.5 years (range 5 – 15 years). 136 children (28%) had MRI evidence of at least one SCI. The mean hemoglobin was 8.1 ± 1.1 g/dL and total WBC count was 12,000 ± 5300 cells/ml. The mean dPVS burden score from the Wardlaw criteria was 3.5 ± 1.5, while that for the Rotterdam criteria was 31 ± 13 (see Table). Global and regional dPVS burden scores from the two rating scales were well correlated (p < 0.001, r = 0.7 – 0.8). There was a significant sex difference in dPVS burden (mean Wardlaw rating: males = 3.7 versus females = 3.4, p = 0.04), but this disappeared in multivariate analysis, which showed that only increasing age was associated with dPVS burden (p = 0.02). Surprisingly, presence or size of SCI were not associated with dPVS burden score. Conclusions: Children with SCD have more evidence of dPVS than would be expected for their age and compared to matched healthy children. There was no significant association between dPVS burden and presence of cerebral injury such as SCI. Table