Honors College Thesis

 

Validation of early predictors of outcome in the Dallas Newborn Cohort Public

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  • Sickle cell disease (SCD) is a phenotypically variable disorder of hemoglobin that leads to abnormally shaped and dysfunctional red blood cells. Several studies have attempted to construct early predictive models for SCD, including Miller et al.’s (2000) model which uses easily identifiable predictors in early life. The primary aim of this project is to validate the model of Miller et al. in the Dallas Newborn Cohort, the world’s largest newborn inception cohort of sickle cell disease. Specifically, we tested the predictive utility of three clinical findings in the first years of life: painful swelling of the hands and feet (dactylitis), severe anemia (Hgb <7 g/dL), and elevation of white blood cell count (leukocytosis) for later complications, which includes death, stroke, painful episodes, and acute chest syndrome. The predictive utility of the Miller model was found to be fairly poor in the Dallas Newborn Cohort. None of the patients with an adverse outcome was properly identified by the model. Thus, the Miller model is not practical to operate as a guide to influence clinical decisions about early disease-modifying interventions for young children with SCD.
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