학술논문
Neonatal screening improves sickle cell disease clinical outcome in Belgium.
Document Type
Article
Author
Source
Subject
*BACTEREMIA prevention
*SICKLE cell anemia diagnosis
*BACTEREMIA
*INFECTION
*ANEMIA
*ACUTE chest syndrome
*BRAIN diseases
*CONFIDENCE intervals
*HOSPITAL care
*NEWBORN screening
*MEDICAL cooperation
*SCIENTIFIC observation
*HEALTH outcome assessment
*PROBABILITY theory
*RESEARCH
*SEX distribution
*SICKLE cell anemia
*STATISTICS
*SURVIVAL
*DATA analysis
*TREATMENT effectiveness
*SEVERITY of illness index
*KAPLAN-Meier estimator
*GENOTYPES
*DISEASE complications
*PROGNOSIS
*
*
*
*
*
*
*
*
*
*
*
*
*
*
*
*
*
*
*
*
*
*
*
*
*
Language
ISSN
0969-1413
Abstract
Objectives To compare the outcomes of sickle cell disease patients diagnosed through neonatal screening with those who were not. Methods In an observational multicenter study in Belgium, 167 screened and 93 unscreened sickle cell disease patients were analyzed for a total of 1116 and 958 patient-years of follow-up, respectively. Both groups were compared with propensity score analysis, with patients matched on three covariates (gender, genotype, and central Africa origin). Bonferroni correction was applied for all comparisons. Results Kaplan–Meier estimates of survival without bacteremia were significantly higher in the screened group than the unscreened group (94.47%; [95% CI, 88.64–97.36%] versus 83.78% [95% CI, 72.27–90.42%]), p = 0.032. Non-significant differences between both groups were reported for survival without acute chest syndrome, acute anemia, cerebral complication, severe infection, and vaso-occlusive crisis. Significantly lower hospitalization rate and days per 100 patient-years were observed in the screened compared with the unscreened group (0.27 vs. 0.63 and 1.25 vs. 2.82, p = 0.0006 and <0.0001). Conclusion These data confirm the benefit of a neonatal screening programme in reducing bacteremia and hospitalization. [ABSTRACT FROM AUTHOR]