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Updated: Feb 27, 2026

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
Neonatal screening improves sickle cell disease clinical outcome in Belgium
Phu-Quoc Lê1, Alina Ferster1, Laurence Dedeken1
11 Hemato-Oncology, Hopital Universitaire des Enfants Reine Fabiola, Brussels, Belgium.
Insights
Neonatal screening for sickle cell disease significantly improves survival without bacteremia and reduces hospitalizations. Early diagnosis through newborn screening offers substantial health benefits for children with sickle cell disease.
Area of Science:
- Hematology
- Pediatrics
- Public Health
Background:
- Sickle cell disease (SCD) is a genetic blood disorder requiring early intervention.
- Neonatal screening programs aim to identify SCD at birth for timely management.
- Outcomes for screened versus unscreened SCD populations require comparative analysis.
Purpose of the Study:
- To compare the clinical outcomes of sickle cell disease patients identified through neonatal screening versus those diagnosed later.
- To evaluate the impact of early diagnosis on key health indicators in SCD patients.
Main Methods:
- An observational, multicenter study in Belgium included 167 screened and 93 unscreened SCD patients.
- Propensity score analysis matched patients based on gender, genotype, and origin.
- Kaplan-Meier survival estimates and hospitalization rates were compared using Bonferroni correction.
Main Results:
- Survival without bacteremia was significantly higher in the screened group (94.47%) compared to the unscreened group (83.78%), p=0.032.
- Hospitalization rates and days per 100 patient-years were significantly lower in screened patients (0.27 vs. 0.63 and 1.25 vs. 2.82, respectively).
- No significant differences were found for survival without acute chest syndrome, anemia, cerebral complications, severe infections, or vaso-occlusive crises.
Conclusions:
- Neonatal screening for sickle cell disease demonstrates a significant benefit in reducing bacteremia and hospitalizations.
- Early detection through newborn screening programs positively impacts key health outcomes for SCD patients.
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.

