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.