Master en Medecine - Faculte de Medecine de l’Universite Libre de Bruxelles 3e année Master de specialisation en Pediatrie - ULB
2020-2021, 2021-2022, 2022-2023, 2023-2024: "Bourse en mémoire de Paul Danblon"
Prospective study of inflammatory and hemostatic plasmatic markers of endothelial lesions in a cohort of children with sickle cell disease.
Laboratory of Translational Medicine and Laboratory of Pediatrics of the Free University of Brussels (ULB)
The primary objective is to determine if a specific SCD sub-phenotype can be attributed during infancy based on biomarker profile in order to predict and prevent clinical manifestations more accurately.
The secondary objectives include:
- The evaluation of potential associations between early steady-state inflammatory, and hemostatic plasma markers of endothelial dysfunction and later complications in children with SCD.
- A longitudinal evaluation of these markers during early childhood.
- An evaluation of the repercussions of therapeutic intervention on these markers.
SCD results from a mutation of the ß-globine gene and is the most frequent hemoglobinopathy in Belgium. It is associated with significant morbimortality hence the interest in an early and targeted care. At present, there is no plasmatic marker able to identify infants at higher risk of developping severe complications later in life. However, recent studies have demonstrated a correlation between certain complications of the disease and biomarkers of the endothelial dysfunction characterizing it.
2 Study objectives
These studies being predominantly transversal, the objective of our study is to evaluate prospectively potential associations between these plasmatic markers and the occurrence of later complications. This would enable us to verify if patients at risk of developing severe complications can be detected at a young age and therefore improve their early management.
We followed a cohort of children diagnosed with SCD through the universal neonatal screening programme in one of the three participating IRIS centers prospectively for 8 years. Patients were enrolled after obtaining written consent from both parents and if inclusion/exclusion criteria were met. Biological, clinical and radiographic data were collected prospectively every year according to protocol. We will study the annual evolution of plasmatic levels of inflammatory and hemostatic markers in our cohort. We then will evaluate potential associations between these biological markers and the occurrence of SCD related complications.