Voxelotor improves red blood cell functionality in children with sickle cell anaemia: An ancillary study of the HOPE‐KIDS 1 trial
Voxelotor improves red blood cell functionality in children with sickle cell anaemia: An ancillary study of the HOPE‐KIDS 1 trial
S. Chonat,Earl Fields,6 Authors,David R. Archer
TLDR
It is suggested that voxelotor treatment is associated with reduced RBC sickling and haemolysis in children with SCD.
Abstract
Introduction: Sickle haemoglobin (HbS) polymerisation perturbs red blood cell (RBC) rheology and drives sickle cell disease (SCD) pathophysiology. Voxelotor is an HbS polymerisation inhibitor that increases haemoglobin (Hb)–oxygen affinity. Methods/Results: In this 48‐week, prospective, single‐centre translational study, 10 children aged 4–11 years with SCD were treated with voxelotor. Improvements in RBC deformability were observed using osmotic/oxygen gradient ektacytometry, with increases in minimal and maximal elongation index and reductions in point of sickling. Increased Hb and reduced markers of haemolysis were also observed. Conclusion: These findings suggest that voxelotor treatment is associated with reduced RBC sickling and haemolysis in children with SCD.