Voxelotor improves red blood cell functionality in children with sickle cell anaemia: An ancillary study of the HOPE-KIDS 1 trial.

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.

SUBMITTER: Chonat S 

PROVIDER: S-EPMC10887232 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature