eJHaem (Feb 2024)

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

  • Satheesh Chonat,
  • Earl Fields,
  • Hannah Baratz,
  • Amanda Watt,
  • Mira Pochron,
  • Sandy Dixon,
  • Margaret Tonda,
  • Clark Brown,
  • David Archer

DOI
https://doi.org/10.1002/jha2.831
Journal volume & issue
Vol. 5, no. 1
pp. 125 – 130

Abstract

Read online

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.

Keywords