Affiliation:
1. Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta and Emory University Department of Pediatrics Atlanta Georgia USA
2. Pfizer Inc New York New York USA
3. Pfizer Inc South San Francisco California USA
Abstract
AbstractIntroduction: 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.
Subject
General Earth and Planetary Sciences