Chonat Satheesh, Fields Earl, Baratz Hannah, Watt Amanda, Pochron Mira, Dixon Sandy, Tonda Margaret, Brown Clark, Archer David
Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta and Emory University Department of Pediatrics Atlanta Georgia USA.
Present address: Pfizer Inc New York New York USA.
EJHaem. 2024 Jan 30;5(1):125-130. doi: 10.1002/jha2.831. eCollection 2024 Feb.
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.
These findings suggest that voxelotor treatment is associated with reduced RBC sickling and haemolysis in children with SCD.
镰状血红蛋白（HbS）聚合会扰乱红细胞（RBC）流变学，并推动镰状细胞病（SCD）的病理生理过程。伏洛妥珠单抗是一种HbS聚合抑制剂，可增加血红蛋白（Hb）与氧气的亲和力。
方法/结果：在这项为期48周的前瞻性单中心转化研究中，10名4至11岁的SCD儿童接受了伏洛妥珠单抗治疗。使用渗透/氧梯度血细胞变形性测定法观察到红细胞变形性有所改善，最小和最大伸长指数增加，镰变点降低。还观察到血红蛋白增加和溶血标志物减少。
这些发现表明，伏洛妥珠单抗治疗与SCD儿童红细胞镰变和溶血减少有关。
