Tsitsikas Dimitris A, Kamal Mawlid, Braimoh Adeyinka, Benson Susan, Abukar Jibril
Haemoglobinopathy Service, Department of Haematology, Homerton University Hospital, National Health Service Foundation Trust, London, UK.
Hemoglobin. 2021 Nov;45(6):355-357. doi: 10.1080/03630269.2020.1788074. Epub 2021 Feb 24.
Voxelotor is a novel agent in the management of sickle cell disease. It is an inhibitor of Hb S (: c.20A>T) polymerization that reversibly binds to hemoglobin (Hb), stabilizing it in the oxygenated state that has been shown to reduce hemolysis and to improve anemia. Four patients in our institution are receiving treatment with Voxelotor as part of clinical studies. All four showed a characteristic change in the appearance of Hb S by high performance liquid chromatography (HPLC) soon after commencing treatment. A second peak was identified eluting at the Hb D window. Cellulose acetate membrane and agar gel electrophoresis only identified a band at the Hb S position. The patients' Hb level or clinical conditions were not adversely affected. Our findings indicate that patients receiving Voxelotor invariably display this unique pattern by HPLC. As there will be an increasing number of patients treated with this agent, it is important to be aware of this characteristic HPLC appearance for diagnostic and treatment monitoring purposes.
伏氯托是镰状细胞病治疗中的一种新型药物。它是Hb S（：c.20A>T）聚合反应的抑制剂，可与血红蛋白（Hb）可逆性结合，将其稳定在氧合状态，这已被证明可减少溶血并改善贫血。我们机构的四名患者作为临床研究的一部分正在接受伏氯托治疗。开始治疗后不久，所有四名患者的Hb S在高效液相色谱（HPLC）中的外观均出现了特征性变化。在Hb D窗口洗脱处鉴定出第二个峰。醋酸纤维素膜和琼脂凝胶电泳仅在Hb S位置鉴定出一条带。患者的Hb水平或临床状况未受到不利影响。我们的研究结果表明，接受伏氯托治疗的患者通过HPLC总是呈现这种独特的模式。由于使用这种药物治疗的患者数量将会增加，为了诊断和治疗监测目的，了解这种特征性的HPLC表现很重要。
