Department of Pathology and Immunology, Baylor College of Medicine, Houston, TX, USA.
Department of Pathology, Texas Children's Hospital, Houston, TX, USA.
Ann Clin Lab Sci. 2023 Jul;53(4):647-652.
Voxelotor, a FDA-approved drug for the treatment of patients with sickle cell disease (SCD), inhibits hemoglobin S (HbS) polymerization and increases total hemoglobin via hemolysis reduction. This drug has shown unique patterns in hemoglobin fractionation, affecting its interpretation. We aimed to evaluate whether these voxelotor-induced changes can be linked to improvement of hemolysis markers in pediatric patients on voxelotor.
A total of 15 patients (age 12 to 20 years; 40% females) on voxelotor were evaluated to compare changes in the hemoglobin fractionation by capillary electrophoresis, total hemoglobin, reticulocyte percentage (retic%), lactate dehydrogenase (LDH), and bilirubin measurements before and after the recorded date of voxelotor prescription.
Hemoglobin fractionation showed changes in the profile of 60% (9/15) of the patients studied. Out of the 9 patients for which voxelotor showed changes in the hemoglobin fractionation, 44% (4/9) had an increase of >1 g/dL in their total hemoglobin after voxelotor treatment was started. Assessment of other hemolysis markers available showed decreased LDH (4/4), retic % (6/8), and bilirubin (3/4).
Unique pattern of hemoglobin fractionation analysis following therapy with voxelotor has potential as a tool for the assessment of response and/or compliance to voxelotor for the treatment of SCD.
羟丁酸钠是一种经美国食品药品监督管理局（FDA）批准用于治疗镰状细胞病（SCD）患者的药物，通过减少溶血来抑制血红蛋白 S（HbS）聚合并增加总血红蛋白。这种药物在血红蛋白分馏中表现出独特的模式，影响其解读。我们旨在评估这些羟丁酸钠诱导的变化是否与接受羟丁酸钠治疗的儿科患者溶血标志物的改善相关。
共评估了 15 名接受羟丁酸钠治疗的患者（年龄 12 至 20 岁；40%为女性），以比较毛细管电泳法检测的血红蛋白分馏、总血红蛋白、网织红细胞百分比（网织红细胞）、乳酸脱氢酶（LDH）和胆红素测量值在记录羟丁酸钠处方日期前后的变化。
血红蛋白分馏显示 60%（9/15）的研究患者的图谱发生变化。在 9 名血红蛋白分馏发生变化的患者中，44%（4/9）在开始羟丁酸钠治疗后总血红蛋白增加>1g/dL。对其他可用的溶血标志物的评估显示 LDH（4/4）、网织红细胞（6/8）和胆红素（3/4）降低。
羟丁酸钠治疗后血红蛋白分馏分析的独特模式可能成为评估 SCD 治疗中羟丁酸钠反应和/或依从性的工具。
