Significance of the 50％ hemolytic complement in hemolysis assessment and efficacy of eculizumab in patients with paroxysmal nocturnal hemoglobinuria

TL;DR

This study shows that CH50 levels can indicate the effectiveness of eculizumab in treating PNH by tracking complement inhibition and hemolysis improvement.

Contribution

The study demonstrates CH50 as a potential biomarker for eculizumab efficacy in PNH patients.

Findings

CH50 levels dropped significantly after eculizumab treatment, indicating effective complement inhibition.

LDH levels decreased and HGB levels increased, showing reduced hemolysis and improved anemia.

Baseline CH50 correlated with LDH, but this correlation disappeared after treatment.

Abstract

回顾性收集2023年1月至2024年6月北京协和医院、北京中医药大学东方医院收治的阵发性睡眠性血红蛋白尿症（PNH）患者数据，选择经过足量依库珠单抗治疗至少3个月，并完成了依库珠单抗治疗前、治疗后3个月的总补体活性（CH50）水平检测的25例患者，其中24例患者完成了6个月治疗及CH50检测。依库珠单抗治疗3及6个月后，所有PNH患者症状显著改善，乳酸脱氢酶（LDH）从基线（1 814.4±924.8）U/L下降至（248.5±61.0）U/L和（239.3±44.8）U/L。HGB水平从基线（73.9±14.4）g/L上升至（99.9±21.3）g/L和（99.6±19.8）g/L。CH50基线水平为（32.4±14.7）％，治疗后3和6个月分别下降至2.0％（1.0％～8.0％）和1.0％（1.0％～4.0％）。基线时CH50水平与LDH呈线性相关（P<0.001，r＝0.789），且CH50水平在依库珠治疗后3和6个月与LDH均明显下降，变化趋势相似，但在用药3及6个月的CH50水平与LDH及其他参数不存在线性相关。提示血清CH50水平可能是依库珠单抗诱导的补体阻断的标志物，可一定程度上反映PNH血管内溶血及依库珠单抗疗效。

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