Ueda Yasutaka, Chou Wen-Chien, Goh Yeow-Tee, Rojnuckarin Ponlapat, Kim Jin Seok, Wong Raymond Siu Ming, Lee Wong Lily Lee, Jang Jun Ho, Chiou Tzeon-Jye, Kanakura Yuzuru, Lee Jong Wook
Department of Hematology and Oncology, Graduate School of Medicine, Faculty of Medicine, Osaka University, 1-1 Yamadaoka, Suita 565-0871, Japan.
Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, No. 7, Chung Shan S. Rd. (Zhongshan S. Rd.), Zhongzheng, Taipei City 100225, Taiwan.
Int J Mol Sci. 2025 Mar 11;26(6):2504. doi: 10.3390/ijms26062504.
Thromboembolism (TE) is a major cause of morbidity and mortality in patients with paroxysmal nocturnal hemoglobinuria (PNH). This narrative review summarizes available evidence on TE in Asian patients with PNH and discusses practical considerations and challenges for preventing and managing PNH-associated TE in Asian populations. Evidence suggests that, compared with non-Asians, fewer Asian patients have a history of TE (3.6% vs. 8.9%, < 0.01), receive anticoagulants (8.5% vs. 16.2%, = 0.002), or die from TE (6.9% vs. 43.7%, = 0.000). Independent predictors of TE include lactate dehydrogenase ≥ 1.5 × upper limit of normal, pain, and male sex. Clone size alone does not appear to be a reliable estimate of TE risk. D-dimer levels are a useful marker of hemostatic activation, although they are not specific to PNH. Complement inhibition reduces the incidence of TE, although it does not wholly eliminate TE risk. Eligibility criteria and access to complement inhibitors vary across Asia, with limited availability in some countries. Anticoagulation is required to treat acute TE events and for primary or secondary prophylaxis in selected patients. Physicians and patients must stay alert to the signs and symptoms of TE to ensure prompt and appropriate treatment.
血栓栓塞（TE）是阵发性夜间血红蛋白尿（PNH）患者发病和死亡的主要原因。这篇叙述性综述总结了亚洲PNH患者TE的现有证据，并讨论了亚洲人群预防和管理PNH相关TE的实际考虑因素和挑战。有证据表明，与非亚洲人相比，有TE病史的亚洲患者较少（3.6%对8.9%，P<0.01），接受抗凝治疗的患者较少（8.5%对16.2%，P = 0.002），死于TE的患者较少（6.9%对43.7%，P = 0.000）。TE的独立预测因素包括乳酸脱氢酶≥正常上限的1.5倍、疼痛和男性。仅克隆大小似乎不是TE风险的可靠估计指标。D-二聚体水平是止血激活的有用标志物，尽管它们并非PNH所特有。补体抑制可降低TE的发生率，尽管不能完全消除TE风险。亚洲各地补体抑制剂的资格标准和可及性各不相同，一些国家的供应有限。治疗急性TE事件以及对选定患者进行一级或二级预防需要抗凝治疗。医生和患者必须对TE的体征和症状保持警惕，以确保及时和适当的治疗。
