Hamamura Atsushi, Sakai Kazuya, Mushino Toshiki, Ueda Yasunori, Ogawa Yoshiyuki, Noguchi Hiroyuki, Okamoto Akinao, Yagi Hideo, Mori Takehiko, Matsumoto Masanori
Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan.
Department of Hematology, Institute of Science Tokyo, Bunkyo-ku, Japan.
Res Pract Thromb Haemost. 2025 Jul 18;9(5):102974. doi: 10.1016/j.rpth.2025.102974. eCollection 2025 Jul.
Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a life-threatening condition caused by a severe deficiency of a disintegrin and metalloproteinase with thrombospondin type 1 motif 13 due to autoantibodies. Despite modern treatments, including therapeutic plasma exchange, immunosuppression, and rituximab, early mortality-often due to cardiac and neurologic events-remains a concern.
We analyzed data from 125 patients between 2010 and 2023 in the Japanese thrombotic thrombocytopenic purpura (TTP) registry, examining demographics, electrocardiogram and transthoracic echocardiography findings, and neurologic symptoms. Troponin I was measured. Outcomes were categorized as survivors, TTP-related deaths, and non-TTP-related deaths.
Of the 125 patients, 15 died, with 5 deaths directly related to iTTP. Early cardiac findings and neurologic symptoms were not significant predictors of mortality. However, elevated lactate dehydrogenase levels and reduced von Willebrand factor multimer indices correlated with poorer prognosis. Patients with myocardial hypokinesis finally recovered their condition during the course of treatment. No patient treated with caplacizumab died during the observation period.
These findings suggest that early cardiac and neurologic symptoms may not be definitive predictors of iTTP-related death. Instead, extremely high lactate dehydrogenase levels indicated a worse prognosis, highlighting the need for targeted monitoring and interventions in high-risk cases.
免疫介导的血栓性血小板减少性紫癜（iTTP）是一种危及生命的疾病，由自身抗体导致的具有血小板反应蛋白基序的解聚素和金属蛋白酶13严重缺乏引起。尽管有现代治疗方法，包括治疗性血浆置换、免疫抑制和利妥昔单抗，但早期死亡率（通常由于心脏和神经系统事件）仍然令人担忧。
我们分析了日本血栓性血小板减少性紫癜（TTP）登记处2010年至2023年期间125例患者的数据，检查了人口统计学、心电图和经胸超声心动图检查结果以及神经系统症状。检测了肌钙蛋白I。结果分为幸存者、TTP相关死亡和非TTP相关死亡。
125例患者中，15例死亡，其中5例死亡与iTTP直接相关。早期心脏检查结果和神经系统症状不是死亡率的显著预测因素。然而，乳酸脱氢酶水平升高和血管性血友病因子多聚体指数降低与较差的预后相关。心肌运动减弱的患者在治疗过程中最终恢复了病情。在观察期内，接受卡泊单抗治疗的患者无死亡。
这些发现表明，早期心脏和神经系统症状可能不是iTTP相关死亡的决定性预测因素。相反，极高的乳酸脱氢酶水平表明预后较差，凸显了在高危病例中进行有针对性监测和干预的必要性。
