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首页> 外文期刊>Pediatrics: Official Publication of the American Academy of Pediatrics >A Novel Strategy for Hemolytic Uremic Syndrome: Successful Treatment With Thrombomodulin alpha
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A Novel Strategy for Hemolytic Uremic Syndrome: Successful Treatment With Thrombomodulin alpha

机译：溶血性尿毒症综合征的新策略：血栓调节蛋白α的成功治疗

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摘要

Hemolytic uremic syndrome (HUS) is a life-threatening infectious disease in childhood for which there is no confirmed therapeutic strategy. Endothelial inflammation leading to microthrombosis formation via complement activation is the main pathology of HUS. Thrombomodulin is an endothelial membrane protein that has anticoagulation and anti-inflammatory effects, including the suppression of complement activity. Recombinant human soluble thrombomodulin (rTM) is a novel therapeutic medicine for disseminated intravascular coagulation. We administered rTM to 3 patients with HUS for 7 days and investigated the outcomes in view of the patients' prognoses, changes in biochemical markers, complications, and adverse effects of rTM. Symptoms and laboratory data improved after initiation of rTM in all 3 patients. Abnormal activation of complements was also dramatically suppressed in 1 patient. The patients recovered without any complications or adverse effects of rTM. They were discharged having normal neurologic status and with no renal dysfunction. To our knowledge, this is the first report of rTM being used to treat HUS. These case reports show the positive effect of rTM in patients with HUS. Randomized controlled studies should be performed to assess the efficacy and safety of rTM for children with HUS. Pediatrics 2013;131:e928-e933
机译：溶血性尿毒症综合征（HUS）是威胁儿童生存的传染病，目前尚无确定的治疗策略。内皮炎症通过补体激活导致微血栓形成，是HUS的主要病理。血栓调节蛋白是一种内皮细胞膜蛋白，具有抗凝和抗炎作用，包括抑制补体活性。重组人可溶性血栓调节蛋白（rTM）是一种用于弥散性血管内凝血的新型治疗药物。我们对3例HUS患者进行了7天的rTM给药，并根据患者的预后，生化指标的变化，并发症和rTM的不良反应对结局进行了研究。所有3例患者均在开始rTM后症状和实验室数据得到改善。 1名患者的补体激活异常也得到了显着抑制。患者康复后没有rTM的任何并发症或不良反应。他们出院后神经系统状态正常，没有肾功能不全。据我们所知，这是rTM用于治疗HUS的第一份报告。这些病例报告显示了rTM对HUS患者的积极作用。应进行随机对照研究以评估rTM对HUS儿童的疗效和安全性。儿科2013; 131：e928-e933

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