Lossos Chen, Brown Jenna, Sheikhbahaei Sara, Hubben Anne, Liu Sharon C, McCrae Keith R, Chaturvedi Shruti, Naik Rakhi P, Francischetti Ivo M B
Department of Pathology, Johns Hopkins University School of Medicine, Baltimore, MD.
Division of Adult Hematology, Johns Hopkins University School of Medicine, Baltimore, MD.
Blood Vessel Thromb Hemost. 2024 Jun;1(2). doi: 10.1016/j.bvth.2024.100006. Epub 2024 Apr 3.
Idiopathic multicentric Castleman disease (iMCD) is an inflammatory disease associated with a cytokine storm, activation of the PI3K/AKT/mTOR pathway, coagulopathy, and increased risk of thrombosis. The mechanisms underlying these pathologic processes remain elusive. We studied novel markers of mTOR activation and thrombosis in 1 patient with typical features of iMCD with TAFRO (thrombocytopenia, anasarca, fevers, reticulin myelofibrosis, and organomegaly) syndrome (iMCD-TAFRO). Plasma levels of SVEP1 (Sushi, von Willebrand factor type A, epidermal growth factor, and pentraxin domain-containing 1 protein), a newly identified mTOR activator associated with cardiovascular diseases and dementia, in addition to cytokines, chemokines and components of the coagulation cascade and complement system were evaluated by enzyme-linked immunosorbent assay (ELISA) and arrays. Compared with healthy controls, a 15-fold increase in SVEP1 was observed. High levels of factor VIIa/antithrombin and microparticles expressing functional tissue factor (TF) were detected. The anticoagulants thrombomodulin and soluble endothelial protein C receptor were elevated, indicating shedding from endothelial cells. Plasminogen activator inhibitor 1 was increased, consistent with hypofibrinolysis, whereas high levels of C3b and C5a are in keeping with complement activation. Furthermore, markers of endothelial cell activation (e.g. von Willebrand factor, angiopoietin-2), cell adhesion molecules, and angiogenesis mediators were upregulated. SVEP1 emerges as a potential mechanism of mTOR activation in iMCD-TAFRO, while multiple pathways influence coagulopathy. Immunothrombosis emerges as a potential therapeutic target for iMCD.
特发性多中心Castleman病(iMCD)是一种与细胞因子风暴、PI3K/AKT/mTOR通路激活、凝血病及血栓形成风险增加相关的炎症性疾病。这些病理过程的潜在机制仍不清楚。我们研究了1例具有TAFRO(血小板减少、全身水肿、发热、网状纤维骨髓纤维化和器官肿大)综合征(iMCD-TAFRO)典型特征的iMCD患者中mTOR激活和血栓形成的新标志物。通过酶联免疫吸附测定(ELISA)和蛋白芯片评估了血浆中SVEP1(含寿司、血管性血友病因子A、表皮生长因子和五聚素结构域的1蛋白)水平,SVEP1是一种新发现的与心血管疾病和痴呆相关的mTOR激活剂,此外还评估了细胞因子、趋化因子、凝血级联反应成分和补体系统。与健康对照相比,SVEP1水平升高了15倍。检测到高水平的因子VIIa/抗凝血酶和表达功能性组织因子(TF)的微粒。抗凝剂血栓调节蛋白和可溶性内皮蛋白C受体升高,表明有内皮细胞脱落。纤溶酶原激活物抑制剂1增加,与纤维蛋白溶解功能减退一致,而高水平的C3b和C5a与补体激活相符。此外,内皮细胞激活标志物(如血管性血友病因子、血管生成素-2)、细胞黏附分子和血管生成介质均上调。SVEP1是iMCD-TAFRO中mTOR激活的潜在机制,而多种途径影响凝血病。免疫血栓形成成为iMCD的一个潜在治疗靶点。