The thrombotic thrombocytopenic purpura and hemolytic uremic syndromes: evaluation, management, and long-term outcomes experience of the Oklahoma TTP-HUS Registry, 1989-2007
The thrombotic thrombocytopenic purpura and hemolytic uremic syndromes: evaluation, management, and long-term outcomes experience of the Oklahoma TTP-HUS Registry, 1989-2007
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DOI：
10.1038/ki.2008.622
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发表时间：
2009-02-01
影响因子：
19.6
通讯作者：
George, James N.
中科院分区：
文献类型：
--
作者：
George, James N.
俄克拉荷马州血栓性血小板减少性紫癜-溶血性尿毒综合征（TTP-HUS）登记处，一个包含382例TTP-HUS患者的初始队列，提供了这些综合征的完整社区观点。TTP，定义为血小板减少症和微血管病性溶血性贫血，没有其他病因，是所有成年人的适当术语。这些有限的诊断标准得到了ADAMTS 13缺乏症患者的表现特征的支持，在这些患者中，神经系统和肾脏异常并不常见。HUS是符合这些诊断标准且同时患有肾衰竭的儿童的适当术语。这些定义与目前的治疗方法一致：血浆置换是大多数成人的基本治疗方法;支持性治疗足以治疗HUS儿童。血浆置换治疗使TTP的死亡率从90%降低到10%。获得性自身免疫性ADAMTS 13缺乏症患者也可能需要免疫抑制治疗以实现持久缓解。复苏揭示了以前未被认识到的长期风险。大约40%的获得性ADAMTS 13缺乏症患者发生复发性急性发作;大多数复发发生在第一年内，大多数患者只有一次复发。任何病因的成人TTP都有持续性轻微认知异常的高风险。
The Oklahoma thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS) Registry, an inception cohort of 382 consecutive patients with TTP-HUS, provides a complete community perspective of these syndromes. TTP, as defined by thrombocytopenia and microangiopathic hemolytic anemia without an alternative etiology, is the appropriate term for all adults. These limited diagnostic criteria are supported by the presenting features of patients with ADAMTS13 deficiency, in whom both neurologic and renal abnormalities are uncommon. HUS is the appropriate term for children who fulfill these diagnostic criteria and who also have renal failure. These definitions are consistent with current management: plasma exchange is the essential treatment for most adults; supportive care is sufficient for children with HUS. Plasma exchange treatment has decreased the mortality of TTP from 90 to 10%. Patients with acquired autoimmune ADAMTS13 deficiency may also require immunosuppressive treatment to achieve a durable remission. Recovery has revealed previously unrecognized long-term risks. Recurrent acute episodes occur in approximately 40% of patients with acquired ADAMTS13 deficiency; most relapses occur within the first year and most patients have only one relapse. Adults with TTP of any etiology have a high risk for persistent minor cognitive abnormalities.