Sadler J Evan
Department of Medicine, Washington University School of Medicine, St Louis, MO 63110, USA.
Blood. 2008 Jul 1;112(1):11-8. doi: 10.1182/blood-2008-02-078170.
Discoveries during the past decade have revolutionized our understanding of idiopathic thrombotic thrombocytopenic purpura (TTP). Most cases in adults are caused by acquired autoantibodies that inhibit ADAMTS13, a metalloprotease that cleaves von Willebrand factor within nascent platelet-rich thrombi to prevent hemolysis, thrombocytopenia, and tissue infarction. Although approximately 80% of patients respond to plasma exchange, which removes autoantibody and replenishes ADAMTS13, one third to one half of survivors develop refractory or relapsing disease. Intensive immunosuppressive therapy with rituximab appears to be effective as salvage therapy, and ongoing clinical trials should determine whether adjuvant rituximab with plasma exchange also is beneficial at first diagnosis. A major unanswered question is whether plasma exchange is effective for the subset of patients with idiopathic TTP who do not have severe ADAMTS13 deficiency.
过去十年间的多项发现彻底改变了我们对特发性血栓性血小板减少性紫癜（TTP）的认识。大多数成人TTP病例是由获得性自身抗体引起的，这些抗体抑制ADAMTS13（一种金属蛋白酶），该酶可在新生的富含血小板的血栓内裂解血管性血友病因子，以防止溶血、血小板减少和组织梗死。尽管约80%的患者对血浆置换有反应，血浆置换可清除自身抗体并补充ADAMTS13，但三分之一至一半的幸存者会出现难治性或复发性疾病。利妥昔单抗强化免疫抑制治疗作为挽救疗法似乎有效，正在进行的临床试验应能确定在首次诊断时，血浆置换联合利妥昔单抗是否也有益处。一个主要的未解决问题是，血浆置换对那些没有严重ADAMTS13缺乏的特发性TTP患者亚组是否有效。
