University College Hospital, London, United Kingdom.
Blood Rev. 2010 Jan;24(1):11-6. doi: 10.1016/j.blre.2009.11.001. Epub 2009 Dec 5.
Thrombotic thrombocytopenic purpura is an acute life threatening disorder, characterised by thrombocytopenia, microangiopathic haemolytic anaemia and multi organ microvascular thrombi that results in variable clinical symptoms. Just over a decade ago, the missing enzyme required for von Willebrand cleavage was recognised in TTP patients, subsequently identified as ADAMTS 13. Assays have confirmed that the majority of TTP cases are idiopathic and are associated with inhibitors and or IgG antibodies to ADAMTS 13. Such cases take longer to treat and are more likely to relapse. Evidence to date suggests the majority of antibodies block the spacer domain of ADAMTS 13. There may be other antibodies binding to ADAMTS 13 domains but their overall clinical involvement remains to be determined. Immunosuppressive treatments have until now been unsatisfactory. However, monoclonal anti-CD 20 therapy, acting on B-lymphocytes involved in antibody production results in remission in most patients and prevention of recurrent relapse. Further investigation into the antibodies produced in TTP and other aspects of immune dysfunction, such as T cells will further our understanding of this devastating disorder.
血栓性血小板减少性紫癜是一种严重的急性疾病，其特征是血小板减少症、微血管病性溶血性贫血和多器官微血栓形成，导致不同的临床症状。就在十多年前，人们发现 TTP 患者缺乏 von Willebrand 裂解所需的酶，随后将其鉴定为 ADAMTS13。检测证实，大多数 TTP 病例是特发性的，与 ADAMTS13 的抑制剂和/或 IgG 抗体有关。这类病例的治疗时间更长，更容易复发。迄今为止的证据表明，大多数抗体阻断 ADAMTS13 的间隔区。可能还有其他抗体与 ADAMTS13 结构域结合，但它们的整体临床参与情况仍有待确定。免疫抑制治疗迄今为止一直不尽如人意。然而，作用于参与抗体产生的 B 淋巴细胞的单克隆抗 CD20 治疗使大多数患者得到缓解，并防止复发。进一步研究 TTP 中产生的抗体以及其他免疫功能障碍方面，如 T 细胞，将有助于我们了解这种严重的疾病。
