ADAMTS13 activity in thrombotic thrombocytopenic purpura-hemolytic uremic syndrome:: relation to presenting features and clinical outcomes in a prospective cohort of 142 patients
ADAMTS13 activity in thrombotic thrombocytopenic purpura-hemolytic uremic syndrome:: relation to presenting features and clinical outcomes in a prospective cohort of 142 patients
复制标题
DOI：
10.1182/blood-2003-01-0193
复制
发表时间：
2003-07-01
期刊：
影响因子：
20.3
通讯作者：
Raskob, GE
中科院分区：
文献类型：
--
作者：
Vesely, SK;George, JN;Raskob, GE
血栓性血小板减少性紫癜-溶血性尿毒症综合征（TTP-HUS）患者的初始管理是困难的，因为缺乏具体的诊断标准，不进行血浆置换治疗的死亡率高，以及血浆置换的风险。虽然严重的ADAMTS13（一种具有血小板反应蛋白1型重复的崩解素样和金属蛋白酶）缺乏症可能是TTP特有的，但ADAMTS13活性测量在初始管理决策中的作用尚不清楚。在161例临床诊断为TTP-HUS的连续患者中，142例（88%）在开始血浆交换治疗前测量ADAMTS13，并将其分为以下4类：低于5%（严重缺乏）、5%至9%、10%至25%和超过25%。142例患者中有18例（13%）存在严重的ADAMTS13缺乏症。在6个预先确定的临床类别（干细胞移植、妊娠/产后、药物关联、血性腹泻、附加/替代疾病、特发性）中，仅在妊娠/产后（10例中有2例）和特发性（48例中有16例）患者中发生严重缺乏症。16例患有严重ADAMTS13缺乏症的特发性TTP-HUS患者的表现特征和临床结果是可变的，与32例没有严重ADAMTS13缺乏症的特发性TTP-HUS患者没有区别。在所有ADAMTS13活性类别中，许多患者对血浆交换治疗明显有反应。因此，严重的ADAMTS13缺乏并不能检测出所有可能被正确诊断为TTP-HUS和可能对血浆交换治疗有反应的患者。(C) 2003年由美国血液病学会出版。
Initial management of patients with thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS) is difficult because of lack of specific diagnostic criteria, high mortality without plasma exchange treatment, and risks of plasma exchange. Although severe ADAMTS13 (a disintegrin-like and metalloprotease with thrombospondin type 1 repeats) deficiency may be specific for TTP, the role of ADAMTS13 activity measurements for initial management decisions is unknown. ADAMTS13 was measured before beginning plasma exchange treatment in 142 (88%) of 161 consecutive patients with clinically diagnosed TTP-HUS with assignment to 1 of 4 categories: less than 5% (severe deficiency), 5% to 9%, 10% to 25%, and more than 25%. Eighteen (13%) of 142 patients had severe ADAMTS13 deficiency. Among 6 predefined clinical categories (stem cell transplantation, pregnant/postpartum, drug association, bloody diarrhea, additional/alternative disorder, idiopathic), severe deficiency occurred only among pregnant/postpartum (2 of 10) and idiopathic (16 of 48) patients. The presenting features and clinical outcomes of the 16 patients with idiopathic TTP-HUS who had severe ADAMTS13 deficiency were variable and not distinct from the 32 patients with idiopathic TTP-HUS who did not have severe ADAMTS13 deficiency. Many patients in all ADAMTS13 activity categories apparently responded to plasma exchange treatment. Therefore, severe ADAMTS13 deficiency does not detect all patients who may be appropriately diagnosed with TTP-HUS and who may respond to plasma exchange treatment. (C) 2003 by The American Society of Hematology.