Nato Yuma, Nagaharu Keiki, Okano Motohiko, Suzuki Kei, Matsumoto Takeshi, Tawara Isao
Department of Hematology and Oncology, Mie University Hospital.
Department of Transfusion Medicine and Cell Therapy, Mie University Hospital.
Rinsho Ketsueki. 2021;62(3):176-179. doi: 10.11406/rinketsu.62.176.
Acquired thrombotic thrombocytopenic purpura (aTTP) is a life-threatening systemic thrombotic microangiopathy characterized by the presence of anti-ADAMTS13 antibodies (inhibitor). Here we report the case of a patient with refractory aTTP successfully treated with cyclosporine. A 69-year-old man presenting with hematuria and petechiae was referred to our hospital; he was disoriented and febrile. Laboratory results revealed Coombs-negative hemolytic anemia, thrombocytopenia, and renal failure. Undetectable ADAMTS13 activity and presence of anti-ADAMTS13 antibodies (inhibitor) confirmed the diagnosis of aTTP. Despite performing plasma exchange and administering prednisolone and rituximab (375 mg/m), we were unable to restore his platelet counts to the normal level. Therefore, he was treated with cyclophosphamide (500 mg/bodyweight), vincristine (1.4 mg/m), bortezomib (1.3 mg/m), and cyclosporine (2.5 mg/kg). After the cyclosporine therapy, his platelet counts gradually normalized. Continuous cyclosporine maintenance therapy led to complete disappearance of the inhibitor. Therapeutic strategies for refractory aTTP have not yet been established. Further investigations are warranted to establish a therapeutic strategy for refractory aTTP.
获得性血栓性血小板减少性紫癜（aTTP）是一种危及生命的系统性血栓性微血管病，其特征是存在抗ADAMTS13抗体（抑制剂）。在此，我们报告一例用环孢素成功治疗的难治性aTTP患者。一名69岁男性因血尿和瘀点就诊于我院；他神志不清且发热。实验室检查结果显示库姆斯试验阴性的溶血性贫血、血小板减少和肾衰竭。ADAMTS13活性检测不到以及抗ADAMTS13抗体（抑制剂）的存在确诊为aTTP。尽管进行了血浆置换并给予泼尼松龙和利妥昔单抗（375mg/m²），但我们仍无法将他的血小板计数恢复到正常水平。因此，他接受了环磷酰胺（500mg/体重）、长春新碱（1.4mg/m²）、硼替佐米（1.3mg/m²）和环孢素（2.5mg/kg）治疗。环孢素治疗后，他的血小板计数逐渐恢复正常。持续的环孢素维持治疗使抑制剂完全消失。难治性aTTP的治疗策略尚未确立。有必要进一步开展研究以确立难治性aTTP的治疗策略。
