Musso M, Porretto F, Crescimanno A, Bondì F, Polizzi V, Scalone R, Iannitto E, Mariani G
Unità Trapianti, Cattedra di Ematologia Università di Palermo, Italy.
Bone Marrow Transplant. 1999 Jul;24(2):207-9. doi: 10.1038/sj.bmt.1701859.
The first-line treatment of thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS syndrome) induces a response and survival rate of approximately 85%, even if a considerable number of patients relapse; nevertheless, a number of these patients are resistant to conventional management. Immunoablation followed by stem cell transplantation has been shown to be capable of inducing remissions in a large spectrum of experimental autoimmune disorders. We report here the case of a 20-year-old male patient with the TTP-HUS syndrome who was resistant to conventional treatment and was transplanted with autologous immunoselected CD34+ PBPC after conditioning with cyclosphosphamide, anti-T lymphocyte globulin and prednisone. Seven months after transplant the patient is alive and well, without any further treatment being given.
血栓性血小板减少性紫癜-溶血尿毒综合征（TTP-HUS综合征）的一线治疗可诱导约85%的缓解率和生存率，即便有相当数量的患者会复发；然而，这些患者中有一些对传统治疗有抵抗性。免疫消融后进行干细胞移植已被证明能够在多种实验性自身免疫性疾病中诱导缓解。我们在此报告一例20岁男性TTP-HUS综合征患者，其对传统治疗有抵抗性，在接受环磷酰胺、抗T淋巴细胞球蛋白和泼尼松预处理后，接受了自体免疫选择的CD34+外周血祖细胞移植。移植七个月后，患者存活且状况良好，未再接受任何进一步治疗。
