title: Successful treatment of resistant thrombotic thrombocytopenic purpura/hemolytic uremic syndrome with autologous peripheral blood stem and progenitor (CD34+) cell transplantation.
abstract: 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.
authors.affiliation: Unità Trapianti, Cattedra di Ematologia Università di Palermo, Italy.
authors.forename: M
authors.initials: M
authors.lastname: Musso
authors.forename: F
authors.initials: F
authors.lastname: Porretto
authors.forename: A
authors.initials: A
authors.lastname: Crescimanno
authors.forename: F
authors.initials: F
authors.lastname: Bondì
authors.forename: V
authors.initials: V
authors.lastname: Polizzi
authors.forename: R
authors.initials: R
authors.lastname: Scalone
authors.forename: E
authors.initials: E
authors.lastname: Iannitto
authors.forename: G
authors.initials: G
authors.lastname: Mariani
mesh_terms: D000328:Adult; D018952:Antigens, CD34; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells; D006463:Hemolytic-Uremic Syndrome / Q000628:therapy*; D006801:Humans; D008297:Male; D011697:Purpura, Thrombotic Thrombocytopenic / Q000150:complications / Q000628:therapy*; D014182:Transplantation, Autologous
chemical_list: D018952:Antigens, CD34