Successful treatment of resistant thrombotic thrombocytopenic purpura/hemolytic uremic syndrome with autologous peripheral blood stem and progenitor (CD34+) cell transplantation.
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.
Musso
M
M
Unità Trapianti, Cattedra di Ematologia Università di Palermo, Italy.
Porretto
F
F
Crescimanno
A
A
Bondì
F
F
Polizzi
V
V
Scalone
R
R
Iannitto
E
E
Mariani
G
G
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
D018952:Antigens, CD34