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 u remic syndrome (TTP-HUS syndrome) induces a response and survival rate of a pproximately 85%, even if a considerable number of patients relapse; nevert heless, 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 autoimm une disorders. We report here the case of a 20-year-old male patient with t he TTP-HUS syndrome who was resistant to conventional treatment and was tra nsplanted with autologous immunoselected CD34(+) PBPC after conditioning wi th cyclosphosphamide, anti-T lymphocyte globulin and prednisone. Seven mont hs after transplant the patient is alive and well, without any further trea tment being given.