6533b86cfe1ef96bd12c8b12

RESEARCH PRODUCT

Successful treatment of resistant thrombotic thrombocytopenic purpura/hemolytic uremic syndrome with autologous peripheral blood stem and progenitor (CD34+) cell transplantation

R ScaloneA CrescimannoFerdinando PorrettoEmilio IannittoG MarianiV PolizziF. BondiMaurizio Musso

subject

AdultMaleHemolytic anemiaLymphocyteThrombotic thrombocytopenic purpuraAntigens CD34Transplantation AutologousPrednisonehemic and lymphatic diseasesHumansMedicineProgenitor cellSurvival rateTransplantationPurpura Thrombotic Thrombocytopenicbusiness.industryHematopoietic Stem Cell TransplantationHematologyHematopoietic Stem Cellsmedicine.diseaseTransplantationmedicine.anatomical_structureHemolytic-Uremic SyndromeImmunologyStem cellbusinessmedicine.drug

description

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.

https://doi.org/10.1038/sj.bmt.1701859