Hematopoetic stem cell transplantation for immune thrombocytopenia and other refractory autoimmune cytopenias

Here we summarize data on hematopoietic stem cell transplantation (HSCT) to treat severe, refractory, hematologic autoimmune cytopenia. In the last 10 years, limited experience has been gained with the use of HSCT for hematologic disease. A phase II study by the NIH has presented data on autologous HSCT in 14 patients with immune thrombocytopenia (ITP) or Evans' syndrome with no early deaths and a response rate of 57%. The registry of the European Group of Blood and Marrow Transplantation holds data on 65 transplants, 37 autologous, 28 allogeneic in 59 patients. Disease entities and regimens used were heterogeneous. Patients had autoimmune hemolytic anemia, Evans' syndrome; ITP, pure red cell aplasia, pure white cell aplasia, or thrombotic thrombocytopenic purpura. Patients had longstanding disease having failed multiple prior treatments. Among 36 evaluable patients with a first autologous HSCT, 3 died of treatment-related causes, 4 died of disease progression, 12 were nonresponders, 7 patients had transient responses and 9 had sustained partial or complete remission. Of the 23 patients with a first allogeneic HSCT 5 died of treatment-related complications, 4 died of progressive disease and all but 2 of the remaining patients had a sustained response. Autologous and allogeneic HSCT may induce response in a considerable proportion of patients with autoimmune cytopenia of long duration.