Haematopoietic stem cell transplantation for immune thrombopenia and other refractory autoimmune cytopenias

This review summarizes data on haematopoietic stem cell transplantation (HSCT) to treat severe, refractory, haematological autoimmune cytopenia. A phase II study by the National Institutes of Health of the USA has presented data on autologous HSCT in 14 patients with immune thrombocytopenia or Evans' syndrome, with no early deaths and a response rate of 57%. The registry of the European Group for Blood and Marrow Transplantation holds data on 38 transplants, autologous for 27 and allogeneic for 9 patients. The disease entities and regimens used were more heterogeneous. The conditions encountered were autoimmune haemolytic anaemia, Evans' syndrome, immune thrombocytopenia, pure red cell aplasia, pure white cell aplasia and thrombotic thrombocytopenic purpura. Patients had long-standing disease, having failed multiple prior treatments. Among 26 evaluable patients mobilized for autologous HSCT, 3 died of treatment-related causes, 1 died of disease progression, 7 were non-responders, 6 patients had transient responses, and 9 had sustained partial or complete remission. Of the 7 evaluable patients receiving allogeneic HSCT, 1 died of treatment-related complications, 1 with a transient response died of progressive disease, and 5 showed a sustained response. Autologous and allogeneic HSCT may induce a response in a considerable proportion of patients with autoimmune cytopenia of long duration.