CD34+ cells from immune aplastic anemia patients expanded in vitro with UM171 and SR1

Aplastic anemia (AA) is a human disease characterized by empty bone marrow associated with low peripheral blood cell counts. In the immune AA, the disease is caused by an attack mediated by the oligoclonal expansion of cytotoxic T cells against the hematopoietic stem cells (HSCs). SR1 and UM171, two HSC self-renewal stimulators, have been tested for the ex vivo expansion of human CD34+ cells. Here, we evaluated the effects of these molecules on the in vitro expansion of CD34+ cells of immune AA patients. The two molecules are promising candidate drugs for the treatment of patients with immune AA.