Treatment of severe aplastic anemia with antilymphocyte globulin and androgens.

The etiology of aplastic anemia is unknown. A stem cell lesion caused by a toxin, virus or microenvironment defect is the main hypothesis. An autoimmune origin has been recently suspected. In an attempt to demonstrate the autoimmune origin of the disease, 17 patients with severe aplastic anemia were treated with antilymphocyte globulin (ALG). Nine patients showed no improvement, developed infectious or hemorrhagic complications and died within 1 to 7 months. In contrast, eight patients had a prompt rise of granulocyte and reticulocyte counts. Although the hematological reconstitution is not complete, these eight patients are still alive between 11 months and 24 months after treatment. This study shows that ALG may have a beneficial effect in the treatment of patients with severe aplastic anemia.