Anabolic steroids in aplastic anemia

Despite the successful recovery of young patients with aplastic anemia following bone marrow transplants, additional patients have had benefit from immune suppression therapy, predominantly antithymus globulin (ATG). There exists a large residual pool of patients who have not been able to benefit from these modalities because of 1) lack of compatible siblings, 2) age, 3) duration of illness prior to diagnosis. Historically oral androstanes have been helpful in the treatment of chronic aplastic anemia. The long-range survival in large cooperative groups of patients treated with androstanes have indicated that both severe and chronic aplastic anemia have had responses similar to immune suppressive therapy. There is a need to have further investigations to seek the most effective anabolic agents. Many group studies have shortened treatment schedules when an erythropoietic response has been obtained, rather than continue therapy until the maximum platelet count is achieved. Such abbreviation of therapy may hasten a hematological relapse. Clinics also should evaluate parenteral androstanes since they appear to be more hematopoietic. Also the investigation of different androstane metabolites, i.e. etiocholanolone, should be pursued to determined if a more effective stimulus of stem cell proliferation can be achieved. In the recovery phase of the aplastic anemia patients treated with immune suppression or androstanes the peripheral blood reflects an altered proliferation of the marrow stem cell. The majority of these patients continue to have abnormalities in red cells (macrocytosis) and decreased platelet size.