Oxymetholone Therapy in Aplastic Anemia

Four consecutive children with acquired aplastic anemia, including one refractory to prolonged testosterone treatment, have responded to the synthetic anabolic drug oxymetholone. Three of these four are now off all medication and have remained well for eighteen to twenty-four months. A fifth child, with constitutional aplastic anemia refractory to testosterone therapy, has also improved while on oxymetholone. Mild virilization was the only side effect noted. Significant improvement in peripheral blood was noted in all five children during the second and third month of treatment. Oxymetholone appears to be a potent bone marrow stimulant.