Aplastic anemia associated with bone marrow suppressor T-cell hyperactivity: successful treatment with antithymocyte...

In five patients with idiopathic aplastic anemia, the colony-forming unit (CFU-c) assay was used to quantitate stem cells and test the possible presence of suppressor cells inhibiting bone marrow differentiation. All five marrows failed to form CFU-c. In one out of the five cases tested, coculture of the patient's marrow with normal marrow suppressed the latter to form CFU-c. Removal of T-cells from the patient's bone marrow freed the aplastic marrow to produce more colonies and, in the coculture study, abrogated its inhibitory activity on the normal bone marrow. Treatment of the patient with horse antihuman thymocyte globulin (ATG), improved the peripheral blood count and myelopoiesis. Furthermore, the aplastic marrow increased its colony forming capacity from less than 1% pre-ATG to 46% post-ATG of the normal control, and its inhibitory activity on the normal marrow was ablated. In selected cases of aplastic anemia, with evidence of T-suppressor cell hyperactivity, treatment with ATG may offer an alternative approach to bone-marrow transplantation.