Thrombocytopenia is a serious clinical problem in several different clinical settings. In chronic
bone marrow failure states, which include
aplastic anemia,
myelodysplastic syndrome, and graft failure, the prolonged nature of
thrombocytopenia often leads to alloimunization after repeated
platelet transfusions, the consequence of which is a platelet-refractory state and enhanced risk of
bleeding. Despite the introduction of several thrombopoietic factors into clinical trials, an effective way to alleviate
thrombocytopenia has been elusive, and the problem in chronic
bone marrow failure states has remained poorly addressed by clinical investigations. Even so, several studies by our group and others suggest that a subset of patients suffering from chronic
bone marrow failure can respond to appropriate
growth factor therapy. The temporal pace of response appears, however, to be much slower than that observed after administering
growth factors which act on neutrophils. On the other hand, durable responses can be secured in some patients given thrombopoietic factors for long periods of time. Herein, we provide an overview of the clinical research investigations of thrombopoietic factors in chronic
bone marrow failure, and the emerging insights these studies provide for understanding the process of thrombopoiesis and its
therapy in this setting.