Long-term trials of deferiprone in Cooley's anemia.

Deferoxamine is the currently available agent for the iron-chelation therapy required by Cooley's anemia patients. The difficulties associated with parenteral administration have mandated a search for alternative therapies, especially orally active iron chelators, to remove excess iron that results in damage to the liver, endocrine organs, and heart. Four orally active agents have reached clinical trials in the last decade. The agent under consideration in this paper, deferiprone (1,2-dimethyl-3-hydroxypyridin-4-one), has shown some promise, but, according to the studies discussed here, may not provide adequate sustained control of body iron in a substantial proportion of Cooley's anemia patients.