Oral iron chelation with deferiprone.

Deferiprone is the most widely studied oral iron chelator and, at present, the only one shown to be effective in achieving negative iron balance in long-term clinical trials for chronic iron overload. Because of its adverse effects (e.g., agranulocytosis and arthropathy) its use is presently restricted to clinical trials and to countries where desferrioxamine is unavailable. Deferiprone was licensed for clinical use in India in 1995. Clinical trials are in progress in many centers worldwide that will provide further information on the long-term effectiveness of deferiprone as well as on the incidence of serious adverse effects in patients with iron overload. Trials of combined use of deferiprone and desferrioxamine are also in progress. In the meantime, deferiprone is an acceptable alternative for patients who cannot use desferrioxamine because of serious adverse effects, lack of compliance, or unavailability. Elucidation of the mechanisms involved in the agranulocytosis and arthropathy associated with deferiprone is still needed, as are methods to predict individual susceptibility to these adverse effects and ways of preventing them. In addition, new indications for iron-chelating therapy are continuously being explored.