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.