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Summary Sickle cell disease (SCD) and thalassaemia major are the most important syndromes of the haemoglobinopathies. Complications of SCD can be life threatening and include stroke, hyposplenism and vaso-occlusive crises.Treatment of SCD is generally supportive or by transfusion, hydroxyurea or bone marrow transplant (BMT). b Thalassaemia major is treated with transfusion and chelation for the resulting iron overload which is the cause of most morbidity, with BMT the only curative procedure. c 2002 Elsevier Science Ltd

Summary Regular subcutaneous desferrioxamine therapy has prolonged the life and reduced the incidence of cardiac, endocrine and liver complications of iron overload in patients with thalassaemia major and other refractory transfusion-dependent anaemias. Its full impact will be apparent only when patients who began this treatment as children 10 years ago reach adult life. The major problems with s.c. DF therapy are its cost and the difficulty of patient compliance with a painful and cumbersome method of administration. Attempts to maintain normal iron stores in these patients has resulted in significant toxicity caused by the excessive use of DF. Although recent studies have shown that long-term subcutaneous calcium diethylene triamine penta-acetic acid (Ca-DTPA) can be used as an effective alternative to s.c. DF ( Wonke et al, 1988 ), this treatment presents additional potential problems of zinc depletion which can be prevented only by careful management. The prospects of an inexpensive, orally active iron chelator are now real ( Kontoghiorghes and Hoffbrand, 1988 ) and it is to be hoped that long-term results at least as good as those with s.c. DF will be achievable. Such oral chelators may be available to a far wider group of patients, than s.c. DF, and the problems of cost and compliance may be diminished.