Combined subcutaneous and high-dose intravenous deferoxamine therapy of thalassemia.

Although the preliminary findings are encouraging, to assess the effectiveness of the SHYD program on the long-term course of thalassemia, a larger patient population and longer treatment and follow-up period are needed. The presence of cardiac abnormalities in patients in Group I suggests that to prevent heart damage in thalassemia, it may be necessary to start the HTP and effective chelation therapy at a very young age, realizing that the amount of iron removed will be small. The finding that two of the four patients who survived to receive the largest amount of Fe/kg have intact spleens and another had splenectomy at age 24 suggests that, in these patients, the spleen may have had a protective role for the heart. If this observation is confirmed, it would provide evidence that removal of the spleen may adversely affect the long-term course of patients with thalassemia. Six years' experience with the SHYD program has not provided evidence for significant acute or chronic toxicity. This suggests that it might be safe to administer the i.v. treatments at home, a program that is anticipated for the future, as it would significantly reduce cost and possibly be more convenient.