LONG-TERM INTRAVENOUS DEFEROXAMINE TREATMENT FOR NONCOMPLIANT TRANSFUSION-DEPENDENT BETA-THALASSEMIA PATIENTS

With the introduction of long-term subcutaneous administration of defe roxamine (DFO), there has peen a decline in the morbidity and mortalit y of transfusion-dependent beta-thalassemia patients. However, since t he use of subcutaneous DFO is hindered by poor compliance, long-term i .v. DFO therapy has been attempted in order to improve compliance, pre vent excessive iron accumulation and extend survival. Thirteen patient s (aged 5.4-18.4 years) were started on i.v. home administration of DF O (100 mg/kg per day) via an exteriorized, tunneled right atrial cathe ter (Hickman type). After a median follow-up of 36 months, the mean fe rritin levels had dropped significantly (5,117+/-1,737 to 1,816+/-1,06 2 mu g/l. P = 0.0001). None of the patients developed new endocrine or cardiac diseases due to iron overload. Patients beginning therapy at an early age (less than or equal to 11 years) showed a tendency for im proved growth parameters at the end of the treatment period. Two patie nts developed moderately high frequency sensorineural hearing loss. On e patient developed a right atrial thrombus. The line infection rate w as low (1.7 episodes per 1,000 patient days). In view of the grave pro gnosis for iron overloaded patients and the fact that oral chelators a re not yet readily available, we recommend this form of therapy for th e young, noncompliant beta-thalassemia patient, despite the occasional complications observed.