CHELATION THERAPY IN OLDER PATIENTS WITH THALASSEMIA MAJOR

Abstract

To determine the utility of chronic subcutaneous Desferal (SCDF) therapy (Rx) in the prevention of cardiac disease in patients with thalassemia major who began treatment after the age of 10, we evaluated 36 patients; all of whom were started on SCDF when they were 10 years of age or older and had no pre-existing cardiac disease (defined as clinical congestive heart failure or abnormal left ventricular function). There were 17 compliant patients (mean age 12.3 years at onset of Rx) who used SCDF at least 5 days a week; 8-12 hours each day. Nineteen patients were noncompliant (mean age 16.6 years), they used SCDF periodically. In the group of compliant patients, the mean number of years on Desferal Rx was 4.8 years, in the noncompliant group, 4.0 years. All patients have been on a hypertransfusion regimen (maintaining a hemoglobin of 11%), at least since they began SCDF. Only one patient in the compliant group developed cardiac disease (at age 11) and she died of congestive heart failure at age 18. In contrast, 12 in the noncompliant group developed cardiac disease. Two of these patients have died. The mean age of development of cardiac disease in the noncompliant patients was 20.3 years ± 4.2 years. Therefore, the lack of cardiac complications in the compliant group suggests that compliance with SCDF Rx may reduce the risk of cardiac disease in older patients.