RESULTS OF LONG-PERM IRON-CHELATING THERAPY

The improvement in survival and quality of life of iron-overloaded pat ients achieved by regular subcutaneous chelation has been extensively documented over the years. A review of the long-term results allows on e to establish the following points: (1) with regular subcutaneous che lation, a negative iron balance can be obtained in most patients, exce pt very young ones; (2) severe deferoxamine (DFO) toxicity may be prev ented by skipping high doses and by carefully monitoring and modulatin g chelation, especially in patients with a low iron overload; (3) the maintenance of compliance with DFO over 0.6 and of ferritin levels bel ow 2,000 prevents iron overload complications, at least for the first 20 years of life; (4) long-term chelation can reverse functional compl ications such as liver fibrosis, arrhythmia and echocardiographic abno rmalities, but not complications due to extensive tissue alterations, such as frank diabetes, hypothyroidism and myocardiosclerosis; (5) int ensive intravenous protocols can be successfully applied in heavily ov erloaded patients and represent the only possibility to reverse their dangerous iron burden in a relatively short period of time; (6) surviv al and quality of life in well-chelated patients are approaching a nor mal pattern, and (7) clinical outcome and prognosis are better evaluat ed by parameters that consider iron overload and chelation trends.