Conclusions
The prospect for transfusion-dependent patients to receive effective iron chelation therapy has substantially improved in the last few years. Subcutaneouse DFO 40 mg/kg over 8-12 hours on at least 5 days a week protects most compliant patienst against cardiac disease and other serious complications and remains the first choice. Its cost, frequent lack of compliance, and complication means that alternative approaches are needed. After many years of short-term clinical trials of the orally active agent deferiprone and much controversy about its efficacy and toxicity, recent published data have been favorable on both aspects. These suggest that the drug at a dose of 75 mg/kg/d may be at least as effective as DFO in protecting patients from iron-in-dced cardiomyopathy. Hepatic fibrosis does not appear to be a problem, and the established side effects do not lead to the need for discontinuation of the drug in the majority of patients.
Combination therapy with DFO and deferiprone is an exciting new possibility for those patients inadequately chelated on either drug alone. ICL 670, a new oral iron chelator in early clinical trial, promises to expand further the range of possibilities for effective andsafe iron chelation therapy for patients with TM and other iron-loaded transfusion-dependent or –independent patients with severe refractory anemias. Whichever chelation regimen is chosen, patients must beclosely monitored both for effectiveness of therapy, with particular attention to cardiac iron and function, and for toxic side effects of the chelating drug.
Conclusions
The prospect for transfusion-dependent patients to receive effective iron chelation therapy has substantially improved in the last few years. Subcutaneouse DFO 40 mg/kg over 8-12 hours on at least 5 days a week protects most compliant patienst against cardiac disease and other serious complications and remains the first choice. Its cost, frequent lack of compliance, and complication means that alternative approaches are needed. After many years of short-term clinical trials of the orally active agent deferiprone and much controversy about its efficacy and toxicity, recent published data have been favorable on both aspects. These suggest that the drug at a dose of 75 mg/kg/d may be at least as effective as DFO in protecting patients from iron-in-dced cardiomyopathy. Hepatic fibrosis does not appear to be a problem, and the established side effects do not lead to the need for discontinuation of the drug in the majority of patients.
Combination therapy with DFO and deferiprone is an exciting new possibility for those patients inadequately chelated on either drug alone. ICL 670, a new oral iron chelator in early clinical trial, promises to expand further the range of possibilities for effective andsafe iron chelation therapy for patients with TM and other iron-loaded transfusion-dependent or –independent patients with severe refractory anemias. Whichever chelation regimen is chosen, patients must beclosely monitored both for effectiveness of therapy, with particular attention to cardiac iron and function, and for toxic side effects of the chelating drug.
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