Optimizing Chelation Therapy in Thalasemia patients; Bridging Clinical Data and Patient Management
Date
2008-11-16
Authors
Prof. Hisham Darwish
Journal Title
Journal ISSN
Volume Title
Publisher
Abstract
<p>Patients with thalassemia major who receive regular blood transfusions are likely to develop iron overload. This will result following saturation of the iron carrying capacity of transferring, which generally takes place after 20 transfusions. Consequently, excess plasma iron (labile iron) is cleared rapidly by the liver, heart and endocrine tissues at a rate that exceeds 200 times normal uptake of transferring-bound iron. Excess labile iron within cells will destroy the structure and function of mitochondria, lysosomes, lipid membranes, proteins and DNA. The clinical consequences can include liver cirrhosis and fibroses, cardiomyopathy, diabetes and other endocrine disorders. With proper control of body iron at all times in patients, these effects are preventable but only some are reversible once tissue damage has occurred. The primary rate of iron chelating therapy is to bind and remove iron from the patient body at a rate either equal to or greater than the rate of iron uptake of transfused iron. Complete chelating should aim to achieve both iron balance and iron detoxification. In patients who accumulated dangerous tissue iron levels, removal of this iron is also highly desirable. Several iron cheaters have been developed to help achieve these objectives. The recent introduction of the effective oral cheater deferasirox provideschelation coverage and significant control of LPI levels over the entire 24 hour period. Evidently, administration of the drug at a dose of 20-30 mg/kg/day significantly controls iron levels in plasma, liver and myocrdiocytes in thalassemi a major patients. Accumulating clinical data from key studies indicate that thalassemia patients can lead a happy and enjoyable normal life similar to normal individuals if their treatment is performed right and they receive the proper clinical attention from the medical staff. Definitely, chelating therapy requires close monitoring of patients vital organ status and the treatment protocol should be adjusted accordingly. Long term treatment with iron chelation highlights the importance of titrating the dose of the drug for each patient according to individual rates of iron intake from continued blood transfusion, current iron storage levels, safety markers (renal function for example) and target body iron content desired.26 First International Faculty of Medicine Conference 2008The presentation will highlight some of the recent clinical data from leading clinicians In chelationtherapy from several parts of the world and the lessons that can be learned from their experience and work for the benefit to optimize the treatment of thalassemia major patients in our population.</p>
<p>Patients with thalassemia major who receive regular blood transfusions are likely to develop iron overload. This will result following saturation of the iron carrying capacity of transferring, which generally takes place after 20 transfusions. Consequently, excess plasma iron (labile iron) is cleared rapidly by the liver, heart and endocrine tissues at a rate that exceeds 200 times normal uptake of transferring-bound iron. Excess labile iron within cells will destroy the structure and function of mitochondria, lysosomes, lipid membranes, proteins and DNA. The clinical consequences can include liver cirrhosis and fibroses, cardiomyopathy, diabetes and other endocrine disorders. With proper control of body iron at all times in patients, these effects are preventable but only some are reversible once tissue damage has occurred. The primary rate of iron chelating therapy is to bind and remove iron from the patient body at a rate either equal to or greater than the rate of iron uptake of transfused iron. Complete chelating should aim to achieve both iron balance and iron detoxification. In patients who accumulated dangerous tissue iron levels, removal of this iron is also highly desirable. Several iron cheaters have been developed to help achieve these objectives. The recent introduction of the effective oral cheater deferasirox provideschelation coverage and significant control of LPI levels over the entire 24 hour period. Evidently, administration of the drug at a dose of 20-30 mg/kg/day significantly controls iron levels in plasma, liver and myocrdiocytes in thalassemi a major patients. Accumulating clinical data from key studies indicate that thalassemia patients can lead a happy and enjoyable normal life similar to normal individuals if their treatment is performed right and they receive the proper clinical attention from the medical staff. Definitely, chelating therapy requires close monitoring of patients vital organ status and the treatment protocol should be adjusted accordingly. Long term treatment with iron chelation highlights the importance of titrating the dose of the drug for each patient according to individual rates of iron intake from continued blood transfusion, current iron storage levels, safety markers (renal function for example) and target body iron content desired.26 First International Faculty of Medicine Conference 2008The presentation will highlight some of the recent clinical data from leading clinicians In chelationtherapy from several parts of the world and the lessons that can be learned from their experience and work for the benefit to optimize the treatment of thalassemia major patients in our population.</p>
<p>Patients with thalassemia major who receive regular blood transfusions are likely to develop iron overload. This will result following saturation of the iron carrying capacity of transferring, which generally takes place after 20 transfusions. Consequently, excess plasma iron (labile iron) is cleared rapidly by the liver, heart and endocrine tissues at a rate that exceeds 200 times normal uptake of transferring-bound iron. Excess labile iron within cells will destroy the structure and function of mitochondria, lysosomes, lipid membranes, proteins and DNA. The clinical consequences can include liver cirrhosis and fibroses, cardiomyopathy, diabetes and other endocrine disorders. With proper control of body iron at all times in patients, these effects are preventable but only some are reversible once tissue damage has occurred. The primary rate of iron chelating therapy is to bind and remove iron from the patient body at a rate either equal to or greater than the rate of iron uptake of transfused iron. Complete chelating should aim to achieve both iron balance and iron detoxification. In patients who accumulated dangerous tissue iron levels, removal of this iron is also highly desirable. Several iron cheaters have been developed to help achieve these objectives. The recent introduction of the effective oral cheater deferasirox provideschelation coverage and significant control of LPI levels over the entire 24 hour period. Evidently, administration of the drug at a dose of 20-30 mg/kg/day significantly controls iron levels in plasma, liver and myocrdiocytes in thalassemi a major patients. Accumulating clinical data from key studies indicate that thalassemia patients can lead a happy and enjoyable normal life similar to normal individuals if their treatment is performed right and they receive the proper clinical attention from the medical staff. Definitely, chelating therapy requires close monitoring of patients vital organ status and the treatment protocol should be adjusted accordingly. Long term treatment with iron chelation highlights the importance of titrating the dose of the drug for each patient according to individual rates of iron intake from continued blood transfusion, current iron storage levels, safety markers (renal function for example) and target body iron content desired.26 First International Faculty of Medicine Conference 2008The presentation will highlight some of the recent clinical data from leading clinicians In chelationtherapy from several parts of the world and the lessons that can be learned from their experience and work for the benefit to optimize the treatment of thalassemia major patients in our population.</p>