Improvement of Cardiac Function in Thalassemia Patients Using Deferiprone

Abstract 

Deferoxamine (DFO) therapy is associated with improved survival of thalassemia patients, and yet cardiac disease remains the main cause of death. Deferiprone (L1) is currently one of the orally active chelating agents used as an alternative to DFO. Both DFO and L1 have demonstrated their ability to normalize cardiac function in patients with iron-induced cardiac disease. Some evidence indicates that L1 is more effective than DFO in cardiac iron removal. Our ability to detect and manage the cardiac complications of thalassemia has also improved dramatically over the last 7 years. Noninvasive techniques of quantification of the iron burden using magnetic resonance imaging (MRI) have been validated. Using MRI and echocardiography to monitor cardiac systems, in particular the cardiac functions that are closely associated with iron overload-related complications and mortality, proved to be practical. Our increased understanding of cardiac pathophysiology and our improved ability to detect at-risk populations are yielding improved outcomes and reduced morbidity in these reported patients. The improvement in cardiac function that can be observed during L1 therapy may have several mechanisms. Due to its tiny size and physicochemical characteristics, it can readily penetrate iron-loaded myocytes where it may exert anti oxidant activity or bind the excess iron and carry it out of the cell into the circulation where it is excreted, mainly in the urine. In addition, L1's cardioprotective effects may be related to its ability to mobilize citrate-bound iron or other forms of nontransferrin-bound iron. We have continued to explore the use of readily available bedside tools, such as echocardio-grams and biochemical markers of cardiac dysfunction, to monitor thalassemia patients with cardiac complications. Herein, the literature and our own studies/findings are summarized. L1 chelation was found to have marginal benefits in increasing cardiac function and reducing cardiac iron accumulation.

keywords:  Cardiac function , Deferiprone , Deferoxamine , Iron-overloaded cardiomyopathy , Thalassemia

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