Deferiprone for the treatment of transfusional iron overload in thalassemia

Abstract

Transfusional iron overload can lead to hepatic fibrosis, arrhythmias and congestive heart failure and a number of endocrinopathies. Deferiprone is an oral iron chelator approved for use in the United States as a second line agent for the treatment of transfusional iron overload in patients with thalassemia. Areas covered: This article will review the data regarding the efficacy of deferiprone for iron chelation and prevention and reversal of iron related complications, the drug's adverse effect profile, and the use of this drug in combination regimens. Expert commentary: Extensive data support that deferiprone is particularly efficacious at cardiac iron removal and therefore, a chelator regimen that contains deferiprone is generally recommended when there is significant cardiac iron loading and/or in the setting of iron-related cardiac disease. The most concerning side effects of deferiprone are agranulocytosis and milder forms of neutropenia, which require appropriate monitoring and patient/provider education.

New Perspectives in Iron Chelation Therapy for the Treatment of Neurodegenerative Diseases

“…Successful experiences support the use of iron chelation therapy for the treatment of systemic diseases with an iron accumulation component, such as thalassemia major, sickle cell disease and cardiomyopathy associated with hereditary hemochromatosis [59,60,61,62,63,64,65,66,67]. The chelators used in these therapies are deferoxamine, deferasirox, deferiprone and PBT2.…”

Treatment of iron overload syndrome: a general review

“…It was synthesized in 1982 and initially approved for use in 1995 in India and in 2000 in Europe. The USFDA approved its use only in 2011 as rescue therapy in patients with thalassemia who had an insufficient response to the available treatments 13, 18 .…”

“…Its lipophilic molecule is the smallest among the three chelators, allowing for good oral bioavailability and better penetration in several tissues and in specific structures within the cell, such as the mitochondria. DFP may be a therapeutic option in other conditions related to iron metabolism, such as Friedreich's ataxia 18 .…”

“…The initial dose recommended is 75 to 100 mg/kg a day, divided into three daily doses 14, 18 . It has efficacy equivalent to DFO 18 and in severely loaded pa-tients, especially those with severe cardiac siderosis, DFP can be associated with DFO, showing a synergistic effect 18, 19 .…”

Deferiprone as Adjunctive Treatment for Patients with Invasive Mucormycosis: A Retrospective Case Series

“… 19 DEF was approved for the treatment of iron overload in Asia, Europe and United States. 20 Side effects included transient liver dysfunction, gastrointestinal discomfort, and arthralgia, although none of the patients discontinued therapy. 19 Long-term use of high-dose DEF (100 mg/kg/day for 2 years) showed higher efficacy in reduction of iron overload without increased toxicity.…”