Effect of deferiprone or deferoxamine on right ventricular function in thalassemia major patients with myocardial iron overload

Smith, Gillian; Alpendurada, Francisco; Carpenter, John Paul; Alam, Mahbub; Berdoukas, Vasilios; Karagiorga, Markisia; Ladis, Vassilios; Piga, Antonio; Aessopos, Athanasios; Gotsis, Efstathios D.; Tanner, Mark; Westwood, Mark; Galanello, Renzo; Roughton, Michael; Pennell, Dudley J.

doi:10.1186/1532-429x-13-34

Cited by 33 publications

(23 citation statements)

References 36 publications

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“…Cardiac T2* improved after 1 year of treatment with DFO by 7-13% Pennell et al, 2014;Smith et al, 2011), and after 1 and 3 years of treatment with DFX by 12-29% (Merchant et al, 2012;Pennell et al, 2014) and 43% , respectively. DFO improved LVEF by only 0.3-2% (Maggio et al, 2002;Pennell et al, 2006), and DFX did not alter LVEF (Merchant et al, 2012;Pennell et al, 2014) after 1 year of treatment.…”

Section: Effects Of Dfo+dfxmentioning

confidence: 89%

Current and future treatment strategies for iron overload cardiomyopathy

Wongjaikam

Kumfu

Chattipakorn

et al. 2015

European Journal of Pharmacology

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Section: Effects Of Dfo+dfxmentioning

confidence: 89%

Current and future treatment strategies for iron overload cardiomyopathy

Wongjaikam

Kumfu

Chattipakorn

et al. 2015

European Journal of Pharmacology

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“…Many studies have shown that DFO, DFP and DFX can cause a reduction in myocardial iron and improve cardiac function in b-thalassemia patients. 55,[60][61][62][63][64][65] However, it has been suggested that combined chelation therapy be used in some iron overload b-thalassemia patients who have shown no improvement when treated with a single iron chelator, due to its increased efficacy. [62][63][64][65] At this time, new evidence indicates a greater efficacy for iron chelation when an iron chelator was combined with an antioxidant such as vitamin E and N-acetylcysteine (NAC).…”

Section: Cardiac Complications In Beta-thalassemia Patientsmentioning

confidence: 99%

Cardiac complications in beta-thalassemia: From mice to men

Kumfu

Fucharoen

Chattipakorn

2017

Exp Biol Med (Maywood)

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Iron overload cardiomyopathy is a major cause of morbidity and mortality in patients with thalassemia. Since investigation of iron overload cardiomyopathy in thalassemia patients has many limitations, a search for an animal model for this condition has been ongoing for decades. In the past decades, there is no doubt that the use of b-thalassemic mice as a study model to investigate the pathophysiology of iron overload cardiomyopathy and the role of various pharmacological interventions, has shed some light in understanding this serious complication and in improving the associated cardiac dysfunction. In this review, the effects of iron overload on the hearts of b-thalassemic mice under conditions of iron overload as well as the efficacy of pharmacological interventions to combat these adverse effects on the heart are reviewed and discussed. AbstractBeta-thalassemia is an inherited hemoglobin disorder caused by reduced or absent synthesis of the beta globin chains of hemoglobin. This results in variable outcomes ranging from clinically asymptomatic to severe anemia, which then typically requires regular blood transfusion. These regular blood transfusions can result in an iron overload condition. The iron overload condition can lead to iron accumulation in various organs, especially in the heart, leading to iron overload cardiomyopathy, which is the major cause of mortality in patients with thalassemia. In the past decades, there is no doubt that the use of b-thalassemic mice as a study model to investigate the pathophysiology of iron overload cardiomyopathy and the role of various pharmacological interventions, has shed some light in understanding this serious complication and in improving the associated cardiac dysfunction. In this review, the effects that iron overload has on the hearts of b-thalassemic mice under conditions of iron overload as well as the efficacy of pharmacological interventions to combat these adverse effects on the heart are reviewed and discussed. The in-depth understanding of biomolecular alterations in the heart of these iron overload thalassemic mice will help give guidance for more effective therapeutic approaches in the near future.

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“…However, the price of this medication is unaffordable for most patients. Deferiprone (DFP), a cheaper oral iron chelator proven effective in achieving a negative iron balance in many short- and long-term clinical trials in adult patients with iron overload[3,4,5,6,7,8,9], has recently been studied for its efficacy and safety in thalassemia children receiving chelation [10,11,12,13,14,15,16,17]. To improve the efficacy of chelation treatment, the ‘iron shuttle hypothesis' [18,19] has been proposed, leading to many studies on the combined use of DFP and DFO [10,11,20,21,22,23,24,25,26,27,28,29,30].…”

Section: Introductionmentioning

confidence: 99%

Combined Chelation Therapy with Daily Oral Deferiprone and Twice-Weekly Subcutaneous Infusion of Desferrioxamine in Children with β-Thalassemia: 3-Year Experience

Songdej

Sirachainan²,

Wongwerawattanakoon³

et al. 2014

Acta Haematol

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Objective: To study the efficacy of combined treatment with oral and subcutaneous iron chelators. Material and Methods: 50-100 mg/kg/day of oral deferiprone (DFP) combined with 40 mg/kg/dose s.c. desferrioxamine (DFO) twice weekly were given to transfusion-dependent β-thalassemia children. Results: Enrolled patients (9 with β-thalassemia major and 33 with β-thalassemia hemoglobin E), ranging from 3 to 18 years in age, were divided into 3 groups; group 1 ferritin ≥1,000-2,500 ng/ml (n = 10), group 2 ferritin >2,500-4,000 ng/ml (n = 23) and group 3 ferritin >4,000 ng/ml (n = 9). Of the 42 patients, 28 reached the 36-month follow-up. Ten patients whose ferritin declined <15% while receiving 100 mg/kg/day of DFP were considered nonresponders. The median age and previous transfusion duration before enrollment were significantly higher in nonresponders than responders (p = 0.04 and 0.003, respectively). The responders exhibited a significant fall in median ferritin levels from 2,954.6 to 936.6 ng/ml (p < 0.001). Time to a significant decrease in serum ferritin among responders was 6 months. In 13 patients, 16 episodes of adverse events occurred: hemophagocytosis with cytopenia (n = 1), neutropenia (n = 2), thrombocytopenia (n = 2), elevated alanine aminotransferase (n = 5), elevated serum creatinine (n = 1), proteinuria (n = 1) and gastrointestinal discomfort (n = 4). Conclusion: Combination therapy with daily oral DFP and subcutaneous DFO twice weekly is a safe and effective alternative to chelation monotherapy in β-thalassemia children. © 2014 S. Karger AG, Basel

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Effect of deferiprone or deferoxamine on right ventricular function in thalassemia major patients with myocardial iron overload

Cited by 33 publications

References 36 publications

Current and future treatment strategies for iron overload cardiomyopathy

Current and future treatment strategies for iron overload cardiomyopathy

Cardiac complications in beta-thalassemia: From mice to men

Combined Chelation Therapy with Daily Oral Deferiprone and Twice-Weekly Subcutaneous Infusion of Desferrioxamine in Children with β-Thalassemia: 3-Year Experience

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