Deferiprone for the treatment of transfusional iron overload in thalassemia

References

• Cohen AR, Glimm E, Porter JB. Effect of transfusional iron intake on response to chelation therapy in {beta}-thalassemia major. Blood. 2008;111(2):583–587.
   PubMed Web of Science ®Google Scholar
• Nienhuis AW, Nathan DG. Pathophysiology and clinical manifestations of the beta-Thalassemias. Cold Spring Harb Perspect Med. 2012;2(12):a011726.
   PubMed Web of Science ®Google Scholar
• Tanno T, Miller JL. Iron loading and overloading due to ineffective erythropoiesis. Adv Hematol. 2010;2010:358283.
   PubMedGoogle Scholar
• Piga A, Longo F, Duca L, et al. High nontransferrin bound iron levels and heart disease in thalassemia major. Am J Hematol. 2009;84(1):29–33.
   PubMed Web of Science ®Google Scholar
• Zurlo MG, De Stefano P, Borgna-Pignatti C, et al. Survival and causes of death in thalassaemia major. Lancet. 1989;2(8653):27–30.
   PubMed Web of Science ®Google Scholar
• Jean G, Terzoli S, Mauri R, et al. Cirrhosis associated with multiple transfusions in thalassaemia. Arch Dis Child. 1984;59(1):67–70.
   PubMed Web of Science ®Google Scholar
• Cunningham MJ, Macklin EA, Neufeld EJ, et al. Complications of beta-thalassemia major in North America. Blood. 2004;104(1):34–39.
   PubMed Web of Science ®Google Scholar
• Walter PB, Fung EB, Killilea DW, et al. Oxidative stress and inflammation in iron-overloaded patients with beta-thalassaemia or sickle cell disease. Br J Haematol. 2006;135(2):254–263.
   PubMed Web of Science ®Google Scholar
• Wood JC, Tyszka M, Carson S, et al. Myocardial iron loading in transfusion-dependent thalassemia and sickle cell disease. Blood. 2004;103(5):1934–1936.
   PubMed Web of Science ®Google Scholar
• Anderson LJ, Westwood MA, Holden S, et al. Myocardial iron clearance during reversal of siderotic cardiomyopathy with intravenous desferrioxamine: a prospective study using T2* cardiovascular magnetic resonance. Br J Haematol. 2004;127(3):348–355.
   PubMed Web of Science ®Google Scholar
• Farmaki K, Tzoumari I, Pappa C, et al. Normalisation of total body iron load with very intensive combined chelation reverses cardiac and endocrine complications of thalassaemia major. Br J Haematol. 2009;148(3):466–475.
   PubMed Web of Science ®Google Scholar
• Files B, Brambilla D, Kutlar A, et al. Longitudinal changes in ferritin during chronic transfusion: a report from the stroke prevention trial in sickle cell anemia (STOP). J Pediatr Hematol Oncol. 2002;24(4):284–290.
   PubMed Web of Science ®Google Scholar
• Pakbaz Z, Fischer R, Fung E, et al. Serum ferritin underestimates liver iron concentration in transfusion independent thalassemia patients as compared to regularly transfused thalassemia and sickle cell patients. Pediatr Blood Cancer. 2007;49(3):329–332.
   PubMed Web of Science ®Google Scholar
• Brittenham GM, Cohen AR, McLaren CE, et al. Hepatic iron stores and plasma ferritin concentration in patients with sickle cell anemia and thalassemia major. Am J Hematol. 1993;42(1):81–85.
   PubMed Web of Science ®Google Scholar
• Telfer PT, Prestcott E, Holden S, et al. Hepatic iron concentration combined with long-term monitoring of serum ferritin to predict complications of iron overload in thalassaemia major. Br J Haematol. 2000;110(4):971–977.
   PubMed Web of Science ®Google Scholar
• Adamkiewicz TV, Abboud MR, Paley C, et al. Serum ferritin level changes in children with sickle cell disease on chronic blood transfusion are nonlinear and are associated with iron load and liver injury. Blood. 2009;114(21):4632–4638.
   PubMed Web of Science ®Google Scholar
• Stanley HM, Friedman DF, Webb J, et al. Transfusional iron overload in a cohort of children with sickle cell disease: impact of magnetic resonance imaging, transfusion method, and chelation. Pediatr Blood Cancer. 2016;63(8):1414–1418.
   PubMed Web of Science ®Google Scholar
• Olivieri NF, Nathan DG, MacMillan JH, et al. Survival in medically treated patients with homozygous beta-thalassemia. N Engl J Med. 1994;331(9):574–578.
   PubMed Web of Science ®Google Scholar
• St. Pierre TG, Clark PR, Chua-Anusorn W, et al. Noninvasive measurement and imaging of liver iron concentrations using proton magnetic resonance. Blood. 2005;105(2):855–861.
   PubMed Web of Science ®Google Scholar
• Wood JC, Enriquez C, Ghugre N, et al. MRI R2 and R2* mapping accurately estimates hepatic iron concentration in transfusion-dependent thalassemia and sickle cell disease patients. Blood. 2005;106(4):1460–1465.
   PubMed Web of Science ®Google Scholar
• Kirk P, Roughton M, Porter JB, et al. Cardiac T2* magnetic resonance for prediction of cardiac complications in thalassemia major. Circulation. 2009;120(20):1961–1968.
   PubMed Web of Science ®Google Scholar
• Anderson LJ, Holden S, Davis B, et al. Cardiovascular T2-star (T2*) magnetic resonance for the early diagnosis of myocardial iron overload. Eur Heart J. 2001;22(23):2171–2179.
   PubMed Web of Science ®Google Scholar
• Westwood MA, Wonke B, Maceira AM, et al. Left ventricular diastolic function compared with T2* cardiovascular magnetic resonance for early detection of myocardial iron overload in thalassemia major. J Magn Reson Imaging. 2005;22(2):229–233.
   PubMed Web of Science ®Google Scholar
• Chouliaras G, Berdoukas V, Ladis V, et al. Impact of magnetic resonance imaging on cardiac mortality in thalassemia major. J Magn Reson Imaging. 2011;34(1):56–59.
   PubMed Web of Science ®Google Scholar
• Modell B, Khan M, Darlison M, et al. Improved survival of thalassaemia major in the UK and relation to T2* cardiovascular magnetic resonance. J Cardiovasc Magn Reson. 2008;10:42.
   PubMed Web of Science ®Google Scholar
• Kwiatkowski JL. Current recommendations for chelation for transfusion-dependent thalassemia. Ann N Y Acad Sci. 2016;1368(1):107–114.
   PubMedGoogle Scholar
• Cappellini MD, Cohen A, Piga A, et al. A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood. 2006;107(9):3455–3462.
   PubMed Web of Science ®Google Scholar
• Cohen A, Martin M, Schwartz E. Depletion of excessive liver iron stores with desferrioxamine. Br J Haematol. 1984;58(2):369–373.
   PubMed Web of Science ®Google Scholar
• Pennell DJ, Berdoukas V, Karagiorga M, et al. Randomized controlled trial of deferiprone or deferoxamine in beta-thalassemia major patients with asymptomatic myocardial siderosis. Blood. 2006;107(9):3738–3744.
   PubMed Web of Science ®Google Scholar
• Pennell DJ, Porter JB, Piga A, et al. A 1-year randomized controlled trial of deferasirox vs deferoxamine for myocardial iron removal in beta-thalassemia major (CORDELIA). Blood. 2014;123(10):1447–1454.
   PubMed Web of Science ®Google Scholar
• Delea TE, Edelsberg J, Sofrygin O, et al. Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion. 2007;47(10):1919–1929.
   PubMed Web of Science ®Google Scholar
• Gabutti V, Piga A. Results of long-term iron chelating therapy. Acta Haematologica. 1996;95:26–36.
   PubMed Web of Science ®Google Scholar
• Pennell DJ, Porter JB, Cappellini MD, et al. Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with beta-thalassemia major. Haematologica. 2012;97(6):842–848.
   PubMed Web of Science ®Google Scholar
• Goulas V, Kourakli-Symeonidis A, Camoutsis C. Comparative effects of three iron chelation therapies on the quality of life of greek patients with homozygous transfusion-dependent Beta-thalassemia. ISRN Hematol. 2012;2012:139862.
   PubMedGoogle Scholar
• Trachtenberg F, Vichinsky E, Haines D, et al. Iron chelation adherence to deferoxamine and deferasirox in thalassemia. Am J Hematol. 2011;86(5):433–436.
   PubMed Web of Science ®Google Scholar
• Porter JB, Evangeli M, El-Beshlawy A. Challenges of adherence and persistence with iron chelation therapy. Int J Hematol. 2011;94(5):453–460.
   PubMed Web of Science ®Google Scholar
• Kontoghiorghes GJ, Pattichis K, Neocleous K, et al. The design and development of deferiprone (L1) and other iron chelators for clinical use: targeting methods and application prospects. Curr Med Chem. 2004;11(16):2161–2183.
   PubMed Web of Science ®Google Scholar
• Al-Refaie FN, Sheppard LN, Nortey P, et al. Pharmacokinetics of the oral iron chelator deferiprone (L1) in patients with iron overload. Br J Haematol. 1995;89(2):403–408.
   PubMed Web of Science ®Google Scholar
• Matsui D, Klein J, Hermann C. Relationship between the pharmacokinetics and iron excretion of the new oral iron chelator 1,2-dimethyl-3-hydroxypyrid-4-1 in patients with thalassemia. Clin Pharmacol Ther. 1991;50(3):294–298.
   PubMed Web of Science ®Google Scholar
• ElAlfy MS, Sari TT, Lee CL, et al. The safety, tolerability, and efficacy of a liquid formulation of deferiprone in young children with transfusional iron overload. J Pediatr Hematol Oncol. 2010;32(8):601–605.
   PubMed Web of Science ®Google Scholar
• Ceci A, Baiardi P, Felisi M, et al. The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients. Br J Haematol. 2002;118(1):330–336.
   PubMed Web of Science ®Google Scholar
• Cohen AR, Galanello R, Piga A, et al. Safety profile of the oral iron chelator deferiprone: a multicentre study. Br J Haematol. 2000;108(2):305–312.
   PubMed Web of Science ®Google Scholar
• Cohen AR, Galanello R, Piga A, et al. Safety and effectiveness of long-term therapy with the oral iron chelator deferiprone. Blood. 2003;102(5):1583–1587.
   PubMed Web of Science ®Google Scholar
• Taher A, Sheikh-Taha M, Sharara A, et al. Safety and effectiveness of 100 mg/kg/day deferiprone in patients with thalassemia major: a two-year study. Acta Haematol. 2005;114(3):146–149.
   PubMed Web of Science ®Google Scholar
• Wonke B, Wright C, Hoffbrand AV. Combined therapy with deferiprone and desferrioxamine. Br J Haematol. 1998;103(2):361–364.
   PubMed Web of Science ®Google Scholar
• Fischer R, Longo F, Nielsen P, et al. Monitoring long-term efficacy of iron chelation therapy by deferiprone and desferrioxamine in patients with beta-thalassaemia major: application of SQUID biomagnetic liver susceptometry. Br J Haematol. 2003;121(6):938–948.
   PubMed Web of Science ®Google Scholar
• Mazza P, Amurri B, Lazzari G, et al. Oral iron chelating therapy. A single center interim report on deferiprone (L1) in thalassemia. Haematologica. 1998;83:496–501.
   PubMed Web of Science ®Google Scholar
• Olivieri NF, Brittenham GM, McLaren CE, et al. Long-term safety and effectiveness of iron-chelation therapy with deferiprone for thalassemia major. N Engl J Med. 1998;339(7):417–423.
   PubMed Web of Science ®Google Scholar
• Anderson LJ, Wonke B, Prescott E, et al. Comparison of effects of oral deferiprone and subcutaneous desferrioxamine on myocardial iron concentrations and ventricular function in beta-thalassaemia. Lancet. 2002;360(9332):516–520.
   PubMed Web of Science ®Google Scholar
• Pepe A, Meloni A, Capra M, et al. Deferasirox, deferiprone and desferrioxamine treatment in thalassemia major patients: cardiac iron and function comparison determined by quantitative magnetic resonance imaging. Haematologica. 2011;96(1):41–47.
   PubMed Web of Science ®Google Scholar
• Maggio A, D’Amico G, Morabito A, et al. Deferiprone versus deferoxamine in patients with thalassemia major: a randomized clinical trial. Blood Cells Mol Dis. 2002;28(2):196–208.
   PubMed Web of Science ®Google Scholar
• Berdoukas V, Chouliaras G, Moraitis P, et al. The efficacy of iron chelator regimes in reducing cardiac and hepatic iron in patients with thalassaemia major: a clinical observational study. J Cardiovasc Magn Reson. 2009;11:20.
   PubMed Web of Science ®Google Scholar
• Perifanis V, Christoforidis A, Vlachaki E, et al. Comparison of effects of different long-term iron-chelation regimens on myocardial and hepatic iron concentrations assessed with T2* magnetic resonance imaging in patients with beta-thalassemia major. Int J Hematol. 2007;86(5):385–389.
   PubMed Web of Science ®Google Scholar
• Piga A, Gaglioti C, Fogliacco E, et al. Comparative effects of deferiprone and deferoxamine on survival and cardiac disease in patients with thalassemia major: a retrospective analysis. Haematologica. 2003;88(5):489–496.
   PubMed Web of Science ®Google Scholar
• Filosa A, Vitrano A, Rigano P, et al. Long-term treatment with deferiprone enhances left ventricular ejection function when compared to deferoxamine in patients with thalassemia major. Blood Cells Mol Dis. 2013;51(2):85–88.
   PubMed Web of Science ®Google Scholar
• Smith GC, Alpendurada F, Carpenter JP, et al. Effect of deferiprone or deferoxamine on right ventricular function in thalassemia major patients with myocardial iron overload. J Cardiovasc Magn Reson. 2011;13:34.
   PubMed Web of Science ®Google Scholar
• Borgna-Pignatti C, Cappellini MD, De Stefano P, et al. Cardiac morbidity and mortality in deferoxamine- or deferiprone-treated patients with thalassemia major. Blood. 2006;107(9):3733–3737.
   PubMed Web of Science ®Google Scholar
• Makis A, Chaliasos N, Alfantaki S, et al. Chelation therapy with oral solution of deferiprone in transfusional iron-overloaded children with hemoglobinopathies. Anemia. 2013;2013:121762.
   PubMedGoogle Scholar
• Viprakasit V, Nuchprayoon I, Chuansumrit A, et al. Deferiprone (GPO-L-ONE((R))) monotherapy reduces iron overload in transfusion-dependent thalassemias: 1-year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO-L-ONE; A001) from Thailand. Am J Hematol. 2013;88(4):251–260.
   PubMed Web of Science ®Google Scholar
• Botzenhardt S, Sing CW, Wong IC, et al. Safety profile of oral iron chelator deferiprone in chinese children with transfusion-dependent thalassaemia. Curr Drug Saf. 2016;11(2):137–144.
   PubMed Web of Science ®Google Scholar
• Chuansumrit A, Songdej D, Sirachainan N, et al. Safety profile of a liquid formulation of deferiprone in young children with transfusion-induced iron overload: a 1-year experience. Paediatr Int Child Health. 2016. doi:10.1080/20469047.2015.1109272. [Epub ahead of print]
   Google Scholar
• Evans P, Kayyali R, Hider RC, et al. Mechanisms for the shuttling of plasma non-transferrin-bound iron (NTBI) onto deferoxamine by deferiprone. Transl Res. 2010;156(2):55–67.
   PubMed Web of Science ®Google Scholar
• Tanner MA, Galanello R, Dessi C, et al. A randomized, placebo-controlled, double-blind trial of the effect of combined therapy with deferoxamine and deferiprone on myocardial iron in thalassemia major using cardiovascular magnetic resonance. Circulation. 2007;115(14):1876–1884.
   PubMed Web of Science ®Google Scholar
• Tanner MA, Galanello R, Dessi C, et al. Combined chelation therapy in thalassemia major for the treatment of severe myocardial siderosis with left ventricular dysfunction. J Cardiovasc Magn Reson. 2008;10:12.
   PubMed Web of Science ®Google Scholar
• Porter JB, Wood J, Olivieri N, et al. Treatment of heart failure in adults with thalassemia major: response in patients randomised to deferoxamine with or without deferiprone. J Cardiovasc Magn Reson. 2013;15:38.
   PubMed Web of Science ®Google Scholar
• Alpendurada F, Smith GC, Carpenter JP, et al. Effects of combined deferiprone with deferoxamine on right ventricular function in thalassaemia major. J Cardiovasc Magn Reson. 2012;14:8.
   PubMed Web of Science ®Google Scholar
• Kolnagou A, Kontoghiorghes GJ. Effective combination therapy of deferiprone and deferoxamine for the rapid clearance of excess cardiac IRON and the prevention of heart disease in thalassemia. the protocol of the international committee on oral chelators. Hemoglobin. 2006;30(2):239–249.
   PubMed Web of Science ®Google Scholar
• Farmaki K, Tzoumari I, Pappa C. Oral chelators in transfusion-dependent thalassemia major patients may prevent or reverse iron overload complications. Blood Cells Mol Dis. 2011;47(1):33–40.
   PubMed Web of Science ®Google Scholar
• Totadri S, Bansal D, Bhatia P, et al. The deferiprone and deferasirox combination is efficacious in iron overloaded patients with beta-thalassemia major: a prospective, single center, open-label study. Pediatr Blood Cancer. 2015;62(9):1592–1596.
   PubMed Web of Science ®Google Scholar
• Elalfy MS, Adly AM, Wali Y, et al. Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients. Eur J Haematol. 2015;95(5):411–420.
   PubMed Web of Science ®Google Scholar
• Agarwal MB, Gupte SS, Viswanathan C, et al. Long-term assessment of efficacy and safety of L1, an oral iron chelator, in transfusion dependent thalassaemia: indian trial. Br J Haematol. 1992;82(2):460–466.
   PubMed Web of Science ®Google Scholar
• Tondury P, Zimmerman A, Nielsen P, et al. Liver iron and fibrosis during long-term treatment with deferiprone in Swiss thalassaemic patients. Br J Haematol. 1998;101:413–415.
   PubMed Web of Science ®Google Scholar
• Wanless IR, Sweeney G, Dhillon AP, et al. Lack of progressive hepatic fibrosis during long-term therapy with deferiprone in subjects with transfusion-dependent beta-thalassemia. Blood. 2002;100:1566–1569.
   PubMed Web of Science ®Google Scholar
• Al-Refaie FN, Hershko C, Hoffbrand AV, et al. Results of long-term deferiprone (L1) therapy: a report by the International Study Group on oral iron chelators. Br J Haematol. 1995;91:224–229.
   PubMed Web of Science ®Google Scholar
• Tricta F, Uetrecht J, Galanello R, et al. Deferiprone-induced agranulocytosis: 20 years of clinical observations. Am J Hematol. 2016;91:1026–1031.
   PubMed Web of Science ®Google Scholar
• Elalfy M, Wali YA, Qari M, et al. Deviating from safety guidelines during deferiprone therapy in clinical practice may not be associated with higher risk of agranulocytosis. Pediatr Blood Cancer. 2014;61(5):879–884.
   PubMed Web of Science ®Google Scholar
• El-Beshlawy AM, El-Alfy MS, Sari TT, et al. Continuation of deferiprone therapy in patients with mild neutropenia may not lead to a more severe drop in neutrophil count. Eur J Haematol. 2014;92(4):337–340.
   PubMed Web of Science ®Google Scholar
• Calvaruso G, Vitrano A, Di Maggio R, et al. Deferiprone versus deferoxamine in sickle cell disease: results from a 5-year long-term Italian multi-center randomized clinical trial. Blood Cells Mol Dis. 2014;53(4):265–271.
   PubMed Web of Science ®Google Scholar
• Maggio A, Rigano P, D’Amico G, et al. Treatment with hydroxyurea and iron chelation therapy in patients with hemoglobinopathies. Eur J Haematol. 2005;75(3):267–269.
   PubMed Web of Science ®Google Scholar
• Henter JI, Karlen J. Fatal agranulocytosis after deferiprone therapy in a child with diamond-blackfan anemia. Blood. 2007;109(12):5157–5159.
   PubMed Web of Science ®Google Scholar
• Hoffbrand AV, Bartlett AN, Veys PA, et al. Agranulocytosis and thrombocytopenia in patient with blackfan-diamond anaemia during oral chelator trial. Lancet. 1989;2(8660):457.
   PubMed Web of Science ®Google Scholar
• Naithani R, Chandra J, Sharma S. Safety of oral iron chelator deferiprone in young thalassaemics. Eur J Haematol. 2005;74(3):217–220.
   PubMed Web of Science ®Google Scholar
• Jamuar SS, Lai AH, Tan AM, et al. Use of deferiprone for iron chelation in patients with transfusion-dependent thalassaemia. J Paediatr Child Health. 2011;47(11):812–817.
   PubMed Web of Science ®Google Scholar
• Calvaruso G, Vitrano A, Di Maggio R, et al. Deferiprone versus deferoxamine in thalassemia intermedia: results from a 5-year long-term Italian multicenter randomized clinical trial. Am J Hematol. 2015;90(7):634–638.
   PubMed Web of Science ®Google Scholar
• Collins AF, Fassos FF, Stobie S, et al. Iron-balance and dose-response studies of the oral iron chelator 1,2-dimethyl-3-hydroxypyrid-4-one (L1) in iron-loaded patients with sickle cell disease. Blood. 1994;83(8):2329–2333.
   PubMed Web of Science ®Google Scholar