Scientific Online Resource System

Heart - Lung (Varna)

Heart failure treatment in patients with thalassemia major

Mariya Stoyanova Dimova, Valeria Kaleva, Yavor Kashlov


The modern treatment of heart failure (HF) in patients with thalassemia is based on the prevention of cardiac impair- ment. Iron overload cardiomyopathy is the most serious and life-threatening complication of transfusion-dependent thalassemia, but due to its association with iron deposition it is reversible. Treatment includes chelation therapy and conventional medication for HF. Severely decompensated cases are treated in specialized centers.


thalassemia major, heart failure

Full Text


Capellini MD, Cohen A, Porter J, Taher A, Viprakasit V, Guidelines for the treatment of transfsion dependent talassemia (TDT), 3rd edition 2014

Farmakis D, Triposkiadis F, Lekakis J, Parissis J. Heart failure in haemoglobinopathies: pathophysiology, clinical phenotypes, and management. Eur J Heart Fail 2017; 19: 479–489

Pennell DJ, Udelson JE, Arai AE, Bozkurt B, Cohen AR, Galanello R, Hoffman TM, Kiernan MS, Lerakis S, Piga A, Porter JB, Walker JM, Wood J; American Heart Association Committee on Heart Failure and Transplantation of the Council on Clinical Cardiology and Council on Cardiovascular Radiology and Imaging. Cardiovascular function and treatment in thalassemia major: a consensus statement from the American Heart Association. Circulation 2013;128:281–308

Carpenter JP, He T, Kirk P, Roughton M, Anderson LJ, de Noronha SV, Sheppard MN, Porter JB, Walker JM, Wood JC, Galanello R, Forni G, Catani G, Matta G, Fucharoen S, Fleming A, House MJ, Black G, Firmin DN, St Pierre TG, Pennell DJ.On T2* magnetic resonance and cardiac iron. Circulation 2011;123:1519–1528.

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;127:348-55

Chouliaras GL, Kattamis A, Berdoukas V, et al.Cardiac magnetic resonance in transfusion dependent thalassaemia: assessment of iron load and relationship to left ventricular ejection fraction. Br J Haematol 2010;151:397-401.

Kirk P, Roughton M, Porter JB, Walker JM, Tanner MA, Patel J, Wu D, Taylor J, Westwood MA, Anderson LJ, Pennell DJ. Cardiac T2* magnetic resonance for prediction of cardiac complications in thalassemia major.Circulation. 2009;120:1961–1968

Hider RC, Liu ZD. Emerging understanding of the advantage of small molecules such as hydroxypyridinones in the treatment of iron overload.Curr Med Chem. 2003;10:1051–1064.

Tanner MA, Galanello R, Dessi C, Smith GC, Westwood MA, Agus A, Roughton M, Assomull R, Nair SV, Walker JM, Pennell DJ 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;115:1876–1884

Galanello R, Campus S. Deferiprone chelation therapy for thalassemia major.Acta Haematol. 2009;122:155–16

Ha SY, Mok AS, Chu WC, Rasalkar DD, Cheuk DK, Chiang AK, Ho MH, Chan GC. Intermediate-term evaluation of a practical chelation protocol based on stratification of thalassemic patients by serum ferritin and magnetic resonance imaging cardiac t2*.Hemoglobin. 2011;35:199–205

Au WY, Lee V, Lau CW, Yau J, Chan D, Chan EY, Cheung WW, Ha SY, Kho B, Lee CY, Li RC, Li CK, Lin SY, Ling AS, Mak V, Sun L, Wong KH, Wong R, Yuen HL. A synopsis of current care of thalassaemia major patients in Hong Kong.Hong Kong Med J. 2011;17:261–266.

Smith GC, Alpendurada F, Carpenter JP, Alam MH, Berdoukas V, Karagiorga M, Ladis V, Piga A, Aessopos A, Gotsis ED, Tanner MA, Westwood MA, Galanello R, Roughton M, Pennell DJ. Effect of deferiprone or deferoxamine on right ventricular function in thalassemia major patients with myocardial iron overload.J Cardiovasc Magn Reson, 2011;13:3

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(1):38. Published 2013 May 20. doi:10.1186/1532-429X-15-38

Galanello R, Campus S, Origa R. Deferasirox: pharmacokinetics and clinical experience.Expert Opin Drug Metab Toxicol. 2012;8:123–134

Vichinsky E, Bernaudin F, Forni GL, Gardner R, Hassell K, Heeney MM, Inusa B, Kutlar A, Lane P, Mathias L, Porter J, Tebbi C, Wilson F, Griffel L, Deng W, Giannone V, Coates T. Long-term safety and efficacy of deferasirox (Exjade) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease.Br J Haematol. 2011;154:387–397

Exjade (deferasirox) US Prescribing Information. T2013-04. January 2013.

Pennell DJ, Porter JB, Cappellini MD, Chan LL, El-Beshlawy A, Aydinok Y, Ibrahim H, Li CK, Viprakasit V, Elalfy MS, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A. Continued improvement in myocardial T2* over two years of deferasirox therapy in β-thalassemia major patients with cardiac iron overload.Haematologica. 2011;96:48–54

Pennell DJ, Porter JB, Cappellini MD, Chan LL, El-Beshlawy A, Aydinok Y, Ibrahim H, Li CK, Viprakasit V, Elalfy MS, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A. Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia major.Haematologica. 2012;97:842–848

Wood JC, Glynos T, Thompson A, Giardina P, Harmatz P, Kang BP, Paley C, Coates TD. Follow-up report on the 2-year cardiac data from a deferasirox monotherapy trial.Am J Hematol. 2010;85:818–819

Pathare A, Taher A, Daar S. Deferasirox (Exjade) significantly improves cardiac T2* in heavily iron-overloaded patients with beta-thalassemia major.Ann Hematol. 2010;89:405–409.

Pennell DJ, Porter JB, Piga A, et al. A 1-year randomized controlled trial of deferasirox vs deferoxamine for myocardial iron removal in β-thalassemia major (CORDELIA). Blood. 2014;123(10):1447-54.

Evans P, Kayyali R, Hider RC, Eccleston J, Porter JB. Mechanisms for the shuttling of plasma non-transferrin-bound iron (NTBI) onto deferoxamine by deferiprone.Transl Res. 2010;156:55–67.

Farmaki K, Tzoumari I, Pappa C, Chouliaras G, Berdoukas V. Normalisation of total body iron load with very intensive combined chelation reverses cardiac and endocrine complications of thalassaemia major.Br J Haematol. 2010;148:466–475

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;47:33–40

Alpendurada F, Smith GC, Carpenter JP, Nair SV, Tanner MA, Banya W, Dessi C, Galanello R, Walker JM, Pennell DJ. Effects of combined deferiprone with deferoxamine on right ventricular function in thalassaemia major.J Cardiovasc Magn Reson. 2012;14:8

Telfer PT, Warburton F, Christou S, Hadjigavriel M, Sitarou M, Kolnagou A, Angastiniotis M. Improved survival in thalassemia major patients on switching from desferrioxamine to combined chelation therapy with desferrioxamine and deferiprone.Haematologica. 2009;94:1777–1778

Galanello R, Agus A, Campus S, Danjou F, Giardina PJ, Grady RW. Combined iron chelation therapy.Ann N Y Acad Sci. 2010;1202:79–86

Lal A, Porter J, Sweeters N, Ng V, Evans P, Neumayr L, Kurio G, Harmatz P, Vichinsky E. Combined chelation therapy with deferasirox and deferoxamine in thalassemia.Blood Cells Mol Dis. 2013;50:99–104

Jetsrisuparb A, Komvilaisak P, Wiangnon S, Jetsrisuparb C. Retrospective study on the combination of desferrioxamine and deferasirox for treatment of iron-overloaded thalassemic patients: first evidence of more than 2 years.J Pediatr Hematol Oncol. 2010;32:400–403

Maggio A, Vitrano A, Capra M, Cuccia L, Gagliardotto F, Filosa A, Romeo MA, Magnano C, Caruso V, Argento C, Gerardi C, Campisi S, Violi P, Malizia R, Cianciulli P, Rizzo M, D’Ascola DG, Quota A, Prossomariti L, Fidone C, Rigano P, Pepe A, D’Amico G, Morabito A, Gluud C. Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassaemia major patients: a randomized clinical trial.Br J Haematol. 2009;145:245–254

Pennell DJ, Carpenter JP, Roughton M, Cabantchik Z. On improvement in ejection fraction with iron chelation in thalassemia major and the risk of future heart failure.J Cardiovasc Magn Reson. 2011;13:45.

Patel N, Gonsalves CS, Malik P, Kalra VK. Placenta growth factor augments endothelin-1 and endothelin-B receptor expression via hypoxia-inducible factor-1 alpha. Blood 2008;112(3):856-865

Piga A, Longo F, Duca L, et al. High nontransferrin bound iron levels and heart disease in thalassemia major. Am J Hematol 2009;84:29-33.

Zanninelli G, Breuer W, Cabantchik ZI. Daily labile plasma iron as an indicator of chelator activity in Thalassaemia major patients.Br J Haematol. 2009;147:744–75

Derchi G, Formisano F, Balocco M, Galanello R, Bina P, Dessì C, Piga A, Donato G, Cappellini M, Cassinerio M, Quarta G, Melpignano A, Forni GL; Clinical management of cardiovascular complications in patients with thalassaemia major: a large observational multicenter study. Eur J Echocardiogr 2011; 12 (3): 242-246.

Fernandes JL, Sampaio EF, Fertrin K, Coelho OR, Loggetto S, Piga A, Verissimo M, Saad ST. Amlodipine reduces cardiac iron overload in patients with thalassemia major: a pilot trial. Am J Med 2013;126:834–837.



Font Size