Comparison of Deferiprone to Deferasirox and Deferoxamine to Cardiac and Hepatic T2* MRI in Thalassemia Patients: Evidence-based Case Report

Pustika Amalia Wahidiyat, Mikhael Yosia, Teny Tjitra Sari

Abstract


Background: there are currently three iron chelator readily available for patients Indonesia; deferiprone/DFP (branded as Ferriprox), deferasirox/DFX (branded as Exjade) and deferoxamine/DFO (branded as Desferal). This study aims to determine which iron chelator is the most efficient in reducing cardiac and hepatic iron overload (measured by means of T2* MRI). Methods: journal search with determined MeSH term was done in PubMed and Scopus. Studies that looked upon thalassemia major patient in all ages with usage of monotherapy iron chelation and its effect on myocardial T2* MRI and/or liver T2* MRI was included. Appraisal of studies was done using Oxford’s CEBM appraisal tools and Joanna Brigs Institute critical appraisal tools. Results: total of 11 studies with grand total of 611 samples were included. Mean T2* MRI value or (when available) mean changes in T2* MRI value after usage of specific iron chelator was gained from all the studies included. Comparison study and individual studies shows better control and increase of myocardial T2* MRI in those with DFP, and of liver T2* in those with good adherence to DFO chelation. Conclusion: DFP is superior in controlling or reducing myocardial iron load (as proven by mT2* MRI) and DFO had better capabilities in controlling or reducing hepatic iron load (as proven by liver T2* MRI). Studies with longer observation and larger samples is needed to see a significant changes of T2* MRI in DFX.

Keywords


iron chelation; thalassemia; T2* MRI

References


Luangasanatip N, Chaiyakunapruk N, Upakdee N, Wong P. Iron-chelating therapies in a transfusion-dependent thalassaemia population in Thailand: a cost-effectiveness study. Clin Drug Investig. 2011;31(7):493-505.

Pepe A, Rossi G, Bentley A, et al. Cost-utility analysis of three iron chelators used in monotherapy for the treatment of chronic iron overload in beta-thalassaemia major patients: An Italian perspective. Clin Drug Investig; 2017.

Xia S, Zhang W, Huang L, Jiang H. Comparative efficacy and safety of deferoxamine, deferiprone and deferasirox on severe thalassemia: a meta-analysis of 16 randomized controlled trials. PLoS One. 2013;8(12):e82662.

Ahmed J, Ahmad N, Jankharia B, Krishnan P, Merchant RH. Effect of deferasirox chelation on liver iron and total body iron concentration. Indian J Pediatrics. 2013;80(8):655-8.

Gomber S, Jain P, Sharma S, Narang M. Comparative efficacy and safety of oral iron chelators and their novel combination in children with thalassemia. Indian J Pediatrics. 2016;53(3):207-10.

Ho PJ, Tay L, Teo J, et al. Cardiac iron load and function in transfused patients treated with deferasirox (the MILE study). Eur J Haematol. 2017;98(2):97-105.

Merchant R, Ahmed J, Krishnan P, Jankharia B. Efficacy and safety of deferasirox for reducing total body and cardiac iron in thalassemia. Indian J Pediatrics. 2012;49(4):281-5.

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(4):405-9.

Pennell DJ, Porter JB, Cappellini MD, et al. Continued improvement in myocardial T2* over two years of deferasirox therapy in beta-thalassemia major patients with cardiac iron overload. Haematologica. 2011;96(1):48-54.

Pennell DJ, Berdoukas V, Karagiorga M, Ladis V, Piga A, Aessopos A, Gotsis ED, Tanner MA, Smith GC, Westwood MA, Wonke B. Randomized controlled trial of deferiprone or deferoxamine in beta-thalassemia major patients with asymptomatic myocardial siderosis. Blood. 2006;107(9):3738-44.

Pepe A, Lombardi M, Positano V, et al. Evaluation of the efficacy of oral deferiprone in beta-thalassemia major by multislice multiecho T2*. Eur J Haematol. 2006;76(3):183-92.

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):41-7.

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):251-60.

Zachariah M, Tony S, Bashir W, Al Rawas A, Wali Y, Pathare A. Comparative assessment of deferiprone and deferasirox in thalassemia major patients in the first two decades-single centre experience. Pediatric Hematol Oncol. 2013;30(2):104-12.

Saggar K, Sobti P. MRI Assessment of iron overload in thalassemia: an overview. Rivista Italiana di Medicina dell’Adolescenza-Volume. 2013;11(1).

Ambati SR, Randolph R, Mennitt K, Kleinert DA, Giardina P. Monitoring cardiac siderosis in patients with beta-thalassemia major on various chelation regimens. Blood. 2011;118(21):3177.

Borgna-Pignatti CA, Rugolotto SI, De Stefano P, Zhao HU, Cappellini MD, Del Vecchio GC, Romeo MA, Forni GL, Gamberini MR, Ghilardi RO, Piga AN. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica. 2004;89(10):1187-93.

Anderson LJ, Westwood MA, Holden S, Davis B, Prescott E, Wonke B, Porter JB, Malcolm Walker J, Pennell DJ. Myocardial iron clearance during reversal of siderotic cardiomyopathy with intravenous desferrioxamine: a prospective study using T2* cardiovascular magnetic resonance. British J Haematol. 2004;127(3):348-55.

Porter JB, Tanner MA, Pennell DJ, Eleftheriou P. Improved myocardial T2* in transfusion dependent anemias receiving ICL670 (Deferasirox). Blood. 2005;106(11):3600.

Maggio A, Vitrano A, Lucania G, Capra M, Cuccia L, Gagliardotto F, Pitrolo L, Prossomariti L, Filosa A, Caruso V, Gerardi C. Long‐term use of deferiprone significantly enhances left‐ventricular ejection function in thalassemia major patients. Am J Hematol. 2012;87(7):732-3.

Piga A, Gaglioti C, Fogliacco E, Tricta F. Comparative effects of deferiprone and deferoxamine on survival and cardiac disease in patients with thalassemia major: a retrospective analysis. Haematologica. 2003;88(5):489-96.

Anderson LJ, Wonke B, Prescott E, Holden S, Walker JM, Pennell DJ. Comparison of effects of oral deferiprone and subcutaneous desferrioxamine on myocardial iron concentrations and ventricular function in beta-thalassaemia. Lancet. 2002;360(9332):516-20.

Brittenham GM, Cohen AR, McLaren CE, Martin MB, Griffith PM, Nienhuis AW, Young NS, Allen CJ, Farrell DE, Harris JW. Hepatic iron stores and plasma ferritin concentration in patients with sickle cell anemia and thalassemia major. Am J Hematol. 1993;42(1):81-5.

Cappellini MD, Bejaoui M, Agaoglu L, Porter J, Coates T, Jeng M, Lai ME, Mangiagli A, Strauss G, Girot R, Watman N. Prospective evaluation of patient-reported outcomes during treatment with deferasirox or deferoxamine for iron overload in patients with β-thalassemia. Clinical Therapeutics. 2007;29(5):909-17.

Fisher R. Large-scale study in thalassemia using biomagnetic liver susceptometry. InProc. 11th Int. Conf/Biomagnetism 1998.

Olivieri NF, Brittenham GM. Final results of the randomized trial of deferiprone (L1) and deferoxamine (DFO). InBlood 1997;90(10):1161.


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