The effect of desferrioxamine chelation versus no therapy in patients with non transfusion-dependent thalassaemia: a multicenter prospective comparison from the MIOT network
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We prospectively assessed by magnetic resonance imaging (MRI) the advantages of desferrioxamine (DFO) with respect to the absence of chelation therapy in non transfusion-dependent thalassaemia (NTDT) patients. We considered 18 patients non-chelated and 33 patients who received DFO alone between the two MRI scans. Iron overload was assessed by the T2* technique. Biventricular function parameters were quantified by cine sequences. No patient treated with DFO had cardiac iron. At baseline, only one non-chelated patient showed a pathological heart T2* value (< 20 ms) and he recovered at the follow-up. The percentage of patients who maintained a normal heart T2* value was 100% in both groups. A significant increase in the right ventricular ejection fraction was detected in DFO patients (3.48 ± 7.22%; P = 0.024). The changes in cardiac T2* values and in the biventricular function were comparable between the two groups. In patients with hepatic iron at baseline (MRI liver iron concentration (LIC) ≥ 3 mg/g/dw), the reduction in MRI LIC values was significant only in the DFO group (− 2.20 ± 4.84 mg/g/dw; P = 0.050). The decrease in MRI LIC was comparable between the groups. In conclusion, in NTDT patients, DFO therapy showed no advantage in terms of cardiac iron but its administration allowed an improvement in right ventricular function. Moreover, DFO reduced hepatic iron in patients with significant iron burden at baseline.
KeywordsDesferrioxamine Non transfusion-dependent thalassaemia Magnetic resonance imaging Iron overload
We would like to thank all the colleagues involved in the MIOT project (https://miot.ftgm.it/).
We thank Claudia Santarlasci for her skillful secretarial work. We finally thank all patients for their cooperation.
The MIOT project receives “no-profit support” from industrial sponsorships (Chiesi Farmaceutici S.p.A. and ApoPharma Inc.).
Compliance with ethical standards
Conflict of interest
The authors declare that they have no conflict of interest.
All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.
Informed consent was obtained from all individual participants included in the study.
- 1.Musallam KM, Cappellini MD, Daar S, Karimi M, El-Beshlawy A, Graziadei G, Magestro M, Wulff J, Pietri G, Taher AT (2014) Serum ferritin level and morbidity risk in transfusion-independent patients with beta-thalassemia intermedia: the ORIENT study. Haematologica 99(11):e218–e221CrossRefGoogle Scholar
- 3.Taher AT, Porter J, Viprakasit V, Kattamis A, Chuncharunee S, Sutcharitchan P, Siritanaratkul N, Galanello R, Karakas Z, Lawniczek T, Ros J, Zhang Y, Habr D, Cappellini MD (2012) Deferasirox reduces iron overload significantly in nontransfusion-dependent thalassemia: 1-year results from a prospective, randomized, double-blind, placebo-controlled study. Blood 120(5):970–977CrossRefGoogle Scholar
- 4.Taher AT, Porter JB, Viprakasit V, Kattamis A, Chuncharunee S, Sutcharitchan P, Siritanaratkul N, Galanello R, Karakas Z, Lawniczek T, Habr D, Ros J, Zhu Z, Cappellini MD (2013) Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients: 1-year extension results from the THALASSA study. Ann Hematol 92(11):1485–1493CrossRefGoogle Scholar
- 8.Calvaruso G, Vitrano A, Di Maggio R, Lai E, Colletta G, Quota A, Gerardi C, Rigoli LC, Sacco M, Pitrolo L, Maggio A (2015) Deferiprone versus deferoxamine in thalassemia intermedia: results from a 5-year long-term Italian multicenter randomized clinical trial. Am J Hematol 90(7):634–638CrossRefGoogle Scholar
- 9.Meloni A, Ramazzotti A, Positano V, Salvatori C, Mangione M, Marcheschi P, Favilli B, De Marchi D, Prato S, Pepe A, Sallustio G, Centra M, Santarelli MF, Lombardi M, Landini L (2009) Evaluation of a web-based network for reproducible T2* MRI assessment of iron overload in thalassemia. Int J Med Inform 78(8):503–512CrossRefGoogle Scholar
- 10.Ramazzotti A, Pepe A, Positano V, Rossi G, De Marchi D, Brizi MG, Luciani A, Midiri M, Sallustio G, Valeri G, Caruso V, Centra M, Cianciulli P, De Sanctis V, Maggio A, Lombardi M (2009) Multicenter validation of the magnetic resonance t2* technique for segmental and global quantification of myocardial iron. J Magn Reson Imaging 30(1):62–68CrossRefGoogle Scholar
- 11.Pepe A, Positano V, Santarelli F, Sorrentino F, Cracolici E, De Marchi D, Maggio A, Midiri M, Landini L, Lombardi M (2006) Multislice multiecho T2* cardiovascular magnetic resonance for detection of the heterogeneous distribution of myocardial iron overload. J Magn Reson Imaging 23(5):662–668CrossRefGoogle Scholar
- 14.Meloni A, Positano V, Pepe A, Rossi G, Dell'Amico M, Salvatori C, Keilberg P, Filosa A, Sallustio G, Midiri M, D'Ascola D, Santarelli MF, Lombardi M (2010) Preferential patterns of myocardial iron overload by multislice multiecho T*2 CMR in thalassemia major patients. Magn Reson Med 64(1):211–219CrossRefGoogle Scholar
- 16.Cerqueira MD, Weissman NJ, Dilsizian V, Jacobs AK, Kaul S, Laskey WK, Pennell DJ, Rumberger JA, Ryan T, Verani MS (2002) Standardized myocardial segmentation and nomenclature for tomographic imaging of the heart: a statement for healthcare professionals from the Cardiac Imaging Committee of the Council on Clinical Cardiology of the American Heart Association. Circulation 105(4):539–542CrossRefGoogle Scholar
- 17.Meloni A, Luciani A, Positano V, De Marchi D, Valeri G, Restaino G, Cracolici E, Caruso V, Dell'amico MC, Favilli B, Lombardi M, Pepe A (2011) Single region of interest versus multislice T2* MRI approach for the quantification of hepatic iron overload. J Magn Reson Imaging 33(2):348–355CrossRefGoogle Scholar
- 21.Aquaro GD, Camastra G, Monti L, Lombardi M, Pepe A, Castelletti S, Maestrini V, Todiere G, Masci P, di Giovine G, Barison A, Dellegrottaglie S, Perazzolo Marra M, Pontone G, Di Bella G (2016) Reference values of cardiac volumes, dimensions, and new functional parameters by MR: a multicenter, multivendor study. J Magn Reson Imaging 45(4):1055–1067CrossRefGoogle Scholar
- 22.Marsella M, Borgna-Pignatti C, Meloni A, Caldarelli V, Dell'Amico MC, Spasiano A, Pitrolo L, Cracolici E, Valeri G, Positano V, Lombardi M, Pepe A (2011) Cardiac iron and cardiac disease in males and females with transfusion-dependent thalassemia major: a T2* magnetic resonance imaging study. Haematologica 96(4):515–520CrossRefGoogle Scholar
- 23.Taher A, Vichinsky E, Musallam K, Cappellini MD, Viprakasit V (2013) Guidelines for the management of non transfusion dependent thalassaemia (NTDT) [Internet]. Thalassaemia International Federation, Nicosia, CyprusGoogle Scholar
- 24.Taher AT, Porter JB, Viprakasit V, Kattamis A, Chuncharunee S, Sutcharitchan P, Siritanaratkul N, Origa R, Karakas Z, Habr D, Zhu Z, Cappellini MD (2014) Defining serum ferritin thresholds to predict clinically relevant liver iron concentrations for guiding deferasirox therapy when MRI is unavailable in patients with non-transfusion-dependent thalassaemia. Br J Haematol 168(2):284–290CrossRefGoogle Scholar
- 26.Karimi M, Amirmoezi F, Haghpanah S, Ostad S, Lotfi M, Sefidbakht S, Rezaian S (2017) Correlation of serum ferritin levels with hepatic MRI T2 and liver iron concentration in nontransfusion beta-thalassemia intermediate patients: a contemporary issue. Pediatr Hematol Oncol 34(5):292–297CrossRefGoogle Scholar
- 29.Taher AT, Musallam KM, Karimi M, El-Beshlawy A, Belhoul K, Daar S, Saned MS, El-Chafic AH, Fasulo MR, Cappellini MD (2010) Overview on practices in thalassemia intermedia management aiming for lowering complication rates across a region of endemicity: the OPTIMAL CARE study. Blood 115(10):1886–1892CrossRefGoogle Scholar
- 31.Pepe A, Rossi G, Bentley A, Putti MC, Frizziero L, D'Ascola DG, Cuccia L, Spasiano A, Filosa A, Caruso V, Hanif A, Meloni A (2017) 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 37(5):453–464CrossRefGoogle Scholar
- 32.Taher AT, Porter JB, Viprakasit V, Kattamis A, Chuncharunee S, Sutcharitchan P, Siritanaratkul N, Origa R, Karakas Z, Habr D, Zhu Z, Cappellini MD (2014) Approaching low liver iron burden in chelated patients with non-transfusion-dependent thalassemia: the safety profile of deferasirox. Eur J Haematol 92(6):521–526CrossRefGoogle Scholar