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A 5-year follow-up in deferasirox treatment: improvement of cardiac and hepatic iron overload and amelioration in cardiac function in thalassemia major patients

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Abstract

Deferasirox (DFX) is an oral iron chelator with established efficacy and safety. We evaluated by T2* cardiovascular magnetic resonance (CMR) the efficacy of DFX in preventing and removing cardiac and liver iron load and cardiac volume changes, along 5 years in adult thalassemia major (TM) patients. Twenty-three TM patients (9 males/14 women, mean age 36 ± 4 years) were included in this study. Repeated CMR was performed to assess myocardial and liver iron load (baseline t0, after 2.5 years t1, after 5 years t2). Myocardial T2* values changed progressively and increased significantly between t0 and t2 (t0: 27.15 ± 9.58 vs t2: 36.64 ± 6.68, p = 0.0001). At baseline evaluation, a cardiac T2* value <20 ms was detected in six patients (26 %): they showed an improvement of cardiac T2* values between t0 and t1, with normal T2* levels reached in all patients at t2. In the overall population, a significant reduction of both end-diastolic and end-systolic left ventricular volumes (EDV, ESV) were detected between t0 and t2 (EDV, t0: 132 ± 31 ml vs t2: 124 ± 22 ml, p = 0.033; ESV, t0: 48 ± 14 ml vs t2: 41 ± 10 ml, p = 0.0007). A significant reduction in liver iron concentration (LIC) was detected at t1 (5.36 ± 3.58 mg/g dw at baseline vs 3.35 ± 2.68 mg/g dw at t1, p = 0.004). In patients with cardiac iron overload at baseline (n.6), mean cardiac T2* values doubled at t2, and mean LIC value is reduced of 29 %. After 5 years of treatment, DFX continually and significantly reduced myocardial and liver iron overload, and it prevented further iron deposition.

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The authors declare that they have no conflict of interest.

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Authors and Affiliations

  1. Rare Diseases Center, Department of Medicine and Medical Specialities, “Ca’ Granda” Ospedale Maggiore Policlinico Foundation IRCCS, Milan, Italy

    E. Cassinerio, N. Orofino, L. Zanaboni, M. Giuditta & M. D. Cappellini

  2. CMR Unit, Department of Cardiology, Niguarda Ca’Granda Hospital, Milan, Italy

    A. Roghi & P. Pedrotti

  3. Department of Clinical Science and Community Health, University of Milan, Milan, Italy

    E. Poggiali & M. D. Cappellini

  4. Epidemiology Unit, “Ca’ Granda” Foundation IRCCS Ospedale Maggiore Policlinico, Milan, Italy

    D. Consonni

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  1. E. Cassinerio
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  9. M. D. Cappellini
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Correspondence to E. Cassinerio.

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Cassinerio, E., Roghi, A., Orofino, N. et al. A 5-year follow-up in deferasirox treatment: improvement of cardiac and hepatic iron overload and amelioration in cardiac function in thalassemia major patients. Ann Hematol 94, 939–945 (2015). https://doi.org/10.1007/s00277-014-2291-x

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  • DOI: https://doi.org/10.1007/s00277-014-2291-x

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