title: A 5-year follow-up in deferasirox treatment: improvement of cardiac and hepatic iron overload and amelioration in cardiac function in thalassemia major patients.
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.
authors.affiliation: Rare Diseases Center, Department of Medicine and Medical Specialities, "Ca' Granda" Ospedale Maggiore Policlinico Foundation IRCCS, Milan, Italy, elena.cassinerio@virgilio.it.
authors.forename: E
authors.initials: E
authors.lastname: Cassinerio
authors.forename: A
authors.initials: A
authors.lastname: Roghi
authors.forename: N
authors.initials: N
authors.lastname: Orofino
authors.forename: P
authors.initials: P
authors.lastname: Pedrotti
authors.forename: L
authors.initials: L
authors.lastname: Zanaboni
authors.forename: E
authors.initials: E
authors.lastname: Poggiali
authors.forename: M
authors.initials: M
authors.lastname: Giuditta
authors.forename: D
authors.initials: D
authors.lastname: Consonni
authors.forename: M D
authors.initials: MD
authors.lastname: Cappellini
mesh_terms: D000328:Adult; D001565:Benzoates / Q000494:pharmacology / Q000627:therapeutic use*; D000077588:Deferasirox; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007502:Iron Chelating Agents / Q000494:pharmacology / Q000627:therapeutic use*; D019190:Iron Overload / Q000175:diagnosis / Q000188:drug therapy* / Q000378:metabolism; D008099:Liver / Q000187:drug effects / Q000378:metabolism*; D008297:Male; D009206:Myocardium / Q000378:metabolism*; D016896:Treatment Outcome; D014230:Triazoles / Q000494:pharmacology / Q000627:therapeutic use*; D017086:beta-Thalassemia / Q000175:diagnosis / Q000188:drug therapy* / Q000378:metabolism
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D000077588:Deferasirox