title: Five Years of Deferasirox Therapy for Cardiac Iron in β-Thalassemia Major.
abstract: Myocardial siderosis in β-thalassemia major (β-TM) remains the leading cause of death. Deferasirox (DFX), a new iron chelation treatment, has proved to be effective in reducing or preventing cardiac iron burden in thalassemic patients according to clinical trials with maximum duration of up to 3 years except one that was recently published and lasted 5 years. The aim of this study was to evaluate the efficacy of DFX in reducing or preventing cardiac iron burden in 23 patients with β-TM after 5 years of therapy. All patients had a magnetic resonance imaging (MRI) T2* evaluation of their cardiac iron load before starting DFX therapy and after a period of 5 years. Ferritin levels and left ventricular ejection fraction (LVEF) were also evaluated at the same time. Deferasirox was administered in a starting dose of 30 mg/kg/day and never increased to more than 40 mg/kg/day. The MRI T2* cardiac iron load mean values before DFX was 32.82 ± 10.86 ms, and after 32.13 ± 7.74 ms, showing a stability in MRI T2* myocardial value but a significant improvement in two patients with an intermediate iron load (12 vs. 23 ms). The mean LVEF value was 68.43 ± 7.08% before treatment with DFX and 67.95 ± 5.94% after DFX therapy without significant change. Our results confirm previous studies that DFX is considered an effective chelating agent used as monotherapy for at least 5 years and is more efficacious in moderate to severe cardiac iron loaded thalassemic patients.
authors.affiliation: a Adult Thalassemia Unit, 2nd Department of Internal Medicine , Aristotle University, Hippokration Hospital , Thessaloniki , Greece.
authors.forename: Efthymia
authors.initials: E
authors.lastname: Vlachaki
authors.forename: Alexandra
authors.initials: A
authors.lastname: Agapidou
authors.forename: Georgios
authors.initials: G
authors.lastname: Spanos
authors.forename: Philippos
authors.initials: P
authors.lastname: Klonizakis
authors.forename: Evaggelia
authors.initials: E
authors.lastname: Vetsiou
authors.forename: Melahrini
authors.initials: M
authors.lastname: Mavroudi
authors.forename: Panagiota
authors.initials: P
authors.lastname: Boura
mesh_terms: D000328:Adult; D001565:Benzoates / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D009202:Cardiomyopathies / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology* / Q000378:metabolism; D000077588:Deferasirox; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007501:Iron / Q000378:metabolism; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000150:complications* / Q000209:etiology* / Q000378:metabolism; D008279:Magnetic Resonance Imaging; D008297:Male; D008875:Middle Aged; D013318:Stroke Volume; D016896:Treatment Outcome; D014230:Triazoles / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016277:Ventricular Function, Left; D055815:Young Adult; D017086:beta-Thalassemia / Q000150:complications*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D007501:Iron; D000077588:Deferasirox
keywords: 5 years; cardiac magnetic resonance imaging (MRI) T2*; deferasirox (DFX); ferritin; left ventricular ejection fraction (LVEF); β-Thalassemia major (β-TM)