title: Follow-up report on the 2-year cardiac data from a deferasirox monotherapy trial.
abstract: The trial CICL670AUS04 was a single-arm, open-label study of the cardiac efficacy of 18 months of deferasirox monotherapy [1]. Cardiac response in this study was related to the degree of liver siderosis. Patients with mild to moderate liver siderosis improved their cardiac T2* while more severely siderotic patients did not, regardless of initial cardiac iron burden. In this letter, we report 2-year data in those patients who completed a 6-month extension phase (N 5 10). Cardiac and liver iron improved steadily during the 24-month period, with final cardiac T2* and LIC improving 37% and 27%, respectively, in this cohort. Serum ferritin and LVEF were not statistically different at anytime-point. When the extension phase (18-24 months) was considered in isolation, serum ferritin, liver iron concentration, and left ventricular ejection fraction were nearly identical to 18 month results. Despite this, cardiac T2* continued to trend higher, increasing 12.7% from 9.5 ms to 10.7 ms (P 5 0.06). Thus defersirox continued to demonstrate cardiac efficacy in patients with mild to moderate hepatic siderosis throughout 2 years of therapy.
authors.affiliation: Department of Pediatrics and Radiology, Divisions of Cardiology and Radiology, Children's Hospital of Los Angeles, CA.
authors.forename: John C
authors.initials: JC
authors.lastname: Wood
authors.forename: Tara
authors.initials: T
authors.lastname: Glynos
authors.forename: Alexis
authors.initials: A
authors.lastname: Thompson
authors.forename: Patricia
authors.initials: P
authors.lastname: Giardina
authors.forename: Paul
authors.initials: P
authors.lastname: Harmatz
authors.forename: Barinder P
authors.initials: BP
authors.lastname: Kang
authors.forename: Carole
authors.initials: C
authors.lastname: Paley
authors.forename: Thomas D
authors.initials: TD
authors.lastname: Coates
mesh_terms: D001565:Benzoates / Q000627:therapeutic use*; D009202:Cardiomyopathies / Q000188:drug therapy* / Q000209:etiology; D015913:Chelation Therapy; D000077588:Deferasirox; D005293:Ferritins / Q000032:analysis; D005500:Follow-Up Studies; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D008107:Liver Diseases / Q000188:drug therapy / Q000209:etiology; D012806:Siderosis / Q000188:drug therapy* / Q000209:etiology; D013318:Stroke Volume; D065227:Transfusion Reaction; D016896:Treatment Outcome; D014230:Triazoles / Q000627:therapeutic use*; D017086:beta-Thalassemia / Q000150:complications
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D000077588:Deferasirox