title: Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients: 1-year extension results from the THALASSA study.
abstract: Patients with non-transfusion-dependent thalassemia (NTDT) often develop iron overload that requires chelation to levels below the threshold associated with complications. This can take several years in patients with high iron burden, highlighting the value of long-term chelation data. Here, we report the 1-year extension of the THALASSA trial assessing deferasirox in NTDT; patients continued with deferasirox or crossed from placebo to deferasirox. Of 133 patients entering extension, 130 completed. Liver iron concentration (LIC) continued to decrease with deferasirox over 2 years; mean change was -7.14 mg Fe/g dry weight (dw) (mean dose 9.8 ± 3.6 mg/kg/day). In patients originally randomized to placebo, whose LIC had increased by the end of the core study, LIC decreased in the extension with deferasirox with a mean change of -6.66 mg Fe/g dw (baseline to month 24; mean dose in extension 13.7 ± 4.6 mg/kg/day). Of 166 patients enrolled, 64 (38.6 %) and 24 (14.5 %) patients achieved LIC <5 and <3 mg Fe/g dw by the end of the study, respectively. Mean LIC reduction was greatest in patients with the highest pretreatment LIC. Deferasirox progressively decreases iron overload over 2 years in NTDT patients with both low and high LIC. Safety profile of deferasirox over 2 years was consistent with that in the core study.
authors.affiliation: American University of Beirut, Beirut, Lebanon, ataher@aub.edu.lb.
authors.forename: Ali T
authors.initials: AT
authors.lastname: Taher
authors.forename: John B
authors.initials: JB
authors.lastname: Porter
authors.forename: Vip
authors.initials: V
authors.lastname: Viprakasit
authors.forename: Antonis
authors.initials: A
authors.lastname: Kattamis
authors.forename: Suporn
authors.initials: S
authors.lastname: Chuncharunee
authors.forename: Pranee
authors.initials: P
authors.lastname: Sutcharitchan
authors.forename: Noppadol
authors.initials: N
authors.lastname: Siritanaratkul
authors.forename: Renzo
authors.initials: R
authors.lastname: Galanello
authors.forename: Zeynep
authors.initials: Z
authors.lastname: Karakas
authors.forename: Tomasz
authors.initials: T
authors.lastname: Lawniczek
authors.forename: Dany
authors.initials: D
authors.lastname: Habr
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Ros
authors.forename: Zewen
authors.initials: Z
authors.lastname: Zhu
authors.forename: M Domenica
authors.initials: MD
authors.lastname: Cappellini
mesh_terms: D001565:Benzoates / Q000627:therapeutic use*; D001803:Blood Transfusion; D018592:Cross-Over Studies; D000077588:Deferasirox; D004311:Double-Blind Method; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D019190:Iron Overload / Q000097:blood / Q000188:drug therapy* / Q000453:epidemiology; D011446:Prospective Studies; D013789:Thalassemia / Q000097:blood / Q000188:drug therapy* / Q000453:epidemiology; D013997:Time Factors; D016896:Treatment Outcome; D014230:Triazoles / Q000627:therapeutic use*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D000077588:Deferasirox