title: Achieving treatment goals of reducing or maintaining body iron burden with deferasirox in patients with β-thalassaemia: results from the ESCALATOR study.
abstract: This analysis evaluated the effects of deferasirox on liver iron concentration in moderate and heavily iron-overloaded patients with β-thalassaemia from the ESCALATOR trial (n = 231). Mean liver iron concentrations (LIC) decreased significantly from 21.1 ± 8.2 to 14.2 ± 12.1 mg Fe/g dry weight (dw) at 2 yr (P < 0.001) in patients with LIC ≥ 7 mg Fe/g dw at baseline; patients with LIC < 7 mg Fe/g dw maintained these levels over the treatment period. The proportion of patients with LIC < 7 mg Fe/g dw increased from 9.4% at core baseline to 39.3% by the end of year 2. The results showed that deferasirox enabled therapeutic goals to be achieved, by maintaining LIC in patients with LIC < 7 mg Fe/g dw at a mean dose of 22.4 ± 5.2 mg/kg/d and significantly reducing LIC in patients with LIC ≥ 7 mg Fe/g dw at a mean dose of 25.7 ± 4.2 mg/kg/d, along with a manageable safety profile.
authors.affiliation: American University of Beirut Medical Center, Beirut, Lebanon. ataher@aub.edu.lb
authors.forename: Ali
authors.initials: A
authors.lastname: Taher
authors.forename: Mohsen S
authors.initials: MS
authors.lastname: Elalfy
authors.forename: Kusai
authors.initials: K
authors.lastname: Al Zir
authors.forename: Shahina
authors.initials: S
authors.lastname: Daar
authors.forename: Abdullah
authors.initials: A
authors.lastname: Al Jefri
authors.forename: Dany
authors.initials: D
authors.lastname: Habr
authors.forename: Ulrike
authors.initials: U
authors.lastname: Kriemler-Krahn
authors.forename: Bernard
authors.initials: B
authors.lastname: Roubert
authors.forename: Amal
authors.initials: A
authors.lastname: El-Beshlawy
mesh_terms: D000293:Adolescent; D001565:Benzoates / Q000627:therapeutic use*; D001822:Body Burden; D002648:Child; D002675:Child, Preschool; D000077588:Deferasirox; D005260:Female; D006801:Humans; D007501:Iron / Q000378:metabolism*; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D008297:Male; D011446:Prospective Studies; D014230:Triazoles / Q000627:therapeutic use*; D017086:beta-Thalassemia / Q000188:drug therapy*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D007501:Iron; D000077588:Deferasirox