Approaching low liver iron burden in chelated patients with non-transfusion-dependent thalassemia: the safety profile of deferasirox.
Bottom Line: Mean ± SD deferasirox treatment duration up to reaching LIC<3 was 476 ± 207 d, and deferasirox dose was 9.7 ± 3.0 mg/kg/d.The exposure-adjusted AE incidence regardless of causality was similar in periods 1 (1.026) and 2 (1.012).There were no clinically relevant differences in renal and hepatic laboratory parameters measured close to the time of LIC<3 compared with measurements near the previous LIC assessment.
Affiliation: American University of Beirut, Beirut, Lebanon.Show MeSH
Related in: MedlinePlus
Mentions: Full study design and patient inclusion/exclusion criteria for the THALASSA study (NCT00873041) have been described previously 7. In brief, a prospective, randomized, placebo-controlled, double-blind, 1-yr trial was performed to evaluate deferasirox (starting dose 5 or 10 mg/kg/d) or placebo in patients with NTDT aged ≥10 yr with iron overload (LIC ≥5 mg Fe/g dw and serum ferritin >300 ng/mL). This was followed by a preplanned 1-yr extension in which all eligible patients, irrespective of initial treatment received, were treated with deferasirox. The starting dose in the extension phase was based on absolute LIC and LIC decrease by the end of the core study (Fig.1). For this post hoc analysis, patients reaching low iron burden (LIC <3; the predefined target for interruption of chelation) in the core or extension phases of the trial were included. Adverse events (AEs) and safety laboratory parameters were monitored throughout the study and the extension period. Auditory and ocular examinations were performed at baseline and at the end of the core and extension phases. Safety parameters during the period 6 months before reaching the LIC <3 target (Period 2) were compared with those during the period from baseline up to 6 months before reaching LIC <3 (Period 1; Fig.2).