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.Electronic supplementary materialThe online version of this article (doi:10.1007/s00277-013-1808-z) contains supplementary material, which is available to authorized users.

Deferiprone versus deferoxamine in thalassemia intermedia: Results from a 5‐year long‐term Italian multicenter randomized clinical trial

“…These findings are consistent with the LIC reduction observed in this cohort, which was the greatest in the patients with the highest pre-treatment LIC [5,6].…”

“…DFX has been shown to effectively reduce iron overload in TI patients in both a 1-year prospective, randomized, double-blind, placebo-controlled trial and a 1-year extension of the THALASSA study [5,6]. These findings are consistent with the LIC reduction observed in this cohort, which was the greatest in the patients with the highest pre-treatment LIC [5,6].…”

“…Although the 1-year data from the phase 2, prospective, randomized, double-blind, placebo-controlled trial and the 1-year extension results from the THALASSA study assessing the efficacy and safety of deferasirox (DFX) in TI patients have been published, no data have been reported regarding deferiprone (DFP) treatment [5,6]. Moreover, no randomized clinical trials comparing oral chelation with deferoxamine (DFO) treatment in patients with TI have been reported.…”

Iron Deficiency and Anaemia in Pregnant Women in Malaysia ? Still a Significant and Challenging Health Problem

“…Iron overload may cause dysfunction of the organs, especially the heart, and necessitate treatment with iron chelators [56]. In this category of patients, implementation of pregnancy is a complicated and controversial issue and iron supplements are contraindicated.…”

Efficacy and safety of deferasirox in non-thalassemic patients with elevated ferritin levels after allogeneic hematopoietic stem cell transplantation

“…[22][23][24][25][26][27][28] However, data regarding the use of deferasirox after allogeneic HSCT are limited. 15,17,19,20 This prospective multicenter trial is, to the best of our knowledge, the largest trial evaluating the efficacy and safety of deferasirox after allogeneic HSCT.…”