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: Ali T Taher, John B Porter, Vip Viprakasit, Antonis Kattamis, Suporn Chuncharunee, Pranee Sutcharitchan, Noppadol Siritanaratkul, Renzo Galanello, Zeynep Karakas, Tomasz Lawniczek, Dany Habr, Jacqueline Ros, Zewen Zhu, M Domenica Cappellini
• Journal: Annals of hematology (Ann Hematol) Vol. 92 Issue 11 Pg. 1485-93 (Nov 2013) ISSN: 1432-0584 [Electronic] Germany
• PMID: 23775581 (Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)

Chemical References

• Benzoates
• Iron Chelating Agents
• Triazoles
• Deferasirox

Topics

• Benzoates (therapeutic use)
• Blood Transfusion
• Cross-Over Studies
• Deferasirox
• Double-Blind Method
• Humans
• Iron Chelating Agents (therapeutic use)
• Iron Overload (blood, drug therapy, epidemiology)
• Prospective Studies
• Thalassemia (blood, drug therapy, epidemiology)
• Time Factors
• Treatment Outcome
• Triazoles (therapeutic use)