Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients: 1-year extension results from the THALASSA study

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

doi:10.1007/s00277-013-1808-z

Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients: 1-year extension results from the THALASSA study

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

Fondazione IRCCS Ca' Granda- Ospedale Maggiore Policlinico

Research output: Contribution to journal › Article › peer-review

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

Original language	English
Pages (from-to)	1485-1493
Number of pages	9
Journal	Annals of Hematology
Volume	92
Issue number	11
DOIs	https://doi.org/10.1007/s00277-013-1808-z
Publication status	Published - Nov 2013

Keywords

Deferasirox
Iron chelation
Iron overload
Non-transfusion-dependent thalassemia

ASJC Scopus subject areas

Hematology
Medicine(all)

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Taher, A. T., Porter, J. B., Viprakasit, V., Kattamis, A., Chuncharunee, S., Sutcharitchan, P., Siritanaratkul, N., Galanello, R., Karakas, Z., Lawniczek, T., Habr, D., Ros, J., Zhu, Z., & Cappellini, M. D. (2013). Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients: 1-year extension results from the THALASSA study. Annals of Hematology, 92(11), 1485-1493. https://doi.org/10.1007/s00277-013-1808-z

Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients : 1-year extension results from the THALASSA study. / Taher, Ali T.; Porter, John B.; Viprakasit, Vip; Kattamis, Antonis; Chuncharunee, Suporn; Sutcharitchan, Pranee; Siritanaratkul, Noppadol; Galanello, Renzo; Karakas, Zeynep; Lawniczek, Tomasz; Habr, Dany; Ros, Jacqueline; Zhu, Zewen; Cappellini, M. Domenica.

In: Annals of Hematology, Vol. 92, No. 11, 11.2013, p. 1485-1493.

Research output: Contribution to journal › Article › peer-review

Taher, AT, Porter, JB, Viprakasit, V, Kattamis, A, Chuncharunee, S, Sutcharitchan, P, Siritanaratkul, N, Galanello, R, Karakas, Z, Lawniczek, T, Habr, D, Ros, J, Zhu, Z & Cappellini, MD 2013, 'Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients: 1-year extension results from the THALASSA study', Annals of Hematology, vol. 92, no. 11, pp. 1485-1493. https://doi.org/10.1007/s00277-013-1808-z

Taher, Ali T. ; Porter, John B. ; Viprakasit, Vip ; Kattamis, Antonis ; Chuncharunee, Suporn ; Sutcharitchan, Pranee ; Siritanaratkul, Noppadol ; Galanello, Renzo ; Karakas, Zeynep ; Lawniczek, Tomasz ; Habr, Dany ; Ros, Jacqueline ; Zhu, Zewen ; Cappellini, M. Domenica. / Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients : 1-year extension results from the THALASSA study. In: Annals of Hematology. 2013 ; Vol. 92, No. 11. pp. 1485-1493.

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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 ",

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