TY - JOUR
T1 - Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients
T2 - 1-year extension results from the THALASSA study
AU - Taher, Ali T.
AU - Porter, John B.
AU - Viprakasit, Vip
AU - Kattamis, Antonis
AU - Chuncharunee, Suporn
AU - Sutcharitchan, Pranee
AU - Siritanaratkul, Noppadol
AU - Galanello, Renzo
AU - Karakas, Zeynep
AU - Lawniczek, Tomasz
AU - Habr, Dany
AU - Ros, Jacqueline
AU - Zhu, Zewen
AU - Cappellini, M. Domenica
PY - 2013/11
Y1 - 2013/11
N2 - 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
AB - 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
KW - Deferasirox
KW - Iron chelation
KW - Iron overload
KW - Non-transfusion-dependent thalassemia
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UR - http://www.scopus.com/inward/citedby.url?scp=84885662128&partnerID=8YFLogxK
U2 - 10.1007/s00277-013-1808-z
DO - 10.1007/s00277-013-1808-z
M3 - Article
C2 - 23775581
AN - SCOPUS:84885662128
VL - 92
SP - 1485
EP - 1493
JO - Revue d'hématologie
JF - Revue d'hématologie
SN - 0939-5555
IS - 11
ER -