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Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients
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Zeitschriftentitel: | American Journal of Hematology |
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Personen und Körperschaften: | , , , , , , , , , , , , , |
In: | American Journal of Hematology, 88, 2013, 6, S. 503-506 |
Format: | E-Article |
Sprache: | Englisch |
veröffentlicht: |
Wiley
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Schlagwörter: |
author_facet |
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 Zhang, Yiyun Cappellini, M. Domenica 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 Zhang, Yiyun Cappellini, M. Domenica |
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author |
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 Zhang, Yiyun Cappellini, M. Domenica |
spellingShingle |
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 Zhang, Yiyun Cappellini, M. Domenica American Journal of Hematology Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients Hematology |
author_sort |
taher, ali t. |
spelling |
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 Zhang, Yiyun Cappellini, M. Domenica 0361-8609 1096-8652 Wiley Hematology http://dx.doi.org/10.1002/ajh.23445 <jats:p>The 1‐year THALASSA study enrolled 166 patients with various non‐transfusion‐dependent thalassemia (NTDT) syndromes, degrees of iron burden and patient characteristics, and demonstrated the overall efficacy and safety of deferasirox in reducing liver iron concentration (LIC) in these patients. Here, reduction in LIC with deferasirox 5 and 10 mg/kg/day starting dose groups is shown to be consistent across the following patient subgroups—baseline LIC/serum ferritin, age, gender, race, splenectomy (yes/no), and underlying NTDT syndrome (β‐thalassemia intermedia, HbE/β‐thalassemia or α‐thalassemia). These analyses also evaluated deferasirox dosing strategies for patients with NTDT. Greater reductions in LIC were achieved in patients dose‐escalated at Week 24 from deferasirox 10 mg/kg/day starting dose to 20 mg/kg/day. Patients who received an average actual dose of deferasirox >12.5–≤17.5 mg/kg/day achieved a greater LIC decrease compared with the ≥7.5–≤12.5 mg/kg/day and >0–<7.5 mg/kg/day subgroups, demonstrating a dose–response efficacy. LIC reduction across patient subgroups was generally consistent with the primary efficacy analysis with a similar safety profile. Am. J. Hematol. 88:503–506, 2013. © 2013 Wiley Periodicals, Inc.</jats:p> Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients American Journal of Hematology |
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title |
Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
title_unstemmed |
Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
title_full |
Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
title_fullStr |
Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
title_full_unstemmed |
Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
title_short |
Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
title_sort |
deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
topic |
Hematology |
url |
http://dx.doi.org/10.1002/ajh.23445 |
publishDate |
2013 |
physical |
503-506 |
description |
<jats:p>The 1‐year THALASSA study enrolled 166 patients with various non‐transfusion‐dependent thalassemia (NTDT) syndromes, degrees of iron burden and patient characteristics, and demonstrated the overall efficacy and safety of deferasirox in reducing liver iron concentration (LIC) in these patients. Here, reduction in LIC with deferasirox 5 and 10 mg/kg/day starting dose groups is shown to be consistent across the following patient subgroups—baseline LIC/serum ferritin, age, gender, race, splenectomy (yes/no), and underlying NTDT syndrome (β‐thalassemia intermedia, HbE/β‐thalassemia or α‐thalassemia). These analyses also evaluated deferasirox dosing strategies for patients with NTDT. Greater reductions in LIC were achieved in patients dose‐escalated at Week 24 from deferasirox 10 mg/kg/day starting dose to 20 mg/kg/day. Patients who received an average actual dose of deferasirox >12.5–≤17.5 mg/kg/day achieved a greater LIC decrease compared with the ≥7.5–≤12.5 mg/kg/day and >0–<7.5 mg/kg/day subgroups, demonstrating a dose–response efficacy. LIC reduction across patient subgroups was generally consistent with the primary efficacy analysis with a similar safety profile. Am. J. Hematol. 88:503–506, 2013. © 2013 Wiley Periodicals, Inc.</jats:p> |
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author | 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, Zhang, Yiyun, Cappellini, M. Domenica |
author_facet | 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, Zhang, Yiyun, Cappellini, M. Domenica, 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, Zhang, Yiyun, Cappellini, M. Domenica |
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description | <jats:p>The 1‐year THALASSA study enrolled 166 patients with various non‐transfusion‐dependent thalassemia (NTDT) syndromes, degrees of iron burden and patient characteristics, and demonstrated the overall efficacy and safety of deferasirox in reducing liver iron concentration (LIC) in these patients. Here, reduction in LIC with deferasirox 5 and 10 mg/kg/day starting dose groups is shown to be consistent across the following patient subgroups—baseline LIC/serum ferritin, age, gender, race, splenectomy (yes/no), and underlying NTDT syndrome (β‐thalassemia intermedia, HbE/β‐thalassemia or α‐thalassemia). These analyses also evaluated deferasirox dosing strategies for patients with NTDT. Greater reductions in LIC were achieved in patients dose‐escalated at Week 24 from deferasirox 10 mg/kg/day starting dose to 20 mg/kg/day. Patients who received an average actual dose of deferasirox >12.5–≤17.5 mg/kg/day achieved a greater LIC decrease compared with the ≥7.5–≤12.5 mg/kg/day and >0–<7.5 mg/kg/day subgroups, demonstrating a dose–response efficacy. LIC reduction across patient subgroups was generally consistent with the primary efficacy analysis with a similar safety profile. Am. J. Hematol. 88:503–506, 2013. © 2013 Wiley Periodicals, Inc.</jats:p> |
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spelling | 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 Zhang, Yiyun Cappellini, M. Domenica 0361-8609 1096-8652 Wiley Hematology http://dx.doi.org/10.1002/ajh.23445 <jats:p>The 1‐year THALASSA study enrolled 166 patients with various non‐transfusion‐dependent thalassemia (NTDT) syndromes, degrees of iron burden and patient characteristics, and demonstrated the overall efficacy and safety of deferasirox in reducing liver iron concentration (LIC) in these patients. Here, reduction in LIC with deferasirox 5 and 10 mg/kg/day starting dose groups is shown to be consistent across the following patient subgroups—baseline LIC/serum ferritin, age, gender, race, splenectomy (yes/no), and underlying NTDT syndrome (β‐thalassemia intermedia, HbE/β‐thalassemia or α‐thalassemia). These analyses also evaluated deferasirox dosing strategies for patients with NTDT. Greater reductions in LIC were achieved in patients dose‐escalated at Week 24 from deferasirox 10 mg/kg/day starting dose to 20 mg/kg/day. Patients who received an average actual dose of deferasirox >12.5–≤17.5 mg/kg/day achieved a greater LIC decrease compared with the ≥7.5–≤12.5 mg/kg/day and >0–<7.5 mg/kg/day subgroups, demonstrating a dose–response efficacy. LIC reduction across patient subgroups was generally consistent with the primary efficacy analysis with a similar safety profile. Am. J. Hematol. 88:503–506, 2013. © 2013 Wiley Periodicals, Inc.</jats:p> Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients American Journal of Hematology |
spellingShingle | 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, Zhang, Yiyun, Cappellini, M. Domenica, American Journal of Hematology, Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients, Hematology |
title | Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
title_full | Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
title_fullStr | Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
title_full_unstemmed | Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
title_short | Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
title_sort | deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
title_unstemmed | Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients |
topic | Hematology |
url | http://dx.doi.org/10.1002/ajh.23445 |