Document Detail

Improved efficacy and tolerability of oral deferasirox by twice-daily dosing for patients with transfusion-dependent β-thalassemia.
MedLine Citation:
PMID:  21072825     Owner:  NLM     Status:  MEDLINE    
BACKGROUND: Deferasirox is an oral iron-chelating agent taken once-daily by patients with transfusion-dependent iron overload. However, some patients are unresponsive or unable to tolerate once-daily deferasirox. The current study evaluated whether twice-daily deferasirox treatment showed increased efficacy or tolerability in unresponsive or intolerant patients.
PROCEDURE: Patients from two Taiwanese hospitals with transfusion-dependent β-thalassemia, including those who showed increasing serum ferritin levels for six consecutive months, with at least one level >2,500 ng/dl, while treated with >30 mg/kg/day of once-daily deferasirox (unresponsive) or developed deferasirox-related adverse events (AEs) at the dosage required to maintain the iron burden balance (intolerant) and were treated twice-daily with the same total daily dose of deferasirox since 2008, were enrolled in the study and evaluated retrospectively by medical record review.
RESULTS: Eighteen patients were included for analysis. A statistically significant median decrease in serum ferritin levels was detected in the 11 unresponsive patients after 6 months of continuous twice-daily deferasirox treatment. Five out of the seven intolerant patients experienced either no deferasirox-related AEs or less severe AEs. The 12 patients from both groups (11 unresponsive, 1 intolerant) who received continuous twice-daily deferasirox for 6 months showed a mild but significant median increase in serum creatinine levels.
CONCLUSIONS: Twice-daily deferasirox dosing is effective in iron chelation and improves tolerability in transfusion-dependent β-thalassemia patients who are unresponsive to or intolerant of once-daily deferasirox. Future studies with greater patient numbers will be required to confirm the results reported herein.
Hsiu-Hao Chang; Meng-Yao Lu; Yu-Mei Liao; Pei-Chin Lin; Yung-Li Yang; Dong-Tsamn Lin; Shyh-Shin Chiou; Shiann-Tarng Jou; Kai-Hsin Lin; Tai-Tsung Chang
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Publication Detail:
Type:  Journal Article; Research Support, Non-U.S. Gov't    
Journal Detail:
Title:  Pediatric blood & cancer     Volume:  56     ISSN:  1545-5017     ISO Abbreviation:  Pediatr Blood Cancer     Publication Date:  2011 Mar 
Date Detail:
Created Date:  2011-01-12     Completed Date:  2011-03-01     Revised Date:  2013-06-03    
Medline Journal Info:
Nlm Unique ID:  101186624     Medline TA:  Pediatr Blood Cancer     Country:  United States    
Other Details:
Languages:  eng     Pagination:  420-4     Citation Subset:  IM    
Copyright Information:
© 2010 Wiley-Liss, Inc.
Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan.
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MeSH Terms
Administration, Oral
Benzoates / administration & dosage*
Blood Transfusion / adverse effects*
Drug Administration Schedule
Ferritins / blood
Iron Chelating Agents / administration & dosage*
Iron Overload / blood,  chemically induced,  drug therapy*
Maximum Tolerated Dose
Retrospective Studies
Treatment Outcome
Triazoles / administration & dosage*
Young Adult
beta-Thalassemia / blood,  drug therapy*
Reg. No./Substance:
0/Benzoates; 0/Iron Chelating Agents; 0/Triazoles; 9007-73-2/Ferritins; V8G4MOF2V9/deferasirox
Comment In:
Expert Rev Hematol. 2011 Aug;4(4):411-4   [PMID:  21801132 ]

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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