| Improved efficacy and tolerability of oral deferasirox by twice-daily dosing for patients with transfusion-dependent β-thalassemia. | |
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MedLine Citation:
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PMID: 21072825 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
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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. |
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Authors:
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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:
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Type: Journal Article; Research Support, Non-U.S. Gov't |
Journal Detail:
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Title: Pediatric blood & cancer Volume: 56 ISSN: 1545-5017 ISO Abbreviation: Pediatr Blood Cancer Publication Date: 2011 Mar |
Date Detail:
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Created Date: 2011-01-12 Completed Date: 2011-03-01 Revised Date: 2011-09-14 |
Medline Journal Info:
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Nlm Unique ID: 101186624 Medline TA: Pediatr Blood Cancer Country: United States |
Other Details:
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Languages: eng Pagination: 420-4 Citation Subset: IM |
Copyright Information:
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© 2010 Wiley-Liss, Inc. |
Affiliation:
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Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan. |
Export Citation:
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APA/MLA Format Download EndNote Download BibTex |
| MeSH Terms | |
Descriptor/Qualifier:
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Administration, Oral Adolescent Adult Benzoic Acids / administration & dosage* Blood Transfusion / adverse effects* Child Drug Administration Schedule Female Ferritins / blood Humans Iron Chelating Agents / administration & dosage* Iron Overload / blood, chemically induced, drug therapy* Male Maximum Tolerated Dose Retrospective Studies Treatment Outcome Triazoles / administration & dosage* Young Adult beta-Thalassemia / blood, drug therapy* |
| Chemical | |
Reg. No./Substance:
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0/Benzoic Acids; 0/Iron Chelating Agents; 0/Triazoles; 0/deferasirox; 9007-73-2/Ferritins |
| Comments/Corrections | |
Comment In:
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Expert Rev Hematol. 2011 Aug;4(4):411-4
[PMID:
21801132
]
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From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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