Improved efficacy and tolerability of oral deferasirox by twice-daily dosing for patients with transfusion-dependent β-thalassemia.

Abstract

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.

• Authors: 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
• Journal: Pediatric blood & cancer (Pediatr Blood Cancer) Vol. 56 Issue 3 Pg. 420-4 (Mar 2011) ISSN: 1545-5017 [Electronic] United States
• PMID: 21072825 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2010 Wiley-Liss, Inc.

Chemical References

• Benzoates
• Iron Chelating Agents
• Triazoles
• Ferritins
• Deferasirox

Topics

• Administration, Oral
• Adolescent
• Adult
• Benzoates (administration & dosage)
• Child
• Deferasirox
• 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
• Transfusion Reaction
• Treatment Outcome
• Triazoles (administration & dosage)
• Young Adult
• beta-Thalassemia (blood, drug therapy)