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

Authors

  • Hsiu-Hao Chang MD,

    1. Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan
    2. Graduate Institute of Clinical Medicine, National Taiwan University College of Medicine, Taipei, Taiwan
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  • Meng-Yao Lu MD,

    1. Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan
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  • Yu-Mei Liao MD,

    1. Department of Pediatrics, Kaohsiung Medical University Hospital, Kaoshiung, Taiwan
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  • Pei-Chin Lin MD,

    1. Department of Pediatrics, Kaohsiung Medical University Hospital, Kaoshiung, Taiwan
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  • Yung-Li Yang MD,

    1. Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan
    2. Graduate Institute of Clinical Medicine, National Taiwan University College of Medicine, Taipei, Taiwan
    3. Department of Laboratory Medicine, National Taiwan University Hospital, Taipei, Taiwan
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  • Dong-Tsamn Lin MD,

    1. Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan
    2. Department of Laboratory Medicine, National Taiwan University Hospital, Taipei, Taiwan
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  • Shyh-Shin Chiou MD,

    1. Department of Pediatrics, Kaohsiung Medical University Hospital, Kaoshiung, Taiwan
    2. Kaohsiung Medical University, Kaoshiung, Taiwan
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  • Shiann-Tarng Jou MD, PhD,

    1. Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan
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  • Kai-Hsin Lin MD,

    1. Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan
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  • Tai-Tsung Chang MD

    Corresponding author
    1. Department of Pediatrics, Kaohsiung Medical University Hospital, Kaoshiung, Taiwan
    2. Kaohsiung Medical University, Kaoshiung, Taiwan
    • Division of Hematology/Oncology, Department of Pediatrics, Kaohsiung Medical University Hospital, Kaohsiung Medical University, No.100, Tzyou 1st Rd., Kaohsiung 807, Taiwan.
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  • Conflict of interest: Nothing to declare.

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. Pediatr Blood Cancer 2011;56:420–424. © 2010 Wiley-Liss, Inc.

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