International Journal of Hematology

DOI: 10.1007/s12185-017-2396-9 Pages: 1-8

Deferasirox for the treatment of iron overload after allogeneic hematopoietic cell transplantation: multicenter phase I study (KSGCT1302)

1. Yokohama City University Graduate School of Medicine, Department of Hematology and Clinical Immunology

2. Jichi Medical University, Division of Hematology, Saitama Medical Center

3. Tokai University School of Medicine, Department of Hematology and Oncology

4. Jikei University School of Medicine, Division of Clinical Oncology and Hematology, Department of Internal Medicine

5. Yokohama City University Medical Center, Department of Hematology

6. Komagome Hospital, Hematology Division, Tokyo Metropolitan Cancer and Infectious Diseases Center

7. Chiba University Hospital, Department of Hematology

8. Kyoto University Graduate School of Medicine, Department of Biomedical Statistics and Bioinformatics

9. Kanagawa Cancer Center

10. Keio University School of Medicine, Division of Hematology, Department of Medicine

Correspondence to:
Takayoshi Tachibana
Tel: 81-45-520-2222
Email: tcbn@kcch.jp

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Abstract

Abstract

The aim of this study was to assess the safety and optimal dose of deferasirox for the treatment of iron overload after allogeneic hematopoietic cell transplantation (HCT). The primary endpoint was the maximum tolerated dose of deferasirox that was determined by the intrapatient dose escalation methods. A total of 16 patients with post-HCT iron overload were enrolled in the study. After excluding one case of early relapse, 15 remained evaluable. Their median age was 42 years (range 22–68). Median time from HCT to deferasirox administration was 9 months (range 6–84). Deferasirox was started at a dose of 5 mg/kg, and the dose was increased to 7.5 and 10 mg/kg every 4 weeks unless there were no grade ≥ 2 of adverse events. Achievement rates of planned medication were 80% in 5 mg/kg (12 of 15), 73% in 7.5 mg/kg (11 of 15), and 60% in 10 mg/kg (9 of 15), respectively. The reasons for discontinuation of the drug were grade 2 of adverse events (n = 4), late relapse (n = 1), and self-cessation (n = 1). None of the patients developed grade ≥ 3 of adverse events or exacerbation of GVHD. Among 11 evaluable cases, mean value of ferritin decreased from 1560 ng/ml pre-treatment to 1285 ng/ml post-treatment. These data suggested that 10 mg/kg of deferasirox may be maximum tolerated dose when given after HCT. Our dose escalating method of deferasirox is useful to identify the optimal dosage of the drug in each patient.

Trial Registration

UMIN000011251

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