Authors: Harmatz, Paul¹; Grady, Robert W.²; Dragsten, Paul³; Vichinsky, Elliott⁴; Giardina, Patricia²; Madden, Jacqueline¹; Jeng, Michael⁵; Miller, Becky⁶; Hanson, Gregory³; Hedlund, Bo³

Source: British Journal of Haematology, Volume 138, Number 3, August 2007 , pp. 374-381(8)

Publisher: Wiley-Blackwell

Abstract:

Summary

The most widely used drug for iron chelation is deferoxamine (DFO) mesylate. While effective in promoting iron excretion, it requires prolonged daily infusions, often resulting in poor compliance. A clinical trial was conducted using starch-conjugated DFO (S-DFO; 40SD02), a high-molecular-weight iron chelator possessing prolonged vascular retention. Single doses of S-DFO were infused intravenously into groups of four transfusion-dependent patients with β-thalassaemia at doses of 150, 300, 600 and 900 mg/kg. Urinary iron excretion and various pharmacologic parameters were evaluated for 1 week and safety for 3 weeks. No drug-related effects were observed on clinical chemistries, haematological and coagulation parameters, urinalyses, vital signs or electrocardiograms. Drug-related adverse events were limited to four urticarial reactions, none requiring termination of the infusion. The drug stimulated clinically significant urinary iron excretion, with the highest dose (900 mg/kg) inducing excretion of 1·31 mg of iron/kg (range 0·79-1·90 mg/kg) over 1 week, with residual iron-binding capacity present in the plasma for over 6 d. In summary, treatment with S-DFO, administered weekly, has the potential to achieve iron balance in the poorly compliant patient.

Keywords: thalassaemia; iron; iron chelator; deferoxamine; urinary iron excretion

Document Type: Research article

DOI: http://dx.doi.org/10.1111/j.1365-2141.2007.06651.x

Affiliations: 1: Department of Gastroenterology, The Children's Hospital & Research Center Oakland, CA 2: Biomedical Frontiers, Inc, Minneapolis, MN 3: Olympia Medical Center, Los Angeles, CA, USA 4: Department of Pediatrics, Division of Haematology/Oncology, Children's Blood Foundation Laboratories, Weill Medical College of Cornell University, New York-Presbyterian Hospital, NY 5: Department of Haematology, The Children's Hospital & Research Center Oakland, CA 6: Department of Pediatrics, Stanford University Medical Center, Stanford, CA

Publication date: 2007-08-01

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