Phase Ib clinical trial of starch-conjugated deferoxamine (40SD02): A novel long-acting iron chelator

British Journal of Haematology

Volumn 138, Issue 3, 2007, Pages 374-381

  Harmatz, Paul ^a   Grady, Robert W ^b   Dragsten, Paul ^c   Vichinsky, Elliott ^a   Giardina, Patricia ^b   Madden, Jacqueline ^a   Jeng, Michael ^d   Miller, Becky ^e   Hanson, Gregory ^c   Hedlund, Bo ^c  

^a CHILDREN S HOSPITAL OAKLAND   (United States)

^b WEILL CORNELL MEDICAL COLLEGE   (United States)

^c Biomedical Frontiers Inc   (United States)

^d STANFORD UNIVERSITY MEDICAL CENTER   (United States)

^e Olympia Medical Center   (United States)

Author keywords

Deferoxamine; Iron; Iron chelator; Thalassaemia; Urinary iron excretion

Indexed keywords

4 0SD02; ANTIHISTAMINIC AGENT; IRON; IRON CHELATING AGENT; STARCH DEFEROXAMINE CONJUGATE; UNCLASSIFIED DRUG;

ADOLESCENT; ADULT; ARTICLE; BETA THALASSEMIA; BLOOD CLOTTING; CLINICAL CHEMISTRY; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DRUG RETENTION; DRUG SAFETY; ELECTROCARDIOGRAM; FEMALE; HEMATOLOGY; HUMAN; IRON BALANCE; IRON BINDING CAPACITY; MALE; MOLECULAR WEIGHT; PATIENT COMPLIANCE; PRIORITY JOURNAL; SINGLE DRUG DOSE; URINALYSIS; URINARY EXCRETION; URTICARIA; VITAL SIGN;

ADOLESCENT; ADULT; AGED; AGED, 80 AND OVER; BETA-THALASSEMIA; CHELATION THERAPY; DEFEROXAMINE; FEMALE; HUMANS; INFUSIONS, INTRAVENOUS; IRON; IRON CHELATING AGENTS; MALE; MIDDLE AGED; PROSPECTIVE STUDIES;

EID: 34447120731     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/j.1365-2141.2007.06651.x     Document Type: Article

References (42)

1. Anderson, L.J., Westwood, M.A., Holden, S., Davis, B., Prescott, E., Wonke, B., Porter, J.B., Walker, J.M. Pennell, D.J. (2004) Myocardial iron clearance during reversal of siderotic cardiomyopathy with intravenous desferrioxamine: a prospective study using T2* cardiovascular magnetic resonance. British Journal of Haematology, 127, 348 355.
2. Artiss, J.D., Vinogradov, S. Zak, B. (1981) Spectrophotometric study of several sensitive reagents for serum iron. Clinical Biochemistry, 14, 311 315.
3. Bergeron, R.J., Streiff, R.R., Wiegand, J., Vinson, J.R.T., Luchetta, G., Evans, K.M., Peter, M. Jenny, H.-B. (1990) A comparative evaluation of iron clearance models. Ann NY Acad Sci, 612, 378 393.
4. Bergeron, R.J., Wiegand, J. Brittenham, G.M. (1998) HBED: a potential alternative to deferoxamine for iron-chelating therapy. Blood, 91, 1446 1452.
5. Borgna-Pignatti, C. Cohen, A. (1997) Evaluation of a new method of administration of the iron chelating agent deferoxamine. Journal of Pediatrics, 130, 86 88.
6. Cabantchik, Z.I., Breuer, W., Zanninelli, G. Cianciulli, P. (2005) LPI-labile plasma iron in iron overload. Best practice and research. Clinical Haematology, 18, 277 287.
7. Cappellini, M.D., Cohen, A., Piga, A., Bejaoui, M., Perrotta, S., Agaoglu, L., Aydinok, Y., Kattamis, A., Kilinc, Y., Porter, J., Capra, M., Galanello, R., Fattoum, S., Drelichman, G., Magnano, C., Verissimo, M., Athanassiou-Metaxa, M., Giardina, P., Kourakli-Symeonidis, A., Janka-Schaub, G., Coates, T., Vermylen, C., Olivieri, N., Thuret, I., Opitz, H., Ressayre-Djaffer, C., Marks, P. Alberti, D. (2006) A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood, 107, 3455 3462.
8. Cohen, A. Schwartz, E. (1978) Iron chelation therapy with deferoxamine in Cooley anemia. Journal of Pediatrics, 92, 643 647.
9. Cohen, A., Cohen, I.J. Schwartz, E. (1981) Scurvy and altered iron stores in thalassemia major. New England Journal of Medicine, 304, 158 160.
10. Collins, A.F., Fassos, F.F., Stobie, S., Lewis, N., Shaw, D., Fry, M., Templeton, D.M., McClelland, R.A., Koren, G. Olivieri, N.F. (1994) Iron-balance and dose-response studies of the oral iron chelator 1,2-dimethyl-3-hydroxypyrid- 4-one (L1) in iron-loaded patients with sickle cell disease. Blood, 83, 2329 2333.
11. Davis, B.A. Porter, J.B. (2000) Long-term outcome of continuous 24-hour deferoxamine infusion via indwelling intravenous catheters in high-risk beta-thalassemia. Blood, 95, 1229 1236.
12. Dragsten, P.R., Hallaway, P.E., Hanson, G.J., Berger, A.E., Bernard, B. Hedlund, B.E. (2000) First human studies with a high-molecular-weight iron chelator. Journal of Laboratory and Clinical Medicine, 135, 57 65.
13. Evans, P.J. Halliwell, B. (1994) Measurement of iron and copper in biological systems: bleomycin and copper-phenanthroline assays. Methods in Enzymology, 233, 82 92.
14. Gehlbach, P.L., Purple, R.L., Hallaway, P.E. Hedlund, B.E. (1993) Polymer conjugation reduces deferoxamine induced retinopathy in an albino rat model. Investigative Ophthalmology and Visual Science, 34, 2871 2877.
15. Gelmi, C., Borgna-Pignatti, C., Franchin, S., Tacchini, M. Trimarchi, F. (1988) Electroretinographic and visual-evoked potential abnormalities in patients with beta-thalassemia major. Ophthalmologica, 196, 29 34.
16. Giardina, P.J. Grady, R.W. (1995) Chelation therapy in beta-thalassemia: the benefits and limitations of desferrioxamine. Seminars in Hematology, 32, 304 312.
17. Giardina, P.J. Grady, R.W. (2001) Chelation therapy in beta-thalassemia: an optimistic update. Seminars in Hematology, 38, 360 366.
18. Gibaldi, M. (1991) Compartmental and Noncompartmental Pharmacokinetics. In : Biopharmaceutics and Clinical Pharmacokinetics pp. 14 23. Lea & Febiger, Philadelphia, PA.
19. Gosriwatana, I., Loreal, O., Lu, S., Brissot, P., Porter, J. Hider, R.C. (1999) Quantification of non-transferrin-bound iron in the presence of unsaturated transferrin. Analytical Biochemistry, 273, 212 220.
20. Grady, R.W. Giardina, P.J. (2000) Iron chelation: the rationale for combination therapy. In : Iron Chelators: New Development Strategies (ed. by D.G. Badman, R.J. Bergeron G.M. Brittenham pp. 293 310, The Saratoga Group, Ponte Vedra Beach, FL.
21. Grady, R.W., Giardina, P.J. Hilgartner, M.W. (1986) Total iron balance in response to desferrioxamine. Blood, 68, 46a.
22. Grady, R.W., Giardina, P.J. Hilgartner, M.W. (1987) Intravenous desferrioxamine and total iron balance. Blood, 70, 47a.
23. Hallaway, P.E., Eaton, J.W., Panter, S.S. Hedlund, B.E. (1989) Modulation of deferoxamine toxicity and clearance by covalent attachment to biocompatible polymers. Proceedings of the National Academy of Sciences of the United States of America, 86, 10108 10112.
24. Hershko, C. Rachmilewitz, E.A. (1979) Mechanism of desferrioxamine- induced iron excretion in thalassaemia. British Journal of Haematology, 42, 125 132.
25. Hershko, C., Graham, G., Bates, G.W. Rachmilewitz, E.A. (1978) Non-specific serum iron in thalassaemia: an abnormal serum iron fraction of potential toxicity. British Journal of Haematology, 40, 255 263.
26. Hussain, M.A., Green, N., Flynn, D.M., Hussein, S. Hoffbrand, A.V. (1976) Subcutaneous infusion and intramuscular injection of desferrioxamine in patients with transfusional iron overload. Lancet, 2, 1278 1280.
27. Jensen, P.D., Jensen, F.T., Christensen, T., Eiskjaer, H., Baandrup, U. Nielsen, J.L. (2003) Evaluation of myocardial iron by magnetic resonance imaging during iron chelation therapy with desferrioxamine: indication of close relation between myocardial iron content and chelatable iron pool. Blood, 101, 4632 4639.
28. Kattamis, A. (2005) Combined therapy with deferoxamine and deferiprone. Annals of the New York Academy of Sciences, 1054, 175 182.
29. Kattamis, A., Ladis, V., Berdousi, H., Kelekis, N.L., Alexopoulou, E., Papasotiriou, I., Drakaki, K., Kaloumenou, I., Galani, A. Kattamis, C. (2006) Iron chelation treatment with combined therapy with deferiprone and deferioxamine: a 12-month trial. Blood Cells, Molecules, and Diseases, 36, 21 25.
30. Lai, M.E., Vacquer, S., Pepe, A., Maggio, A., Carta, M.P., Cianciulli, P., Bina, P., Sau, F. Grady, R.W. (2006) Long-term outcome of iron-induced cardiac disease in patients with thalassemia major treated with combined DFP/DFO or DFO alone. Blood, 108, 500a.
31. Nisbet-Brown, E., Olivieri, N.F., Giardina, P.J., Grady, R.W., Neufeld, E.J., Sechaud, R., Krebs-Brown, A.J., Anderson, J.R., Alberti, D., Sizer, K.C. Nathan, D.G. (2003) Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: a randomised, double-blind, placebo-controlled, dose-escalation trial. Lancet, 361, 1597 1602.
32. O'Brien, R.T. (1974) Ascorbic acid enhancement of desferrioxamine-induced urinary iron excretion in thalassemia major. Annals of the New York Academy of Sciences, 232, 221 225.
33. Olivieri, N.F. Brittenham, G.M. (1997) Iron-chelating therapy and the treatment of thalassemia. Blood, 89, 739 761.
34. Olivieri, N.F., Koren, G., Hermann, C., Bentur, Y., Chung, D., Klein, J., St Louis, P., Freedman, M.H., McClelland, R.A. Templeton, D.M. (1990) Comparison of oral iron chelator L1 and desferrioxamine in iron-loaded patients. Lancet, 336, 1275 1279.
35. Olivieri, N.F., Nisbet-Brown, E., Srichairatanakool, S., Dragsten, P.R., Hallaway, P.E., Hedlund, B. Porter, J.B. (1996) Studies of iron excretion and non-transferrin-bound plasma iron (NTBPI) following a single infusion of hydroxyethyl starch-deferoxamine (HES-DFO): a new approach to iron chelation therapy. Blood, 88, 310a.
36. Pennell, D.J., Berdoukas, V., Karagiorga, M., Ladis, V., Piga, A., Aessopos, A., Gotsis, E.D., Tanner, M.A., Smith, G.C., Westwood, M.A., Wonke, B. Galanello, R. (2006) Randomized controlled trial of deferiprone or deferoxamine in beta-thalassemia major patients with asymptomatic myocardial siderosis. Blood, 107, 3738 3744.
37. Pippard, M.J., Callender, S.T. Finch, C.A. (1982) Ferrioxamine excretion in iron-loaded man. Blood, 60, 288 294.
38. Pootrakul, P., Breuer, W., Sametband, M., Sirankapracha, P., Hershko, C. Cabantchik, Z.I. (2004) Labile plasma iron (LPI) as an indicator of chelatable plasma redox activity in iron-overloaded beta-thalassemia/HbE patients treated with an oral chelator. Blood, 104, 1504 1510.
39. Porter, J.B., Jaswon, M.S., Huehns, E.R., East, C.A. Hazell, J.W. (1989) Desferrioxamine ototoxicity: evaluation of risk factors in thalassaemic patients and guidelines for safe dosage. British Journal of Haematology, 73, 403 409.
40. Porter, J.B., Abeysinghe, R.D., Marshall, L., Hider, R.C. Singh, S. (1996) Kinetics of removal and reappearance of non-transferrin-bound plasma iron with deferoxamine therapy. Blood, 88, 705 713.
41. Propper, R.D., Cooper, B., Rufo, R.R., Nienhuis, A.W., Anderson, W.F., Bunn, H.F., Rosenthal, A. Nathan, D.G. (1977) Continuous subcutaneous administration of deferoxamine in patients with iron overload. New England Journal of Medicine, 297, 418 423.
42. Sorcinelli, R., Sitzia, A., Figus, A. Lai, M.E. (1990) Ocular findings in beta-thalassemia. Metabolic, Pediatric and Systemic Ophthalmology, 13, 23 25.