The controversial role of deferiprone in the treatment of thalassemia

Journal of Laboratory and Clinical Medicine

Volumn 137, Issue 5, 2001, Pages 324-329

  Richardson, Des R ^a,b  

^a HEART RESEARCH INSTITUTE   (Australia)

^b NONE   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 0035336822     PISSN: 00222143     EISSN: None     Source Type: Journal    
DOI: 10.1067/mlc.2001.114105     Document Type: Article

References (61)

1. Cerami A. The future of new iron chelating drugs. In: Martell AE, Anderson WF, Badman DG, editors. Development of Iron Chelators for Clinical Use. New York: Elsevier North Holland; 1981. p. 197-200.
2. Richardson DR, Ponka P. The development of iron chelators to treat iron overload disease and their use as experimental tools to probe intracellular iron metabolism. Am J Hematol 1998;58:299-305.
3. Olivieri NF, Brittenham GM. Iron chelation therapy and the treatment of thalassemia. Blood 1997;89:739-61.
4. Richardson DR. Therapeutic potential of iron chelators. Exp Opin Invest Drugs 1999;8:2141-58.
5. Olivieri NF, Koren G, Matsui D, Liu PP, Blendis L, Cameron R, et al. Reduction of tissue iron stores and normalisation of serum ferritin during treatment with the oral iron chelator L1 in thalassemia intermedia. Blood 1992;79:2741-8.
6. Olivieri NF, Brittenham GM, Matsui D, Berkovitch M, Blendis LM, Cameron RG, et al. Iron chelation therapy with oral deferiprone in patients with thalassemia major. N Engl J Med 1995;332:918-22.
7. Olivieri NF, Brittenham GM, McLaren CE, Templeton DM, Cameron RG, McClelland RA, et al. Long-term safety and effectiveness of iron-chelation therapy with deferiprone for thalassemia major. N Engl J Med 1998;339:417-23.
8. Hoffbrand AV, A1-Refaie F, Davis B, Siritanakatkul N, Jackson BFA, Cochrane J, Long-term trial of deferiprone in 51-transfusion-dependent iron overloaded patients. Blood 1998;91:295-300.
9. Hoyes KP, Porter JB. Subcellular distribution of desferrioxamine and hydroxypyridin-4-one chelators in K562 cells affects chelation of intracellular iron pools. Br J Haematol 1993;85:393-400.
10. al Refaie FN, Sheppard LN, Nortey P, Wonke B, Hoffbrand AV. Pharmacokinetics of the oral iron chelator deferiprone (L1) in patients with iron overload. Br J Haematol 1995;89:403-8.
11. Porter JB, Gyparaki M, Burke LC, Huehns ER, Sarpong P, Saez V, et al. Iron mobilization from hepatocyte monolayer cultures by chelators: The importance of membrane permeability and the iron-binding constant. Blood 1988;72:1497-503.
12. Kontoghiorghes GJ, Evans RW. Site specificity of iron removal from transferrin by α-ketohydroxypyridone chelators. FEBS Lett 1985;189:141-4.
13. al Refaie FN, De-Silva CE, Wonke B, Hoffbrand AV. Changes in transferrin saturation after treatment with the oral iron chelator deferiprone in patients with iron overload. J Clin Pathol 1995;48:110-4.
14. Cragg L, Hebbel RP, Miller W, Solovey A, Selby S, Enright H. The iron chelator L1 potentiates oxidative DNA damage in iron-loaded liver cells. Blood 1998;92:632-8.
15. Cooper CE, Lynagh GR, Hoyes KP, Hider RC, Cammack R, Porter JB. The relationship of intracellular iron chelation to the inhibition and regeneration of human ribonucleotide reductase. J Biol Chem 1996;271:20291-9.
16. Hileti D, Panayiotidis P, Hoffbrand AV. Iron chelators induce apoptosis in proliferating cells. Br J Haematol 1995;89:181-7.
17. Berkovitch M, Laxer RM, Inman R, Koren G, Pritzker KP, Fritzler MJ, Olivieri NF. Arthropathy in thalassemic patients receiving deferiprone. Lancet 1994;343:1471-2.
18. Collins AF, Fassos FF, Stobie S, Lewis N, Shaw D, Fry M, 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 1994;83:2329-33.
19. Dogherty P, Einarson T, Koren G, Sher G. The effectiveness of deferiprone in thalassemia. Blood 1997;90:894.
20. Richardson DR, Ponka P. Pyridoxal isonicotinoyl hydrazone and its analogues: Potential orally effective iron-chelating agents for the treatment of iron overload disease. J Lab Clin Med 1998;131:306-15.
21. Richardson DR, Ponka P. Orally effective iron chelators for the treatment of iron overload disease: The case for a further look at pyridoxal isonicotinoyl hydrazone (PIH) and its analogs. J Lab Clin Med 1998;132:351-2.
22. Florence A, Longueville A, Crichton RR. Oral iron chelators may do more harm than good. In: Proceedings of the Ninth International Conference on Proteins of Iron Transport and Storage, July 9-13, Brisbane, Australia, 1989:P118
23. Berdoukas V, Lindeman R, Eagle C, Bohane T, Tobias V, DeSilve K, et al. The Sydney children's hospital experience with the oral iron chelator deferiprone. In: Proceedings of the Tenth International Conference on Oral Chelators in the Treatment of Thalassemia and Other Diseases and Biomed Meeting. March 22-26, 2000, Limassol, Cyprus. p. 6.
24. Gogtey J, Agarwal MB, Rajadhyaksha G, Munot S. Deferiprone (Kelfer-L1). A review of Indian study over 10 years. In: Proceedings of the Tenth International Conference on Oral Chelators in the Treatment of Thalassemia and Other Diseases and Biomed Meeting. March 22-26, 2000, Limassol, Cyprus. p. 4.
25. Agarwal MB, Rajadhyaksha G. Deferiprone (Kelfer). A report of 22 patients who have taken it for over a decade. In: Proceedings of the Tenth International Conference on Oral Chelators in the Treatment of Thalassemia and Other DisEases and Biomed Meeting. March 22-26, 2000. Limassol, Cyprus. p. 3.
26. Töndury P, Nielsen P, Fischer R, Zimmerman A, von der Weid N, Hirt A. Liver iron and fibrosis during long-term treatment with deferiprone in swiss thalassaemic patients, 1989-2000. In: Proceedings of the Tenth International Conference on Oral Chelators in the Treatment of Thalassemia and Other Diseases and Biomed Meeting. March 22-26 2000. Limassol, Cyprus. p. 9.
27. Töndury P, Zimmermann A, Nielsen P, Hirt A. Liver iron and fibrosis during long-term treatment wih deferiprone in swiss thalassaemic patients. Br J Haematol 1998;101:413-5.
28. Wonke B, Wright C, Hoffbrand AV. Combined therapy with deferiprone and desferrioxamine. Br J Haematol 1998;103:361-4.
29. Aydinok Y, Nisli G, Kavakli K, Coker C, Kantar M, Centingul N. Sequential use of deferiprone and desferrioxamine in primary school children with thalassemia major in turkey. Acta Haematol 1999;102:17-21.
30. Grady RW, Berdoukas VA, Rachmilewitz EA, Giardina PJ. Combining deferiprone and desferrioxamine to optimize chelation. In: Proceedings of the Tenth International Conference on Oral Chelators in the Treatment of Thalassemia and Other Diseases and Biomed Meeting. March 22-26, 2000. Limassol, Cyprus. p. 9.
31. Tsafack A, Loyevsky M, Ponka P, Cabantchik ZI. Mode of action of iron(III) chelators as anti-malarials. IV. Potentiation of desferal action by benzoyl and isonicotinoyl hydrazone derivatives. J Lab Clin Med 1996;127:574-82.
32. Tsafack A, Golenser J, Libman J, Shanzer A, Cabantchik ZI. Mode of action of iron(III) chelators as antimalarials. 3. Overadditive effects in the combined action of hydroxamate-based agents on in vitro growth of plasmodium falciparum. Mol Pharmacol 1995;47:403-9.
33. al Refaie FN, Wonke B, Hoffbrand AV. Deferiprone-associated myelotoxicity. Eur J Haematol 1994;53:298-301.
34. al Refaie FN, Hershko C, Hoffbrand AV, Kosaryan M, Olivieri NF, Tondury P, et al. Results of long-term deferiprone (L1) therapy: A report by the international study group on oral iron chelators. Br J Haematol 1995;91:224-9.
35. Castriota-Scanderbeg A, Sacco M. Agranulocytosis, arthritis and systemic vasculitis in a patient receiving the oral iron chelator L1 (deferiprone). Br J Hematol 1997;96:254-5.
36. Kersten MJ, Lange R, Smeets ME, Vreugdenhil G, Roozendaal KJ, Lameijer W, et al. Long-term treatment of transfusional iron overload with the oral iron chelator deferiprone (L1): A Dutch multicenter trial. Ann Hematol 1996;73:247-52.
37. Cohen AR, Galanello R, Piga A, Dipalma A, Vullo C, Tricta F. Safety profile of the oral iron chelator deferiprone: A multicentre study. Br J Haematol 2000;108:305-12.
38. Yokel RA, Fredenburg AM, Durbin PW, Xu J, Rayens MK, Raymond KN. The hexadentate hydroxypyridinonate TREN-(Me-3,2-HOPO) is a more orally active iron chelator than its bidentate analogue. J Pharm Sci 2000;89:545-55.
39. Hider RC, Singh S, Porter JB, Huehns ER. The development of hydroxypyridin-4-ones as orally active iron chelators. Ann NY Acad Sci 1990;612:327-38.
40. Hershko C, Link G, Pinson A, Peter HH, Dobbin P, Hider RC. Iron mobilization from myocardial cells by 3-hydroxypyridin-4-one chelators: Studies in rat heart cells in culture. Blood 1991;77:2049-53.
41. Liu ZD, Khodr HH, Liu DY, Lu SL, Hider RC. Synthesis, physicochemical characterisation, and biological evaluation of 2-(1′-hydroxyalkyl)-3-hydroxypyridin-4-ones: Novel iron chelators with enhanced pFe(3+) values. J Med Chem 1999; 42:4814-23.
42. Ponka P, Borova J, Neuwirt J, Fuchs O. Mobilization of iron from reticulocytes. Identification of pyridoxal isonicotinoyl hydrazone as a new iron chelating agent. FEBS Lett 1979;97:317-21.
43. Ponka P, Borova J, Neuwirt J, Fuchs O, Necas E. A study of intracellular iron metabolism using pyridoxal isonicotinoyl hydrazone and other synthetic chelating agents. Biochim Biophys Acta 1979;586:278-97.
44. Baker E, Vitolo ML, Webb JM. Iron chelation by pyridoxal isonicotinoyl hydrazone and analogues in hepatocytes in culture. Biochem Pharmacol 1985;34:3011-7.
45. Richardson D, Baker E, Ponka P, Wilairat P, Vitolo ML, Webb J. Effects of pyridoxal isonicotinoyl hydrazone and analogs on iron metabolism in hepatocytes and macrophages in culture. Birth Defects 1988;23:81-8.
46. Ponka P, Richardson D, Baker E, Schulman HM, Edwart JT. Effect of pyridoxal isonicotinoyl hydrazone and other hydrazones on iron release from macrophages, reticulocytes and hepatocytes. Biochim Biophys Acta 1988;967:122-9.
47. Baker E, Richardson DR, Gross S, Ponka P. Evaluation of the iron chelation potential of hydrazones of pyridoxal, salicylaldehyde and 2-hydroxy-1-naphthylaldehyde using the hepatocyte in culture. Hepatology 1992;15:492-501.
48. Johnson DK, Pippard MJ, Murphy TB, Rose NJ. An in vivo evaluation of iron chelating drugs derived from pyridoxal and its analogues. J Pharmacol Exp Ther 1982;221:399-403.
49. Blaha K, Cikrt M, Nerudova J, Fornuskova H, Ponka P. Biliary iron excretion in rats following treatment with analogues of pyridoxal isonicotinoyl hydrazone. Blood 1998;91:4368-72.
50. Richardson DR, Hefter GT, May PM, Webb J, Baker E. Iron chelators of the pyridoxal isonicotinoyl hydrazone class III. Formation constants with calcium(II), magnesium(II) and zinc(II). Biol met 1989;2:161-7.
51. Vitolo LMW, Hefter GT, Clare BW, Webb J. Iron chelators of the pyridoxal isonicotinoyl hydrazone class part 2. Formation constants with iron(III) and iron(II). Inorg Chim Acta 1990;170:171-4.
52. Richardson DR, Wis Vitolo ML, Hefter GT, May PM, Clare BW, Webb J, et al. Iron chelators of the pyridoxal isonicotinoyl hydrazone class part 1. Ionisation characteristics of the ligands and their relevance to biological properties. Inorg Chim Acta 1990;170:165-70.
53. Richardson DR, Baker E. The release of iron and transferrin from the human melanoma cell. Biochim Biophys Acta 1991;1091:294-302.
54. Richardson DR, Tran E, Ponka P. The potential of iron chelators of the pyridoxal isonicotinoyl hydrazone class as effective antiproliferative agents. Blood 1995;86:4295-306.
55. Richardson DR, Milnes K. The potential of iron chelators of the pyridoxal isonicotinoyl hydrazone class as effective anti-proliferative agents II: The mechanism of action of ligands derived from salicylaldehyde benzoyl hydrazone and 2-hydroxy-1-naphthylaldehyde benzoyl hydrazone. Blood 1997; 89:3025-38.
56. Darnell G, Richardson DR. The potential of analogues of the pyridoxal isonicotinoyl hydrazone class as effective anti-proliferative agents III: The effect of the ligands on molecular targets involved in proliferation. Blood 1999;94:781-92.
57. Richardson DR, Bernhardt P. Crystal and molecular structure of 2-hydroxy-1-naphthylaldehyde isonicotinoyl hydrazone (NIH) and its iron(III) complex: An iron chelator with anti-proliferative activity. J Biol Inorg Chem 1999;4:266-73.
58. Becker E, Richardson DR. Development of novel aroylhydrazone ligands for iron chelation therapy: The 2-pyridylcarboxaldehyde isonicotinoyl hydrazone (PCIH) analogues. J Lab Clin Med 1999;134:510-21.
59. Richardson DR, Becker E, Bernhardt PV. The biologically active iron chelators 2-pyridylcarboxaldehyde isonicotinoyl hydrazone, 2-pyridylcarboxaldehyde benzoyl hydrazone and 2-furfural isonicotinoyl hydrazone. Acta Crystallogr C 1999; C55:2102-05.
60. Richardson DR, Becker E, Bernhardt PV. Iron Chelators and Uses Thereof. International Patent Application No. PCT/AU00/01050.
61. Martell AE, Motekaitis RJ, Sun Y, Ma R, Welch MJ, Pajeau T. New chelating agents suitable for the treatment of iron overload. Inorg Chim Acta 1999;291:238-46.