Future Chelation Monotherapy and Combination Therapy Strategies in Thalassemia and Other Conditions. Comparison of Deferiprone, Deferoxamine, ICL670, GT56-252, L1NAll and Starch Deferoxamine Polymers

REFERENCES

• Andrews NC. A genetic view of haemochromatosis. Semin Hematol 2002; 39(4)227–234, [INFOTRIEVE], [CSA]
   PubMedGoogle Scholar
• Weatherall DJ, Glegg JB. The Thalassaemia Syndromes 3rd. Blackwell Scientific Publications, Oxford 1981
   Google Scholar
• Brown EB. Thalassemia. Prog Clin Biol Res 1983; 121: 33–42, [CSA]
   Google Scholar
• Modell B, Berdoukas V. The Clinical Approach to Thalassaemia. Grune & Stratton, London 1984
   Google Scholar
• Kontoghiorghes GJ. Present status and future prospects of oral iron chelation therapy in thalassemia and other diseases. Indian J Paediatr 1993; 60(4)485–507, [CSA]
   PubMedGoogle Scholar
• Kontoghiorghes GJ, Neocleous K, Kolnagou A. Benefits and risks of deferiprone in iron overload in thalassemia and other conditions. Drug Saf 2003; 26(8)553–584, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Gross F. Iron Metabolism. Springer-Verlag, Berlin 1964
   Google Scholar
• Anderson WF, Hiller MC. Development of Iron Chelators for Clinical Use. National Institute of Health, Bethesda 1975
   Google Scholar
• Martell AE, Anderson WF, Badman DG. Development of Iron Chelators for Clinical Use. Elsevier, Amsterdam 1980
   Google Scholar
• Kontoghiorghes GJ, Pattichis K, Neocleous K, Kolnagou A. The design and development of deferiprone (L1) and other iron chelators for clinical use: targeting methods and application prospects. Curr Med Chem 2004; 11(16)2161–2183, [INFOTRIEVE], [CSA]
   PubMed Web of Science ®Google Scholar
• Kontoghiorghes GJ. Comparative efficacy and toxicity of desferrioxamine, deferiprone and other iron and aluminium chelating drugs. Toxicol Lett 1995; 80(1–3)1–18, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Keberle H. The biochemistry of desferrioxamine and its relation to iron metabolism. Ann NY Acad Sci 1964; 119: 758–768, [INFOTRIEVE], [CSA]
   PubMed Web of Science ®Google Scholar
• Tenenbein M, Kowalski S, Sienko A, Bowden DH, Adamson IYR. Pulmonary toxic effects of continuous desferrioxamine administration in acute iron poisoning. Lancet 1992; 1(8795)699–701, [CROSSREF], [CSA]
   Google Scholar
• Sofroniadou K, Drossou M, Foundoulaki M, Konstantopoulos K, Kyriakou D, Zervas J. Acute bone marrow aplasia associated with intravenous administration of deferoxamine (desferrioxamine). Drug Saf 1990; 5(2)152–154, [INFOTRIEVE], [CSA]
   PubMedGoogle Scholar
• Sharnetzky M, Konig R, Lamomek M, Tillmann W, Schroter W. Prophylaxis of systemic yersiniasis in thalassemia major. Lancet 1984; 2(8380)791, [CROSSREF], [CSA]
   PubMedGoogle Scholar
• Modell B, Khan M, Darlison M. Survival in β-thalassaemia major in the UK: data from the UK register. Lancet 2000; 355(9220)2051–2052, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Lee P, Mohammed N, Marshall RD, Abeysinghe R, Hider RC, Porter J, Singh S. Intravenous infusion pharmacokinetics of desferrioxamine and its relation to iron metabolism. Drug Metabol Dispos 1993; 21(4)640–644, [CSA]
   PubMedGoogle Scholar
• Marcus RE, Davies SG, Bantock HM, Underwood SR, Walton S, Huehns ER. Desferrioxamine to improve cardiac function in iron-overloaded patients with thalassemia major. Lancet 1984; 1(8373)392–393, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Anderson LJ, Westwood MA, Holden S, Davis B, Prescott E, Wonke B, Porter JB, Walker JM, Pennell DJ. Myocardial iron clearance during reversal of siderotic cardiomyopathy with intravenous desferrioxamine: a prospective study using T2* cardiovascular magnetic resonance. Br J Haematol 2004; 127(3)348–355, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Borgna-Pignatti C, Cohen A. Evaluation of a new method of administration of the iron chelating agent deferoxamine. J Pediatr 1997; 130(1)86–88, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Harmatz P, Grady RW, Vichinsky E, Giardina PJ, Jeng M, Madden J, Nugent KA, Dragsten P. Phase 1b study of the safety, pharmacokinetics, acute tolerability, and efficacy of ascending single doses of 40SD02 (CHF5140, GS460) in iron loaded patients. Blood 2003; 102(11), Abstract 423[CSA]
   Google Scholar
• Kontoghiorghes GJ. L1 (1,2-dimethyl-3-hydroxypyrid-4-one). Drugs Future 1988; 13(5)413–415, [CSA]
   Google Scholar
• Kontoghiorghes GJ, Goddard JG, Bartlett AN, Sheppard L. Pharmacokinetic studies in humans with the oral iron chelator 1,2-dimethyl-3-hydroxypyrid-4-one. Clin Pharm Ther 1990; 48(3)255–261, [CSA]
   PubMed Web of Science ®Google Scholar
• Kontoghiorghes GJ. New concepts of iron and aluminium chelation therapy with oral L1 (Deferiprone) and other chelators. Analyst 1995; 120(3)845–851, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMedGoogle Scholar
• Sheppard L, Kontoghiorghes GJ. Synthesis and metabolism of L1 and other novel α-ketohydroxypyridine iron chelators and their metal complexes. Drugs Today 1992; 28(Suppl A)3–10, [CSA]
   Google Scholar
• Kontoghiorghes GJ, Bartlett AN, Hoffbrand AV, Goddard JG, Sheppard L, Barr J, Nortey P. Long term trial with the oral chelator 1,2-dimethyl-3-hydroxypyrid-4-one (L1). (I) Iron chelation and metabolic studies. Br J Haematol 1990; 76(2)295–300, [INFOTRIEVE], [CSA]
   PubMed Web of Science ®Google Scholar
• Kontoghiorghes GJ, Bartlett AN, Sheppard L, Barr J, Nortey P. Oral iron chelation therapy with deferiprone (L1): monitoring of biochemical, drug and iron excretion changes. Arzn Forsch/Drug Res 1995; 45(1)65–69, [CSA]
   PubMedGoogle Scholar
• Kontoghiorghes GJ, Evans RW. Site specificity of iron removal from transferrin by α-kytohydroxypyridine chelators. FEBS Lett 1985; 189(1)141–144, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Kontoghiorghes GJ. The study of iron mobilization from transferrin using α-ketohydroxy heteroaromatic chelators. Biochim Biophys Acta 1986; 869(2)141–146, [INFOTRIEVE], [CSA]
   PubMed Web of Science ®Google Scholar
• Evans RW, Sharma M, Ogwang W, Patel KJ, Bartlett AN, Kontoghiorghes GJ. The effect of α-ketohydroxypyridine chelators on transferrin saturation in vitro and in vivo. Drugs Today 1992; 28(Suppl A)19–23, [CSA]
   Google Scholar
• Turcot I, Stintzi A, Xu J, Raymond KN. Fast biological iron chelators: kinetics of iron removal from human diferric transferrin by multidentate hydroxypyridonates. J Biol Inorg Chem 2000; 5(5)634–641, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Olivieri NF, Koren G, Matsuii D, Liu PP, Blendis L, Cameron R, McClelland RA, Templeton DM. Reduction of tissue iron stores and normalization of serum ferritin during treatment with the oral iron chelator L1 in thalassemia intermedia. Blood 1992; 79(10)2741–2748, [INFOTRIEVE], [CSA]
   PubMed Web of Science ®Google Scholar
• Tondury P, Zimmerman A, Nielsen P, Hirt A. Liver iron fibrosis during long-term treatment with deferiprone in Swiss thalassaemic patients. Br J Haematol 1998; 101(3)413–415, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Kolnagou A, Fessas Ch, Papatryphonas A, Economides Ch, Kontoghiorghes GJ. Prophylactic use of deferiprone (L1) and magnetic resonance imaging T2* or T2 for preventing heart disease in thalassaemia. Br J Haematol 2004; 127(3)360–361, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Anderson LJ, Wonke B, Prescott E, Holden S, Walker JM, Pennel DJ. Comparison of effects of oral deferiprone and subcutaneous desferrioxamine on myocardial iron concentrations and ventricular function in β-thalassaemia. Lancet 2002; 360(9332)516–520, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Kontoghiorghes GJ. Do we need more iron chelating drugs?. Lancet 2003; 362(9382)495–496, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Peng CT, Chow KC, Chen JH, Chiang YP, Lin TY, Tsai CH. Safety monitoring of cardiac and hepatic systems in β-thalassemia patients with chelating treatment in Taiwan. Eur J Haematol 2003; 70(6)392–397, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Kontoghiorghes GJ, Aldouri MA, Hoffbrand AV, Barr J, Wonke B, Kourouclaris T, Sheppard L. Effective chelation of iron in β-thalassaemia with the oral chelator 1, 2-dimethyl-3-hydroxypyrid-4-one. Br Med J 1987; 295(6612)1509–1512, [CSA]
   PubMed Web of Science ®Google Scholar
• Kontoghiorghes GJ, Aldouri MA, Sheppard LN, Hoffbrand AV. 1, 2-Dimethyl-3-hydroxypyrid-4-one, an orally active chelator for the treatment of transfusional iron overload. Lancet 1987; 1(8545)1294–1295, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Goudsmit R, Kersten MJ. Long term treatment of transfusion hemosiderosis with the oral iron chelator L1. Drugs Today 1992; 28(suppl. A)133–135, [CSA]
   Google Scholar
• Jaeger M, Aul C, Sohngen D, Germing U, Schneider W. Iron overload in polytransfused patients with MDS; use of L1 for oral iron chelation. Drugs Today 1992; 28(Suppl A)143–147, [CSA]
   Google Scholar
• Agarwal MB, Gupta SS, Viswanathan C, Vasandani D, Ramanathan J, Desai N, Puniyani RR, Chhablani AT. Long term assessment of efficacy and toxicity of L1 (1,2-dimethyl-3-hydroxypyrid-4‐one) in transfusion dependent thalassemia. Indian trial. Drugs Today 1992; 28(Suppl A)107–114, [CSA]
   Google Scholar
• Cohen AR, Galanello R, Piga A, Di Palma A, Voullo C, Tricta F. Safety profile of the oral iron chelator deferiprone: a multicentre study. Br J Haematol 2000; 108(2)305–312, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Agarwal MB, Gupta SS, Viswanathan C, Vasantani D, Ramanathan J, Desai N, Puniyani RR, C­hablani AT. Long term assessment of efficacy and safety of L1, an oral iron chelator in transfusion dependent thalassaemia: Indian trial. Br J Haematol 1992; 82(2)460–466, [INFOTRIEVE], [CSA]
   PubMed Web of Science ®Google Scholar
• Kontoghiorghes GJ, Bartlett AH, Hoffbrand AV. Prospects for effective oral iron chelation therapy in man with 1,2-dimethyl-3-hydroxypyrid-4-one and other α-ketohydroxypyridines. Prog Clin Biol Res 1989; 309: 107–114, [INFOTRIEVE], [CSA]
   PubMedGoogle Scholar
• Kellenberger CJ, Schmugge M, Saurenmann T, Gennaro LD, Eber SW, Willi UV, Babyn PS. Radiographic and MRI features of deferiprone-related arthropathy of the knees in patients with β-thalassemia. Am J Roentgenol 2004; 183(4)989–994, [CSA]
   PubMed Web of Science ®Google Scholar
• Kontoghiorghes GJ. Advances in oral iron chelation in man. Int J Haematol 1992; 55(1)27–38, [CSA]
   PubMed Web of Science ®Google Scholar
• Wonke B, Wright C, Hoffbrand AV. Combined therapy with deferiprone and deferoxamine. Br J Haematol 1998; 103(2)361–364, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Giardina PJ, Grady RW. Chelation therapy in β-thalassemia: an optimistic update. Semin Hematol 2001; 38(4)360–366, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Davies SC, Marcus RE, Hungerford JL, Miller MH, Arden GB, Huehns ER. Ocular toxicity of high dose intravenous desferrioxamine. Lancet 1983; 2(8343)181–184, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Rahi AHS, Hungerford JL, Ahmed AI. Ocular toxicity of desferrioxamine: light microscopic histochemical and ultrastructural findings. Br J Ophthal 1986; 70(5)373–381, [CSA]
   PubMed Web of Science ®Google Scholar
• Rubinstein M, Dupont P, Doppel JP, Ducobu J, Hainant J. Ocular toxicity of desferrioxamine. ­Lancet 1985; 1(8432)817–818, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Borgna-Pignatti C, De Stefano P, Broglia AM. Visual loss in patient on high dose subcutaneous desferrioxamine. Lancet 1984; 1(8378)68, [CSA]
   Google Scholar
• Guerin A, London G, Marchais S, Mativier F, Pelisse JM. Acute deafness and desferrioxamine. Lancet 1985; 2(8445)39–40, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMedGoogle Scholar
• Cappelini MD. Iron-chelating therapy with the new oral agent ICL670 (Exjade). Best Prac Res Clin Hematol 2005; 18(2)289–298, [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Nick H, Acklin P, Lattmann R, Buechlmayer P, Hauffe S, Hauffe S, Schupp J, Alberti D. Development of tridentate iron chelators: from desferrithiocin to ICL670. Curr Med Chem 2003; 10(12)1065–1076, [INFOTRIEVE], [CSA]
   PubMed Web of Science ®Google Scholar
• Nisbet-Brown E, Olivieri NF, Giardina PJ, Grady RW, Neufeld EJ, Sechaud R, Krebs-Brown AJ, Anderson JR, Alberti D, Sizer KC, Nathan DG. Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: a randomised, double-blind, placebo-controlled, dose-escalation trial. Lancet 2003; 361(9369)1597–1602, [INFOTRIEVE], [CSA]
   PubMed Web of Science ®Google Scholar
• Galanello R, Piga A, Alberti D, Rouan MC, Bigler H, Sechaud R. Safety, tolerability, and pharmacokinetics of ICL670, a new orally active iron-chelating agent in patients with transfusion-dependent iron overload due to β-thalassemia. J Clin Pharmacol 2003; 43(6)565–572, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Hershko C, Konjin MA, Nick HP, Breuer W, Cabantchik Z I, Link G. ICL670A: a new synthetic oral chelator: evaluation in hypertransfused rats with selective radio iron probes of hepatocellular and reticuloendothelial iron stores and in iron-loaded rat heart cells in culture. Blood 2001; 97(4)1115–1122, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Wood JC, Otto-Duessel M, Aguilar M, Nick H, Coates TD, Rex M. Dose response of deferoxamine, deferiprone, and ICL670 chelation therapy in a Gerbil model of iron overload. Blood 2004; 104(4), Abstract 3621[CSA]
   Google Scholar
• Piga A, Galanello R, Foschini ML, Zappu A, Bordone E, Longo F, Forni GL, Lavagetto A, Bertrand Y, Maseruka H, Latham N, Alberti D. Once-daily treatment with the oral iron chelator ICL670 (Exjade): results of a phase II study in pediatric patients with β-thalassemia major. Blood 2004; 104(11), Abstract 3614[CSA]
   PubMedGoogle Scholar
• Cappelini M, Bejaoui M, Perrota S, Agaoglou L, Kattamis A, Giardina P, Janka-Schaub G, Opitz H, Ressayre-Djaffer C, Alberti D. Phase III evaluation of once-daily oral therapy with ICL670 (Exjade) versus deferoxamine in patients with β-thalassemia and transfusional hemosiderosis. Blood 2004; 104(11), Abstract 3619[CSA]
   Google Scholar
• Porter J, Vichinsky E, Rose C, Piga A, Olivieri N, Gattermann I, Maertens J, Rabault B, Gathmann I, Alberti D. A phase II study with ICL670 (Exjade), a once-daily oral iron chelator, in patients with various transfusion-dependent anemias and iron overload. Blood 2004; 104(11), Abstract 3193[CSA]
   Google Scholar
• Kontoghiorghes GJ, Eracleous E, Economides Ch, Kolnagou A. Advances in iron overload therapies. Prospects for effective use of deferiprone (L1), deferoxamine, the new experimental chelators ICL670, GT56-252, L1Nall and their combinations. Curr Med Chem 2005; 12(23)2663–2681, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Kontoghiorghes GJ. Effects of ICL670 (deferasirox) on cardiac iron concentration. Lancet 2005; 366(9488)804, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Bergeron RJ, Wiegand J, Weimar WR, Vinson JR, Bussenious J, Yao GW, MacManis JS. Desazadesmethyldesferrithiocin analogues as orally effective iron chelators. J Med Chem 1999; 42(1)95–108, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMedGoogle Scholar
• Marquis J, Dagher R, Bree M, Appelqvist T, Guillaumat PO, Forster R. Pharmacokinetic of GT56-252, a novel orally available iron chelator in rat, dog and monkey. Biomarkers Environment 2003; 6(1)7–9, [CSA]
   Google Scholar
• Donovan JM, Palmer PA, Plone MA, Wonke B. The safety and pharmacokinetics of GT56-252, a novel orally available iron chelator. Eur J Clin Invest 2004; 34(Supp I)39, Abstract 139[CSA]
   Google Scholar
• Kontoghiorghes GJ, Sheppard L. Simple synthesis of the potent iron chelators 1-alkyl-3-hydroxy-2-methylpyrid-4-ones. Inorg Chim Acta 1987; 136: L11–L12, [CROSSREF], [CSA]
   Web of Science ®Google Scholar
• Kontoghiorghes GJ. L1NAll. 1-Allyl-2-methyl-3-hydroxypyrid-4-one. Drugs Future 1990; 15(3)230–232, [CSA]
   Google Scholar
• Kontoghiorghes GJ. New orally active iron chelators. Lancet 1985; 1(8432)817, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMedGoogle Scholar
• Kontoghiorghes GJ, Barr J, Nortey P, Sheppard L. Selection of a new generation of orally active α‐ketohydroxypyridine iron chelators intended for use in the treatment of iron overload. Am J Hematol 1993; 42(4)340–349, [INFOTRIEVE], [CSA]
   PubMed Web of Science ®Google Scholar
• Kontoghiorghes GJ, Sheppard LN. Production of pyrid-4-ones. European patent application 0335745 1989, [CSA]
   Google Scholar
• Nortey P, Barr J, Matsakis M, Nasseri-Sina P, Kontoghiorghes GJ. Effect on iron excretion and animal toxicology of L1 and other α-ketohydroxypyridine chelators. Drugs Today 1992; 28(Suppl A)81–88, [CSA]
   Google Scholar
• Sauvage C, Rouyer-Fessard R, Turlin B, Brissot P, Beuzard Y. Study of L1NAll, a new oral iron chelator in two experimental models of human β-thalassemia. Drugs Today 1992; 28(Suppl A)65–71, [CSA]
   Google Scholar
• Nasseri-Sina P, Murray BP, Boobis AR, Kontoghiorghes GJ. Results of the mutagenecity tests of 1,2-dimethyl-3-hydroxypyrid-4-one (L1) 1-allyl-2-methyl-3-hydroxypyrid-4-one (L1NAll) and their iron complexes. Plzen Lek Sborn 1990; 62: 221–224, [CSA]
   Google Scholar
• Eybl V, Svihovcova P, Koutensky J, Kontoghiorghes GJ. Interaction of L1, L1NAll and deferoxamine with gallium in vivo. Drugs Today 1992; 28(Suppl A)173–175, [CSA]
   Google Scholar
• Dragsten PR, Hallaway PE, Hanson GJ, Berger AE, Bernard B, Hedlund BE. First human studies with a high-molecular-weight iron chelator. J Lab Clin Med 2000; 135(1)57–65, [INFOTRIEVE], [CROSSREF], [CSA]
   PubMed Web of Science ®Google Scholar
• Grady RW. An update on development of new iron-chelating drugs. Abstract Book, Proceedings of the 15th International Committee on Oral Chelators (ICOC), TaichungTaiwan, 2005; 66
   Google Scholar
• Kontoghiorghes GJ. New oral iron-chelating drugs for the treatment of transfusional iron overload and other diseases. Drugs Future 2006; 30(12)1241–1251, [CROSSREF], [CSA]
   Web of Science ®Google Scholar