Deferasirox for managing iron overload in people with thalassaemia

Cochrane Database of Systematic Reviews

Volumn , Issue 4, 2008, Pages

  Meerpohl, Joerg J ^a,c   Antes, Gerd ^a   Rücker, Gerta ^a   McLeod, Claire ^b   Fleeman, Nigel ^b   Niemeyer, Charlotte ^c   Bassler, Dirk ^d  

^a German Cochrane Center   (Germany)

^b UNIVERSITY OF LIVERPOOL   (United Kingdom)

^c UNIVERSITY OF FREIBURG   (Germany)

^d UNIVERSITY HOSPITAL TÜBINGEN   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

CHELATING AGENT; DEFERASIROX; DEFERIPONE; DEFEROXAMINE; FERRITIN; IRON; UNCLASSIFIED DRUG;

AGRANULOCYTOSIS; BLOOD TRANSFUSION; CLINICAL TRIAL; COUGHING; CREATININE BLOOD LEVEL; DATA BASE; DATA EXTRACTION; DIARRHEA; DRUG BIOAVAILABILITY; DRUG EFFICACY; DRUG SAFETY; FERRITIN BLOOD LEVEL; FEVER; GASTROINTESTINAL SYMPTOM; HALF LIFE TIME; HEADACHE; HEARING LOSS; HUMAN; INFORMATION PROCESSING; IRON BLOOD LEVEL; IRON OVERLOAD; KIDNEY FAILURE; NAUSEA; NEUTROPENIA; RASH; RETINAL TOXICITY; REVIEW; SIDE EFFECT; STOMACH PAIN; THALASSEMIA; TREATMENT OUTCOME;

EID: 55049112755     PISSN: 1469493X     EISSN: None     Source Type: Journal    
DOI: 10.1002/14651858.CD007476     Document Type: Review

References (56)

1. Angastiniotis 1998 Angastiniotis M, Modell B. Global epidemiology of hemoglobin disorders. Annals of the New York Academy of Sciences 1998;850:251-69.
2. Borgna-Pignatti 2004 Borgna-Pignatti C, Rugolotto S, De Stefano P, Zhao H, Cappellini MD, Del Vecchio GC, et al.Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica 2004;89(10):1187-93.
3. Borgna-Pignatti 2005 Borgna-Pignatti C, Cappellini MD, De Stefano P, Del Vecchio GC, Forni GL, Gamberini MR, et al.Survival and complications in thalassemia. Annals of the New York Academy of Sciences 2005;1054:40-7.
4. Brittenham 1994 Brittenham GM, Griffith PM, Nienhuis AW, McLaren CE, Young NS, Tucker EE, et al.Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major. The New England Journal of Medicine 1994;331(9):567-73.
5. Cappellini 2005 Cappellini MD. Overcoming the challenge of patient compliance with iron chelation therapy. Seminars in Hematology 2005;42(2 Suppl 1):S19-21.
6. Cappellini 2006 Cappellini MD, Cohen A, Piga A, Bejaoui M, Perrotta S, Agaoglu L, et al.A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood 2006;107(9):3455-62.
7. Cappellini 2007 Cappellini MD, Bejaoui M, Agaoglu L, Porter J, Coates T, Jeng M, et al.Prospective evaluation of patient-reported outcomes during treatment with deferasirox or deferoxamine for iron overload in patients with beta-thalassemia. Clinical Therapeutics 2007;29(5):909-17.
8. Ceci 2002 Ceci A, Baiardi P, Felisi M, Cappellini MD, Carnelli V, De Sanctis V, et al.The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients. British Journal of Haematology 2002;118(1):330-6.
9. Cooley 1925 Cooley TB, Lee P. A series of cases of splenomegaly in children with anemia and peculiar bone changes. Transactions of the American Pediatric Society 1925;37:29-30.
10. Curtin 2002a Curtin F, Altman DG, Elbourne D. Meta-analysis combining parallel and cross-over clinical trials. I: Continuous outcomes. Statistics in Medicine 2002;21(15):2131-44.
11. Curtin 2002b Curtin F, Elbourne D, Altman DG. Meta-analysis combining parallel and cross-over clinical trials. II: Binary outcomes. Statistics in Medicine 2002;21(15):2145-59.
12. Curtin 2002c Curtin F, Elbourne D, Altman DG. Meta-analysis combining parallel and cross-over clinical trials. III: The issue of carry-over. Statistics in Medicine 2002;21(15):2161-73.
13. Delea 2008 Delea TE, Hagiwara M, Thomas SK, Baladi JF, Phatak PD, Coates TD. Outcomes, utilization, and costs among thalassemia and sickle cell disease patients receiving deferoxamine therapy in the United States. American Journal of Hematology 2008;83(4):263-70.
14. Elbourne 2002 Elbourne DR, Altman DG, Higgins JP, Curtin F, Worthington HV, Vail A. Meta-analyses involving cross-over trials: methodological issues. International Journal of Epidemiology 2002;31(1):140-9.
15. EMEA 2007 European Medicines Agency, London, UK. Exjade: European Public Assessment Report: Summary of Product Characteristics. www.emea.europa.eu/humandocs/Humans/EPAR/exjade/exjade.htm (accessed 14 February 2008).
16. Farmaki 2006 Farmaki K, Angelopoulos N, Anagnostopoulos G, Gotsis E, Rombopoulos G, Tolis G. Effect of enhanced iron chelation therapy on glucose metabolism in patients with beta-thalassaemia major. British Journal of Haematology 2006;134(4):438-44.
17. FDA 2005 Food, Drug Administration (FDA). USA. Exjade. www.fda.gov/cder/foi/label/2007/021882s002lbl/pdf (accessed 14 February 2008).
18. Freedman 1990 Freedman MH, Grisaru D, Olivieri N, MacLusky I, Thorner PS. Pulmonary syndrome in patients with thalassemia major receiving intravenous deferoxamine infusions. American Journal of Diseases of Children 1990;144(5):565-9.
19. Gabutti 1996 Gabutti V, Piga A. Results of long-term iron-chelating therapy. Acta Haematologica 1996;95(1):26-36.
20. Galanello 2006 Galanello R, Kattamis A, Piga A, Fischer R, Leoni G, Ladis V, et al.A prospective randomized controlled trial on the safety and efficacy of alternating deferoxamine and deferiprone in the treatment of iron overload in patients with thalassemia. Haematologica 2006;91(9): 1241-3.
21. Higgins 2002 Higgins JP, Thompson SG. Quantifying heterogeneity in a meta-analysis. Statistics in Medicine 2002;21(11):1539-58.
22. Higgins 2003 Higgins JP, Thompson SG, Deeks JJ, Altman DG. Measuring inconsistency in meta-analyses. BMJ 2003;327(7414):557- 60.
23. Higgins 2008 Higgins JPT, Altman DG (editors). Chapter 8: Assessing risk of bias in included studies. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.0.0 (updated February 2008). The Cochrane Collaboration, 2008. Available from www.cochrane-handbook.org. Chichester, UK: John Wiley & Sons, Ltd, 2008.
24. Hoffbrand 1989 Hoffbrand AV, Bartlett AN, Veys PA, O'Connor NT, Kontoghiorghes GJ. Agranulocytosis and thrombocytopenia in patient with Blackfan-Diamond anaemia during oral chelator trial. Lancet 1989;2(8660):457.
25. Hoffbrand 2003 Hoffbrand AV, Cohen A, Hershko C. Role of deferiprone in chelation therapy for transfusional iron overload. Blood 2003;102(1):17-24.
26. Kattamis 2003 Kattamis A, Kassou C, Berdousi H, Ladis V, Papassotiriou I, Kattamis C. Combined therapy with desferrioxamine and deferiprone in thalassemic patients: effect on urinary iron excretion. Haematologica 2003;88(12):1423-25.
27. Kolnagou 2008 Kolnagou A, Economides C, Eracleous E, Kontoghiorghes GJ. Long term comparative studies in thalassemia patients treated with deferoxamine or a deferoxamine/deferiprone combination. Identification of effective chelation therapy protocols. Hemoglobin 2008;32(1-2):41- 7.
28. Kontoghiorghes 1987a Kontoghiorghes GJ, Aldouri MA, Hoffbrand AV, Barr J, Wonke B, Kourouclaris T, et al.Effective chelation of iron in beta thalassaemia with the oral chelator 1,2-dimethyl-3-hydroxypyrid-4-one. BMJ (Clinical Research Edition) 1987;295(6612):1509-12.
29. Kontoghiorghes 1987b Kontoghiorghes GJ, Aldouri MA, Sheppard L, Hoffbrand AV. 1,2-Dimethyl-3-hydroxypyrid-4-one, an orally active chelator for treatment of iron overload. Lancet 1987;1(8545):1294-95.
30. Koren 1991 Koren G, Kochavi-Atiya Y, Bentur Y, Olivieri NF. The effects of subcutaneous deferoxamine administration on renal function in thalassemia major. International Journal of Hematology 1991;54(5):371-5.
31. Kushner 2001 Kushner JP, Porter JP, Olivieri NF. Secondary iron overload. Hematology. American Society of Hematology. Education Program 2001:47-61.
32. Maggio 2002 Maggio A, D'Amico G, Morabito A, Capra M, Ciaccio C, Cianciulli P, et al.Deferiprone versus deferoxamine in patients with thalassemia major: a randomized clinical trial. Blood Cells, Molecules and Diseases 2002;28(2):196-208.
33. Modell 2000 Modell B, Khan M, Darlison M. Survival in beta-thalassaemia major in the UK: data from the UK Thalassaemia Register. Lancet 2000;355(9220):2051-2.
34. Modell 2008 Modell B, Darlison M. Global epidemiology of haemoglobin disorders and derived service indicators. Bulletin of the World Health Organization 2008;86(6):480-7.
35. Nisbet-Brown 2003 Nisbet-Brown E, Olivieri NF, Giardina PJ, Grady RW, Neufeld EJ, Sechaud R, et al.Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: a randomised, double-blind, placebo-controlled, dose-escalation trial. Lancet 2003;361(9369): 1597-602.
36. Olivieri 1994 Olivieri NF, Nathan DG, MacMillan JH, Wayne AS, Liu PP, McGee A, et al.Survival inmedically treated patients with homozygous beta-thalassemia. The New England Journal of Medicine 1994;331(9):574-8.
37. Olivieri 1997 Olivieri NF, Brittenham GM. Iron-chelating therapy and the treatment of thalassemia. Blood 1997;89(3):739-61.
38. Olivieri 1998 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. The New England Journal of Medicine 1998;339(7):417-23.
39. Olivieri 1999 Olivieri NF. The beta-thalassemias. The New England Journal of Medicine 1999;341(2):99-109.
40. Origa 2005 Origa R, Bina P, Agus A, Crobu G, Defraia E, Dessi C, et al.Combined therapy with deferiprone and desferrioxamine in thalassemia major. Haematologica 2005;90(10):1309-14.
41. Parmar 1998 Parmar MK, Torri V, Stewart L. Extracting summary statistics to perform meta-analyses of the published literature for survival endpoints. Statistics in Medicine 1998;17(24):2815-34.
42. Peng 2008 Peng CT, Tsai CH, Wu KH. Effects of chelation therapy on cardiac function improvement in thalassemia patients: literature review and the Taiwanese experience. Hemoglobin 2008;32(1-2):49-62.
43. Review Manager 2008 The Nordic Cochrane Centre, The Cochrane Collaboration. Review Manager (RevMan). 5.0. Copenhagen: The Nordic Cochrane Centre, The Cochrane Collaboration, 2008.
44. Richer 2005 Richer J, Chudley AE. The hemoglobinopathies andmalaria. Clinical Genetics 2005;68(4):332-6.
45. Roberts 2005 Roberts DJ, Rees D, Howard J, Hyde C, Alderson P, Brunskill S. Desferrioxamine mesylate for managing transfusional iron overload in people with transfusion-dependent thalassaemia. Cochrane Database of Systematic Reviews 2005, Issue 4. [DOI: 10.1002/14651858.CD004450.pub2]
46. Roberts 2007 Roberts DJ, Brunskill S, Doree C, Williams S, Howard J, Hyde C. Oral deferiprone for iron chelation in people with thalassaemia. Cochrane Database of Systematic Reviews 2007, Issue 3. [DOI: 10.1002/14651858.CD004839.pub2]
47. Rund 2005 Rund D, Rachmilewitz E. Beta-thalassemia. The New England Journal of Medicine 2005;353(11):1135-46.
48. Taher 2006 Taher A, Isma'eel H, Cappellini MD. Thalassemia intermedia: revisited. Blood Cells, Molecules & Diseases 2006;37(1):12-20.
49. Tanner 2007 Tanner MA, Galanello R, Dessi C, Smith GC, Westwood MA, Agus A, et al.A randomized, placebo-controlled, double-blind trial of the effect of combined therapy with deferoxamine and deferiprone on myocardial iron in thalassemia major using cardiovascular magnetic resonance. Circulation 2007;115(14):1876-84.
50. Wanless 2002 Wanless IR, Sweeney G, Dhillon AP, Guido M, Piga A, Galanello R, et al.Lack of progressive hepatic fibrosis during long-term therapy with deferiprone in subjects with transfusion-dependent beta-thalassemia. Blood 2002;100(5):1566-9.
51. Weatherall 1998 Weatherall DJ. Thalassemia in the nextmillennium. Keynote address. Annals of the New York Academy of Sciences 1998; Vol. 850:1-9.
52. Weatherall 2000 Weatherall DJ, Clegg JB. The Thalassemia Syndromes. 4th Edition. Oxford: Blackwell Sciences Ltd., 2000.
53. Williamson 2002 Williamson PR, Smith CT, Hutton JL, Marson AG. Aggregate data meta-analysis with time-to-event outcomes. Statistics in Medicine 2002;21(22):3337-51.
54. Witte 2004 Witte S, Victor N. Some problems with the investigation of noninferiority in meta-analysis. Methods of Information in Medicine 2004;43(5):470-4.
55. Wu 2006 Wu SF, Peng CT, Wu KH, Tsai CH. Liver fibrosis and iron levels during long-term deferiprone treatment of thalassemia major patients. Hemoglobin 2006;30(2):215-8.
56. Zurlo 1989 Zurlo MG, De Stefano P, Borgna-Pignatti C, Di Palma A, Piga A, Melevendi C, et al.Survival and causes of death in thalassaemia major. Lancet 1989;2(8653):27-30.