Deferasirox for managing transfusional iron overload in people with sickle cell disease
Referencias
Additional references
Adams RJ, McKie VC, Hsu L, Files B, Vichinsky E, Pegelow C, et al. Prevention of a first stroke by transfusions in children with sickle cell anemia and abnormal results on transcranial Doppler ultrasonography. The New England Journal of Medicine 1998;339(1):5‐11.
Adams RJ, Brambilla D. Discontinuing prophylactic transfusions used to prevent stroke in sickle cell disease. The New England Journal of Medicine 2005;353(26):2769‐78.
Angastiniotis M, Modell B. Global epidemiology of hemoglobin disorders. Annals of the New York Academy of Sciences 1998;850:251‐69.
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.
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.
Cappellini MD. Overcoming the challenge of patient compliance with iron chelation therapy. Seminars in Hematology 2005;42(2 Suppl 1):S19‐21. [MEDLINE: 8]
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.
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.
Ceci A, Baiardi P, Felisi M, Cappellini MD, Carnelli V, De S, 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.
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.
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.
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.
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.
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.
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).
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.
Food, Drug Administration (FDA). USA. Exjade. www.fda.gov/cder/foi/label/2007/021882s002lbl/pdf (accessed 14 February 2008).
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.
Gabutti V, Piga A. Results of long‐term iron‐chelating therapy. Acta Haematologica 1996;95(1):26‐36.
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.
Higgins JP, Thompson SG. Quantifying heterogeneity in a meta‐analysis. Statistics in Medicine 2002;21(11):1539‐58.
Higgins JPT, Thompson SG, Deeks JJ, Altman DG. Measuring inconsistency in meta‐analyses. BMJ 2003;327(7414):557‐60.
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.
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.
Hoffbrand AV, Cohen A, Hershko C. Role of deferiprone in chelation therapy for transfusional iron overload. Blood 2003;102(1):17‐24.
Josephson CD, Su LL, Hillyer KL, Hillyer CD. Transfusion in the patient with sickle cell disease: a critical review of the literature and transfusion guidelines. Transfusion Medicine Reviews 2007;21(2):118‐33.
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):1423‐5.
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.
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.
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):1294‐5.
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.
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 & Diseases 2002;28(2):196‐208.
Miller ST, Sleeper LA, Pegelow CH, Enos LE, Wang WC, Weiner SJ, et al. Prediction of adverse outcomes in children with sickle cell disease. The New England Journal of Medicine 2000;342(2):83‐9.
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.
Modell B, Darlison M. Global epidemiology of haemoglobin disorders and derived service indicators. Bulletin of the World Health Organization 2008;86(6):480‐7.
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.
Olivieri NF, Nathan DG, MacMillan JH, Wayne AS, Liu PP, McGee A, et al. Survival in medically treated patients with homozygous beta‐thalassemia. The New England Journal of Medicine 1994;331(9):574‐8.
Olivieri NF, Brittenham GM. Iron‐chelating therapy and the treatment of thalassemia. Blood 1997;89(3):739‐61.
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.
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.
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.
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.
Platt OS, Thorington BD, Brambilla DJ, Milner PF, Rosse WF, Vichinsky E, et al. Pain in sickle cell disease. Rates and risk factors. The New England Journal of Medicine 1991;325(1):11‐6.
Powars DR, Chan LS, Hiti A, Ramicone E, Johnson C. Outcome of sickle cell anemia: a 4‐decade observational study of 1056 patients. Medicine (Baltimore) 2005;84(6):363‐76.
The Nordic Cochrane Centre, The Cochrane Collaboration. Review Manager (RevMan) [Computer program]. Version 5.0. Copenhagen: The Nordic Cochrane Centre, The Cochrane Collaboration, 2008.
Richer J, Chudley AE. The hemoglobinopathies and malaria. Clinical Genetics 2005;68(4):332‐6.
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]
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]
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.
Vichinsky E, Butensky E, Fung E, Hudes M, Theil E, Ferrell L, et al. Comparison of organ dysfunction in transfused patients with SCD or beta thalassemia. American Journal of Hematology 2005;80(1):70‐4.
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.
Weatherall DJ. Thalassemia in the next millennium. Keynote address. Annals of the New York Academy of Sciences 1998;850:1‐9.
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.
Witte S, Victor N. Some problems with the investigation of noninferiority in meta‐analysis. Methods of Information in Medicine 2004;43(5):470‐4.
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.
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.
