, Volume 67, Issue 15, pp 2211–2230 | Cite as


A Review of its Use in the Management of Transfusional Chronic Iron Overload
  • Lily P. H. Yang
  • Susan J. Keam
  • Gillian M. Keating
Adis Drug Evaluation




Deferasirox (Exjade®) is an oral, once-daily iron chelator widely approved for the treatment of transfusional chronic iron overload. In the EU, deferasirox is indicated in patients with β-thalassaemia major aged ≥6 years and, in the US, in all transfusional chronic iron overload patients aged ≥2 years. Deferasirox is highly selective for iron as Fe3+.

In ≈1-year clinical trials of patients with transfusional chronic iron overload associated with β-thalassaemia, sickle cell disease, myelodysplastic syndrome or other rare chronic anaemias, deferasirox 20 or 30 mg/kg/day had a beneficial effect on liver iron concentrations (LIC) and serum ferritin levels; tolerability issues were clinically manageable with regular patient monitoring. Although longer-term efficacy and tolerability data are required, in particular examining the prevention of iron overload-related complications and the effect of deferasirox on renal function, deferasirox is an easily administered iron chelator and is a valuable option in the management of transfusional chronic iron overload.

Pharmacological Properties

Deferasirox is a tridentate iron chelator with high affinity for iron as Fe3+; two molecules of deferasirox bind to one Fe3+. In clinical trials, most (≈70%) patients with transfusional chronic iron overload treated with deferasirox 20 or 30 mg/kg/ day (the approved regimen) achieved net iron excretion or maintained iron balance. Preliminary data indicate that after treatment with deferasirox for ≈1 year, patients with transfusional chronic iron overload demonstrated an improvement in myocadial iron load and labile plasma iron.

The deferasirox maximum plasma concentration (Cmax) and the area under the plasma concentration-time curve (AUC) were dose dependent, although time to reach Cmax was independent of dose (1–2 hours). Time to steady state is 3 days, and at steady state, AUC was increased by an accumulation factor of 1.3–2.3. Deferasirox is mainly metabolised by hepatic glucuronidation and is excreted in bile. The mean elimination half-life of deferasirox is 7.2–18.5 hours and, although variable between study patients, permits once-daily administration. Deferasirox is predominantly excreted through the faecal route (83%), mostly as the unchanged drug. Renal excretion accounts for 6–8% of an administered dose. At therapeutic dosages, deferasirox did not induce or inhibit cytochrome P450 enzymes.

Therapeutic Efficacy

The iron chelation efficacy of deferasirox was investigated in five trials of ≈1 year’s duration in paediatric and adult patients, including a phase III noninferi-oritytrial. The underlying anaemic condition in study patients was predominantly β-thalassaemia major; other conditions included sickle cell disease, myelodysplastic syndrome, and Diamond-Blackfan and other rare anaemias.

In the phase III β-thalassaemia trial, dosages of deferasirox or deferoxamine were assigned according to baseline LIC. Although noninferiority to deferoxamine was not established in the overall study population, deferasirox was noninferior to deferoxamine in a predefined subgroup analysis of patients with higher baseline LIC (≥7mg Fe/g dry weight) who were treated with 20 or 30 mg/ kg/day. In addition, LIC was significantly reduced from baseline in this group of patients; reductions in serum ferritin levels were also observed, although no statistical analyses were reported. Limited efficacy data for deferasirox are available in the treatment of patients with sickle cell disease, myelodysplastic syndrome or other chronic rare anaemias; similar changes in LIC and serum ferritin levels to those seen in patients with β-thalassaemia were observed in patients with these conditions.

Preliminary data indicate that a larger number of patients indicated satisfaction, convenience and less time lost to treatment with deferasirox than deferox-amine. These data suggest that treatment with deferasirox may be beneficial in patients with a history of noncompliance to infusional deferoxamine.

Cost-utility analyses from a US or UK perspective in patients with β-thalassaemia, sickle cell disease or myelodysplastic syndrome suggest that deferasirox is a cost-effective treatment relative to branded or generic deferoxamine. Incremental quality-adjusted life years gained from treatment of patients with β-thalassaemia major with deferasirox relative to deferoxamine were mainly attributed to patient preference for an oral iron chelator.


Deferasirox was associated with a clinically manageable tolerability profile. The most common adverse events associated with deferasirox treatment included transient gastrointestinal adverse events (nausea, vomiting, abdominal pain, constipation and diarrhoea) and skin rash. Other adverse events include elevated serum creatinine, and less frequently, elevated ALT levels and visual and auditory disturbances; regular monitoring and careful management are recommended. Postmarketing cases of renal failure and cytopenia have been reported.


Unable to display preview. Download preview PDF.

Unable to display preview. Download preview PDF.


  1. 1.
    Rund D, Rachmilewitz E. β-Thalssemia. N Engl J Med 2005 Sep 15; 353(11): 1135–46PubMedCrossRefGoogle Scholar
  2. 2.
    Stuart MJ, Nagel R. Sickle-cell disease. Lancet 2004 Oct 9; 364(9442): 1343–60PubMedCrossRefGoogle Scholar
  3. 3.
    Neumeister P, Pestell R, Balent B, et al. Myelodysplastic syndrome: etiology, prognosis and management. Am J Cancer 2002; 1(5): 301–11CrossRefGoogle Scholar
  4. 4.
    Barton JC. Optimal management strategies for chronic iron overload. Therapy in Practice 2007; 67(5): 685–700Google Scholar
  5. 5.
    World Health Organization. Thalassaemia and other haemoglobinopathies [online]. Available from URL: http:// www.who.int/gb/ebwha/pdf_files/EB118/B118_5-en.pdf [Accessed 2007 Apr 27]Google Scholar
  6. 6.
    Kwiatkowski JL, Cohen AR. Iron chelation therapy in sickle-cell disease and other transfusion-dependent anemias. Hematol Oncol Clin North Am 2004; 18(6): 1355–77PubMedCrossRefGoogle Scholar
  7. 7.
    American Sickle Cell Anemia Association. How common is sickle cell anemia? [online]. Available from URL: http:// www.ascaa.org/comm.htm [Accessed 2007 Apr 27]Google Scholar
  8. 8.
    Quirolo K, Coates T, Singer T, et al. Clinical practice guidelines for the management of thalassemia patients California consensus [online]. Available from URL: http://www.tha-lassemia.com/Thal_SOC_guide.pdf [Accessed 2007 Mar 1]Google Scholar
  9. 9.
    Adams R, Ataga KI, Ballard H, et al. The management of sickle cell disease. National Institutes of Health, National Heart, Lung, and Blood Institute [online]. Available from URL: http:/ /www.nhlbi.nih.gov/health/prof/blood/sickle/sc_mngt.pdf [Accessed 2007 Jun 15]Google Scholar
  10. 10.
    Adams RJ, McKie VC, Hsu L, et al. Prevention of a first stroke by transfusions in children with sickle cell anemia and abnormal results on transcranial Doppler ultrasonography [published erratum appears in N Engl J Med 1998; 339 (20): 1478]. N Engl J Med 1998 Jul 2; 339(1): 5–11PubMedCrossRefGoogle Scholar
  11. 11.
    Adams RJ, Brambilla D,Optimizing Primary Stroke Prevention in Sickle Cell Anemia (STOP 2) Trial Investigators. Discontinuing prophylactic transfusions used to prevent stroke in sickle cell disease. N Engl J Med 2005 Dec 29; 353(26): 2769–78PubMedCrossRefGoogle Scholar
  12. 12.
    Vichinsky EP, Neumayr LD, Earles AN, et al. Causes and outcomes of the acute chest syndrome in sickle cell disease. N Engl J Med 2000 Jun 22; 342(25): 1855–65PubMedCrossRefGoogle Scholar
  13. 13.
    Kushner JP, Porter JP, Olivieri NF. Secondary iron overload. Hematology Am Soc Hematol Educ Program 2001: 47–61Google Scholar
  14. 14.
    European Medicines Agency. Summary of product characteristics (product information, Exjade) [online]. Available from URL: http://www.emea.europa.eu/humandocs/PDFs/EPAR/ exjade/H-670-PI-en.pdf [Accessed 2007 Aug 3]Google Scholar
  15. 15.
    Novartis Pharmaceuticals Corporation. Exjade® (deferasirox) tablets for oral suspension: prescribing information (US) [online]. Available from URL: http://www.fda.gov/cder/foi/label/ 2007/021882s002lbl.pdf [Accessed 2007 Apr 26]Google Scholar
  16. 16.
    Borgna-Pignatti C, Rugolotto S, De Stefano P, et al. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica 2004 Oct; 89(10): 1187–93PubMedGoogle Scholar
  17. 17.
    Olivieri NF, Nathan DG, MacMillan JH, et al. Survival in medically treated patients with homozygous β-thalassemia. N Engl J Med 1994 Sep 1; 331(9): 574–8PubMedCrossRefGoogle Scholar
  18. 18.
    Wood JC, Tyszka JM, Carson S, et al. Myocardial iron loading in transfusion-dependent thalassemia and sickle cell disease. Blood 2004 Mar 1; 103(5): 1934–6PubMedCrossRefGoogle Scholar
  19. 19.
    Raman SV, Simonetti OP, Cataland SR, et al. Myocardial ischemia and right ventricular dysfunction in adult patients with sickle cell disease. Haematologica 2006 Oct; 91(10): 1329–35PubMedGoogle Scholar
  20. 20.
    Konen E, Ghoti H, Goitein O, et al. No evidence for myocardial iron overload in multitransfused patients with myelodysplastic syndrome using cardiac magnetic resonance T2* technique. Am J Hematol. Epub 2007 Jul 25Google Scholar
  21. 21.
    Roberts DJ, Rees D, Howard J, et al. Desferrioxamine mesylate for managing transfusional iron overload in people with transfusion-dependent thalassaemia. Cochrane Database of Systematic Reviews 2005; (4): CD004450Google Scholar
  22. 22.
    Tavill AS. Diagnosis and management of hemochromatosis. Hepatology 2001; 33(5): 1321–8PubMedCrossRefGoogle Scholar
  23. 23.
    Novartis Pharmaceuticals Corporation. Desferal® (deferoxamine mesylate) for injection USP: prescribing information (US) [online]. Available from URL: http://www.pharma.us.-novartis.com/product/pi/pdf/desferal.pdf [Accessed 2007 Mar 9]Google Scholar
  24. 24.
    Brittenham GM, Griffith PM, Nienhuis AW, et al. Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major. N Engl J Med 1994 Sep 1; 331(9): 567–73PubMedCrossRefGoogle Scholar
  25. 25.
    Abetz L, Baladi J-F, Jones P, et al. The impact of iron overload and its treatment on quality of life: results from a literature review. Health Qual Life Outcomes 2006; 4(73): e1–6Google Scholar
  26. 26.
    Ceci A, Baiardi P, Catapano M, et al. Risk factors for death in patients with β-thalassemia major: results of a case-control study. Haematologica 2006; 91(10): 1420–1PubMedGoogle Scholar
  27. 27.
    European Medicines Agency. European public assessment report (EPAR): summary of product characteristics (Ferriprox) [online]. Available from URL: http://www.emea.eu.int/humandocs/PDFs/EPAR/Ferriprox/H-236-PI-en.pdf [Accessed 2007 Apr 17]Google Scholar
  28. 28.
    Roberts DJ, Brunskill SJ, Doree C, et al. Oral deferiprone for iron chelation in people with thalassaemia. Cochrane Database of Systematic Reviews 2007 Jul 18; (3): CD004839Google Scholar
  29. 29.
    Neufeld EJ. Oral chelators deferasirox and deferiprone for trans-fusional iron overload in thalassemia major: new data, new questions. Blood 2006 May 1; 107(9): 3436–41PubMedCrossRefGoogle Scholar
  30. 30.
    US Food and Drug Administration. FDA approves first oral drug for chronic iron overload [online]. Available from URL: http://www.fda.gov/bbs/topics/news/2005/NEW01258.html [Accessed 2007 Apr 1]Google Scholar
  31. 31.
    European Medicines Agency. Committee for Medicinal Products for Human Use summary of positive opinion for Exjade [online]. Available from URL: http://www.emea.europa.eu/ pdfs/human/opinion/22855406en.pdf [Accessed 2007 Aug 6]Google Scholar
  32. 32.
    Steinhauser S, Heinz U, Bartholoma M, et al. Complex formation of ICL670 and related ligands with FeIII and FeII [published erratum appears in Eur J Inorg Chem 2005; (11): 2262]. Eur J Inorg Chem 2004; (21): 4177–92Google Scholar
  33. 33.
    Piga A, Galanello R, Forni GL, et al. Randomized phase II trial of deferasirox (Exjade®, ICL670), a once-daily, orally-administered iron chelator, in comparison to deferoxamine in thalassemia patients with transfusional iron overload. Haematologica 2006 Jul; 91(7): 873–80PubMedGoogle Scholar
  34. 34.
    Nisbet-Brown E, Olivieri NF, Giardina PJ, et al. Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: a randomised, double-blind, placebo-controlled, dose-escalation trial. Lancet 2003 May 10; 361(9369): 1597–602PubMedCrossRefGoogle Scholar
  35. 35.
    Tanner MA, Galanello R, Dessi C, et al. Myocardial iron loading in patients with thalassemia major on deferoxamine chelation. J Cardiovasc Magn Resonance 2006; 8(3): 543–7CrossRefGoogle Scholar
  36. 36.
    Anderson LJ, Holden S, Davis B, et al. Cardiovascular T2-star (T2*) magnetic resonance for the early diagnosis of myocardial iron overload. Eur Heart J 2001 Dec; 22(23): 2171–9PubMedCrossRefGoogle Scholar
  37. 37.
    Esposito BP, Breuer W, Sirankapracha P, et al. Labile plasma iron in iron overload: redox activity and susceptibility to chelation. Blood 2003 Oct 1; 102(7): 2670–7PubMedCrossRefGoogle Scholar
  38. 38.
    Pootrakul P, Breuer W, Sametband M, et al. Labile plasma iron (LPI) as an indicator of chelatable plasma redox activity in iron-overloaded β-thalassemia/HbE patients treated with an oral chelator. Blood 2004 Sep 1; 104(5): 1504–10PubMedCrossRefGoogle Scholar
  39. 39.
    Cappellini MD, Cohen A, Piga A, et al. A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with β-thalassemia. Blood 2006 May 1; 107(9): 3455–62PubMedCrossRefGoogle Scholar
  40. 40.
    Galanello R, Piga A, Forni GL, et al. Phase II clinical evaluation of deferasirox, a once-daily oral chelating agent, in pediatric patients with β-thalassemia major. Haematologica 2006 Oct; 91(10): 1343–51PubMedGoogle Scholar
  41. 41.
    Glickstein H, El RB, Shvartsman M, et al. Intracellular labile iron pools as direct targets of iron chelators: a fluorescence study of chelator action in living cells. Blood 2005 Nov 1; 106(9): 3242–50PubMedCrossRefGoogle Scholar
  42. 42.
    Wood JC, Otto-Duessel M, Gonzalez I, et al. Deferasirox and deferiprone remove cardiac iron in the iron-overloaded gerbil. Transl Res 2006 Nov; 148(5): 272–80PubMedCrossRefGoogle Scholar
  43. 43.
    Porter JB, Tanner MA, Pennell DJ, et al. Improved myocardial T2* in transfusion dependent anemias receiving ICL670 (deferasirox) [abstract no. 3600]. Blood 2005 Nov 16; 106 (11 Pt 1): 1003aCrossRefGoogle Scholar
  44. 44.
    Eleftheriou P, Tanner M, Pennell D, et al. Response of myocardial T2* to oral deferasirox monotherapy for 1 year in 29 patients with transfusion-dependent anaemias: a subgroup analysis [abstract no. 0999]. 1 1th Congress of the European Hem-atology Association; 2006 Jun 15–18; AmsterdamGoogle Scholar
  45. 45.
    Daar S, Taher A, Pathare A, et al. Sustained protection from labile plasma iron (LPI) with the once-daily, oral iron chelator deferasirox (Exjade®, ICL670) in iron-overloaded β-thalassemia patients [abstract no. 1773]. Blood 2006 Nov 16; 108 (11 Pt 1): 503aGoogle Scholar
  46. 46.
    Porter J, Vichinsky E, Rose C, et al. A phase II study with ICL670 (Exjade®), a once-daily oral iron chelator, in patients with various transfusion-dependent anemias and iron overload [abstract no. 3193]. Blood 2004 Nov 16; 104 (11 Pt 1): 872a. Plus poster presented at the 46th Annual Meeting and Exposition of the American Society of Hematology; 2004Dec 4–7; San Diego (CA)Google Scholar
  47. 47.
    Galanello R, Piga A, Alberti D, et al. Safety, tolerability, and pharmacokinetics of ICL670, a new orally active iron-chelating agent in patients with transfusion-dependent iron overload due to beta-thalassemia. J Clin Pharmacol 2003 Jun; 43(6): 565–72PubMedGoogle Scholar
  48. 48.
    Porter JB, Waldmeier F, Bruin G, et al. Pharmacokinetics, metabolism and elimination of the iron chelator drug ICL670 in beta-thalassemia patients [abstract no. 3720]. Blood 2003 Nov 16; 102 (11 Pt 2): 5bCrossRefGoogle Scholar
  49. 49.
    Sechaud R, Belleli R, Balez S, et al. Single oral dose of ICL670 has no effect on safety, tolerability and steady state pharmacokinetics of digoxin in healthy volunteers. Haematologica 2005 Jun; 90 Suppl. 2: 436–7Google Scholar
  50. 50.
    Olivieri NF, Brittenham GM. Iron-chelating therapy and the treatment of thalassemia [published addendum appears in Blood 1997; 89 (7): 2621]. Blood 1997 Feb 1; 89(3): 739–61PubMedGoogle Scholar
  51. 51.
    Vichinsky E, Onyekwere O, Porter J, et al. A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease. Br J Haematol 2007 Feb; 136(3): 501–8PubMedCrossRefGoogle Scholar
  52. 52.
    Brittenham GM, Sheth S, Allen CJ, et al. Noninvasive methods for quantitative assessment of transfusional iron overload in sickle cell disease. Semin Hematol 2001 Jan; 38 (1 Suppl. 1): 37–56PubMedCrossRefGoogle Scholar
  53. 53.
    European Medicines Agency. European public assessment report (EPAR): scientific discussion [online]. Available from URL: http://www.emea.europa.eu/humandocs/PDFs/EPAR/ exjade/H-670-en6.pdf [Accessed 2007Aug 3]Google Scholar
  54. 54.
    Shashaty G. Food and Drug Administration Blood Products Advisory Committee: FDA briefing document (clinical review) [online]. Available from URL: http://www.fda.gov/ ohrms/dockets/ac/05/briefing/2005-4177B1_02_b.pdf [Accessed 2007 Apr 23]Google Scholar
  55. 55.
    Cappellini MD, Bejaoui M, Agaoglu L, et al. Prospective evaluation of patient-reported outcomes during treatment with deferasirox or deferoxamine for iron overload in patients with β-thalassemia. Clin Ther 2007 May; 29(5): 909–17PubMedCrossRefGoogle Scholar
  56. 56.
    Vichinsky E, Fischer R, Pakbaz Z et al. Satisfaction and convenience of chelation therapy in patients with sickle cell disease (SCD): comparison between deferasirox (Exjade®, ICL670) and deferoxamine (DFO) [abstract no. 2334]. Blood 2005 Nov 16; 106 (11 Pt 1): 656–7a. Plus poster presented at the 47th Annual Meeting and Exposition of the American Society of Hematology; 2005 Dec 10–13; Atlanta (GA)Google Scholar
  57. 57.
    Porter JB, Cohen A, Agaoglu L, et al. Long-term effects of deferasirox (Exjade®) on serum ferritin: outcome of dose adjustments in achieving maintenance or reduction in body iron stores [abstract no. 1769]. Blood 2006 Nov 16; 108 (11 Pt 1): 502a. Plus poster presented at the 48th Annual Meeting of the American Society of Hematology; 2006 Dec 9–12; Orlando (FL)CrossRefGoogle Scholar
  58. 58.
    Piga A, Bejaoui M, Kilinc Y, et al. Long-term treatment with the once-daily oral iron chelator deferasirox (Exjade®, ICL670) is effective and generally well tolerated in pediatric patients [abstract no. 1781]. Blood 2006 Nov; 108 (11 Pt 1): 506a. Plus poster presented at the 48th Annual Meeting and Exposition of the American Society of Hematology; 2006 Dec 9–12; Orlando (FL)Google Scholar
  59. 59.
    Delea TE, Sofrygin O, Thomas SK, et al. Cost effectiveness of once-daily oral chelation therapy with deferasirox versus infusional deferoxamine in transfusion-dependent thalassaemia patients: US Healthcare System Perspective. Pharmacoeco-nomics 2007; 25(4): 329–42CrossRefGoogle Scholar
  60. 60.
    Delea E, Sofrygin O, Baladi JF, et al. Chelation therapy with deferasirox versus deferoxamine in transfusion-dependent sickle-cell disease: a cost-effectiveness analysis from the US perspective [abstract no. 3]. Haematologica 2006 Jun; 91 Suppl. 1: 2Google Scholar
  61. 61.
    Delea E, Baladi JF, Thomas SK, et al. Cost-effectiveness of chelation therapy with deferasirox versus deferoxamine in transfusion-dependent myelodysplastic syndrome [abstract no. 952]. Haematologica 2006 Jun; 91 Suppl. 1: 348Google Scholar
  62. 62.
    Karnon J, Akehurst RL, Jewitt K, et al. Cost-utility analysis of deferasirox versus deferoxamine (Desferal) for patients requiring iron chelation therapy in the United Kingdom [abstract no. 598]. Haematologica 2007 Jun; 92 Suppl. 1: 222–3. Plus poster presented at the 12th Congress of the European Hematology Association; 2007 Jun 7–10; ViennaGoogle Scholar
  63. 63.
    Jönsson B. Changing health environment: the challenge to demonstrate cost-effectiveness of new compounds. Pharmacoeconomics 2004; 22 Suppl. 4: 5–10PubMedCrossRefGoogle Scholar
  64. 64.
    Cappellini MD, Giardina P, Porter T, et al. Long-term safety and tolerability of the once-daily, oral iron chelator deferasirox (Exjade®, ICL670) in patients with transfusional iron overload [abstract no. 1768]. Blood 2006 Nov; 108 (11 Pt 1): 501a. Plus poster presented at the 48th Annual Meeting and Exposition of the American Society of Hematology; 2006 Dec 9–12; Orlando (FL)CrossRefGoogle Scholar
  65. 65.
    Cappellini MD. Deferasirox (ICL670) is clinically effective and generally well tolerated, and demonstrates dose-responsiveness [letter]. Blood 2006 Jul 15; 108(2): 775–6Google Scholar
  66. 66.
    Hohneker JA. Important information about Exjade® (deferasirox) tablets for oral suspension: important drug warning [online]. Available from URL: http://www.fda.gov/medwatch/ safety/2007/Exjade_DHCPL_May2007.pdf [Accessed 2007 Aug3]Google Scholar
  67. 67.
    Greenberg PL. Myelodysplastic syndromes: iron overload consequences and current chelating therapies. J Natl Compr Canc Netw 2006 Jan; 4(1): 91–6PubMedGoogle Scholar
  68. 68.
    Splendiani G, Tozzo C, Mazzarella V, et al. Deferoxamine in thalassemia major [letter]. N Engl J Med 1995 Jan 26; 332(4): 270–1PubMedCrossRefGoogle Scholar
  69. 69.
    Cable R, Carlson B, Chambers L, et al. Practice guidelines for blood transfusion: a compilation from recent peer-reviewed literature [online]. Available from URL: www.newengland-blood.org/professional/pgbtscreen.pdf [Accessed 2007 Mar 1]Google Scholar
  70. 70.
    Lane PA, Buchanan GR, Hutter JJ, et al. Sickle cell disease in children and adolescents: diagnosis, guidelines for comprehensive care, and care paths and protocols for management of acute and chronic complications [online]. Available from URL: http://www.scinfo.org/Protocol-2002.PDF [Accessed 2007 Jun 15]Google Scholar
  71. 71.
    Myelodysplastic Syndromes Foundation I. Transfusion-dependent iron overload and MDS: a handbook for patients [online]. Available from URL: www.mds-foundation.org/pdf/iron-overload-2ndedition.pdf [Accessed 2007 Mar 1]Google Scholar
  72. 72.
    Arboretti R, Tognoni G, Alberti D. Pharmacosurveillance and quality of care of thalassaemic patients. Eur J Clin Pharmacol 2001 Mar; 56(12): 915–22PubMedCrossRefGoogle Scholar
  73. 73.
    Modell B, Khan M, Darlison M. Survival in β-thalassaemia major in the UK: data from the UK Thalassaemia Register. Lancet 2000 Jun 10; 355(9220): 2051–2PubMedCrossRefGoogle Scholar
  74. 74.
    Angelucci E, Brittenham GM, McLaren CE, et al. Hepatic iron concentration and total body iron stores in thalassemia major [published erratum appears in N Engl J Med 2000 Dec 7; 343 (23): 1740]. N Engl J Med 2000 Aug 3; 343(5): 327–31PubMedCrossRefGoogle Scholar
  75. 75.
    Nielsen P, Güunther U, Dürken M, et al. Serum ferritin iron in iron overload and liver damage: correlation to body iron stores and diagnostifc relevance. J Lab Clin Med 2000; 135(5): 413–8PubMedCrossRefGoogle Scholar
  76. 76.
    Fischer R, Harmatz P, Nielsen P. Does liver biopsy overestimate liver iron concentration? [letter]. Blood 2006 Sep 1; 108(5): 1775–6PubMedCrossRefGoogle Scholar
  77. 77.
    Cappellini MD. Deferasirox (ICL670) effectively reduces liver iron concentration, whether assessed by biopsy or SQUID [letter]. Blood 2006 Sep 1; 108(5): 1776CrossRefGoogle Scholar
  78. 78.
    Pazdur R. Session II: Exjade® phase 4 requirements [online]. Available from URL: http://www.fda.gov/ohrms/dockets/AC/ 06/briefing/2006_4204B1_05_FDA.Exjade.pdf [Accessed 2007 Mar 1]Google Scholar

Copyright information

© Adis Data Information BV 2007

Authors and Affiliations

  • Lily P. H. Yang
    • 1
    • 2
  • Susan J. Keam
    • 1
    • 2
  • Gillian M. Keating
    • 1
    • 2
  1. 1.Wolters Kluwer Health ¦ AdisMairangi Bay, North Shore 0754, AucklandNew Zealand
  2. 2.Wolters Kluwer HealthConshohockenUSA

Personalised recommendations