Combined chelation therapy with deferasirox and deferoxamine in thalassemia

Blood Cells, Molecules, and Diseases

Volumn 50, Issue 2, 2013, Pages 99-104

  Lal, Ashutosh ^a   Porter, John ^b   Sweeters, Nancy ^a   Ng, Vivian ^a   Evans, Patricia ^b   Neumayr, Lynne ^a   Kurio, Gregory ^a   Harmatz, Paul ^a   Vichinsky, Elliott ^a  

^a CHILDREN S HOSPITAL OAKLAND   (United States)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

Deferasirox; Deferoxamine; Iron overload; Thalassemia major

Indexed keywords

DEFERASIROX; DEFEROXAMINE; FERRITIN; IRON; TRANSFERRIN;

ABDOMINAL PAIN; ADOLESCENT; ADULT; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ARTICLE; ASCITES; CHELATION THERAPY; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; CREATININE BLOOD LEVEL; DIARRHEA; DRUG EFFICACY; DRUG FATALITY; DRUG INFUSION; DRUG SAFETY; HUMAN; IRON BLOOD LEVEL; IRON OVERLOAD; LEUKOCYTOSIS; LIVER TOXICITY; MONOTHERAPY; NEPHROTOXICITY; ORGAN INJURY; PHASE 2 CLINICAL TRIAL; PILOT STUDY; PRIORITY JOURNAL; SCHOOL CHILD; SIDE EFFECT; THALASSEMIA;

ADOLESCENT; ADULT; BENZOATES; CHELATION THERAPY; CHILD; DEFEROXAMINE; DRUG SYNERGISM; FEMALE; FERRITINS; HUMANS; IRON; IRON CHELATING AGENTS; IRON OVERLOAD; LIVER; MALE; MYOCARDIUM; PILOT PROJECTS; THALASSEMIA; TRANSFERRIN; TRIAZOLES; YOUNG ADULT;

EID: 84871941628     PISSN: 10799796     EISSN: 10960961     Source Type: Journal    
DOI: 10.1016/j.bcmd.2012.10.006     Document Type: Article

References (38)

1. Rund D., Rachmilewitz E. Beta-thalassemia. N. Engl. J. Med. 2005, 353:1135-1146.
2. Brittenham G.M., Griffith P.M., Nienhuis A.W., et al. Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major. N. Engl. J. Med. 1994, 331:567-573.
3. Cohen A.R., Galanello R., Piga A., De Sanctis V., Tricta F. Safety and effectiveness of long-term therapy with the oral iron chelator deferiprone. Blood 2003, 102:1583-1587.
4. Cappellini M.D., Cohen A., Piga A., et al. A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood 2006, 107:3455-3462.
5. 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, 89:1187-1193.
6. Kirk P., Roughton M., Porter J.B., et al. Cardiac T2* magnetic resonance for prediction of cardiac complications in thalassemia major. Circulation 2009, 120:1961-1968.
7. Davis B.A., Porter J.B. Long-term outcome of continuous 24-hour deferoxamine infusion via indwelling intravenous catheters in high-risk β-thalassemia. Blood 2000, 95:1229-1236.
8. Taher A., Cappellini M.D., Vichinsky E., et al. Efficacy and safety of deferasirox doses of >30mg/kg per d in patients with transfusion-dependent anaemia and iron overload. Br. J. Haematol. 2009, 147:752-759.
9. Tanner M.A., Galanello R., Dessi C., 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, 115:1876-1884.
10. Evans P., Kayyali R., Hider R.C., Eccleston J., Porter J.B. Mechanisms for the shuttling of plasma non-transferrin-bound iron (NTBI) onto deferoxamine by deferiprone. Transl. Res. 2010, 156:55-67.
11. Glickstein H., El R.B., Link G., et al. Action of chelators in iron-loaded cardiac cells: accessibility to intracellular labile iron and functional consequences. Blood 2006, 108:3195-3203.
12. Hoyes K.P., Porter J.B. 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.
13. Porter J.B., Rafique R., Srichairatanakool S., et al. Recent insights into interactions of deferoxamine with cellular and plasma iron pools: implications for clinical use. Ann. N. Y. Acad. Sci. 2005, 1054:155-168.
14. De Domenico I., Ward D.M., Kaplan J. Specific iron chelators determine the route of ferritin degradation. Blood 2009, 114:4546-4551.
15. Fischer R., Piga A., Harmatz P., Nielsen P. Monitoring long-term efficacy of iron chelation treatment with biomagnetic liver susceptometry. Ann. N. Y. Acad. Sci. 2005, 1054:350-357.
16. Evans R.W., Rafique R., Zarea A., et al. Nature of non-transferrin-bound iron: studies on iron citrate complexes and thalassemic sera. J. Biol. Inorg. Chem. 2008, 13:57-74.
17. Silva A.M., Hider R.C. Influence of non-enzymatic post-translation modifications on the ability of human serum albumin to bind iron. Implications for non-transferrin-bound iron speciation. Biochim. Biophys. Acta 2009, 1794:1449-1458.
18. Singh S., Hider R.C., Porter J.B. A direct method for quantification of non-transferrin-bound iron. Anal. Biochem. 1990, 186:320-323.
19. Zanninelli G., Breuer W., Cabantchik Z.I. Daily labile plasma iron as an indicator of chelator activity in Thalassaemia major patients. Br. J. Haematol. 2009, 147:744-751.
20. Esposito B.P., Breuer W., Sirankapracha P., et al. Labile plasma iron in iron overload: redox activity and susceptibility to chelation. Blood 2003, 102:2670-2677.
21. Porter J.B. Optimizing iron chelation strategies in beta-thalassaemia major. Blood Rev. 2009, 23:S3-S7.
22. Cohen A.R., Glimm E., Porter J.B. Effect of transfusional iron intake on response to chelation therapy in beta-thalassemia major. Blood 2008, 111:583-587.
23. Wood J.C., Kang B.P., Thompson A., et al. The effect of deferasirox on cardiac iron in thalassemia major: impact of total body iron stores. Blood 2010, 116:537-543.
24. Hoffbrand A.V., AL-Refaie F., Davis B., et al. Long-term trial of deferiprone in 51 transfusion-dependent iron overloaded patients. Blood 1998, 91:295-300.
25. Cappellini M.D., Bejaoui M., Agaoglu L., et al. Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5years' follow-up. Blood 2011, 118:884-893.
26. Taher A., Elalfy M.S., Al Zir K., et al. Importance of optimal dosing ≥30mg/kg/d during deferasirox treatment: 2.7-yr follow-up from the ESCALATOR study in patients with beta-thalassaemia. Eur. J. Haematol. 2011, 87:355-365.
27. Pennell D.J., Porter J.B., Cappellini M.D., et al. Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with beta-thalassemia major. Haematologica 2012, 10.3324/haematol.2011.049957.
28. Grady R.W., Galanello R., Randolph R.E., et al. Toward optimizing the use of deferasirox: potential benefits of combined use with deferoxamine. Haematologica 2012, 10.3324/haematol.2012.070607.
29. Porter J.B., Abeysinghe R.D., Marshall L., Hider R.C., Singh S. Kinetics of removal and reappearance of non-transferrin-bound plasma iron with deferoxamine therapy. Blood 1996, 88:705-713.
30. Walter P.B., Macklin E.A., Porter J., et al. Inflammation and oxidant-stress in beta-thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trial. Haematologica 2008, 93:817-825.
31. Aydinok Y., Evans P., Manz C.Y., Porter J.B. Timed non-transferrin bound iron determinations probe the origin of chelatable iron pools during deferiprone regimens and predict chelation response. Haematologica 2012, 97:835-841.
32. Cabantchik Z.I., Breuer W., Zanninelli G., Cianciulli P. LPI-labile plasma iron in iron overload. Best Pract. Res. Clin. Haematol. 2005, 18:277-287.
33. Daar S., Pathare A., Nick H., et al. Reduction in labile plasma iron during treatment with deferasirox, a once-daily oral iron chelator, in heavily iron-overloaded patients with beta-thalassaemia. Eur. J. Haematol. 2009, 82:454-457.
34. Porter J.B., Lin K.H., Beris P., et al. Response of iron overload to deferasirox in rare transfusion-dependent anaemias: equivalent effects on serum ferritin and labile plasma iron for haemolytic or production anaemias. Eur. J. Haematol. 2011, 87:338-348.
35. Pootrakul P., Breuer W., Sametband M., et al. Labile plasma iron (LPI) as an indicator of chelatable plasma redox activity in iron-overloaded {beta}-thalassemia/HbE patients treated with an oral chelator. Blood 2004, 104:1504-1510.
36. Pennell D.J., Porter J.B., Cappellini M.D., et al. Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia. Blood 2010, 115:2364-2371.
37. Vichinsky E. Clinical application of deferasirox: practical patient management. Am. J. Hematol. 2008, 83:398-402.
38. Galanello R., Agus A., Campus S., et al. Combined iron chelation therapy. Ann. N. Y. Acad. Sci. 2010, 1202:79-86.