Safety and efficacy of iron chelation therapy with deferiprone in patients with transfusion-dependent thalassemia

Therapeutic Advances in Hematology

Volumn 3, Issue 5, 2012, Pages 299-307

  Jamuar, Saumya S ^a   Lai, Angeline H M ^b  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b KK Hospital   (Singapore)

Author keywords

deferiprone; iron chelation; iron overload; thalassemia

Indexed keywords

DEFERIPRONE; DEFEROXAMINE; FERRITIN; IRON; ZINC;

ABDOMINAL PAIN; AGRANULOCYTOSIS; ARTHRITIS; ARTHROPATHY; BLOOD CELL COUNT; BLOOD TRANSFUSION; BREAST FEEDING; CARDIOVASCULAR MAGNETIC RESONANCE; CONCENTRATION (PARAMETERS); DRUG ABSORPTION; DRUG BIOAVAILABILITY; DRUG EFFICACY; DRUG GLUCURONIDATION; DRUG METABOLISM; DRUG SAFETY; ENDOCRINE DISEASE; FERRITIN BLOOD LEVEL; HOMEOSTASIS; HUMAN; HYPERTRANSAMINASEMIA; IRON CHELATION; LONG TERM SURVIVAL; NAUSEA; NEUROTOXICITY; NEUTROPENIA; PRIORITY JOURNAL; REVIEW; SIDE EFFECT; THALASSEMIA; THROMBOCYTOPENIA; TRANSFUSION DEPENDENT THALASSEMIA; TREATMENT RESPONSE; ZINC BLOOD LEVEL;

EID: 84993724894     PISSN: 20406207     EISSN: 20406215     Source Type: Journal    
DOI: 10.1177/2040620712450252     Document Type: Review

References (50)

1. Aessopos A. Berdoukas V. Tsironi M. (2008) The heart in transfusion dependent homozygous thalassaemia today-prediction, prevention and management. Eur J Haematol 80: 93–106.
2. Alpendurada F. Smith G.C. Carpenter J.-P. Nair S.V. Tanner M.A. Banya W. et al (2012) Effects of combined deferiprone with deferoxamine on right ventricular function in thalassaemia major. J Cardiovasc Magn Reson 14:8.
3. Al-Refaie F.N. Hershko C. Hoffbrand A.V. Kosaryan M. Olivieri N.F. Tondury P. et al (1995 a) Results of long-term deferiprone (L1) therapy: a report by the International Study Group on Oral Iron Chelators. Br J Haematol 91: 224–229.
4. Al-Refaie F.N. Sheppard L.N. Nortey P. Wonke B. Hoffbrand A.V. (1995 b) Pharmacokinetics of the oral iron chelator deferiprone (L1) in patients with iron overload. Br J Haematol 89: 403–408.
5. Anderson L.J. Holden S. Davis B. Prescott E. Charrier C.C. Bunce N.H. et al (2001) Cardiovascular T2-star (T2*) magnetic resonance for the early diagnosis of myocardial iron overload. Eur Heart J 22: 2171–2179.
6. Anderson L.J. Wonke B. Prescott E. Holden S. Malcolm Walker J. Pennell D.J. (2002) Comparison of effects of oral deferiprone and subcutaneous desferrioxamine on myocardial iron concentrations and ventricular function in beta-thalassaemia. Lancet 360: 516–520.
7. Bartakke S. Bavdekar S.B. Kondurkar P. Muranjan M.N. Manglani M.V. Sharma R. (2005) Effect of deferiprone on urinary zinc excretion in multiply transfused children with thalassemia major. Indian Pediatr 42: 150–154.
8. Beau-Salinas F. Guitteny M.A. Donadieu J. Jonville-Bera A.P. Autret-Leca E. (2009) High doses of deferiprone may be associated with cerebellar syndrome. BMJ 338:653.
9. Berkovitch M. Laxer R.M. Koren G. Inman R. Olivieri N.F. Laxer R.M. et al (1994) Arthropathy in thalassaemia patients receiving deferiprone. Lancet 343: 1471–1472.
10. Borgna-Pignatti C. Cappellini M.D. de Stefano P. Del Vecchio G.C. Forni G.L. Gamberini M.R. et al (2006) Cardiac morbidity and mortality in deferoxamine- or deferiprone-treated patients with thalassemia major. Blood 107: 3733–3737.
11. Ceci A. Baiardi P. Catapano M. Felisi M. Cianciulli P. de Sanctis V. et al (2006) Risk factors for death in patients with beta-thalassemia major: results of a case–control study. Haematologica 91: 1420–1421.
12. Ceci A. Baiardi P. Felisi M. Cappellini M.D. Carnelli V. de Sanctis V. et al (2002) The safety and effectiveness of deferiprone in a large-scale, 3-year study in Italian patients. Br J Haematol 118: 330–336.
13. Christoforidis A. Perifanis V. Spanos G. Vlachaki E. Economou M. Tsatra I. et al (2009) MRI assessment of liver iron content in thalassamic patients with three different protocols: comparisons and correlations. Eur J Haematol 82: 388–392.
14. Cohen A.R. Galanello R. Piga A. de Sanctis V. Tricta F. (2003) Safety and effectiveness of long-term therapy with the oral iron chelator deferiprone. Blood 102: 1583–1587.
15. Collins A.F., Fassos F.F., Stobie S., Lewis N., Shaw D., Fry M., et al (1994) Iron-balance and dose-response studies of the oral iron chelator 1,2- dimethyl-3-hydroxypyrid-4-one (L1) in iron-loaded patients with sickle cell disease. Blood 83: 2329–2333.
16. de Sanctis V. Eleftheriou A. Malaventura C. (2004) Prevalence of endocrine complications and short stature in patients with thalassaemia major: a multicenter study by the Thalassaemia International Federation (TIF). Pediatr Endocrinol Rev 2: 249–255.
17. El Alfy M. Sari T.T. Lee C.L. Tricta F. El-Beshlawy A. (2010) The safety, tolerability, and efficacy of a liquid formulation of deferiprone in young children with transfusional iron overload. J Pediatr Hematol Oncol 32: 601–605.
18. Farmaki K. Tzoumari I. Pappa C. (2011) Oral chelators in transfusion-dependent thalassemia major patients may prevent or reverse iron overload complications. Blood Cells Mol Dis 47: 33–40.
19. Galanello R. (2007) Deferiprone in the treatment of transfusion-dependent thalassemia: a review and perspective. Ther Clin Risk Manag 3: 795–805.
20. Gamberini M.R. de Sanctis V. Gilli G. (2008) Hypogonadism, diabetes mellitus, hypothyroidism, hypoparathyroidism: incidence and prevalence related to iron overload and chelation therapy in patients with thalassaemia major followed from 1980 to 2007 in the Ferrara Centre. Pediatr Endocrinol Rev 6: 158–169.
21. Gardenghi S. Marongiu M.F. Ramos P. Guy E. Breda L. Chadburn A. et al (2007) Ineffective erythropoiesis in beta-thalassemia is characterized by increased iron absorption mediated by down-regulation of hepcidin and up-regulation of ferroportin. Blood 109: 5027–5035.
22. Henter J.-I. Karlén J. (2007) Fatal agranulocytosis after deferiprone therapy in a child with Diamond-Blackfan anemia. Blood 109: 5157–5159.
23. Hoffbrand A.V. Al-Refaie F. Davis B. Siritanakatkul N. Jackson B.F. Cochrane J. et al (1998) Long-term trial of deferiprone in 51 transfusion-dependent iron overloaded patients. Blood 91: 295–300.
24. Hoffbrand A.V. Cohen A. Hershko C. (2003) Role of deferiprone in chelation therapy for transfusional iron overload. Blood 102: 17–24.
25. Jamuar S.S. Lai A.H.M. Tan A.M. Chan M.Y. Tan E.S. Ng I.S.L. (2011) Use of deferiprone for iron chelation in patients with transfusion-dependent thalassaemia. J Paediatr Child Health 47: 812–817.
26. Kontoghiorghes G.J. Aldouri M.A. Sheppard L. Hoffbrand A.V. (1987) 1,2-Dimethyl-3-hydroxypyrid-4-one, an orally active chelator for treatment of iron overload. Lancet 1: 1294–1295.
27. Maggio A. D'Amico G. Morabito A. Capra M. Ciaccio C. Cianciulli P. et al (2002) Deferiprone versus deferoxamine in patients with thalassemia major: a randomized clinical trial. Blood Cells Mol Dis 28: 196–208.
28. Maggio A. Filosa A. Vitrano A. Aloj G. Kattamis A. Ceci A. et al (2011) Iron chelation therapy in thalassemia major: a systematic review with meta-analyses of 1520 patients included on randomized clinical trials. Blood Cells Mol Dis 47: 166–175.
29. Masera N. Tavecchia L. Longoni D.V. Maglia O. Biondi A. Masera G. (2011) Agranulocytosis due to deferiprone: a case report with cytomorphological and functional bone marrow examination. Blood Transfus 9: 462–465.
30. Modell B. Khan M. Darlison M. (2000) Survival in beta-thalassaemia major in the UK: data from the UK Thalassaemia Register. Lancet 355: 2051–2052.
31. Naithani R. Chandra J. Sharma S. (2005) Safety of oral iron chelator deferiprone in young thalassaemics. Eur J Haematol 74: 217–220.
32. Olivieri N.F. Brittenham G.M. McLaren C.E. Templeton D.M. Cameron R.G. McClelland R.A. et al (1998) Long-term safety and effectiveness of iron-chelation therapy with deferiprone for thalassemia major. N Engl J Med 339: 417–423.
33. Olivieri N.F. Freedman M.H. Koren G. Hermann C. Bentur Y. Chung D. et al (1990) Comparison of oral iron chelator L 1 and desferrioxamine in iron-loaded patients. Lancet 336: 1275–1279.
34. Panigrahi I. Marwaha R.K. Das R.R. (2010) Long-term response to deferiprone therapy in Asian Indians. Ann Hematol 89: 135–140.
35. Pashalidis I. Kontoghiorghes G.J. (2000) Competition studies of L1-deferiprone with copper and iron. Possible implications on efficacy, toxicity and new therapeutic applications. Transfus Sci 23: 259–261.
36. Pennell D.J. Berdoukas V. Karagiorga M. Ladis V. Piga A. Aessopos A. et al (2006) Randomized controlled trial of deferiprone or deferoxamine in beta-thalassemia major patients with asymptomatic myocardial siderosis. Blood 107: 3738–3744.
37. Pennell D.J. Carpenter J.P. Roughton M. Cabantchik Z. (2011) On improvement in ejection fraction with iron chelation in thalassemia major and the risk of future heart failure. J Cardiovasc Magn Reson 13: 45.
38. Perifanis V. Christoforidis A. Vlachaki E. Tsatra I. Spanos G. Athanassiou-Metaxa M. (2007) Comparison of effects of different long-term iron-chelation regimens on myocardial and hepatic iron concentrations assessed with T2* magnetic resonance imaging in patients with beta-thalassemia major. Int J Hematol 86: 385–389.
39. Piga A. Gaglioti C. Fogliacco E. Tricta F. (2003) Comparative effects of deferiprone and deferoxamine on survival and cardiac disease in patients with thalassemia major: a retrospective analysis. Haematologica 88: 489–496.
40. Piga A. Roggero S. Salussolia I. Massano D. Serra M. Longo F. (2010) Deferiprone. Ann N Y Acad Sci 1202: 75–78.
41. Pontikoglou C. Papadaki H.A. (2010) Idiosyncratic drug-induced agranulocytosis: the paradigm of deferiprone. Hemoglobin 34: 291–304.
42. Porcu M. Landis N. Salis S. Corda M. Orrù P. Serra E. et al (2007) Effects of combined deferiprone and desferrioxamine iron chelating therapy in beta-thalassemia major end-stage heart failure: a case report. Eur J Heart Fail 9: 320–322.
43. Roberts D.J. Brunskill S.J. Doree C. Williams S. Howard J. Hyde C.J. (2007) Oral deferiprone for iron chelation in people with thalassaemia. Cochrane Database Syst Rev (3): CD004839.
44. Smith G.C. Alpendurada F. Carpenter J.P. Alam M.H. Berdoukas V. Karagiorga M. et al (2011) Effect of deferiprone or deferoxamine on right ventricular function in thalassemia major patients with myocardial iron overload. J Cardiovasc Magn Reson 13:34.
45. Telfer P.T. Warburton F. Christou S. Hadjigavriel M. Sitarou M. Kolnagou A. et al (2009) Improved survival in thalassemia major patients on switching from desferrioxamine to combined chelation therapy with desferrioxamine and deferiprone. Haematol 94: 1777–1778.
46. Wanless I.R. Sweeney G. Dhillon A.P. Guido M. Piga A. Galanello R. et al (2002) Lack of progressive hepatic fibrosis during long-term therapy with deferiprone in subjects with transfusion-dependent beta-thalassemia. Blood 100: 1566–1569.
47. Weatherall D.J. (1997) The thalassaemias. BMJ 314: 1675–1678.
48. Weizer-Stern O. Adamsky K. Amariglio N. Levin C. Koren A. Breuer W. et al (2006) Downregulation of hepcidin and haemojuvelin expression in the hepatocyte cell-line HepG 2 induced by thalassaemic sera. Br J Haematol 135: 129–138.
49. Wu K.H. Chang J.S. Tsai C.H. Peng C.T. (2004) Combined therapy with deferiprone and desferrioxamine successfully regresses severe heart failure in patients with beta-thalassemia major. Ann Hematol 83: 471–473.
50. Zurlo M. de Stefano P. Borgna-Pignatti C. Di Palma A. Melevendi C. Piga A. et al (1989) Survival and causes of death in thalassaemia major. Lancet 334: 27–30.