Cross-talk between available guidelines for the management of patients with beta-thalassemia major

Acta Haematologica

Volumn 130, Issue 2, 2013, Pages 64-73

  Musallam, Khaled M ^a   Angastiniotis, Michael ^b   Eleftheriou, Androulla ^b   Porter, John B ^c  

^a UNIVERSITY OF MILAN   (Italy)

^b Thalassaemia International Federation   (Cyprus)

^c UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

Iron chelation; Iron overload; Splenectomy; Transfusion

Indexed keywords

DEFERASIROX; DEFERIPRONE; DEFEROXAMINE; IRON CHELATING AGENT;

BETA THALASSEMIA; BLOOD TRANSFUSION; CHELATION THERAPY; DRUG EFFICACY; DRUG SAFETY; HUMAN; IRON CHELATION; IRON OVERLOAD; MEDICAL RESEARCH; MONOTHERAPY; PATIENT CARE; PRACTICE GUIDELINE; PRIORITY JOURNAL; RECOMMENDED DRUG DOSE; REVIEW; SPLENECTOMY; THALASSEMIA MAJOR; TREATMENT OUTCOME;

EID: 84874814802     PISSN: 00015792     EISSN: 14219662     Source Type: Journal    
DOI: 10.1159/000345734     Document Type: Review

References (49)

1. Weatherall DJ: The inherited diseases of hemoglobin are an emerging global health burden. Blood 2010; 115: 4331-4336.
2. Rund D, Rachmilewitz E: Beta-thalassemia. N Engl J Med 2005; 353: 1135-1146.
3. Steinberg MH, Forget BG, Higgs DR, Weatherall DJ: Disorders of Hemoglobin: Genetics, Pathophysiology, and Clinical Management, ed 2. New York, Cambridge University Press, 2009.
4. Charafeddine K, Isma'eel H, Charafeddine M, Inati A, Koussa S, Naja M, Taher A: Survival and complications of beta-thalassemia in Lebanon: a decade's experience of centralized care. Acta Haematol 2008; 120: 112-116.
5. Chern JP, Su S, Lin KH, Chang SH, Lu MY, Jou ST, Lin DT, Ho WL, Lin KS: Survival, mortality, and complications in patients with beta-thalassemia major in northern Taiwan. Pediatr Blood Cancer 2007; 48: 550-554.
6. Borgna-Pignatti C, Cappellini MD, De Stefano P, Del Vecchio GC, Forni GL, Gamberini MR, Ghilardi R, Origa R, Piga A, Romeo MA, Zhao H, Cnaan A: Survival and complications in thalassemia. Ann NY Acad Sci 2005; 1054: 40-47.
7. Telfer P: Update on survival in thalassemia major. Hemoglobin 2009; 33(suppl 1):S76-S80.
8. Modell B, Khan M, Darlison M: Survival in beta-thalassaemia major in the UK: data from the UK Thalassaemia Register. Lancet 2000; 355: 2051-2052.
9. Telfer P, Coen PG, Christou S, Hadjigavriel M, Kolnakou A, Pangalou E, Pavlides N, Psiloines M, Simamonian K, Skordos G, Sitarou M, Angastiniotis M: Survival of medically treated thalassemia patients in cyprus. Trends and risk factors over the period 1980-2004. Haematologica 2006; 91: 1187-1192.
10. Fung EB, Harmatz P, Milet M, Ballas SK, De Castro L, Hagar W, Owen W, Olivieri N, Smith-Whitley K, Darbari D, Wang W, Vichinsky E, Group M-CSoIOR: Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: a report from the Multi-Center Study of Iron Overload. Am J Hematol 2007; 82: 255-265.
11. Cario H, Stahnke K, Sander S, Kohne E: Epidemiological situation and treatment of patients with thalassemia major in Germany: results of the German multicenter beta-thalassemia study. Ann Hematol 2000; 79: 7-12.
12. Thuret I, Pondarre C, Loundou A, Steschenko D, Girot R, Bachir D, Rose C, Barlogis V, Donadieu J, de Montalembert M, Hagege I, Pegourie B, Berger C, Micheau M, Bernaudin F, Leblanc T, Lutz L, Galacteros F, Simeoni MC, Badens C: Complications and treatment of patients with β-thalassemia in France: results of the National Registry. Haematologica 2010; 95: 724-729.
13. Rachmilewitz EA, Giardina PJ: How I treat thalassemia. Blood 2011; 118: 3479-3488.
14. Cappellini MD, Cohen A, Eleftheriou A, Piga A, Porter J, Taher A: Guidelines for the Clinical Management of Thalassemia, ed 2. Nicosia, Thalassaemia International Federation, 2009.
15. Vichinsky E, Levine L, Bhatia S, Bojanowski J, Coates T, Foote D, Fung E, Harmatz P, Jeng M, Knudsen JM, Lal A, Pakbaz Z, Schroepfer C, Singer ST, Sweeters N, Walters M, Wood J, Levine M: Standards of Care Guidelines for Thalassemia. Oakland, Children's Hospital and Research Center Oakland, 2009.
16. Sayani F, Warner M, Wu J, Wong-Rieger D, Humphreys K, Odame I: Guidelines for the Clinical Care of Patients with Thalassemia in Canada. Tornonto, Anemia Institute for Research and Education, 2009.
17. Yardumian A, Telfer P, Darbyshire P: Standards for the Clinical Care of Children and Adults with Thalassaemia in the UK, ed 2. London, United Kingdom Thalassaemia Society, 2008.
18. Angelucci E, Barosi G, Camaschella C, Cappellini MD, Cazzola M, Galanello R, Marchetti M, Piga A, Tura S: Italian Society of Hematology practice guidelines for the management of iron overload in thalassemia major and related disorders. Haematologica 2008; 93: 741-752.
19. Ho PJ, Tay L, Lindeman R, Catley L, Bowden DK: Australian guidelines for the assessment of iron overload and iron chelation in transfusion- dependent thalassaemia major, sickle cell disease and other congenital anaemias. Intern Med J 2011; 41: 516-524.
20. Allen A, Fisher C, Premawardhena A, Peto T, Allen S, Arambepola M, Thayalsutha V, Olivieri N, Weatherall D: Adaptation to anemia in hemoglobin E-β thalassemia. Blood 2010; 116: 5368-5370.
21. Anderson LJ, Holden S, Davis B, Prescott E, Charrier CC, Bunce NH, Firmin DN, Wonke B, Porter J, Walker JM, Pennell DJ: Cardiovascular T2-star (T2 ∗) magnetic resonance for the early diagnosis of myocardial iron overload. Eur Heart J 2001; 22: 2171-2179.
22. Garbowski MW, Carpenter JP, Smith G, Pennell DJ, Porter JB: Calibration of improved T2 ∗ method for the estimation of liver iron concentration in transfusional iron overload (abstract). Blood 2009; 114: 2004.
23. Kirk P, He T, Anderson LJ, Roughton M, Tanner MA, Lam WW, Au WY, Chu WC, Chan G, Galanello R, Matta G, Fogel M, Cohen AR, Tan RS, Chen K, Ng I, Lai A, Fucharoen S, Laothamata J, Chuncharunee S, Jongjirasiri S, Firmin DN, Smith GC, Pennell DJ: International reproducibility of single breathhold T2 ∗ MR for cardiac and liver iron assessment among five thalassemia centers. J Magn Reson Imaging 2010; 32: 315-319.
24. Carpenter JP, He T, Kirk P, Roughton M, Anderson LJ, de Noronha SV, Sheppard MN, Porter JB, Walker JM, Wood JC, Galanello R, Forni G, Catani G, Matta G, Fucharoen S, Fleming A, House MJ, Black G, Firmin DN, St Pierre TG, Pennell DJ: On T2 ∗ magnetic resonance and cardiac iron. Circulation 2011; 123: 1519-1528.
25. Noetzli LJ, Carson SM, Nord AS, Coates TD, Wood JC: Longitudinal analysis of heart and liver iron in thalassemia major. Blood 2008; 112: 2973-2978.
26. Haidar R, Musallam KM, Taher AT: Bone disease and skeletal complications in patients with beta thalassemia major. Bone 2011; 48: 425-432.
27. ElAlfy MS, Sari TT, Lee CL, Tricta F, El-Beshlawy A: The safety, tolerability, and efficacy of a liquid formulation of deferiprone in young children with transfusional iron overload. J Pediatr Hematol Oncol 2010; 32: 601-605.
28. Cappellini MD, Cohen A, Piga A, Bejaoui M, Perrotta S, Agaoglu L, Aydinok Y, Kattamis A, Kilinc Y, Porter J, Capra M, Galanello R, Fattoum S, Drelichman G, Magnano C, Verissimo M, Athanassiou-Metaxa M, Giardina P, Kourakli-Symeonidis A, Janka-Schaub G, Coates T, Vermylen C, Olivieri N, Thuret I, Opitz H, Ressayre-Djaffer C, Marks P, Alberti D: A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood 2006; 107: 3455-3462.
29. Cappellini MD, Bejaoui M, Agaoglu L, Canatan D, Capra M, Cohen A, Drelichman G, Economou M, Fattoum S, Kattamis A, Kilinc Y, Perrotta S, Piga A, Porter JB, Griffel L, Dong V, Clark J, Aydinok Y: Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5 years' follow-up. Blood 2011; 118: 884-893.
30. Aydinok Y, Unal S, Oymak Y, Vergin C, Turker ZD, Yildiz D, Yesilipek A: Observational study comparing long-term safety and efficacy of deferasirox with desferrioxamine therapy in chelation-naive children with transfusional iron overload. Eur J Haematol 2012; 88: 431-438.
31. Smith GC, Alpendurada F, Carpenter JP, Alam MH, Berdoukas V, Karagiorga M, Ladis V, Piga A, Aessopos A, Gotsis ED, Tanner MA, Westwood MA, Galanello R, Roughton M, Pennell DJ: Effect of deferiprone or deferoxamine on right ventricular function in thalassemia major patients with myocardial iron overload. J Cardiovasc Magn Reson 2011; 13: 34.
32. Alpendurada F, Smith GC, Carpenter JP, Nair SV, Tanner MA, Banya W, Dessi C, Galanello R, Walker JM, Pennell DJ: Effects of combined deferiprone with deferoxamine on right ventricular function in thalassaemia major. J Cardiovasc Magn Reson 2012; 14: 8.
33. Maggio A, Vitrano A, Capra M, Cuccia L, Gagliardotto F, Filosa A, Magnano C, Rizzo M, Caruso V, Gerardi C, Argento C, Campisi S, Cantella F, Commendatore F, D'Ascola DG, Fidone C, Ciancio A, Galati MC, Giuffrida G, Cingari R, Giugno G, Lombardo T, Prossomariti L, Malizia R, Meo A, Roccamo G, Romeo MA, Violi P, Cianciulli P, Rigano P: Improving survival with deferiprone treatment in patients with thalassemia major: a prospective multicenter randomised clinical trial under the auspices of the Italian Society for Thalassemia and Hemoglobinopathies. Blood Cells Mol Dis 2009; 42: 247-251.
34. Lai ME, Grady RW, Vacquer S, Pepe A, Carta MP, Bina P, Sau F, Cianciulli P, Maggio A, Galanello R, Farci P: Increased survival and reversion of iron-induced cardiac disease in patients with thalassemia major receiving intensive combined chelation therapy as compared to desferoxamine alone. Blood Cells Mol Dis 2010; 45: 136-139.
35. Deugnier Y, Turlin B, Ropert M, Cappellini MD, Porter JB, Giannone V, Zhang Y, Griffel L, Brissot P: Improvement in liver pathology of patients with beta-thalassemia treated with deferasirox for at least 3 years. Gastroenterology 2011; 141: 1202-1211, 1211.e1201-e1203.
36. Pennell DJ, Porter JB, Cappellini MD, Chan LL, El-Beshlawy A, Aydinok Y, Ibrahim H, Li CK, Viprakasit V, Elalfy MS, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A: Deferasirox for up to 3 years leads to continued improvement of myocardial T2∗ in patients with beta-thalassemia major. Haematologica 2012; 97: 842-848.
37. Wood JC, Kang BP, Thompson A, Giardina P, Harmatz P, Glynos T, Paley C, Coates TD: The effect of deferasirox on cardiac iron in thalassemia major: impact of total body iron stores. Blood 2010; 116: 537-543.
38. Pepe A, Meloni A, Capra M, Cianciulli P, Prossomariti L, Malaventura C, Putti MC, Lippi A, Romeo MA, Bisconte MG, Filosa A, Caruso V, Quarta A, Pitrolo L, Missere M, Midiri M, Rossi G, Positano V, Lombardi M, Maggio A: Deferasirox, deferiprone and desferrioxamine treatment in thalassemia major patients: cardiac iron and function comparison determined by quantitative magnetic resonance imaging. Haematologica 2011; 96: 41-47.
39. Voskaridou E, Christoulas D, Terpos E: Successful chelation therapy with the combination of deferasirox and deferiprone in a patient with thalassaemia major and persisting severe iron overload after single-agent chelation therapies. Br J Haematol 2011; 154: 654- 656.
40. Farmaki K, Tzoumari I, Pappa C: Oral chelators in transfusion-dependent thalassemia major patients may prevent or reverse iron overload complications. Blood Cells Mol Dis 2011; 47: 33-40.
41. Balocco M, Carrara P, Pinto V, Forni GL: Daily alternating deferasirox and deferiprone therapy for 'hard-to-chelate' beta-thalassemia major patients. Am J Hematol 2010; 85: 460-461.
42. Berdoukas V, Chouliaras G, Moraitis P, Zannikos K, Berdoussi E, Ladis V: The efficacy of iron chelator regimes in reducing cardiac and hepatic iron in patients with thalassaemia major: a clinical observational study. J Cardiovasc Magn Reson 2009; 11: 20.
43. Cassinerio E, Roghi A, Pedrotti P, Brevi F, Zanaboni L, Graziadei G, Pattoneri P, Milazzo A, Cappellini MD: Cardiac iron removal and functional cardiac improvement by different iron chelation regimens in thalassemia major patients. Ann Hematol 2012; 91: 1443-1449.
44. Neufeld EJ, Galanello R, Viprakasit V, Aydinok Y, Piga A, Harmatz P, Forni GL, Shah FT, Grace RF, Porter JB, Wood JC, Peppe J, Jones A, Rienhoff HY Jr: A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload. Blood 2012; 119: 3263- 3268.
45. Cohen AR, Glimm E, Porter JB: Effect of transfusional iron intake on response to chelation therapy in beta-thalassemia major. Blood 2008; 111: 583-587.
46. Taher A, Cappellini MD, Vichinsky E, Galanello R, Piga A, Lawniczek T, Clark J, Habr D, Porter JB: Efficacy and safety of deferasirox doses of >30 mg/kg per d in patients with transfusion-dependent anaemia and iron overload. Br J Haematol 2009; 147: 752-759.
47. Porter JB, Piga A, Cohen A, Ford JM, Bodner J, Rojkjaer L, Cappellini MD: Safety of deferasirox (Exjade ®) in patients with transfusiondependent anemias and iron overload who achieve serum ferritin levels <1,000 ng/ml during long-term treatment (abstract). Blood 2008; 112: 5423.
48. Ponticelli C, Musallam KM, Cianciulli P, Cappellini MD: Renal complications in transfusion-dependent beta thalassaemia. Blood Rev 2010; 24: 239-244.
49. Taher AT, Musallam KM, Karimi M, El-Beshlawy A, Belhoul K, Daar S, Saned M, Cesaretti C, Cappellini MD: Splenectomy and thrombosis: the case of thalassemia intermedia. J Thromb Haemost 2010; 8: 2152-2158.