Contemporary approaches to treatment of beta-thalassemia intermedia

Blood Reviews

Volumn 26, Issue SUPPL.1, 2012, Pages

  Taher, Ali T ^a   Musallam, Khaled M ^b   Karimi, Mehran ^c   Cappellini, Maria D ^b  

^a AMERICAN UNIVERSITY OF BEIRUT   (Lebanon)

^b UNIVERSITY OF MILAN   (Italy)

^c SHIRAZ UNIVERSITY OF MEDICAL SCIENCES   (Iran)

Author keywords

Beta thalassemia intermedia; HbF induction; Iron chelation; Splenectomy; Transfusion; Treatment

Indexed keywords

ANTIBIOTIC AGENT; CARNITINE; DEFERASIROX; DEFERIPRONE; DEFEROXAMINE; HEMOGLOBIN; HYDROXYUREA; IRON CHELATING AGENT; MAGNESIUM; PLACEBO; SILDENAFIL;

AGRANULOCYTOSIS; APPLICATION SITE REACTION; ARTHRALGIA; ARTICLE; BETA THALASSEMIA; CHELATION THERAPY; CLINICAL ASSESSMENT; DRUG EFFICACY; DRUG ERUPTION; DRUG EXCRETION; DRUG HALF LIFE; DRUG HYPERSENSITIVITY; DRUG TOLERABILITY; ERYTHROCYTE TRANSFUSION; EYE DISEASE; GASTROINTESTINAL HEMORRHAGE; GASTROINTESTINAL SYMPTOM; GROWTH RETARDATION; HEARING DISORDER; HEMOGLOBIN BLOOD LEVEL; HUMAN; INFECTION RISK; KIDNEY DYSFUNCTION; KIDNEY FAILURE; LIVER DYSFUNCTION; LIVER FAILURE; NEUTROPENIA; POSTOPERATIVE INFECTION; PRIORITY JOURNAL; QUALITY OF LIFE; RISK FACTOR; SIDE EFFECT; SPLENECTOMY; THROMBOEMBOLISM; TREATMENT INDICATION; TREATMENT RESPONSE; VEIN THROMBOSIS;

BETA-THALASSEMIA; BLOOD TRANSFUSION; HUMANS; IRON CHELATING AGENTS; SPLENECTOMY;

EID: 84861362043     PISSN: 0268960X     EISSN: 15321681     Source Type: Journal    
DOI: 10.1016/S0268-960X(12)70008-5     Document Type: Article

References (46)

1. Sturgeon P, Itano HA, Bergren WR Genetic and biochemical studies of intermediate types of Cooley's anaemia. Br J Haematol 1955, 1(3):264-277.
2. Taher AT, Musallam KM, Cappellini MD, Weatherall DJ Optimal management of beta thalassaemia intermedia. Br J Haematol 2011, 152(5):512-523.
3. Taher A, Isma'eel H, Cappellini MD Thalassemia intermedia: revisited. Blood Cells Mol Dis 2006, 37(1):12-20.
4. Haidar R, Mhaidli H, Taher AT Paraspinal extramedullary hematopoiesis in patients with thalassemia intermedia. Eur Spine J 2010, 19(6):871-878.
5. Ataga KI, Cappellini MD, Rachmilewitz EA Beta-thalassaemia and sickle cell anaemia as paradigms of hypercoagulability. Br J Haematol 2007, 139(1):3-13.
6. Taher A, Isma'eel H, Mehio G, et al. Prevalence of thromboembolic events among 8,860 patients with thalassaemia major and intermedia in the Mediterranean area and Iran. Thromb Haemost 2006, 96(4):488-491.
7. Taher AT, Otrock ZK, Uthman I, Cappellini MD Thalassemia and hypercoagulability. Blood Rev 2008, 22(5):283-292.
8. Aessopos A, Farmakis D, Karagiorga M, et al. Cardiac involvement in thalassemia intermedia: a multicenter study. Blood 2001, 97(11):3411-3416.
9. Taher A, Abou-Mourad Y, Abchee A, Zalouaa P, Shamseddine A Pulmonary thromboembolism in beta-thalassemia intermedia: are we aware of this complication?. Hemoglobin 2002, 26(2):107-112.
10. Karimi M, Musallam KM, Cappellini MD, et al. Risk factors for pulmonary hypertension in patients with beta thalassemia intermedia. European J Intern Med 2011, 22(6):607-610.
11. Taher A, Hershko C, Cappellini MD Iron overload in thalassaemia intermedia:reassessment of iron chelation strategies. Br J Haematol 2009, 147(5):634-640.
12. Musallam KM, Cappellini MD, Wood JC, et al. Elevated liver iron concentration is a marker of increased morbidity in patients with {beta} thalassemia intermedia. Haematologica 2011, 96(11):1605-1612.
13. Taher AT, Musallam KM, El-Beshlawy A, et al. Age-related complications in treatment-naive patients with thalassaemia intermedia. Br J Haematol 2010, 150(4):486-489.
14. Taher AT, Musallam KM, Karimi M, et al. Overview on practices in thalassemia intermedia management aiming for lowering complication rates across a region of endemicity: the OPTIMAL CARE study. Blood 2010, 115(10):1886-1892.
15. Borgna-Pignatti C Modern treatment of thalassaemia intermedia. Br J Haematol 2007, 138(3):291-304.
16. Cappellini MD, Robbiolo L, Bottasso BM, Coppola R, Fiorelli G, Mannucci AP Venous thromboembolism and hypercoagulability in splenectomized patients with thalassaemia intermedia. Br J Haematol 2000, 111(2):467-473.
17. Aessopos A, Farmakis D Pulmonary hypertension in beta-thalassemia. Ann NY Acad Sci 2005, 1054:342-349.
18. Atichartakarn V, Likittanasombat K, Chuncharunee S, et al. Pulmonary arterial hypertension in previously splenectomized patients with beta-thalassemic disorders. Int J Hematol 2003, 78(2):139-145.
19. Taher AT, Musallam KM, Nasreddine W, Hourani R, Inati A, Beydoun A Asymptomatic brain magnetic resonance imaging abnormalities in splenectomized adults with thalassemia intermedia. J Thromb Haemost 2010, 8(1):54-59.
20. Bisharat N, Omari H, Lavi I, Raz R Risk of infection and death among post-splenectomy patients. J Infect 2001, 43(3):182-186.
21. Taher AT PJ, Viprakasit V, et al. Deferasirox significantly reduces liver iron concentration in non-transfusion-dependent thalassemia patients with iron overload: results form the 1-year randomized, double-blind, placebo-controlled phase II THALASSA study. 53rd American Society of Hematology Annual Meeting, December 10-13, 2011; San Diego, California. Abstract 902.
22. Davies JM, Lewis MP, Wimperis J, Rafi I, Ladhani S, Bolton-Maggs PH Review of guidelines for the prevention and treatment of infection in patients with an absent or dysfunctional spleen: prepared on behalf of the British Committee for Standards in Haematology by a working party of the Haemato-Oncology task force. Br J Haematol 2011, 155(3):308-317.
23. Borgna-Pignatti C, Marsella M, Zanforlin N The natural history of thalassemia intermedia. Ann N Y Acad Sci 2010, 1202:214-220.
24. Eder AF, Chambers LA Noninfectious complications of blood transfusion. Arch Pathol Lab Med 2007, 131(5):708-718.
25. Aessopos A, Kati M, Farmakis D Heartdisease in thalassemia intermedia: a review of the underlying pathophysiology. Haematologica 2007, 92(5):658-665.
26. Musallam KM, Khoury B, Abi-Habib R, et al. Health-related quality of life in adults with transfusion-independent thalassaemia intermedia compared to regularly transfused thalassaemia major: new insights. Eur J Haematol 2011, 87(1):73-79.
27. Kontoghiorghes GJ, Spyrou A, Kolnagou A Iron chelation therapy in hereditary hemochromatosis and thalassemia intermedia: regulatory and non regulatory mechanisms of increased iron absorption. Hemoglobin 2010, 34(3):251-264.
28. Cossu P, Toccafondi C, Vardeu F, et al. Iron overload and desferrioxamine chelation therapy in beta-thalassemia intermedia. Eur J Pediatr 1981, 137(3):267-271.
29. Taher A, El Rassi F, Isma'eel H, Koussa S, Inati A, Cappellini MD Correlation of liver iron concentration determined by R2 magnetic resonance imaging with serum ferritin in patients with thalassemia intermedia. Haematologica 2008, 93(10):1584-1586.
30. Desferal [package insert] 2011, Novartis Pharmaceuticals Corporation, East Hanover, NJ, 2010. Available at: http://www.pharma.us.novartis.com/product/pi/pdf/desferal.pdf. Accessed November 30.
31. EXJADE [package insert] 2011, Novartis Pharmaceuticals Corporation, East Hanover, NJ, 2010. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2010/021882s010lbl.pdf. Accessed November 30.
32. EXJADE [summary of product characteristics] 2011, Novartis Europharm Limited, Hosham, West Sussex, United Kingdom, Available at: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000670/WC500033925.pdf. Accessed November 30.
33. Ferriprox [summary of product characteristics] 2011, Apotex Europe B.V., Leiden, Netherlands, Accessed November 30. http://www.ferriprox.com/aboutferriprox/SPC_tablet.pdf.
34. Cappellini MD Overcoming the challenge of patient compliance with iron chelation therapy. Semin Hematol 2005, 42(2 Suppl 1):S19-S21.
35. Treadwell MJ, Weissman L Improving adherence with deferoxamine regimens for patients receiving chronic transfusion therapy. Semin Hematol 2001, 38(1Suppl 1):77-84.
36. Ladis V, Berdousi H, Gotsis E, Kattamis A Deferasirox administration for the treatment of non-transfusional iron overload in patients with thalassaemia intermedia. Br J Haematol 2010, 151(5):504-508.
37. Voskaridou E, Plata E, Douskou M, et al. Treatment with deferasirox (Exjade) effectively decreases iron burden in patients with thalassaemia intermedia: results of a pilot study. Br J Haematol 2010, 148(2):332-334.
38. Porter J, Cappellini M, Kattamis A, Viprakasit V, Lawniczek T, Ros J, Deng W, Taher A. Relationship between plasma non-transferrin-bound iron and markers of iron overload, anaemia and ineffective erythropoiesis in non-transfusion-dependent thalassaemia syndromes. Presented at the 16th Congress of the European Hematology Association, June 9-12, 2011, London, UK. Abstract 0536.
39. Karimi M, Cohan N, Moosavizadeh K, Falahi MJ, Haghpanah S Adverse effects of hydroxyurea in beta-thalassemia intermedia patients: 10 years' experience. Pediatr Hematol Oncol 2010, 27(3):205-211.
40. Rigano P, Pecoraro A, Calzolari R, et al. Desensitization to hydroxycarbamide following long-term treatment of thalassaemia intermedia as observed in vivo and in primary erythroid cultures from treated patients. Br J Haematol 2010, 151(5):509-515.
41. Karimi M, Mohammadi F, Behmanesh F, et al. Effect of combination therapy of hydroxyurea with L-carnitine and magnesium chloride on hematologic parameters and cardiac function of patients with beta-thalassemia intermedia. Eur J Haematol 2010, 84(1):52-58.
42. Amoozgar H, Farhani N, Khodadadi N, Karimi M, Cheriki S Comparative study of pulmonary circulation and myocardial function in patients with beta-thalassemia intermedia with and without hydroxyurea, a case-control study. Eur J Haematol 2011, 87(1):61-67.
43. Gardenghi S, Ramos P, Marongiu MF, et al. Hepcidin as a therapeutic tool to limit iron overload and improve anemia in beta-thalassemic mice. J Clin Invest 2010, 120(12):4466-4477.
44. Li H, Rybicki AC, Suzuka SM, et al. Transferrin therapy ameliorates disease in beta-thalassemic mice. Nat Med 2010, 16(2):177-182.
45. Sankaran VG, Nathan DG Reversing the hemoglobin switch. N Engl J Med 2010, 363(23):2258-2260.
46. Porter JB Deferoxamine pharmacokinetics. Semin Hematol 2001, 38(1 Suppl1):63-68.