Beta thalassemia

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24. Bender MA, Hulihan M, Dorley MC, et al. Newborn Screening practices for beta-thalassemia in the United States. Int J Neonatal Screen. 2021 Dec 13;7(4):83.[Abstract][Full Text]

25. Farmakis D, Angastiniotis M, Eleftheriou, A. A short guide for the management of transfusion dependent thalassaemia. 2017 [internet publication].[Full Text]

26. Taher A, Musallam K, Cappellini MD. Guidelines for the management of non transfusion dependent thalassaemia (NTDT) 2nd Edition. 2017 [internet publication].[Full Text]

27. Karimi M, Cohan N, De Sanctis V, et al. Guidelines for diagnosis and management of beta-thalassemia intermedia. Pediatr Hematol Oncol. 2014 Oct;31(7):583-96.[Abstract]

28. Berdoukas V, Farmaki K, Wood JC, et al. Iron chelation in thalassemia: time to reconsider our comfort zones. Expert Rev Hematol. 2011 Feb;4(1):17-26.[Abstract]

29. Farmaki K, Tzoumari I, Pappa C, et al. Normalisation of total body iron load with very intensive combined chelation reverses cardiac and endocrine complications of thalassaemia major. Br J Haematol. 2010 Feb;148(3):466-75.[Abstract][Full Text]

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31. Taher AT, Viprakasit V, Musallam KM, et al. Treating iron overload in patients with non-transfusion-dependent thalassemia. Am J Hematol. 2013 May;88(5):409-15.[Abstract][Full Text]

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33. Wood JC. Magnetic resonance imaging measurement of iron overload. Curr Opin Hematol. 2007 May;14(3):183-90.[Abstract]

34. Olivieri NF, Brittenham GM. Iron-chelating therapy and the treatment of thalassemia. Blood. 1997 Feb 1;89(3):739-61.[Abstract][Full Text]

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38. Telfer PT, Prestcott E, Holden S, et al. Hepatic iron concentration combined with long-term monitoring of serum ferritin to predict complications of iron overload in thalassaemia major. Br J Haematol. 2000 Sep;110(4):971-7. [Abstract]

39. Maggio A, Filosa A, Vitrano A, et al. Iron chelation therapy in thalassemia major: a systematic review with meta-analyses of 1520 patients included on randomized clinical trials. Blood Cells Mol Dis. 2011 Oct 15;47(3):166-75.[Abstract]

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41. Sheth S. Iron chelation: an update. Curr Opin Hematol. 2014 May;21(3):179-85.[Abstract]

42. 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):567-73.[Abstract][Full Text]

43. Fisher SA, Brunskill SJ, Doree C, et al. Desferrioxamine mesylate for managing transfusional iron overload in people with transfusion-dependent thalassaemia. Cochrane Database Syst Rev. 2013 Aug 21;(8):CD004450.[Abstract][Full Text]

44. Pennell DJ, Porter JB, Cappellini MD, et al. Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia. Blood. 2010 Mar 25;115(12):2364-71.[Abstract]

45. Taher AT, Porter J, Viprakasit V, et al. Deferasirox reduces iron overload significantly in nontransfusion-dependent thalassemia: 1-year results from a prospective, randomized, double-blind, placebo-controlled study. Blood. 2012 Aug 2;120(5):970-7.[Abstract][Full Text]

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47. Bollig C, Schell LK, Rücker G, et al. Deferasirox for managing iron overload in people with thalassaemia. Cochrane Database Syst Rev. 2017 Aug 15;8:CD007476.[Abstract][Full Text]

48. Cappellini MD, Bejaoui M, Agaoglu L, et al. Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5 years' follow-up. Blood. 2011 Jul 28;118(4):884-93.[Abstract][Full Text]

49. Pennell DJ, Berdoukas V, Karagiorga M, et al. Randomized controlled trial of deferiprone or deferoxamine in beta-thalassemia major patients with asymptomatic myocardial siderosis. Blood. 2006 May 1;107(9):3738-44.[Abstract][Full Text]

50. Hider RC, Hoffbrand AV. The role of deferiprone in iron chelation. N Engl J Med. 2018 Nov 29;379(22):2140-50.[Abstract][Full Text]

51. Fisher SA, Brunskill SJ, Doree C, et al. Oral deferiprone for iron chelation in people with thalassaemia. Cochrane Database Syst Rev. 2013 Aug 21;(8):CD004839.[Abstract][Full Text]

52. Delea TE, Edelsberg J, Sofrygin O, et al. Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion. 2007 Oct;47(10):1919-29.[Abstract]

53. Davis BA, Porter JB. Long-term outcome of continuous 24-hour deferoxamine infusion via indwelling intravenous catheters in high-risk beta-thalassemia. Blood. 2000 Feb 15;95(4):1229-36.[Abstract][Full Text]

54. Lucarelli G, Andreani M, Angelucci E. The cure of thalassemia by bone marrow transplantation. Blood Rev. 2002 Jun;16(2):81-5.[Abstract]

55. Gaziev D, Galimberti M, Lucarelli G, et al. Bone marrow transplantation from alternative donors for thalassemia: HLA-phenotypically identical relative and HLA-nonidentical sibling or parent transplants. Bone Marrow Transplant. 2000 Apr;25(8):815-21.[Abstract][Full Text]

56. Kanathezhath B, Walters MC. Umbilical cord blood transplantation for thalassemia major. Hematol Oncol Clin North Am. 2010 Dec;24(6):1165-77.[Abstract]

57. Lucarelli G, Galimberti M, Polchi P, et al. New approach to bone marrow transplantation in thalassemia. Haematologica. 1990 Sep-Oct;75(suppl 5):111-21.[Abstract]

58. Cappellini MD, Cohen A, Eleftheriou A, et al. Splenectomy in beta-thalassaemia. In: Guidelines for the Clinical Management of Thalassaemia [Internet]. 2nd Revised edition. 2008.[Full Text]

59. Sharma A, Easow Mathew M, Puri L. Splenectomy for people with thalassaemia major or intermedia. Cochrane Database Syst Rev. 2019 Sep 17;9:CD010517.[Abstract][Full Text]

60. Taher AT, Musallam KM, Cappellini MD, et al. Optimal management of beta thalassaemia intermedia. Br J Haematol. 2011 Mar;152(5):512-23.[Abstract]

61. Porter JB. Optimizing iron chelation strategies in beta-thalassaemia major. Blood Rev. 2009 Dec;23(suppl 1):S3-7.[Abstract]

62. Thompson AA, Walters MC, Kwiatkowski J, et al. Gene therapy in patients with transfusion-dependent beta-thalassemia. N Engl J Med. 2018 Apr 19;378(16):1479-93.[Abstract][Full Text]

63. Samakoglu S, Lisowski L, Budak-Alpdogan T, et al. A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference. Nat Biotechnol. 2006 Jan;24(1):89-94.[Abstract]

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66. Gardenghi S, Ramos P, Follenzi A, et al. Hepcidin and Hfe in iron overload in beta-thalassemia. Ann N Y Acad Sci. 2010 Aug;1202:221-5.[Abstract]

67. Suwanmanee T, Sierakowska H, Lacerra G, et al. Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides. Mol Pharmacol. 2002 Sep;62(3):545-53.[Abstract][Full Text]

68. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion-dependent beta-thalassemia. N Engl J Med. 2020 Mar 26;382(13):1219-31.[Abstract][Full Text]

69. European Medicines Agency. Reblozyl. Jul 2020 [internet publication].[Full Text]

70. US Food and Drug Administration. FDA approves luspatercept-aamt for anemia in patients with beta thalassemia. Aug 2019 [internet publication].[Full Text]

71. Foong WC, Ho JJ, Loh CK, et al. Hydroxyurea for reducing blood transfusion in non-transfusion dependent beta thalassaemias. Cochrane Database Syst Rev. 2016 Oct 18;10(10):CD011579.[Abstract][Full Text]

72. Wilber A, Nienhuis AW, Persons DA. Transcriptional regulation of fetal to adult hemoglobin switching: new therapeutic opportunities. Blood. 2011 Apr 14;117(15):3945-53.[Abstract][Full Text]

73. Cogliandro T, Derchi G, Mancuso L, et al. Guideline recommendations for heart complications in thalassemia major. J Cardiovasc Med (Hagerstown). 2008 May;9(5):515-25.[Abstract][Full Text]

74. Borgna-Pignatti C, Cappellini MD, De Stefano P, et al. Survival and complications in thalassemia. Ann N Y Acad Sci. 2005 Jul;1054(1):40-7.[Abstract]

75. Cunningham MJ. Update on thalassemia: clinical care and complications. Hematol Oncol Clin North Am. 2010 Feb;24(1):215-27.[Abstract]

76. Alavian S-MT. Treatment of chronic hepatitis C in polytransfused thalassaemic patients: a meta-analysis. J Viral Hepat. 2010 Apr;17(4):236-44.[Abstract]

77. Di Marco V, Capra M, Angelucci E, et al. Management of chronic viral hepatitis in patients with thalassemia: recommendations from an international panel. Blood. 2010 Oct 21;116(16):2875-83.[Abstract][Full Text]

78. Kremastinos DT, Farmakis D, Aessopos A, et al. Beta-thalassemia cardiomyopathy: history, present considerations, and future perspectives. Circ Heart Fail. 2010 May;3(3):451-8.[Abstract][Full Text]

79. Toumba M, Sergis A, Kanaris C, et al. Endocrine complications in patients with thalassaemia major. Pediatr Endocrinol Rev. 2007 Dec;5(2):642-8.[Abstract]

80. Delvecchio M, Cavallo L. Growth and endocrine function in thalassemia major in childhood and adolescence. J Endocrinol Invest. 2010 Jan;33(1):61-8.[Abstract]

81. Haidar R, Musallam KM, Taher AT. Bone disease and skeletal complications in patients with beta thalassemia major. Bone. 2011 Mar 1;48(3):425-32.[Abstract]

82. Bhardwaj A, Swe KM, Sinha NK, et al. Treatment for osteoporosis in people with ß-thalassaemia. Cochrane Database Syst Rev. 2016 Mar 10;3:CD010429.[Abstract][Full Text]

83. Skordis N, Toumba M. Bone disease in thalassaemia major: recent advances in pathogenesis and clinical aspects. Pediatr Endocrinol Rev. 2011 Mar;8 Suppl 2:300-6.[Abstract]

84. Mulimani P, Abas AB, Karanth L, et al. Treatment of dental and orthodontic complications in thalassaemia. Cochrane Database Syst Rev. 2019 Aug 2;8:CD012969.[Abstract][Full Text]

85. Galanello R, Piras S, Barella S, et al. Cholelithiasis and Gilbert's syndrome in homozygous beta-thalassaemia. Br J Haematol. 2001 Dec;115(4):926-8.[Abstract][Full Text]

86. Farmakis D, Aessopos A. Pulmonary hypertension associated with hemoglobinopathies: prevalent but overlooked. Circulation. 2011 Mar 22;123(11):1227-32.[Abstract]

87. Cappellini MD, Motta I, Musallam KM, et al. Redefining thalassemia as a hypercoagulable state. Ann N Y Acad Sci. 2010 Aug;1202:231-6.[Abstract]