A thalassemic child becomes adult

Reviews in Clinical and Experimental Hematology

Volumn 7, Issue 1, 2003, Pages 4-21

  Galanello, Renzo ^a,b  

^a UNIVERSITY OF CAGLIARI   (Italy)

^b Osp Regionale per le Microcitemie   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

DEFERIPRONE; DEFEROXAMINE; IRON CHELATING AGENT;

AGRANULOCYTOSIS; ARTHRALGIA; ARTHROPATHY; BETA THALASSEMIA; BONE MARROW TRANSPLANTATION; COST OF ILLNESS; ERYTHROCYTE TRANSFUSION; EYE TOXICITY; FAMILY LIFE; GASTROINTESTINAL SYMPTOM; GENE THERAPY; GROWTH RETARDATION; HLA SYSTEM; HUMAN; IRON CHELATION; KNEE ARTHRITIS; LIFE EXPECTANCY; LIVER FIBROSIS; NEUTROPENIA; OTOTOXICITY; PATIENT COMPLIANCE; PROGNOSIS; PSYCHOLOGICAL ASPECT; QUALITY OF LIFE; REVIEW; SOCIAL SUPPORT; SURVIVAL; THALASSEMIA; WORK; ZINC DEFICIENCY;

ADULT; AGE DISTRIBUTION; CHILD; DISEASE MANAGEMENT; HUMANS; PROGNOSIS; SURVIVORS; THALASSEMIA;

EID: 0141522686     PISSN: 11270020     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (105)

1. Economidou J. Problem related to treatment of beta-thalassemia major. Paediatrician, 11: 157-177, 1982.
2. Zurlo MG, De Stefano P, Borgna-Pignatti C, et al. Survival and causes of death in thalassemia major. Lancet, ii: 27-30, 1989.
3. Borgna-Pignatti C, Rugolotto S, De Stefano P, et al. Survival and disease complications in thalassemia major. Annals NY Acad Sci, 850: 227-231, 1998.
4. Modell B, Khan M, Darlison M, et al. Survival in beta-thalassemia major in the UK: data from the UK Thalassemia Register. Lancet, 355: 2051-2052, 2000.
5. Lucarelli G, Giardini C, Baronciani D. Bone marrow transplantation in β-thalassemia, Semin Hematol, 32: 297, 1995.
6. Atweh GF, Lokopoulos D. Pharmacological induction of fetal hemoglobin in sickle cell disease and β-thalassemia. Semin Hematol, 38: 367-373, 2001.
7. May C, Rivella S, Callegari J, et al. Therapeutic hemoglobin synthesis in beta-thalassemic mice expressing lentivirus-encoded human beta-globin. Nature, 406: 82-86, 2000.
8. May C, Rivella S, Chadburn A, et al. Successful treatment of murine β-thalassemia intermedia by transfer of the human β-globin gene. Blood, 99: 1902-1908, 2002.
9. Tosti ME, Solinas S, Prati D, et al. An estimate of the current risk of transmitting blood-borne infections through blood transfusion in Italy. Br J Haematol, 117: 215-219, 2002.
10. Sprogoe-Jakobsen U, Sactre AM, Georgsen J. Preparation of white cell reduced red cells by filtration: comparison of bedside filter and two blood bank filter systems. Transfusions, 35: 421-426, 1995.
11. Williamson LM. Leucocyte depletion of the blood supply. How will patients benefit? Br J Hematol, 103: 897-901, 1998.
12. Piomelli S, Karpatkin MH, Arzanian M, et al. Hypertransfusion regimen in patients with Cooley's anemia. Ann NY Acad Sci, 232: 186-192, 1974.
13. Propper RD, Button LN, Nathan DG. New approaches to the transfusion management of thalassemia. Blood, 55: 55-60, 1980.
14. Cazzola M, De Stefano P, Pronchio L, et al. Relationship between transfusion regimen and suppression of erythropoiesis in β thalassemia major. Br J Hematol, 89: 471-478, 1995.
15. Cazzola M, Borgna-Pignatti C, Locatelli F, et al. A moderate transfusion regimen may reduce iron loading in β thalassemia major without producing excessive expansion of erythropoiesis. Transfusion, 37: 135-139, 1997.
16. Cappellini N, Cohen A, Elefteriou A, et al. Guidelines for the Clinical Management of Thalassemia. Thalassemia International Federation: April 2000.
17. Cohen AR, Schmidt JM, Martin MB et al. Clinical trial of young red cell transfusions. J Pediatr, 104: 865, 1984.
18. Marcus RE, Wonke B, Bantock HM. A prospective trial of young red cells in 48 patients with transfusion-dependent thalassaemia. Br J Haematol, 60: 153, 1985.
19. Gabutti V and Piga A. Results of long-term iron-chelating therapy. Acta Haematol, 95: 26-36, 1996.
20. Olivieri NF and Brittenham GM. Iron-chelating therapy and the treatment of thalassemia. Blood, 89: 739-761, 1997.
21. Porter JB, Abeysinghe RB, Marshall L, et al. Kinectis of removal and reappearence of non-transferrin-bound plasma iron with desferrioxamine therapy. Blood, 88: 705-714, 1996.
22. Chatterjee R, Katz M, Cox TF, et al. Prospective study of the hypothalamic-pituitary axis in thalassaemic patients who developed secondary amenorrhoea. Clin Endocrinol (Oxf), 39: 287-96, 1993.
23. De Virgiliis S, Congia M, Frau F, et al. Desferrioxamine-induced growth retardation in patients with thalassemia major. J Pediatr, 113: 661-669, 1988.
24. Olivieri NF, Koren G, Harris J, et al. Growth failure and bony changes induced by desferoxamine. Am J Pediatr Hematol Oncol, 14: 48-56, 1992.
25. Davis BA, Porter JB. Long-term outcome of continuous 24-hour deferoxamine infusion via indwelling intravenous catheters in high-risk beta-thalassemia. Blood, 95: 1229-1236, 2000.
26. Olivieri NF, Nathan DG, MacMillan HJ, et al. Survival in medically treated patients with homozygous beta-thalassemia. N Engl J Med, 331: 574-578, 1994.
27. Al-Refaie FN, Hersko C, Hoffbrand AV, et al. Results of long-term deferiprone (L1) therapy: a report by the International Study Group on Oral Chelators. Br J Haematol, 91: 224-229, 1995.
28. Barman Balfour JA, Foster RH. Deferiprone: A review of its clinical potential in iron overload in beta-thalassemia major and other transfusion-dependent diseases. Drugs, 58: 553-578, 1999.
29. Cohen AR, Galanello R, Piga A, et al. Safety profile of the oral iron chelator deferiprone: a multicentre study. Br J Haematol, 108: 305-312, 2000.
30. Agarwal MB, Gupte SS, Viswanathan C, et al. Long term assessment of efficacy and safety of L1, an oral iron chelator in transfusion dependent thalassemia. Br J Haematol, 82: 460-466, 1992.
31. Olivieri NF, Brittenham GM, McLaren CE, et al. Long-term safety and effectiveness of iron-chelation therapy with deferiprone for thalassemia major. N Engl J Med, 339: 417-423, 1998.
32. Tondury P, Zimmermann A, Nielsen P, et al. Liver iron and fibrosis during long-term treatment with deferiprone in Swiss thalassemic patients. Br J Haematol, 101: 413-415, 1998.
33. Berdoukas V, Boxane T, Eagle C, et al. The Sydney Children's Hospital experience with the oral iron chelator deferiprone (L1). Transfus Sci, 23: 239-240, 2000.
34. Maggio A, D'Amico G, Morabito A, et al. Deferiprone versus Deferoxamine in patients with thalassemia major: a randomized clinical trial. Blood Cells, 28: 196-208, 2002.
35. Wanless IR, Sweeney G, Dhillon AP, et al. Lack of progressive hepatic fibrosis during long term therapy with deferiprone in subjecys with transfusion- dependent beta thalassemia. Blood, 100: 1566-1569, 2002.
36. Anderson LJ, Wonke B, Prescott E, et al. Comparison of effects of oral deferiprone and subcutaneous desferrioxamine on myocardial iron concentration and ventricular function in beta-thalassemia. The Lancet, 360: 516-520, 2002.
37. Sherman M, Koch D, Giardina P, et al. Thalassemic children's understanding of illness: a study of cognitive and emotional factors. Ann NY Acad Sci, 445: 327-336, 1985.
38. Massaglia P, Carpignano M. Psychology of the thalassemia patient and his family. Thalassemia Today; The Mediterranean Experience. Proceedings of the 2nd Mediterranean Meeting, Milano 28-30, 1985.
39. Tsiantis J. Family reactions and relationships in thalassemia. Ann NY Acad Sci, 612: 451-461, 1990.
40. Beratis S. Psychological status in pre-adolescent children with β-thalassemia. J Psychosomat Res, 37: 271-279, 1993.
41. Ratip S, Skuse D, Porter J, et al. Psychological and clinical burden of thalassemia intermedia and its implications for prenatal diagnosis. Arch Dis Child, 72: 408-412, 1995.
42. Ratip S, Modell B. Psychological and sociological aspects of the thalassemias. Semin Hematol, 33: 55-65, 1996.
43. Modell B, Berdoukas V. The clinical approach to thalassemia. London, England, Grune and Stratton, 1984.
44. Savona-Ventura C, Bonello F. Beta-thalassemia syndromes and pregnancy. Obstet Gynecol Surv, 49: 129-37, 1994.
45. Skordis N, Christou S, Koliou M, Pavlides N, Angastiniotis M. Fertility in female patients with thalassemia. J Pediatr Endocrinol Metab, 11 Suppl 3; 935-943, 1998.
46. Eleftheriou A. Compliance to iron chelation therapy with desferrioxamine. In: Thalassemia International Federation, Cyprus, July 2000.
47. Kuo G, Choo QL, Alter HJ, et al. An assay for circulating antibodies to a major etiologic virus of human non-A, non-B hepatitis. Science, 244: 362-364, 1989.
48. De Montalembert M, Costagliola DG, Lefrere JJ, et al. Prevalence of markers for human immunodeficiency virus types 1 and 2, human T-lymphotropic virus type I, cytomegalovirus, and hepatitis B and C virus in multiply transfused thalassemia patients. The French Study Group on Thalassaemia. Transfusion, 32: 509-512, 1992.
49. Prati D, Zanella A, Farma E, et al. A multicenter prospective study on the risk of acquiring liver disease in anti-hepatitis C virus negative patients affected from homozygous beta-thalassemia. Blood, 92: 3460, 1998.
50. Wonke B, Hoffbrand AV, Brown D et al. Antibody to hepatitis C virus in multiply transfused patients with thalassaemia major. J Clin Pathol, 43: 638-640, 1990.
51. Cao A, Galanello R, Rosatelli MC, et al. Clinical Experience of management of thalassemia: the Sardinian experience. Semin Hematol, 33: 66-75, 1996.
52. Alter HJ. To C or not to C. There are the questions. Blood, 85: 1681, 1995.
53. Resnick RH and Koff R. Hepatitis C-related hepatocellular carcinoma. Prevalence and significance. Arch Intern Med, 153: 1672-1677, 1993.
54. Donohue SM, Wonke B, Hoffbrand AV, et al. Alpha interferon in the treatment of chronic hepatitis C virus in multiply transfused patients with thalassaemia major. Br J Haematol, 83: 491-497, 1993.
55. Wedemeyer H, Wiegand J, Cornberg M, et al. Polyethylene glycol-interferon: current status in hepatitis C virus therapy. J Gastroenterol Hepatol, 3: 344-350, 2002.
56. Telfer PT, et al. Combination therapy with interferon alpha and ribavirin for chronic hepatitis C virus infection in thalassemic patients. Br J Haematol, 98: 850-855, 1997.
57. Girot R, Lefrere JJ. HIV infection and AIDS in thalassemia and sickle cell disease. Haematol, 5: 128-131, 1990.
58. Choudhury NV, Dubey ML, Jolly JG, et al. Post-transfusion malaria in thalassaemia patients. Blut, 61: 314, 1990.
59. Lisboa PE. Experimental hepatic cirrhosis in dogs caused by chronic massive iron overload. Gut, 12: 363-368, 1971.
60. Niederau C, Fischer R, Sonnenberg A, et al. Survival and causes of death in cirrhotic and in noncirrhotic patients with primary hemochromatosis. N Engl J Med, 313: 1256-1262, 1985.
61. Fracanzani A, Conte D, Fraquelli M, et al. Increased cancer risk in a cohort of 230 patients with hereditary hemochromatosis in comparison to matched control patients with non-iron-related chronic liver disease. Hepatology, 33: 647, 2001.
62. De Luca G, Maggiolini M, Bria M, et al. Growth hormone secretion in thalassemia patients with short stature. Hormone Res, 44: 158-163, 1995.
63. Wonke B. Clinical Management of beta-Thalassemia Major. Semin Hematol, 38: 350-359, 2001.
64. Canale VC, Steinherz P, New M, et al. Endocrine function in thalassemia major. Ann N Y Acad Sci, 232: 333-345, 1974.
65. De Sanctis V, Pintor C, Aliquò MC et al. Prevalence of endocrine complications in patients with Beta-thalassemia Major: an Italian Multicenter Study. In: C. Pintor, EE Muller, S. Loche MI, eds. New Advances in Pediatric Endocrinology. Milano: Pythagora Press and Berlin-Hedelberg: Springer-Verlag, 127-133, 1992.
66. 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, 331: 567, 1994.
67. Merkel PA, Simonson DC, Amiel SA, et al. Insulin resistance and hyperinsulinemia in patiens with thalassemia major treated by hypertransfusion. N Eng J Med, 318: 809, 1988.
68. Olivieri NF, Britenham GM, Matsui D, et al. Iron chelation therapy with oral deferiprone in patients with thalassemia major. N Eng J Med, 332: 918, 1995.
69. Angelucci E, Brittenham GM, McLaren CE, et al. Hepatic iron concentration and total body iron stores in thalassemia major. N Eng J Med, 343: 327, 2000.
70. Fischer R, Tiemann CD, Engelhardt R, et al. Assessment of iron stores in children with transfusion siderosis by biomagnetic liver susceptometry. Am J Hematol, 60: 289-2999, 1999.
71. Chan YL, Li CK, Lam CW, et al. Liver iron estimation in beta-thalassemia: comparison of MRI biochemical assay and histological grading. Clin Radiol, 56: 911-916, 2001.
72. Olivieri NF, Koren G, Matsui D, et al. Reduction of tissue iron stores and normalization of serum ferritin during treatment with the oral iron chelator L1 in thalassemia intermedia. Blood, 79: 2741-2748, 1992.
73. Berkovitch M, Bistritzer T, Milone SD, et al. Iron deposition in the anterior pituitary in homozygous beta-thalassemia: MRI evaluation and correlation with gonadal function. J Pediatr Endocrinol Metab, 13: 179-184, 2000.
74. Chezmar JL, Nelson RC, Malko JA, et al. Hepatic iron overload: diagnosis and quantification by noninvasive imaging. Gastrointest Radiol, 15: 27-31, 1990.
75. Angelucci E, Giovagnoni A, Valeri G, et al. Limitations of magnetic resonance imaging in measurement of hepatic iron. Blood, 90: 4736-4742, 1997.
76. Anderson LJ, Holden S, Davis B, et al. Cardiovascular T2* magnetic resonance for the early diagnosis of myocardial iron overload. Eur Heart J, 22: 2171-2179, 2001.
77. Anderson L, Porter JB, Wonke B, et al. Cardiac iron deposition is not predicted by conventional markers of iron overload in homozygous beta-thalassemia. Blood, 96 (suppl I): 606a (abstract), 2000.
78. Modell B. Total management of thalassemia major. Arch Dis Child, 52: 489-500, 1977.
79. Cohen A, Markenson AL, Scwarz E. Transfusion requirements and splenectomy in thalassemia major. Journal of Pediatric, 97: 100-102, 1980.
80. Eldor A and Rachmilewitz EA. The hypercoagulable state in thalassemia. Blood, 99: 36-43, 2002.
81. Borgna-Pignatti C, Carnelli V, Caruso V, et al. Thromboembolic events in beta thalassemia major: an Italian multicenter study. Acta Haematol, 99: 76-79, 1998.
82. Lasco A, Morabito N, Gaudio A, et al. Effects of hormonal replacement therapy on bone metabolism in young adults with beta-thalassemia major. Osteoporos Int, 12: 570-575, 2001.
83. Dewey KW, Grossman H, Canale VC. Cholelithiasis in thalassemia major. Radiology, 96: 385-388, 1970.
84. Borgna-Pignatti C, De Stefano P, Panjo D et al. Cholelithiasis in children with thalassemia major. An ultrasonographic study. Pediatrics, 99: 243-244, 1981.
85. Galanello R, Piras S, Barella S, et al. Cholelithiasis and Gilbert's syndrome in homozygous β-thalassemia. Br J Haematol, 115: 926-928, 2001.
86. Weatherall DJ and Clegg JB. The thalassaemia syndromes. In: Blackwell Science Ltd eds. Osney Mead, Oxford, 2001.
87. Karnon J, Zeuner D, Brown J, et al. Lifetime treatment costs of beta-thalassemia major. Clin Lab Haematol, 21: 377-385, 1999.
88. Nick HP, Acklin P, Faller B, et al. ICL670A: A new, potent, orally active chelator. World Congress on Iron Metabolism Bioiron, Sorrento, Italy, May 23-28, 1999 (abstr).
89. Piga A, Galanello R, Cappellini MD, et al. Phase II study of oral chelator ICL670 in thalassaemia patients with transfusional iron overload: efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) after 6 months of therapy. Blood, 100: 5a, 2002.
90. Galanello R, Loehrer F, Alberti D, et al. A novel oral iron chelator (ICL670A): Update on a phase I single-dose safety study. 7th International Conference on Thalassemia and the Haemoglobinopathies, Bangkok, Thailond, May 31-June 4, 1999 (abstr).
91. Giardina PJ, Grady R. Chelation therapy in β-thalassemia: an optimistic update. Semin Hematol, 38: 360-366, 2001.
92. Wonke B, Wright C, Hoffbrand V. Combined therapy with deferipron and desferrioxamine. Br J Haematol, 103: 361-364, 1998.
93. Galanello R, Doneddu I, Origa R, et al. Combination therapy with desferrioxamine and deferiprone. 12th International Conference on Oral Chelation in the Treatment of Thalassemia & Other Disease. 105: Ref ID 1659, 2002.
94. Kattamis A, Kassou Ch, Ladis V, et al. Combination therapy with desferrioxamine and deferiprone in thalassemic patients. 12th International Conference of Oral Chelation in the Treatment of Thalassemia & Other Disease, 105: Ref ID1660, 2002
95. Giardini C, Lucarelli G. Bone marrow transplantation for beta-thalassemia. Hematol Oncol Clin North Am, 13: 1059-1064, 1999.
96. Lucarelli G, Andreani M, Angelucci E. The cure of thalassemia by bone marrow transplantation. Blood Rev, 16: 81-85, 2002.
97. Kernan NA, Bartsch G, Ash RC, et al. Analysis of 462 transplantation from unrelated donors facilitated by the National Marrow Donor Program. New Engl J Med, 328: 593-602, 1993
98. Balduzzi A, Gooley T, Anasetti C, et al. Unrelated donor marrow transplantation in children. Blood, 86: 3247-3256, 1995.
99. La Nasa G, Giardini C, Argiolu F, et al. Unrelated donor bone marrow transplantation for thalassemia: the effect of extended haplotypes. Blood, 99: 4350-4356, 2002.
100. Locatelli F, Rocha V, Reed W. et al. Related umbilical cord blood transplant in patients with thalassemia and Sickle Cell Disease. Blood online, November 7, 2002.
101. Orofino MG, Argiolu F, Sanna MA, et al. Fetal HLA typing in couples at risk for thalassemia: implications for genetic counselling and haematopoietic stem cell transplantation. Blood, 100: 236a, 2002.
102. Pace BS, White GL, Dover GJ, et al. Short-chain fatty acid derivatives induce fetal globin expression and erythropoiesis in vivo. Blood, 100: 4640-4648, 2002
103. Sorrentino BP, Niehuis AW. Prospects for gene therapy of sickle cell disease and thalassemia. In: Steinberg MH, Forget BG, Higgs DR, Nagel RL, eds. Disorders of Hemoglobin: Genetics. Pathopysiology, and Clinical Management. Cambridge, England: Cambridge University Press, 1084-1118, 2001
104. Weatherall DJ. Thalassemia in the next millenium. Annals of the New York Academy of Science. Volume 850, Cooley's Anemia Seventh Symposium, 850: 1-9, 1998.
105. Angastiniotis M, Eleftheriou A, Galanello R, et al. Prevention of thalassemia. TIF 2002.