Thalassaemia: Clinical management

Bailliere's Clinical Haematology

Volumn 11, Issue 1, 1998, Pages 147-162

  Olivieri, N ^a  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

Bone marrow transplantation; Chelation; Haemoglobinopathy; Iron overload; Management; Thalassaemia; Transfusions

Indexed keywords

DEFEROXAMINE;

ARTICLE; BLOOD TRANSFUSION; BONE DISEASE; BONE MARROW TRANSPLANTATION; EDEMA; ERYTHROCYTE CONCENTRATE; HEMOGLOBINOPATHY; HEPATITIS C; HUMAN; IRON CHELATION; IRON OVERLOAD; PRIORITY JOURNAL; PROGNOSIS; PRURITUS; SPLENECTOMY; SURVIVAL; THALASSEMIA; THALASSEMIA MAJOR;

EID: 0032445486     PISSN: 09503536     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0950-3536(98)80073-5     Document Type: Article

References (116)

1. Agarwal MB Gupte SS Viswanathan C Long-term assessment of efficacy and safety of L1, an oral iron chelator, in transfusion-dependent thalassaemia: Indian trial British Journal of Haematology 82 1992 460 466
2. Angelucci E Ripalti M Baronciani D Phlebotomy to reduce iron overload in patients cured of thalassemia by marrow transplantation Bone Marrow Transplantation 19 1997 123 125
3. Araujo A Kosaryan M MacDowell A A novel delivery system for continuous desferrioxamine infusion in transfusional iron overload British Journal of Haematology 93 1996 835 837
4. Arruda VR Lima CS Saad ST Costa FF Successful use of hydroxyurea in β-thalassemia major New England Journal of Medicine 336 1997 964
5. (abstract) Atweh GF Dover GJ Faller DV Sustained hematologic response to pulse butyrate therapy in beta globin disorders Blood 88 1996 652a
6. Bachir D Drame M Lee K Clinical effects of hydroxyurea in thalassemia intermedia patients Beuzard Y Lubin B Rosa J Sickle Cell Disease and Thalassemia: New Trends in Therapy Colloques INSERM 1995 195
7. Bard H Prosmanne J Relative rates of fetal hemoglobin and adult hemoglobin synthesis in cord blood of infants of insulin-dependent diabetic mothers Pediatrics 75 1985 1143 1147
8. Borgna-Pignatti C Cohen AR Evaluation of a new method of administration of the iron chelating agent deferoxamine Journal of Pediatrics 130 1997 86 88
9. Borgna-Pignatti C de Stefano P Bongo IG Spleen iron content is low in thalassemia American Journal of Pediatric Hematology/Oncology 6 1984 340 343
10. Bousquet J Navarro M Robert G Rapid desensitization for desferrioxamine anaphylactoid reactions Lancet 2 1983 859 860
11. Brittenham GM Development of iron-chelating agents for clinical use Blood 80 1992 569 574
12. Brittenham GM Disorders of iron metabolism: deficiency and overload Hoffman R Benz E Shattil S Hematology: Basic Principles and Practice 1994 Churchill Livingstone New York 492 523
13. Brittenham GM Farrell DE Harris JW Magnetic-susceptibility measurement of human iron stores New England Journal of Medicine 307 1982 1671 1675
14. Brittenham GM Cohen AR McLaren CE Hepatic iron stores and plasma ferritin concentration in patients with sickle cell anemia and thalassemia major American Journal of Hematology 42 1993 81 85
15. Brittenham GM Griffith PM Nienhuis AW Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major New England Journal of Medicine 331 1994 567 573
16. Bronspiegel-Weintrob N Olivieri NF Tyler BJ Effect of age at the start of iron chelation therapy on gonadal function in β-thalassemia major New England Journal of Medicine 323 1990 713 719
17. Camaschella C Cappellini MD Thalassemia intermedia Haematologica 80 1995 58 68
18. Camaschella C Mazza U Roetto A Genetic interactions in thalassemia intermedia: analysis of β-mutations, α-genotype, γ-promoters, and β-LCR hypersensitive sites 2 and 4 in Italian patients American Journal of Hematology 48 1995 82 87
19. Canatan D Akar N Arcasoy A Effects of calcitonin therapy on osteoporosis in patients with thalassemia Acta Haematologica 93 1995 20 24
20. (abstract) Cappellini MD Graziadei G Ciceri L Phase II open study of oral isobutyramide in patients with thalassemia intermedia Blood 88 1996 311a
21. Carthew P Smith AG Hider RC Potentiation of iron accumulation in cardiac myocytes during the treatment of iron overload with the hydroxypyridinone iron chelator CP94 BioMetals 7 1994 267 271
22. Cartwright GE Edwards CQ Kravitz K Hereditary hemochromatosis: phenotypic expression of the disease New England Journal of Medicine 301 1979 175 179
23. Cazzola M Borgna-Pignatti C Locatelli F A moderate transfusion regimen may reduce iron loading in β-thalassemia major without producing excessive expansion of erythropoiesis Transfusion 37 1997 135 140
24. Clemente MG Congia M Lai ME Effect of iron overload on the response to recombinant interferon-alfa treatment in transfusion-dependent patients with thalassemia major and chronic hepatitis C Journal of Pediatrics 125 1994 123 128
25. Clift RA Johnson FL Marrow transplants for thalassemia: the U.S.A. experience Bone Marrow Transplantation 19 1997 57 59
26. Cohen A Gayer R Mizanin J Longterm effect of splenectomy on transfusion requirements in thalassemia major American Journal of Hematology 30 1989 254 256
27. Cohen AR Martin MB Schwartz E Hydroxyurea therapy in thalassemia intermedia Beuzard Y Lubin B Rosa J Sickle Cell Disease and Thalassemia: New Trends in Therapy Colloques INSERM 1995 193 194
28. Cohen AR Schmidt JM Martin MB Clinical trial of young red cell transfusions Journal of Pediatrics 104 1984 865 868
29. (abstract) Collins AF Belluzzo N Lorenzana A Osteopenia in young adults with homozygous beta thalassemia: persistence of an old problem Blood 82 1993 358a
30. Collins AF Dias GC Haddad S Evaluation of a new neocyte transfusion preparation vs. washed cell transfusion in patients with homozygous beta thalassemia Transfusion 34 1994 517 520
31. Collins AF Pearson HA Giardina P Oral sodium phenylbutyrate therapy in homozygous beta thalassemia Blood 85 1995 43 49
32. Contu L La Nasa G Arras M Successful unrelated bone marrow transplantation in thalassemia Bone Marrow Tranplantation 13 1994 329 331
33. Donohue SM Wonke B Hoffbrand AV Alpha interferon in the treatment of chronic hepatitis C infection in thalassaemia major British Journal of Haematology 83 1993 491 497
34. Fosburg MT Nathan DG Treatment of Cooley's anaemia Blood 76 1990 435 444
35. Fucharoen S Siritanaratkul N Winichagoon P Hydroxyurea increases hemoglobin F levels and improves the effectiveness of erythropoiesis in β-thalassemia/hemoglobin E disease Blood 87 1996 887 892
36. Gaziev D Polchi P Galimberti M Graft-versus-host disease following bone marrow transplantation for thalassemia: an analysis of incidence and risk factors Transplantation 63 1997 854 860
37. Giardini C Treatment of β-thalassemia Current Opinion in Hematology 4 1997 79 87
38. Giardini C Galimberti M Lucarelli P Desferrioxamine therapy accelerates clearance of iron deposits after bone marrow transplantation for thalassemia British Journal of Haematology 89 1995 868 873
39. Graziano JH Piomelli S Hilgartner M Chelation therapy in β-thalassemia major, III. The role of splenectomy in achieving iron balance Journal of Pediatrics 99 1981 695 699
40. Hajjar FM Pearson HA Pharmacologic treatment of thalassemia intermedia with hydroxyurea Journal of Pediatrics 125 1994 490 492
41. Hallaway PE Eaton JW Panter SS Hedlund BE Modulation of deferoxamine toxicity and clearance by covalent attachment to biocompatible polymers Proceedings of the National Academy of Sciences of the USA 86 1989 10108 10112
42. Hider RC, Kontoghiorghes GJ & Silver J (1982) UK Patent GB-2118176.
43. Ho PJ Hall GW Luo LY Beta-thalassaemia intermedia: is it possible consistently to predict phenotype from genotype? British Journal of Haematology 100 1998 70 78
44. Hoffbrand AV Oral iron chelation Seminars in Hematology 33 1996 1 8
45. Hoffbrand AV al-Refaie F Davis B Long-term trial of deferiprone in 51 transfusion-dependent iron overloaded patients Blood 91 1998 295 300
46. Huang S-Z Ren Z-R Chen M-J Treatment of β-thalassemia with hydroxyurea. Effects of HU on globin gene expression Science in China (Series B) 37 1994 1350 1359
47. Issagrisil S Visuthisakchai S Suvatte V Brief report: transplantation of cord-blood stem cells into a patient with severe thalassemia New England Journal of Medicine 332 1995 367 369
48. Jensen PD Jensen FT Christensen T Ellegaard J Evaluation of transfusional iron overload before and during iron chelation by magnetic resonance imaging of the liver and determination of serum ferritin in adult non-thalassaemic patients British Journal of Haematology 89 1995 880 889
49. Kevy SV Jacobson MS Fosburg M A new approach to neocyte transfusion: preliminary report Journal of Clinical Apheresis 4 1988 194 197
50. Kheradpir MH Albouyeh M Partial splenectomy in the treatment of thalassaemia major Kinderchirurgie 40 1985 195 198
51. Kontoghiorghes GJ Bartlett AN Hoffbrand AV Long-term trial with the oral iron chelator 1,2-dimethyl-3-hydroxypyrid-4-one (L1) British Journal of Haematology 76 1990 295 300
52. Lai ME De Virgiliis S Argiolu F Evaluation of antibodies to hepatitis C virus in a long-term prospective study of post-transfusion hepatitis among thalassemic children: comparison between first and second-generation assay Journal of Pediatric Gastroenterology and Nutrition 16 1993 458 464
53. (abstract). Lai E Belluzzo N Muraca MF The prognosis for adults with thalassemia major: Sardinia, 1995 Blood 86 1995 251a
54. Liu P Olivieri N Iron overload cardiomyopathies: new insights into an old disease Cardiovascular Drugs and Therapy 8 1994 101 110
55. Lombardo T Ferro G Frontini V Percolla S High-dose intravenous desferrioxamine (DFO) delivery in four thalassemic patients allergic to subcutaneous DFO administration American Journal of Hematology 51 1996 90 92
56. Lombardo T Frontini V Ferro G Laboratory evaluation of a new delivery system to improve patient compliance with chelation therapy Clinical and Laboratory Haematology 18 1996 13 17
57. Loreal O Deugnier Y Moirand R Liver fibrosis in genetic hemochromatosis. Respective roles of iron and non-iron related factors in 127 homozygous patients Journal of Hepatology 16 1992 122 127
58. Intercept Loukopoulos D Voskaridou E Stamoulakatou A Clinical trials with hydroxyurea and recombinant human erythropoietin Stamatoyannopoulos G Molecular Biology of Hemoglobin Switching 1995 365 372
59. Lucarelli G Giardini C Baronciani D Bone marrow transplantation in β-thalassemia Seminars in Hematology 32 1995 297 303
60. Lucarelli G Clift R Galimberti M Marrow transplantation for patients with thalassemia: results in class 3 patients Blood 87 1996 2082 2088
61. (abstract). McDonagh KT Orringer EP Dover GJ Nienhuis AW Hydroxurea improves erythropoiesis in a patient with homozygous beta thalassemia Clinical Research 38 1990 346a
62. Di Marco V Lo Iacono O Almasio P Long-term efficacy of a-interferon in β-thalassemics with chronic hepatitis C Blood 90 1997 2207 2212
63. Mariotti E Agostini A Angelucci E Reduced left ventricular contractile reserve identified by low dose dobutamine echocardiography as an early marker of cardiac involvement in asymptomatic patients with thalassemia major Echocardiography 13 1996 463 472
64. Mariotti E Agostini A Angelucci E Reversal of the initial cardiac damage in thalassemic patients treated with bone marrow transplantation and phlebotomy Bone Marrow Transplantation 19 1997 139 141
65. Miller KB Rosenwasser LJ Bessette JA Rapid desensitisation for desferrioxamine anaphylactic reaction Lancet 1 1981 1059
66. de Montalembert M Girot R Revillon Y Partial splenectomy in homozygous β thalassaemia Archives of Diseases in Childhood 65 1989 304 307
67. Neinhuis AW Ley TJ Humphries RK Pharmacological manipulation of fetal hemoglobin synthesis in patients with severe beta thalassemia Annals of the New York Academy of Sciences 445 1985 198 211
68. Niederau C Fischer R Purschel A Long-term survival in patients with hereditary hemochromatosis Gastroenterology 110 1996 1304 1307
69. Okon E Levij IS Rachmilewitz EA Splenectomy, iron overload and liver cirrhosis in β-thalassemia major Acta Haematologica 56 1976 142 150
70. (abstract). Olivieri NF Long-term followup of body iron in patients with thalassemia major during therapy with the orally active iron chelator deferiprone (L1) Blood 88 1996 310a
71. (abstract). Olivieri NF Randomized trial of deferiprone (L1) and deferoxamine (DFO) in thalassemia major Blood 88 1996 651a
72. Olivieri NF Reactivation of fetal hemoglobin in patients with β-thalassemia Seminars in Hematology 33 1996 24 42
73. Olivieri NF Brittenham GM Iron-chelating therapy and the treatment of thalassemia Blood 89 1997 739 761
74. Olivieri NF Koren G Hermann C Comparison of oral iron chelator L1 and desferrioxamine in iron-loaded patients Lancet 336 1990 1275 1279
75. Olivieri NF Berriman AM Davis SA Continuous intravenous administration of deferoxamine in adults with severe iron overload American Journal of Hematology 41 1992 61 63
76. Olivieri NF Koren G Matsui D Reduction of tissue iron stores and normalization of serum ferritin during treatment with the oral iron chelator L1 in thalassemia intermedia Blood 79 1992 2741 2748
77. Olivieri NF Nathan DG MacMillan JH Survival of medically treated patients with homozygous β thalassemia New England Journal of Medicine 331 1994 574 578
78. Olivieri NF Brittenham GM Matsui D Iron-chelation therapy with oral deferiprone in patients with thalassemia major New England Journal of Medicine 332 1995 918 922
79. (abstract). Olivieri NF Nisbet-Brown E Dragsten P Studies of iron excretion and non-transferrin-bound iron following a single infusion of hydroxyethyl starch-deferoxamine: a new approach to iron chelation therapy Blood 88 1996 310a
80. Olivieri NF Rees DC Ginder GD Treatment of thalassemia major with phenylbutyrate and hydroxyurea Lancet 350 1997 491 492
81. Olson LJ Edwards WD McCall JT Cardiac iron deposition in idiopathic hemochromatosis: histologic and analytic assessment of 14 hearts from autopsy Journal of the American College of Cardiology 10 1987 1239 1243
82. Orvieto R Leichter I Rachmilewitz EA Margulies JY Bone density, mineral content, and cortical index in patients with thalassemia major and the correlation to their bone fractures, blood transfusions, and treatment with desferrioxamine Calcified Tissue International 50 1992 397 399
83. Perrine SP Greene MF Faller DV Delay in the fetal globin switch in infants of diabetic monthers New England Journal of Medicine 312 1985 334 338
84. Perrine SP Ginder GD Faller DV A short-term trial of butyrate to stimulate fetal-globingene expression in the β-globin disorders New England Journal of Medicine 328 1993 81 86
85. Perrine SP Dover GH Daftari P Isobutyramide, an orally bioavailable butyrate analogue, stimulates fetal globin gene expression in vitro and in vivo British Journal of Haematology 88 1994 555 561
86. Piomelli S Danoff S Becker M Prevention of bone malformations and cardiomegaly in Cooley's amemia by early hypertransfusion regimen Annals of the New York Academy of Sciences 165 1969 427 436
87. Piomelli S Seaman C Reibman J Separation of younger red cells with improved survival in vivo: an approach to chronic transfusion therapy Proceedings of the National Academy of Sciences of the USA 75 1978 3474 3478
88. Pippard MJ Measurement of iron status Progress in Clinical and Biological Research 309 1989 85 92
89. Pippard MJ Callender ST Warner GT Weatherall DJ Iron absorption and loading in betathalassaemia intermedia Lancet 2 1979 819 821
90. Politis C Spigos DG Georgiopoulou P Partial splenic embolisation for hypersplenism of thalassaemia major: five year follow-up British Medical Journal Clinical Research Edition 294 1987 665 667
91. Pootrakul P Rugkiatsakul R Wasi P Increased transferrin iron saturation in splenectomized thalassaemic patients British Journal of Haematology 46 1980 143 145
92. Pootrakul P Kitcharoen KM Yansukon P The effect of erythroid hypreplasia on iron balance Blood 71 1988 1124 1129
93. Porter J Huehns ER The toxic effects of desferrioxamine Baillière's Clinical Haematology vol. 2 1989 Baillière Tindall London 459 474
94. Porter JB Jaswon MS Huehns ER Desferrioxamine ototoxicity: evaluation of risk factors in thalassaemic patients and guidelines for safe dosage British Journal of Haematology 73 1989 403 409
95. Preston H Wright TL Interferon therapy for hepatitis C Lancet 348 1996 973 974
96. al-Refaie FN Hershko C Hoffbrand AV Results of long-term deferiprone (L1) therapy. A report by the International Study Group on Oral Iron Chelators British Journal of Haematology 91 1995 224 229
97. Richardson ME Mathews RN Alison JF Prevention of heart disease by subcutaneous desferrioxamine in patients with thalassaemia major Australia and New Zealand Journal of Medicine 23 1993 656 661
98. Rigano P Manfré L La Galla R Clinical and hematologic response to hydroxyurea in a patient with Hb Lepore/β-thalassemia Hemoglobin 21 1997 219 226
99. Roberts IAG Darbyshire PJ Will AM B.M.T. for children with b-thalassemia major in the U.K. Bone Marrow Transplantation 19 1997 60 61
100. Rund D Oron-Karni V Filon D Genetic analysis of β-thalassemia intermedia in Israel: diversity of mechanisms and unpredictability of phenotype American Journal of Hematology 54 1997 16 22
101. Shalit M Tedeschi A Miadonna A Levi-Shaffer A Desferal (desferrioxamine)—a novel activator of connective tissue-type mast cells Journal of Allergy and Clinical Immunology 6 1991 854 860
102. Sher GD Ginder G Little JA Extended therapy with arginine butyrate in patients with thalassemia and sickle cell disease New England Journal of Medicine 332 1995 106 110
103. Sherlock S Antiviral therapy for chronic hepatitis C viral infection Journal of Hepatology 23 1995 3 7
104. Simon TL Sohmer P Nelson EF Extended survival of neocytes produced by a new system Transfusion 29 1989 221 225
105. Spanos T Ladis V Palamidou F The impact of neocyte transfusion in the management of thalassaemia Vox Sanguinis 70 1996 217 223
106. Tinè F Magrin S Craxì A Pagliaro L Interferon for non-A, non-B chronic hepatitis: a metaanalysis of randomized clinical trials Journal of Hepatology 13 1991 192 199
107. Tondury P Kontoghiorghes GJ Ridolfi-Luthy AR L1 (1,2-dimethyl-3-hydroxypyrid-4-one) for oral iron chelation in patients with beta-thalassaemia major British Journal of Haematology 76 1990 550 553
108. Tong MT El-Farra NS Reikes AR Co RL Clinical outcomes after transfusion-associated hepatitis C New England Journal of Medicine 332 1995 1463 1466
109. Vellodi A Picton S Downie CJC Bone marrow transplantation for thalassemia: experience of two British centres Bone Marrow Transplantation 13 1994 559 562
110. Walters MC Thomas ED Bone marrow transplantation for thalassemia: the United States experience American Journal of Pediatric Hematology/Oncology 16 1994 1 17
111. Weatherall DJ Clegg JB The β thalassaemias Weatherall DJ Clegg JB The Thalassaemia Syndromes 1981 Blackwell Scientific Oxford 148 319
112. Westgren M Ringden O Eik-Nes S Lack of evidence of permanent engraftment after in utero fetal stem cell transplantation in congenital hemoglobinopathies Transplantation 61 1996 1176 1179
113. Wong A Alder V Robertson D Liver iron depletion and toxicity of the iron chelator deferiprone (L1, CP20) in the guinea pig Biometals 10 1997 247 256
114. Wood WG Increased HbF in adult life Clinical Haematology 6 1993 177 213
115. Zeng Y-T Huang S-Z Ren Z-R Hydroxyurea therapy in β-thalassaemia intermedia: improvement in haematological parameters due to enhanced β-globin synthesis British Journal of Haematology 90 1995 557 563
116. Zurlo MG De Stefano P Borgna-Pignatti C Survival and causes of death in thalassaemia major Lancet 2 1989 27 30