Pharmacological therapy

Bailliere's Clinical Haematology

Volumn 11, Issue 1, 1998, Pages 239-255

  Rodgers, G P ^a  

^a NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES   (United States)

Author keywords

Fetal haemoglobin; Gene therapy; Haemoglobinopathies; Sickle cell disease; Thalassaemia

Indexed keywords

GLOBIN; HEMOGLOBIN F; HEMOGLOBIN S; HYDROXYUREA;

ARTICLE; BETA THALASSEMIA; BONE MARROW TRANSPLANTATION; CLINICAL FEATURE; ERYTHROCYTE; GENE EXPRESSION REGULATION; GENE THERAPY; GLOBIN GENE; HEMOGLOBINOPATHY; HEMOLYTIC ANEMIA; HUMAN; LIFE EXPECTANCY; PHENOTYPE; PRIMARY PREVENTION; PRIORITY JOURNAL; SECONDARY PREVENTION; SICKLE CELL ANEMIA; TISSUE INJURY;

EID: 0032418810     PISSN: 09503536     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0950-3536(98)80077-2     Document Type: Article

References (83)

1. Abboud M Lauer J Blau CA Granulocytosis causing sickle cell crisis Lancet 351 1998 959
2. Aker M Perry D Dover G Rachmilewitz EA The effect of combined recombinant human erythropoietin and hydroxyurea on erythropoiesis in β-thalassemia intermedia Blood 84 1994 257a
3. Al-Khatti A Papayannopoulou T Knitter G Cooperative enhancement of F-cell formation in baboons treated with erythropoietin and hydroxyurea Blood 72 1988 817 819
4. Arruda VR Lima CSP Saad STO Costa FF Successful use of hydroxyurea in betathalassemia major New England Journal of Medicine 328 1997 964
5. Bachir D Galacteros F Potential alternatives to erythrocyte transfusion in hemoglobinopathies: hydroxyurea (HU), erythropoietin (EPO), butyrate derivatives, blood substitutes Transfusion Clinical Biology 1 1994 35 39
6. Belt RS Haas CD Kennedy J Taylor S Studies of hydroxyurea administered by continuous infusion Cancer 46 1980 455 462
7. Beutler E The effect of methemoglobin formation in sickle cell disease Journal of Clinical Investigation 40 1961 1856 1871
8. Beutler E Effects of sex hormones on fetal hemoglobin levels Journal of the American Medical Association 207 1969 2284 2285
9. Blau CA Constantoulakis P Shaw CM Stamatoyannopoulos G Fetal hemoglobin induction with butyric acid: efficacy and toxicity Blood 81 1993 529 537
10. 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
11. Bunn HF Forget BG Hemoglobin: Molecular, Genetic and Clinical Aspects 1986 W.B. Saunders Philadelphia, PA
12. De Ceulaer K Gruber C Hayes RJ Serjeant GR Medroxyprogesterone acetate and homozygous sickle cell disease Lancet ii 1982 229 231
13. Charache S Dover G Moyer M More J Hydoxyurea-induced augmentation of fetal hemoglobin production in patients with sickle cell anemia Blood 69 1987 109 116
14. Charache S Dover G Smith K Treatment of sickle cell anemia with 5-azacytidine results in increased fetal hemoglobin production and is associated with non random hypomethylation of DNA around the gamma-delta-beta-globin gene complex Proceedings of the National Academy of Sciences of the USA 80 1983 4842 4846
15. Charache S Dover GJ Moore RD Hydroxyurea: effects on hemoglobin F production in patients with sickle cell anemia Blood 69 1992 2555 2565
16. Charache S Terrin ML Moore RD Design of the multicenter study of hydroxyurea in sickle cell anemia Controlled Clinical Trials 16 1995 432 446
17. Charache S Terrin ML Moore RD Effect of hydroxyurea on frequency of painful crises in sickle cell anemia New England Journal of Medicine 332 1995 1317 1322
18. (letter). Collins AF Luban NLC Dover GJ Increased fetal hemoglobin production in patients receiving valporic acid for epilepsy Blood 84 1994 1690
19. Collins AF Pearson HA Giardina P Oral sodium phenylbutyrate therapy in homozygous beta thalassemia: a clinical trial Blood 85 1995 43 49
20. Constantoulakis P Knitter G Stamatoyannopoulos G On the induction of fetal hemoglobin by butyrates: in vivo and in vitro studies with sodium butyrate and comparsion of combination treatment with 5-Aza C and Ara C Blood 74 1989 1963 1971
21. Dover GJ Brusilow S Samid D Increased fetal hemoglobin in patients receiving sodium-4-phenylbutyrate New England Journal of Medicine 327 1992 569 570
22. Dover GJ Brusilow S Charach S Induction of fetal hemoglobin production in subjects with sickle cell anemia by oral sodium phenylbutyrate Blood 84 1994 339 343
23. Dover GJ Charache S Boyer SH 5-Azacytidine increases HbF production and reduces anemia in sickle cell disease: dose-response analysis of subcutaneous and oral dosage regimens Blood 66 1985 527 532
24. Dunbar C Travis W Kan YW Nienhuis A 5-Azacytidine treatment in beta-thalassemic patient unable to be transfused due to multiple alloantibodies British Journal of Haematology 72 1989 467 474
25. Ferster A Vermylen C Cornu G Hydroxyurea for treatment of severe sickle cell anemia: a pediatric clinical trial Blood 88 1996 1960 1964
26. Fibach E Prasanna P Rodgers GP Samid D Enhanced fetal hemoglobin production by phenylacetate and 4-phenylbutyrate in erythroid precusors derived from normal donors and patients with sickle cell disease Blood 82 1993 2203 2206
27. Fucharoen S Siritanaratkul N Winchagoon P Hydroxyurea increases HbF levels and improves the effectiveness of erythropoiesis in b-thalassaemia/HbF disease Blood 1996 887 892
28. Ginder G Whitters M Pohlman J Activation of a chicken-embryonic globin gene in adult erythroid cells by 5-azacytidine and sodium butyrate Proceedings of the National Academy of Sciences of the USA 81 1984 3954 3958
29. Goldberg MA Brugnara C Dover GJ Treatment of sickle cell anemia with hydroxyurea and erythropoietin New England Journal of Medicine 323 1990 366 372
30. Hajjar FM Pearson HA Pharmacologic treatment of thalassemia intermedia with hydroxyurea Journal of Pediatrics 125 1994 490 492
31. el-Hazmi MA Warsy AS Al-Momen A Harakati M Hydroxyurea for the treatment of sickle cell disease Acta Haematologica 88 1992 170 174
32. el-Hazmi MA Al-Momen A Kandaswamy S On the use of hydroxyurea erythropoietin combination therapy for sickle cell disease Acta Haematologica 94 1995 128 134
33. Jayabose S Tugal O Sandoral C Clinical and hematologic effects of hydroxyurea in children with sickle cell anemia Journal of Pediatrics 129 1996 559 565
34. Ley TJ Nienhuis AW Induction of hemoglobin F synthesis in patients with b-thalassemia Annual Review of Medicine 36 1985 485 498
35. Ley TJ DeSimone J Anagnou N 5-Azacytidine selectively increases gamma-globin synthesis in a patient with b-thalassemia New England Journal of Medicine 307 1982 1469 1475
36. Ley TJ DeSimone J Noguchi CT 5-Azacytidine increases gamma-globin synthesis and reduces the proportion of dense cells in patients with sickle cell anemia Blood 62 1983 370 380
37. Little JA Dempsey NJ Tuchman M Ginder GD Metabolic persistence of fetal hemoglobin Blood 85 1995 1712 1718
38. Liu DP Liang CC Ao ZH Treatment of severe beta-thalassemia (patients) with myleran American Journal of Hematology 33 1 1990 50 55
39. Loukopoulos D Vosakaridou E Cozma G Effective stimulation of erythropoietins in thalassemia intermedia with r-hu-erythropoietin and hydroxyurea Blood 82 1993 357a
40. Lowrey C Nienhuis AW Brief report: treatment with azacytidine of patients with end-stage beta-thalassemia New England Journal of Medicine 329 1993 845 848
41. Lucarelli G Giardini C Angelucci E Bone marrow transplantation in thalassemia Winter JN Blood Stem Cell Transplantation 1997 Kluwer Dordrecht 305 315
42. McDonagh KT Nienhuis AW Induction of the human gamma globin gene promoter in K562 cells by sodium butyrate: reversal repression by CCAAT displacement protein Blood 78 1991 255a
43. McDonagh KT Orringer EP Dover GJ Nienhuis AW Hydroxyurea improves erythropoiesis in a patient with homozygous beta-thalassemia Clinical Research 38 1990 346A
44. McDonagh KT Dover GJ Donahue RE Hydroxyurea-induced HbF production in anemic primates: augmentation by erythropoietin, hematopoietic growth factors, and sodium butyrate Experimental Hematology 20 1992 1156 1164
45. de Montalembert M Belloy M Bernaudin F Three year follow-up of hydroxyurea treatment in severely ill children with sickle cell disease Journal of Pediatric Hematology and Oncology 19 1997 313 318
46. Nagel RL Vichinsky E Shah M F reticulocyte response in sickle cell anemia treated with recombinant human erythropoietin: a double-blind study Blood 81 1993 9 14
47. Ohene-Frempong K Horiuchi K Bulgarelli W Hydroxyurea increases HbF production in children with sickle cell disease Blood 82 1993 472a
48. Olivieri NP Vichinsky BP Hydroxyurea in children with sickle cell disease: impact on splenic function and compliance with therapy Journal of Pediatric Hematology and Oncology 20 1998 26 31
49. Olivieri NF Freedman MH Perrine SP Trial of recombinant human erythropoietin: three patients with thalassemia intermedia Blood 80 1992 3258 3260
50. Olivieri NF Nathan DG MacMillan JH Survival in medically treated patients with homozygous beta-thalassemia New England Journal of Medicine 331 1994 574 578
51. Oury AP Hoyoux C Dresse MF Chantraine JM Value of hydroxyurea in children severely affected with sickle cell disease Archives of Pediatrics 4 1997 839 844
52. Papayannoupoulou T De Torrealba R Veith R Arabinosylcytosine induces fetal hemoglobin in baboons by perturbing erythroid differentiation kinetics Science 224 1984 617 619
53. Perrine SP Greene MF Faller DV Delay in the fetal globin switch in infants of diabetic mothers New England Journal of Medicine 312 1985 334 338
54. Perrine SP Rudolph A Faller DV Butyrate infusions in the ovine fetus delay the biologic clock for globin gene switching Proceedings of the National Academy of Sciences of the USA 85 1988 8540 8542
55. Perrine SP Ginder GD Faller DV A short-term trial of butyrate to stimulate fetal-globingene expression in the beta-globin disorders New England Journal of Medicine 328 1993 81 86
56. Platt OS Orkin SH Dover G Hydroxyurea enhances fetal hemoglobin production in sickle cell anemia Journal of Clinical Investigation 74 1984 652 656
57. Platt OS Branbil DJ Rosse WF Mortality in sickle cell disease. Life expectancy and risk factors for early death New England Journal of Medicine 330 1994 1639 1644
58. Rachmilewitz EA, Aker M, Perry D & Dover G (in press) Sustained increase in haemoglobin and RBC following long-term administration of recombinant human erythropoietin to patients with homozygous beta-thalassaemia. British Journal of HaematologyI 90: 341–345.
59. Rigano P Manfrè L La Galla R Clinical and hematological response to hydroxyurea in a patient with Hb Lepore/beta-thalassemia Hemoglobin 21 1997 219 226
60. Riggs MG Whittaker RG Neumann JR Ingram VM N-butyrate causes histone modification in HeLa and Friend erythroleukemia cells Nature 268 1997 462 464
61. Rodgers GP Overview of pathophysiology and rationale for treatment of sickle cell anemia Seminars in Hematology 34 1997 2 7 supplement 3
62. Rodgers GP Dover GJ Noguchi CT Hematologic responses of patients with sickle cell disease to treatment with hydroxyurea New England Journal of Medicine 322 1990 1037 1045
63. Rodgers GP Dover GJ Uyesaka N Augmentation by erythropoietin of the fetal-hemoglobin response to hydroxyurea in sickle cell disease New England Journal of Medicine 328 1993 73 80
64. Rogers Z Hydroxyurea therapy for diverse pediatric populations with sickle cell disease Seminars in Hematology 34 1997 42 47 supplement 3
65. Salim KA Salim A Therapy with sodium valproate is as effective as that with hydroxyurea in preventing painful crisis of sickle cell disease Blood 86 1995 141a
66. Schechter AN Rodgers GP Sickle cell anemia—basic research reaches the clinic New England Journal of Medicine 332 1995 1372 1374
67. Scott JP Hilleeny CA Brown ER Hydroxyurea therapy in children severely affected with sickle cell disease Journal of Pediatrics 128 1996 820 828
68. Selby R Nisbet-Brown E Basran RK Valproic acid and augmentation of fetal hemoglobin in individuals with and without sickle cell disease Blood 90 1997 891 893
69. Sher GD Olivieri FA McCusker PJ Long-term treatment with recombinant human erythropoietin (rHuEpo) in thalassemia: reduction in requirement for transfusion and chelation therapy Blood 84 1994 258a
70. Sher GD Ginder GD Little J Extended therapy with intravenous arginine butyrate in patients with b-hemoglobinopathies New England Journal of Medicine 332 1995 1606 1610
71. DeSimone J Heller P Hall I Zwiers D 5-Azacytidine stimulates fetal hemoglobin synthesis in anemic baboons Proceedings of the National Academy of Sciences of the USA 79 1982 4428 4431
72. Steinberg MH Determinants of fetal hemoglobin response to hydroxyurea Seminars in Hematology 34 1997 8 14 supplement 3
73. Steinberg MH Nagel RL Brugnara C Cellular effects of hydroxyurea in Hb SC disease British Journal of Haematology 98 1997 838 844
74. Sumoza A Bissotti RS Treatment of sickle cell anemia with hydroxyurea: results in twenty-six patients Blood 68 1986 67a
75. Umemura T al-Khatti A Donahue RE Effect of interleukin-3 and erythropoietin on in vivo erythropoiesis and F cell formation in primates Blood 74 1989 1571 1576
76. Veith R Galanello R Papayannopoulou T Stamatoyannopoulos G Stimulation of F-cell production in patients with sickle cell anemia treated with cytarabine or hydroxyurea New England Journal of Medicine 313 1985 1571 1575
77. Veith R Papayannopoulou T Kuracho S Stamatoyannopoulos G Treatment of baboon with vinblastine: insights into the mechanisms of pharmacologic stimulation of HbF in the adult Blood 66 1985 456 459
78. Vermylen C Cornu G Bone marrow transplantation for sickle cell disease: the European experience American Journal of Paediatric Haematology and Oncology 16 1994 18 21
79. Vodopick H Rupp EM Edwardo CL Spontaneous cyclic leukocytosis and thrombosis in chronic granulocytic leukemia New England Journal of Medicine 286 1972 284 290
80. Voskaridou E Kalotychou V Loukopoulos D Clinical and laboratory effects of long-term administration of hydroxyurea to patients with sickle-cell/b-thalassemia British Journal of Haematology 89 1995 479 484
81. Walters MC Patience M Leisenring W Bone marrow transplantation for sickle cell disease New England Journal of Medicine 335 1996 369 376
82. Wood A Davies SC Induction of haemoglobin F in sickle cell anemia by sodium valproate Blood 86 1995 141a
83. Zeng YT Huang SZ Ren ZR Hydroxyurea therapy in b-thalassemia intermedia: improvement in hematological parameters due to enhanced b-globin synthesis British Journal of Haematology 90 1995 557 563