The therapeutic reactivation of fetal haemoglobin

Human Molecular Genetics

Volumn 7, Issue 10, 1998, Pages 1655-1658

  Olivieri, Nancy F ^a   Weatherall, D J ^b  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

^b JOHN RADCLIFFE HOSPITAL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

AZACITIDINE; BUTYRIC ACID DERIVATIVE; CYTARABINE; HEMOGLOBIN F; HYDROXYUREA; RECOMBINANT ERYTHROPOIETIN;

ARTICLE; BETA THALASSEMIA; CLINICAL TRIAL; DUCHENNE MUSCULAR DYSTROPHY; HEMOGLOBIN SYNTHESIS; HUMAN; INTRAVENOUS DRUG ADMINISTRATION; PRIORITY JOURNAL; SICKLE CELL ANEMIA;

ANEMIA, SICKLE CELL; ANTISICKLING AGENTS; BETA-THALASSEMIA; BUTYRATES; CLINICAL TRIALS; FETAL HEMOGLOBIN; HUMANS; HYDROXYUREA; UP-REGULATION;

EID: 0031712378     PISSN: 09646906     EISSN: None     Source Type: Journal    
DOI: 10.1093/hmg/7.10.1655     Document Type: Article

References (46)

1. Wood, W.G. and Weatherall, D.J. (1983) Developmental genetics of the human hemoglobins. Biochem. J., 215, 1-10.
2. Deconinck, N., Tinsley, J., De-Backer, F., Fisher, R., Kahn, D., Phelps, S., Davies, K. and Gillis, J.M. (1997) Expression of truncated utrophin leads to major functional improvements in dystrophin-deficient muscles of mice. Nature Med., 3, 1216-1221.
3. Wood, W.G. (1993) Increased HbF in adult life. Clin. Haematol., 6, 177-213.
4. Dover, G.J., Smith, K.D., Chang, Y.C., Purvis, S., Mays. A., Meyers, D.A., Sheils, C. and Serjeant, G. (1992) Fetal hemoglobin levels in sickle cell disease and normal individuals are partially controlled by an X-linked gene located at Xp22.2. Blood, 80, 816-824.
5. Grosveld, F., Dillon, N. and Higgs, D. (1993) The regulation of human globin gene expression. Clin. Haematol., 6, 31-55.
6. Weatherall, D.J. and Clegg, J.B. (1981) The Thalassaemia Syndromes. Blackwell Scientific. Oxford.
7. Rees, D.C., Clegg, J.B. and Weatherall, D.J. (1998) Why are haemoglobin F levels raised in Hb E/β thalassaemia? Br. J. Haematol., 101, 103.
8. Craig, J.E., Rochette, J., Fisher, C.A., Weatherall, D.J., Marc, S., Lathrop, G.M., Demenais, F. and Thein, S.L. (1996) Haemoglobin switch: dissecting the loci controlling fetal haemoglobin production on chromosomes 11p and 6q by the regressive approach. Nature Genet., 12, 58-64.
9. Sheridan, B.L., Weatherall, D.J., Clegg, J.B., Pritchard, J., Wood, W.G., Callender, S.T., Durrant, I.J., McWhirter, W.R., Ali, M., Partridge, J.W. and Thompson, E.N. (1976) The patterns of fetal haemoglobin production in leukaemia. Br. J. Haematol., 32, 487.
10. Alter, B.P., Rappeport, J.M., Huisman, T.H.J., Schroeder, W.A. and Nathan, D.G. (1976) Fetal erythropoiesis following bone marrow transplantation. Blood, 48, 843-854.
11. DeSimone, J., Heller, P., Hall, L. and Zwiers, D. (1982) 5-Azacytidine stimulates fetal hemoglobin synthesis in anemic baboons. Proc. Natl Acad. Sci. USA, 79, 4428-4431.
12. Stamatoyannopoulos, J.A. and Nienhuis, A.W. (1992) Therapeutic approaches to hemoglobin switching in treatment of hemoglobinopathies. Anna. Rev. Med., 43, 497-521.
13. Bard, H. and Prosmanne, J. (1985) Relative rates of fetal hemoglobin and adult hemoglobin synthesis in cord blood of infants of insulin-dependent diabetic mothers. Pediatrics, 75, 1143-1147.
14. Perrine, S.P., Greene, M.F. and Faller, D.V. (1985) Delay in the fetal globin switch in infants of diabetic mothers. N. Engl. J Med., 312, 334-338.
15. Samid, D., Yeh, A. and Prasanna, P. (1992) Induction of erythroid differentiation and fetal hemoglobin production in human leukemic cells treated with phenylacetate. Blood, 80, 1576-1581.
16. Olivieri, N.F. (1996) Reactivation of fetal hemoglobin in patients with β thalassemia. Semin. Hematol., 33, 24-42.
17. Platt, O., Orkin, S., Dover, G.J., Beardsley, G.P., Miller, B. and Nathan, D.G. (1984) Hydroxyurea enhances fetal hemoglobin production in sickle cell anemia. J. Clin. Invest., 74, 652-656.
18. Charache, S. and Dover, G.J. (1987) Hydroxyurea therapy for sickle cell anemia. Blood. 69, 109-116.
19. Dover, G.J., Humphries, R.K., Moore, J.G., Ley, T.J., Young, N.S., Charache, S. and Nienhuis, A.W. (1986) Hydroxyurea induction of hemoglobin F production in sickle cell disease: relationship between cytotoxicity and F cell production. Blood, 67, 735-738.
20. Charache, S. (1997) Mechanism of action of hydroxyurea in the management of sickle cell anemia in adults. Sem. Hematol., 34 (suppl. 3), 15-21.
21. Scott, J.P., Hillery, C.A., Brown, E.R., Misiewicz, V.M. and Labotka, R.J. (1996) Hydroxyurea therapy in children severely affected with sickle cell disease. J. Pediatr., 128, 820-828.
22. Ferster, A., Vermylen, C., Cornu, G., Buyse, M., Corazza, F., Devalck, C., Fondu, P., Toppet, M. and Sariban, E. (1996) Hydroxyurea for treatment of severe sickle cell anemia: a pediatric clinical trial. Blood, 88, 1960-1964.
23. Jayabose, S., Tugal, O., Sandoval, C., Patel, P., Puder, D., Lin, T. and Visintainer, P. (1996) Clinical and hematologic effects of hydroxyurea in children with sickle cell anemia. J. Pediatr., 129, 559-565.
24. McDonagh, K.T., Orringer, E.P., Dover, G.J. and Nienhuis, A.W. (1990) Hydroxyurea improves erythropoiesis in a patient with homozygous beta thalassemia. Clin. Res., 38, 346A.
25. Hajjar, F.M. and Pearson, H.A. (1994) Pharmacologic treatment of thalassemia intermedia with hydroxyurea. J. Pediatr., 125, 490-492.
26. Huang, S.-Z., Ren, Z.-R., Chen, M.-J., Xu, H.-P., Zeng, Y.-T., Rodgers, G.P., Zeng, F.-Y. and Schechter, A.N. (1994) Treatment of β-thalassemia with hydroxyurea. Effects of HU on globin gene expression. Sci. China (Ser. B), 37, 1350-1359.
27. Bachir, D., Drame, M., Lee, K., Portos, J.-L. and Galacteros, F. (1995) Clinical effects of hydroxyurea in thalassemia intermedia patients. In Beuzard, Y., Lubin, B. and Rosa, J. (eds), Sickle Cell Disease and Thalassemia: New Trends in Therapy. Colloque INSERM, 234, 195.
28. Zeng, Y.-T., Huang, S.-R., Ren, Z.-R., Zeng, F-Y., Schechter, A.N. and Rodgers, G.P. (1995) Hydroxyurea therapy in β-thalassaemia: improvement in haematological parameters due to enhanced β-globin synthesis. Br. J. Haematol., 90, 557-563.
29. Cohen, A.R., Martin, M.B. and Schwartz, E. (1995) Hydroxyurea therapy in thalassemia intermedia. In Beuzard, Y., Luhin, B. and Rosa, J. (eds), Sickle Cell Disease and Thalassemia: New Trends in Therapy. Colloque INSERM, 234, 194.
30. Fucharoen, S., Siritanaratkul, N., Winichagoon, P., Chowthaworn, J., Siriboon, W., Muangsup, W., Chaicharoen, S., Poolsup, N., Chindavijak, B., Pootrakul, P., Piankijagum, A., Schechter, A.N. and Rodgers, G.P. (1996) Hydroxyurea increases hemoglobin F levels and improves the effectiveness of erythropoiesis in β-thalassemia/hemoglobin E disease. Blood, 87, 887-892.
31. Ley, T.J., DeSimone, J., Anagnou, N.P., Keller, G.H., Humphries, R.K., Turner, P.H., Young, N.S., Heller, P. and Nienhuis, A.W. (1982) 5-Azacytidine selectively increases γ-globin synthesis in a patient with β-thalassemia. N. Engl. J. Med., 307, 1469.
32. Nienhuis, A.W., Ley, T.J., Humphries, R.K., Young, N.S. and Dover, G.J. (1985) Pharmacological manipulation of fetal hemoglobin synthesis in patients with severe beta thalassemia. Ann. NY Acad. Sci., 445, 198-211.
33. Loukopoulos, D., Voskaridou, E., Stamoulakatou, A. et al. (1995) Clinical trials with hydroxyurea and recombinant human erythropoietin. In Stamatoyannopoulos, G. and Nienhuis, A. W. (eds). Hemoglobin Switching. Alan R. Liss, New York, pp. 365-372.
34. Goldberg, M.A., Brugnara, C., Dover, G.J., Schapira, I., Charache, S. and Bunn, H.F. (1990) Treatment of sickle cell anemia with hydroxyurea and erythropoietin. N. Engl. J. Med., 323, 366-372.
35. Rodgers, G.P., Dover, G.J., Uyesaka, N., Noguchi, C.T., Schechter, A.N. and Nienhuis, A.W. (1993) Augmentation by erythropoietin of the fetal-hemoglobin response to hydroxyurea in sickle cell disease. N. Engl. J. Med., 328, 73-80.
36. Rachmilewitz, E.A., Goldfarb, A. and Dover, G. (1991) Administration of erythropoietin to patients with β-thalassemia intermedia: a preliminary trial. Blood, 78, 1145-1147.
37. Olivieri, N.F., Freedman, M., Perrine, A., Dover, G.J., Sheridan, B., Esseltine, D.L. and Nagel, R.L. (1992) Trial of recombinant human erythropoietin in thalassemia intermedia. Blood, 80, 3258-3260.
38. Aker, M., Dover, G., Schrier, S. and Rachmilewitz, E.A. (1993) Sustained increase in hemoglobin, hematocrit and RBC following long term administration of recombinant human erythropoietin to patients with beta thalassemia intermedia. Blood, 82, 358a.
39. Loukopoulos, D., Voskaridou, E., Cozma, C., Stamoulakatou, A., Papasotiriou, J., Xenaki, M., Paterakis, G., Papadakis, M. and Loutradi, A. (1993) Effective stimulation of erythropoiesis in thalassemia intermedia with recombinant human erythropoietin and hydroxyurea. Blood, 82, 357a.
40. Perrine, S.P., Ginder, G.D., Faller D.V., Dover, G.H., Ikuta, T., Witkowska, E., Cai, S.-P., Vichinsky, E.P. and Olivieri, N.F. (1993) A short-term trial of butyrate to stimulate fetal-globin-gene expression in the β-globin disorders. N. Engl. J. Med., 328, 81-86.
41. Sher, G.D., Ginder, G., Little, J.A., Wang, S.Y., Dover, G. and Olivieri, N.F. (1995) Extended therapy with arginine butyrate in patients with thalassemia and sickle cell disease. N. Engl. J. Med., 332, 106-110.
42. Olivieri, N.F., Rees, D.C., Ginder, G.D., Thein, S.L., Brittenham, G.M., Waye, J.S. and Weatherall, D.J. (1997) Treatment of thalassaemia major with phenylbutyrate and hydroxyurea. Lancet, 350, 491-492.
43. Rigano, P., Manfré, L., La Galla, R., Renda, D., Renda, M.C., Calabrese, A., Calzolari, R. and Maggio, A. (1997) Clinical and hematologic response to hydroxyurea in a patient with Hb Lepore/β-thalassemia. Hemoglobin, 21, 219-226.
44. Collins, A.F., Pearson, H.A., Giardina, P., McDonagh, K.T., Brusilow, S.W. and Dover, G.J. (1995) Oral sodium phenylbutyrate therapy in homozygous beta thalassemia. Blood, 85, 43-49.
45. Dover, G.J., Brusilow, S. and Charache, S. (1994) Induction of fetal hemoglobin production in subjects with sickle cell anemia by oral sodium phenylbutyrate. Blood, 84, 339-343.
46. Faller, D.V. and Perrine, S.P. (1995) Butyrate in the treatment of sickle cell disease and β-thalassemia. Curr. Opin. Hematol., 2, 109-117.