Single and combination drug therapy for fetal hemoglobin augmentation in hemoglobin E-β°-thalassemia: Considerations for treatment

Annals of the New York Academy of Sciences

Volumn 1054, Issue , 2005, Pages 250-256

  Singer, Sylvia T ^a   Kuypers, Frans A ^a   Olivieri, Nancy F ^b   Weatherall, David J ^c   Mignacca, Robert ^d   Coates, Thomas D ^e   Davies, Sally ^f   Sweeters, Nancy ^a   Vichinsky, Elliott P ^a  

^a CHILDREN S HOSPITAL OAKLAND   (United States)

^b UNIVERSITY OF TORONTO   (Canada)

^c UNIVERSITY OF OXFORD   (United Kingdom)

^d CHILDREN S HOSPITAL CENTRAL CALIFORNIA   (United States)

^e CHILDREN S HOSPITAL LOS ANGELES   (United States)

^f CENTRAL MIDDLESEX HOSPITAL   (United Kingdom)

Author keywords

Erythropoietin; Hb E thalassemia; Hemoglobin; Hydroxyurea

Indexed keywords

ARYLBUTYRIC ACID DERIVATIVE; DEFEROXAMINE MESYLATE; HEMOGLOBIN E; HEMOGLOBIN F; HYDROXYUREA; RECOMBINANT ERYTHROPOIETIN;

BETA THALASSEMIA; BLOOD TRANSFUSION; CLINICAL ARTICLE; CLINICAL TRIAL; CONFERENCE PAPER; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; FOLLOW UP; HEMOGLOBINOPATHY; HUMAN; LIVER TOXICITY; NEUTROPENIA; TREATMENT FAILURE;

EID: 29744445074     PISSN: 00778923     EISSN: None     Source Type: Book Series    
DOI: 10.1196/annals.1345.031     Document Type: Conference Paper

References (25)

1. NIENHUIS, A.W., T.J. LEY, R.K. HUMPHRIES, et al. 1985. Pharmacological manipulation of fetal hemoglobin synthesis in patients with severe beta-thalassemia. Ann. N.Y. Acad. Sci. 445: 198-211.
2. OLIVIERI, N.F. 1997. Fetal erythropoiesis and the diagnosis and treatment of hemoglobin disorders in the fetus and child. Semin. Perinatol. 21: 63-69.
3. WEATHERALL, D.J. & J.B. CLEGG. 2001. Inherited haemoglobin disorders: an increasing global health problem. Bull. WHO 79: 704-712.
4. WEATHERALL, D. 2001. The Thalassemia Syndromes. Blackwell Science. Maldon, MA.
5. ANGASTINIOTIS, M. & B. MODELL. 1998. Global epidemiology of hemoglobin disorders. Ann. N. Y. Acad. Sci. 850: 251-269.
6. LOREY, F. 2000. Asian immigration and public health in California: thalassemia in newborns in California. J. Pediatr. Hematol. Oncol. 22: 564-566.
7. MODELL, B., M. KHAN, M. DARLISON, et al. 2001. A national register for surveillance of inherited disorders: beta thalassaemia in the United Kingdom. Bull. WHO 79: 1006-1013.
8. FUCHAROEN, S. & P. WINICHAGOON. 2000. Clinical and hematologic aspects of hemoglobin E beta-thalassemia. Curr. Opin. Hematol. 7: 106-112.
9. DE SILVA, S., C.A. FISHER, A. PREMAWARDHENA, et al. 2000. Thalassaemia in Sri Lanka: implications for the future health burden of Asian populations. Sri Lanka Thalassaemia Study Group. Lancet 355: 786-791.
10. WINICHAGOON, P., V. THONGLAIROAM, S. FUCHAROEN, et al. 1993. Severity differences in beta-thalassaemia/haemoglobin E syndromes: implication of genetic factors. Br. J. Haematol. 83: 633-639.
11. WEATHERALL, D.J. 2000. Introduction to the problem of hemoglobin E-beta thalassemia. J. Pediatr. Hematol. Oncol. 22: 551.
12. FUCHAROEN, S., P. WINICHAGOON, P. POOTRAKUL, et al. 1987. Variable severity of Southeast Asian beta 0-thalassemia/Hb E disease. Birth Defects Orig. Artic. Ser. 23: 241-248.
13. REES, D.C., L. STYLES, E.P. VICHINSKY, et al. 1998. The hemoglobin E syndromes. Ann. N.Y. Acad. Sci. 850: 334-343.
14. DE PAULA, E.V., C.S. LIMA, V.R. ARRUDA, et al. 2003. Long-term hydroxyurea therapy in beta-thalassaemia patients. Eur. J. Haematol. 70: 151-155.
15. KOHLI-KUMAR, M., H. MARANDI, M.A. KELLER, et al. 2002. Use of hydroxyurea and recombinant erythropoietin in management of homozygous beta0 thalassemia. J. Pediatr. Hematol. Oncol. 24: 777-778.
16. BRADAI, M., M.T. ABAD, S. PISSARD, et al. 2003. Hydroxyurea can eliminate transfusion requirements in children with severe beta-thalassemia. Blood 102: 1529-1530.
17. FUCHAROEN, S., N. SIRITANARATKUL, P. WINICHAGOON, et al. 1996. Hydroxyurea increases hemoglobin F levels and improves the effectiveness of erythropoiesis in beta-thalassemia/hemoglobin E disease. Blood 87: 887-892.
18. COLLINS, A.F., H.A. PEARSON, P. GIARDINA, et al. 1995. Oral sodium phenylbutyrate therapy in homozygous beta thalassemia: a clinical trial. Blood 85: 43-49.
19. RACHMILEWITZ, E.A., M. AKER, D. PERRY & G. DOVER. 1995. Sustained increase in haemoglobin and RBC following long-term administration of recombinant human erythropoietin to patients with homozygous beta-thalassaemia. Br. J. Haematol. 90: 341-345.
20. WILD, B.J. & A.D. STEPHENS. 1997. The use of automated HPLC to detect and quantitate haemoglobins. Clin. Lab. Haematol. 19: 171-176.
21. WEATHERALL, D. 1998. Hemoglobin E beta-thalassemia: an increasingly common disease with some diagnostic pitfalls. J. Pediatr. 132: 765-767.
22. WEATHERALL, D. 1999. From genotype to phenotype: genetics and medical practice in the new millennium. Philos. Trans. R. Soc. Lond. B Biol. Sci. 354: 1995-2010.
23. REES, D.C., J.B. PORTER, J.B. CLEGG & D.J. WEATHERALL. 1999. Why are hemoglobin F levels increased in HbE/beta thalassemia? Blood 94: 3199-3204.
24. MCDONAGH, K.T., G.J. DOVER, R.E. DONAHUE, et al. 1992. Hydroxyurea-induced HbF production in anemic primates: augmentation by erythropoietin, hematopoietic growth factors, and sodium butyrate. Exp. Hematol. 20: 1156-1164.
25. OLIVIERI, N.F., D.C. REES, G.D. GINDER, et al. 1997. Treatment of thalassaemia major with phenylbutyrate and hydroxyurea. Lancet 350: 491-492.