Fetal haemoglobin augmentation in E/β0 thalassaemia: Clinical and haematological outcome

British Journal of Haematology

Volumn 131, Issue 3, 2005, Pages 378-388

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

^a CHILDREN S HOSPITAL OAKLAND   (United States)

^b CHILDREN S HOSPITAL OAKLAND RESEARCH INSTITUTE   (United States)

^c UNIVERSITY OF TORONTO   (Canada)

^d HOSPITAL FOR SICK CHILDREN   (Canada)

^e UNIVERSITY OF OXFORD   (United Kingdom)

^f CHILDREN S HOSPITAL CENTRAL CALIFORNIA   (United States)

^g CHILDREN S HOSPITAL LOS ANGELES   (United States)

^h CENTRAL MIDDLESEX HOSPITAL   (United Kingdom)

Author keywords

E 0 thalassaemia; Fetal haemoglobin augmentation; Haemoglobin F; Hydroxyurea; Thalassaemia

Indexed keywords

BUTYRIC ACID DERIVATIVE; ERYTHROPOIETIN; HEMOGLOBIN; HEMOGLOBIN F; HYDROXYUREA; RECOMBINANT ERYTHROPOIETIN; SODIUM PHENYL BUTYRATE; UNCLASSIFIED DRUG; ARYLBUTYRIC ACID DERIVATIVE;

ARTICLE; BETA THALASSEMIA; BLOOD TRANSFUSION; CLINICAL ARTICLE; FOLLOW UP; HUMAN; PRIORITY JOURNAL; ADOLESCENT; BLOOD; CHILD; CLINICAL TRIAL; DRUG COMBINATION; DRUG EFFECT; ERYTHROCYTE LIFESPAN; ERYTHROPOIESIS; FEMALE; IRON OVERLOAD; MALE; METABOLISM; MULTICENTER STUDY; PROSPECTIVE STUDY; TREATMENT OUTCOME;

ADOLESCENT; BETA-THALASSEMIA; BLOOD TRANSFUSION; CHILD; DRUG THERAPY, COMBINATION; ERYTHROCYTE AGING; ERYTHROPOIESIS; ERYTHROPOIETIN, RECOMBINANT; FEMALE; FETAL HEMOGLOBIN; HEMOGLOBINS; HUMANS; HYDROXYUREA; IRON OVERLOAD; MALE; PHENYLBUTYRATES; PROSPECTIVE STUDIES; TREATMENT OUTCOME;

EID: 33644649454     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/j.1365-2141.2005.05768.x     Document Type: Article

References (36)

1. Angastiniotis M. Modell B. (1998) Global epidemiology of hemoglobin disorders. Annals of the New York Academy of Sciences, 850, 251 269.
2. Beguin Y. (2003) Soluble transferrin receptor for the evaluation of erythropoiesis and iron status. Clinica Chimica Acta, 329, 9 22.
3. Cavill I. (2002) Erythropoiesis and iron. Best Practice & Research Clinical Haematology, 15, 399 409.
4. Collins A.F. Pearson H.A. Giardina P. McDonagh K.T. Brusilow S.W. Dover G.J. (1995) Oral sodium phenylbutyrate therapy in homozygous beta thalassemia: a clinical trial. Blood, 85, 43 49.
5. Franco R.S. Lohmann J. Silberstein E.B. Mayfield-Pratt G. Palascak M. Nemeth T.A. Joiner C.H. Weiner M. Rucknagel D.L. (1998) Time-dependent changes in the density and hemoglobin F content of biotin-labeled sickle cells. Journal of Clinical Investigation, 101, 2730 2740.
6. Fucharoen S. Winichagoon P. (2000) Clinical and hematologic aspects of hemoglobin E beta-thalassemia. Current Opinion in Hematology, 7, 106 112.
7. Fucharoen S. Winichagoon P. Pootrakul P. Piankijagum A. Wasi P. (1987) Variable severity of Southeast Asian beta 0-thalassemia/Hb E disease. Birth Defects Original Article Series, 23, 241 248.
8. 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. Rodgers G.P. (1996) Hydroxyurea increases hemoglobin F levels and improves the effectiveness of erythropoiesis in beta-thalassemia/hemoglobin E disease. Blood, 87, 887 892.
9. Galanello R. Barella S. Turco M.P. Giagu N. Cao A. Dore F. Liberato N.L. Guarnone R. Barosi G. (1994) Serum erythropoietin and erythropoiesis in high- and low-fetal hemoglobin beta-thalassemia intermedia patients. Blood, 83, 561 565.
10. Garner C. Tatu T. Reittie J.E. Littlewood T. Darley J. Cervino S. Farrall M. Kelly P. Spector T.D. Thein S.L. (2000) Genetic influences on F cells and other hematologic variables: a twin heritability study. Blood, 95, 342 346.
11. Glader B.E. Look K.A. (1996) Hematologic disorders in children from southeast Asia. Pediatric Clinics of North America, 43, 665 681.
12. Hendy J. (1999) Preventation of thalassemia in Australia. Southeast Asian Journal of Tropical Medicine and Public Health, 30 (Suppl. 2 94 96.
13. Landaw S. (1995) Homeostasis, survival, and red cell kinetics: measurement and imaging of red cell production. In: Hematology: Basic Principles and Practice (ed. by R. Hoffman E.J. Benz Jr. S.J. Shattil B. Furie H.J. Cohen L.E. Silberstein pp. 448 458. Churchill, Livingstone.
14. Lorey F. (2000) Asian immigration and public health in California: thalassemia in newborns in California. Journal of Pediatric Hematology/Oncology, 22, 564 566.
15. Loukopoulos D. Voskaridou E. Stamoulakatou A. (1995) Clinical trials with hydroxyurea and recombinant human erythropoietin. In: Hemoglobin Switching (ed. by G. Stamatoyannopoulos A. Nienhaus pp. 365 372. Alan R. Liss, New York.
16. McDonagh K.T. Dover G.J. Donahue R.E. Nathan D.G. Agricola B. Byrne E. Nienhuis A.W. (1992) Hydroxyurea-induced HbF production in anemic primates: augmentation by erythropoietin, hematopoietic growth factors, and sodium butyrate. Experimental Hematology, 20, 1156 1164.
17. Modell B. Khan M. Darlison M. King A. Layton M. Old J. Petrou M. Varnavides L. (2001) A national register for surveillance of inherited disorders: beta thalassaemia in the United Kingdom. Bulletin of the World Health Organization, 79, 1006 1013.
18. Najean Y. Deschryver F. Henni T. Girot R. (1985) Red cell kinetics in thalassaemia intermedia: its use for a prospective prognosis. British Journal of Haematology, 59, 533 539.
19. Nienhuis A.W. Ley T.J. Humphries R.K. Young N.S. Dover G. (1985) Pharmacological manipulation of fetal hemoglobin synthesis in patients with severe beta-thalassemia. Annals of the New York Academy of Sciences, 445, 198 211.
20. Olivieri N.F. (1997) Fetal erythropoiesis and the diagnosis and treatment of hemoglobin disorders in the fetus and child. Seminars in Perinatology, 21, 63 69.
21. Olivieri N.F. Rees D.C. Ginder G.D. Thein S.L. Brittenham G.M. Waye J.S. Weatherall D.J. (1997) Treatment of thalassaemia major with phenylbutyrate and hydroxyurea. Lancet, 350, 491 492.
22. Premawardhena A. Fisher C.A. Fathiu F. de Silva S. Perera W. Peto T.E. Olivieri N.F. Weatherall D.J. (2001) Genetic determinants of jaundice and gallstones in haemoglobin E beta thalassaemia. Lancet, 357, 1945 1946.
23. Rachmilewitz E.A. Aker M. Perry D. Dover G. (1995) Sustained increase in haemoglobin and RBC following long-term administration of recombinant human erythropoietin to patients with homozygous beta-thalassaemia. British Journal of Haematology, 90, 341 345.
24. Rees D.C. Styles L. Vichinsky E.P. Clegg J.B. Weatherall D.J. (1998) The hemoglobin E syndromes. Annals of the New York Academy of Sciences, 850, 334 343.
25. Rees D.C. Porter J.B. Clegg J.B. Weatherall D.J. (1999) Why are hemoglobin F levels increased in HbE/beta thalassemia Blood, 94, 3199 3204.
26. Rodgers G.P. Dover G.J. Uyesaka N. Noguchi C.T. Schechter A.N. Nienhuis A.W. (1993) Augmentation by erythropoietin of the fetal-hemoglobin response to hydroxyurea in sickle cell disease. New England Journal of Medicine, 328, 73 80.
27. Sher G.D. Ginder G.D. Little J. Yang S. Dover G.J. Olivieri N.F. (1995) Extended therapy with intravenous arginine butyrate in patients with beta-hemoglobinopathies. New England Journal of Medicine, 332, 1606 1610.
28. de Silva S. Fisher C.A. Premawardhena A. Lamabadusuriya S.P. Peto T.E. Perera G. Old J.M. Clegg J.B. Olivieri N.F. Weatherall D.J. (2000) Thalassaemia in Sri Lanka: implications for the future health burden of Asian populations. Sri Lanka Thalassaemia Study Group. Lancet, 355, 786 791.
29. Skikne B.S. Cook J.D. (1992) Effect of enhanced erythropoiesis on iron absorption. Journal of Laboratory and Clinical Medicine, 120, 746 751.
30. Thein S.L. (2004) Genetic insights into the clinical diversity of beta thalassaemia. British Journal of Haematology, 124, 264 274.
31. Viprakasit V. Tanphaichitr V.S. Mahasandana C. Assteerawatt A. Suwantol L. Veerakul G. Kankirawatana S. Pung-Amritt P. Suvatte V. (2001) Linear growth in homozygous beta-thalassemia and beta-thalassemia/hemoglobin E patients under different treatment regimens. Journal of the Medical Association of Thailand, 84, 929 941.
32. Weatherall D.J. (2000) Introduction to the problem of hemoglobin E-beta thalassemia. Journal of Pediatric Hematology/Oncology, 22, 551.
33. Weatherall D. (2001) The Thalassemia Syndromes. Blackwell Science, Malden, MA
34. Clegg J.B. (2001) Inherited haemoglobin disorders: an increasing global health problem. Bulletin of the World Health Organization, 79, 704 712.
35. Wild B.J. Stephens A.D. (1997) The use of automated HPLC to detect and quantitate haemoglobins. Clinical and Laboratory Haematology, 19, 171 176.
36. Winichagoon P. Thonglairoam V. Fucharoen S. Wilairat P. Fukumaki Y. Wasi P. (1993) Severity differences in beta-thalassaemia/haemoglobin E syndromes: implication of genetic factors. British Journal of Haematology, 83, 633 639.