Efficacy of hydroxyurea (HU) in reduction of pack red cell (PRC) transfusion requirement among children having beta-thalassemia major: Karachi HU Trial (KHUT)

Journal of Pediatric Hematology/Oncology

Volumn 29, Issue 11, 2007, Pages 743-746

  Ansari, Saqib H ^a   Shamsi, Tahir S ^a   Siddiqui, Fahad J ^a   Irfan, Muhammad ^a   Perveen, Kausar ^a   Farzana, Tasneem ^a   Panjwani, Vinodh K ^a   Yousuf, Ayesha ^a   Mehboob, Tabassum ^a  

^a Blood Disease Center   (Pakistan)

Author keywords

Beta thalassemia major; Hydroxyurea; Transfusion volume

Indexed keywords

DEFEROXAMINE; HYDROXYUREA;

ADOLESCENT; ADULT; ARTICLE; BETA THALASSEMIA; BLOOD TRANSFUSION; BONE MARROW SUPPRESSION; CLINICAL ARTICLE; CONTROLLED STUDY; DIARRHEA; DRUG EFFICACY; DRUG SAFETY; DRUG WITHDRAWAL; ERYTHROCYTE CONCENTRATE; FEMALE; HEART ARRHYTHMIA; HUMAN; IRON OVERLOAD; MALE; PAKISTAN; PATIENT COMPLIANCE; PRIORITY JOURNAL; SCHOOL CHILD; STATISTICAL SIGNIFICANCE; TREATMENT DURATION;

ADOLESCENT; ANTISICKLING AGENTS; BETA-THALASSEMIA; CHILD; CHILD, PRESCHOOL; ERYTHROCYTE TRANSFUSION; FEMALE; HUMANS; HYDROXYUREA; MALE; PAKISTAN;

EID: 35948998859     PISSN: 10774114     EISSN: 15363678     Source Type: Journal    
DOI: 10.1097/MPH.0b013e318157fd75     Document Type: Article

References (26)

1. Afroze M, Shamsi TS, Syed S. Predictive value of MCV/RBC count ratio to discriminate between iron deficiency anemia and β-thalassaemia trait. J Pak Med Assoc. 1998;48:8-89.
2. Alwan A, Modell B. Community Control of Genetic and Congenital Disorders. EMRO Technical Publication Series 24. Alexandrea, Egypt: WHO Regional Office for the Eastern Mediterranean; 1997.
3. Ahmed S, Petrou M, Saleem M. Molecular genetics of β-thalassaemia in Pakistan: a basis for prenatal diagnosis. Br J Haematol. 1997;97:504.
4. Farzana T, Shamsi TS, Irfan M, et al. Peripheral PRC stem cell transplantation in children with β-thalassaemia major. J Coll Physicians Surgeons Pak. 2003;13:204-206.
5. Thein SL, Wainscoat JS, Sampietro M, et al. Association of thalassaemia intermedia with beta globin gene haplotype. Br J Haematol. 1987;65:367-371.
6. De Simone J, Heller P, Hall L, et al. 5-Azacytidine stimulates fetal hemoglobin synthesis in anemic baboons. Proc Natl Acad Sci U S A. 1982;79:4428-4431.
7. Ley TJ, Desimone J, Anafnou NP, et al. 5-Azacytadine selectively increase gamma-globin synthesis in a patient with beta-+ thalassaemia. N Engl J Med. 1982;307:1469-1475.
8. Chaidos A, Makis A, Hatzimichael E, et al. Treatment of beta-thalassaemia patients with recombinant human erythropoietin: effect on transfusion requirements and soluble adhesion molecule. Acta Haematol. 2004;111:189-195.
9. Collins AF, Pearson HA, Giardina P, et al. Oral sodium phenylbutyrate therapy in homozygous beta thalassemia: a clinical trial. Blood. 1995;85:39-43.
10. Reich S, Buhrer C, Henze G, et al. Oral isobutyramide reduces transfusion requirements in some patients with homozygous beta-thalassemia. Blood. 2000;96:3357-3363.
11. Platt OS, Orkin SH, Dover G, et al. Hydroxyurea enhances fetal hemoglobin production in sickle cell anemia. J Clin Invest. 1984;74:652-656.
12. Rodgers GP, Dover GJ, Noguchi CT, et al. Haematological responses of patients with sickle cell disease to treatment with hydroxyurea. N Engl J Med. 1990;322:1037-1045.
13. Haynes J, Baliga BS, Obiako B, et al. Zileuton induces hemoglobin F synthesis in erythroid progenitors: role of the L-arginine-nitric oxide signaling pathway. Blood. 2004;103:3945-3950.
14. Zeng YT, Huang SZ, Ren ZR, et al. Hydroxyurea therapy in β-thalassaemia intermedia: improvement in haematological parameters due to enhanced β-globin synthesis. Br J Haematol. 1995;90:557-563.
15. Rigano P, Manfre L, La Galla R, et al. Clinical and haematological responses to hydroxyurea in a patient with Hb Lepore/β-thalassaemia. Hemoglobin. 1997;21:219-226.
16. Singer ST, Kuypers FA, Coats TD, et al. The effects of single and combination drug therapy on previously transfused E/β-thalassaemia patients: implications on decision-making for therapy. Blood. 2002(suppl) Abstract 442.
17. Anupam S, Vainer HK, Verma IC, et al. Hydroxyurea and termination of transfusion therapy in transfusion dependent thalassemics. Blood. 2002(suppl) Abstract 3599.
18. Fucharoen S, Siritanaratkul N, Winichagoon P, et al. Hydroxyurea increases hemoglobin F levels and improves the effectiveness of erythropoiesis in β-thalassaemia/Hemoglobin E disease. Blood. 1996;87:887-892.
19. Yavarian M, Karimi M, Bakker E, et al. Response to hydroxyurea treatment in Iranian transfusion-dependent beta-thalassaemia patients Thalassaemia Medical Center. Haematologica. 2004;89:117.
20. Charache S, Dover GJ, Moore RD, et al. Hydroxyurea: effects on hemoglobin F production in patients with sickle cell anemia. Blood. 1992;79:2555-2565.
21. Charache S, Dover GJ. Hydroxyurea therapy for sickle cell anemia. Blood. 1987;69:109-116.
22. Charache S, Terrin ML, Moore RD, et al. Effects of hydroxyurea on the frequency of painful crises in sickle cell anemia. N Eng J Med. 1995;332:1317-1322.
23. Hoppe C, Vichinsky E, Lewis B, et al. Hydroxyurea and sodium phenyl butyrate therapy in thalassaemia intermedia. Am J Haematol. 1999;63:221-227.
24. Olivieri NF. Reactivation of fetal hemoglobin in patients with β-thalassaemia. Semin Haematol. 1996;33:24-42.
25. Choudhry VP, Lal A, Pati HP, et al. Haematological responses to hydroxyurea therapy in multi-transfused thalassaemia children. Indian J Pediatr. 1997;64:395-398.
26. Bradai M, Abad MT, Pissard S, et al. Hydroxyurea can eliminate transfusion requirements in children with severe β-thalassaemia. Blood. 2003;104:114-117.