TY - JOUR
T1 - Hydroxyurea-induced hematological response in transfusion-independent beta-thalassemia intermedia
T2 - Case series and review of literature
AU - Ehsani, Mohammad Ali
AU - Hedayati-Asl, Amir Abbas
AU - Bagheri, Alireza
AU - Zeinali, Syrus
AU - Rashidi, Armin
N1 - Copyright:
Copyright 2010 Elsevier B.V., All rights reserved.
PY - 2009/11/20
Y1 - 2009/11/20
N2 - Background: Hydroxyurea (HU) may improve the symptoms in thalassemia patients by increasing gamma-globin chain expression. However, the efficacy of HU in beta-thalassemia intermedia (TI) is unclear. Methods: The authors treated 16 transfusion-independent TI patients (8 males) aged 10.7 ± 5.0 years with HU, 20 mg/kg/day 4 days per week, for 6 months. Hemoglobin (Hb) and HbF levels were measured prior to treatment, during the treatment period (monthly), and following the completion of treatment. Mutations in the β-globin gene as well as the XmnI polymorphism were determined. Results: Treatment was well tolerated. There was a significant increase in both Hb and HbF (p < .001), and the increments were strongly correlated (r .94; p < .001). XmnI polymorphism was not correlated with hematological response. Hb (p .026) and HbF (p .046) showed a more significant rise in patients with a Fr8/9 allele than those with one or two IVS-II-1 alleles. Conclusion: HU therapy was associated with a significant hematological response in our TI patients. The Fr8/9 mutation, but not the XmnI polymorphism, was a predictor of good hematological response. Studies with larger sample sizes are needed to confirm the results obtained in this study.
AB - Background: Hydroxyurea (HU) may improve the symptoms in thalassemia patients by increasing gamma-globin chain expression. However, the efficacy of HU in beta-thalassemia intermedia (TI) is unclear. Methods: The authors treated 16 transfusion-independent TI patients (8 males) aged 10.7 ± 5.0 years with HU, 20 mg/kg/day 4 days per week, for 6 months. Hemoglobin (Hb) and HbF levels were measured prior to treatment, during the treatment period (monthly), and following the completion of treatment. Mutations in the β-globin gene as well as the XmnI polymorphism were determined. Results: Treatment was well tolerated. There was a significant increase in both Hb and HbF (p < .001), and the increments were strongly correlated (r .94; p < .001). XmnI polymorphism was not correlated with hematological response. Hb (p .026) and HbF (p .046) showed a more significant rise in patients with a Fr8/9 allele than those with one or two IVS-II-1 alleles. Conclusion: HU therapy was associated with a significant hematological response in our TI patients. The Fr8/9 mutation, but not the XmnI polymorphism, was a predictor of good hematological response. Studies with larger sample sizes are needed to confirm the results obtained in this study.
KW - Hydroxyurea
KW - Polymorphism
KW - Thalassemia intermedia
KW - Transfusion
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U2 - 10.3109/08880010903271671
DO - 10.3109/08880010903271671
M3 - Article
C2 - 19954365
AN - SCOPUS:71449113459
VL - 26
SP - 560
EP - 565
JO - Pediatric Hematology and Oncology
JF - Pediatric Hematology and Oncology
SN - 0888-0018
IS - 8
ER -