Hydroxyurea-induced hematological response in transfusion-independent beta-thalassemia intermedia: Case series and review of literature

Mohammad Ali Ehsani, Amir Abbas Hedayati-Asl, Alireza Bagheri, Syrus Zeinali, Armin Rashidi

Research output: Contribution to journalArticlepeer-review

26 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)560-565
Number of pages6
JournalPediatric hematology and oncology
Volume26
Issue number8
DOIs
StatePublished - Nov 20 2009

Keywords

  • Hydroxyurea
  • Polymorphism
  • Thalassemia intermedia
  • Transfusion

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