title: Hydroxyurea-induced hematological response in transfusion-independent beta-thalassemia intermedia: case series and review of literature.
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 beta-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.
  
  
  CONCLUSIONS
  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.
authors: Bahrami Children Hospital, Tehran University of Medical Sciences, Tehran, Iran. ehsanimo@sina.tums.ac.ir
authors: Mohammad Ali
authors: MA
authors: Ehsani
authors: Amir Abbas
authors: AA
authors: Hedayati-Asl
authors: Alireza
authors: A
authors: Bagheri
authors: Syrus
authors: S
authors: Zeinali
authors: Armin
authors: A
authors: Rashidi
mesh_terms: D000293:Adolescent; D001803:Blood Transfusion; D002648:Child; D002675:Child, Preschool; D016903:Drug Monitoring / Q000379:methods; D005260:Female; D005319:Fetal Hemoglobin / Q000032:analysis; D006454:Hemoglobins / Q000032:analysis; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use*; D008297:Male; D009154:Mutation; D011110:Polymorphism, Genetic; D012074:Remission Induction; D055544:beta-Globins / Q000235:genetics; D017086:beta-Thalassemia / Q000188:drug therapy* / Q000235:genetics / Q000628:therapy
chemical_list: D006454:Hemoglobins; D055544:beta-Globins; D005319:Fetal Hemoglobin; D006918:Hydroxyurea