title: Efficacy of hydroxyurea (HU) in reduction of pack red cell (PRC) transfusion requirement among children having beta-thalassemia major: Karachi HU trial (KHUT).
abstract: BACKGROUND
  Packed red blood cell (PRC) transfusion with iron chelation is the mainstay of treatment for patients with beta-thalassemia major. Hemoglobin F augmentation is another approach to treat this hemoglobinopathy. This study evaluates the efficacy and safety of hydroxyurea (HU) in minimizing PRC transfusions in patients with beta-thalassemia major.
  
  
  METHODS
  Twenty-three patients with beta-thalassemia major received HU at a mean dose of 16 mg/kg/d. The results were analyzed at the end of 24 months. Transfusion requirement during the 6 months preceding the study was considered as the control.
  
  
  RESULTS
  Twenty patients were evaluable after 24 months. The mean volume of PRC transfused was reduced from 2126.45 mL to 1489.59 mL (P<0.001). The interval between transfusions was increased by 68.7%. Grade I myelosuppression was observed in 4 patients and diarrhea in 2 patients.
  
  
  CONCLUSIONS
  HU was found to be safe in patients with beta-thalassemia major, and resulted in reduction in the transfusion requirements and in increase of the intervals between transfusions.
authors.affiliation: Bismillah Taqee Institute of Health Sciences and Blood Disease Center, Gulshan-e-Iqbal, Karachi. ansarisaqib@hotmail.com
authors.forename: Saqib H
authors.initials: SH
authors.lastname: Ansari
authors.forename: Tahir S
authors.initials: TS
authors.lastname: Shamsi
authors.forename: Fahad J
authors.initials: FJ
authors.lastname: Siddiqui
authors.forename: Muhammad
authors.initials: M
authors.lastname: Irfan
authors.forename: Kausar
authors.initials: K
authors.lastname: Perveen
authors.forename: Tasneem
authors.initials: T
authors.lastname: Farzana
authors.forename: Vinodh K
authors.initials: VK
authors.lastname: Panjwani
authors.forename: Ayesha
authors.initials: A
authors.lastname: Yousuf
authors.forename: Tabassum
authors.initials: T
authors.lastname: Mehboob
mesh_terms: D000293:Adolescent; D000986:Antisickling Agents / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D017707:Erythrocyte Transfusion; D005260:Female; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use*; D008297:Male; D010154:Pakistan; D017086:beta-Thalassemia / Q000188:drug therapy* / Q000628:therapy
chemical_list: D000986:Antisickling Agents; D006918:Hydroxyurea