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J Pediatr Hematol Oncol. 2007 Nov;29(11):743-6.

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

Author information

1
Bismillah Taqee Institute of Health Sciences and Blood Disease Center, Gulshan-e-Iqbal, Karachi. ansarisaqib@hotmail.com

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.

METHOD:

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.

RESULT:

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.

PMID:
17984691
DOI:
10.1097/MPH.0b013e318157fd75
[Indexed for MEDLINE]

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