Efficacy of hydroxyurea (HU) in reduction of pack red cell (PRC) transfusion requirement among children having beta-thalassemia major: Karachi HU trial (KHUT).
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.
Ansari
Saqib H
SH
Bismillah Taqee Institute of Health Sciences and Blood Disease Center, Gulshan-e-Iqbal, Karachi. ansarisaqib@hotmail.com
Shamsi
Tahir S
TS
Siddiqui
Fahad J
FJ
Irfan
Muhammad
M
Perveen
Kausar
K
Farzana
Tasneem
T
Panjwani
Vinodh K
VK
Yousuf
Ayesha
A
Mehboob
Tabassum
T
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
D000986:Antisickling Agents; D006918:Hydroxyurea