Abstract

Objective: To determine the effectiveness of hydroxyurea on the blood transfusion rate in patients with beta-thalassemia major.
 Materials and Methods: This cross-sectional study was held in the Department of Genetics and Molecular Biology/Pathology at LUMHS Jamshoro, and Diagnostic and Research Laboratory, in Hyderabad, Sindh from February 2015 to August 2015. Patients with beta thalassaemia major, diagnosed with Hb electrophoresis, who had Hb less than 7 g/dl and were regularly transfused every two to four weeks were included. Patients were divided into two groups in equal numbers. Half of the patients underwent blood transfusion with the treatment of hydroxyurea and half without hydroxyurea. In comparison to those who did not receive hydroxyurea treatment. The ability to keep haemoglobin levels over 9 g/dl or a lowering of at least 50% from the initial transfusion needs were considered signs of therapeutic success. Data was entered and analyzed using SPSS 26.
 Results: The mean age of the patients was 11.02+3.93 years and males were in the majority (68.0%). In 56.2% of cases, there was a positive family history. The mean serum ferritin level was 12824.39+300.60 ng/ml and the mean haemoglobin level was 7.52+1.67 gm/dl. Some patients did not report follow-up, because some families had migrated to other areas of Sindh, and some cases went to other welfare hospitals/ centers, for treatment. Therefore, out of 40 patients, 30 were observed with hydroxyurea, and overall, this treatment showed a significant decrease in blood transfusion requirements (P-0.01).
 Conclusion: As per the study’s conclusion, hydroxyurea was observed to be the most effective treatment to decrease the blood transfusion rate, but patients should be treated under proper and responsible observation.

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