HYDROXYUREA TREATMENT AND REDUCTION IN TRANSFUSION DEMAND IN Β-THALASSEMIA MAJOR: EVIDENCE FROM THE HOSPITAL-BASED STUDY IN KARACHI

Background: β-thalassemia major is a hereditary hemoglobin disorder caused by mutations in the β-globin gene, leading to ineffective erythropoiesis and transfusion dependence. Blood transfusions are the primary management strategy of this disorder, but are associated with complications including iron overload and alloimmunization. Hydroxyurea (HU), through production of fetal hemoglobin (HbF), has emerged as a promising therapeutic option to produce better therapeutic outcomes in managing the severity of disease.

Objective: This study was aimed to evaluate the effectiveness of HU in reducing transfusion requirements among patients with β-thalassemia major in a welfare hospital in Karachi, Pakistan.

Methodology: A cross-sectional survey-based study was conducted on 150 patients diagnosed with β-thalassemia major at AL-Mustafa hospital, Karachi. Data was retrieved from diagnostic laboratory records of patients, aged between 3 months and 26 years. Hematological parameters, transfusion frequency, treatment regimens, and adverse effects were assessed and compared between patients receiving conventional transfusion therapy and those on HU. Statistical evaluation was performed by applying chi-square and frequency distribution.

Results: The mean age of patients was 2.17 ± 0.918 years. HU therapy demonstrated a significant improvement in hemoglobin profiles and clinical status. A reduction in transfusion frequency was observed in the majority of patients on HU therapy. Additionally, HU use was associated with fewer transfusion-related complications. The results were statistically significant with a p-value <0.05.

Conclusion: Hydroxyurea showed considerable efficacy in improving hematological outcomes and reducing transfusion dependency in β-thalassemia major patients. These findings support the potential role of HU as an effective therapeutic alternative, particularly in resource-limited settings where transfusion-related complications remain a major clinical concern.