Efficacy and Safety of Hydroxyurea as Adjuvant Therapy in Pediatric Patients of Transfusion-Dependent Beta-Thalassemia Major at Zhob, Balochistan

Background Hydroxyurea is being used effectively in sickle cell anemia and thalassemia intermedia. Its role in transfusion-dependent beta-thalassemia major yet needs to be clearly established. This study has been carried out to assess the efficacy and safety of hydroxyurea as adjuvant therapy in pediatric cases of transfusion-dependent beta-thalassemia major disease. Materials and methods This quasi-experimental study was carried out at District Headquarter Hospital (DHQ), Zhob, from February 2021 to January 2022. One hundred ten cases fulfilling the inclusion-exclusion criteria were selected and divided into groups of 55 each. Group A cases received hydroxyurea (10-20 mg/kg/day) in addition to blood transfusion and chelation therapy. Group B received a blood transfusion and chelation therapy only. Both groups were compared in terms of blood transfusion requirement, mean hemoglobin, and mean serum ferritin levels. All the data were analyzed with SPSS 21 (IBM Corp., Armonk, NY). Results Of Group A cases, three were dropped because of side effects of hydroxyurea, and two were lost to follow-up. Similarly, three cases of Group B lost to follow-up, one patient withdrew consent, and one child died at home; thus both groups were left with 50 cases each. The mean age of participants was 11.98 + 3.74 years. There were 51 males and 49 females. Both the groups were comparable in terms of age and gender. Similarly, mean hemoglobin levels and serum ferritin levels were comparable at the start of the study. After one year, there was a significant improvement in mean hemoglobin level (p<0.001) and a significant reduction in serum ferritin levels (p=0.014) in the group taking adjuvant hydroxyurea. The requirement of packed red blood cells (RBCs) significantly decreased in cases taking hydroxyurea (p<0.001). Conclusion Hydroxyurea is a safe and effective treatment that significantly decreases the packed RBC transfusion requirement in transfusion-dependent thalassemia children, improves hemoglobin levels, and reduces serum ferritin levels compared to the children on blood transfusions alone.