Treatment Response to Hydroxyurea in Children with Beta Thalassemia

Introduction: Thalassemias are a group of haematological disorders due to quantitative defect of globin chain. Patients require continuous transfusion support with chelation therapy throughout the life with resultant iron overload and toxic injury to various organs. So there was always a need for a therapy which could substitute for this transfusion therapy. Hydroxyurea (HU) has been proved as one of the accepted agents which acts by combacting the imbalance between α and non α chains. We conducted this study at Dhaka Shishu Hospital Thalassemia centre to evaluate response of HU in patients of Thalassemia who were under transfusion management. Patients & Methods: A prospective observational study was conducted at Thalassemia Centre of Dhaka Shishu Hospital, Bangladesh. Hundred patients of β Thalassemia who were under transfusion management were treated with HU in a dose of 15-20 mg/kg body weight and they were evaluated for about 24 months for clinical and haematological response. Patients were divided into three groups depending upon their response into; good responder, partial responders and non-responders. Results: In total of 100 (50 Thalassemia major and 50 Thalassemia intermedia), 55 were males and 45 were females with age range of 1-18 years (7.52 + 2.46). Our results showed that 25 children were good responders, 70 partial responders and 5 were non responders. A significant increase in transfusion interval (p-value 0.001) was found after treatment with HU. There was also a significant reduction in size of liver and spleen (p-value 0.003 and 0.001 respectively). Conclusion: Treatment of HU has shown promising results in our study. However, response to treatment varies among patients and there is a need to identify various factors which affect treatment response.