Role of Hydroxyurea in Reducing Transfusion Requirements in Patients with β-Thalassaemia Major

Abstract

Background: β thalassemia major (βTM) is one of the major genetic hematological diseases of Bangladesh. Blood transfusion is yet the mainstay of treatment. Hydroxyurea (HU) is known to reduce the transfusion requirements of patients with thalassemia intermedia and sickle cell anemia, but there are very limited evidences on its role in βTM. Objective: To evaluate the response of patients with βTM to Hydroxyurea. Methods: This prospective interventional study, included 28 diagnosed transfusions dependent βTM patients who took HU for one year (Group-I) and 28 diagnosed transfusions dependent βTM patients who did not take HU for one year (Group-II). After enrollment, baseline CBC was recorded. Patients were treated with HU at an incremental dose from 10 to 20 mg/kg over 10 weeks and then continued with 20 mg/kg for 42 weeks. Both clinical and laboratory response was observed at 3rd, 6th, 9th and at 12th month. Results: The mean Hb at 12 months was 7.42±1.31 g/dl in group I and 6.64±0.61 g/dl in group II. Mean MCV at 6 months was 68.34±7.09 fL (Gr-I) and 65.11±0.85 fL (Gr-II), at 9 month 71.19±6.29 fL (Gr-I) and 64.98±0.59 fL (Gr-II) and at 12 month 73.79±7.38 fL (Gr-I) and 64.76±0.52 fL (Gr-II). Mean serum bilirubin at 9 months was found 1.99±0.73mg/dl (Gr-I) and 2.42±0.44mg/dl (Gr-II) and at 12 month 1.66±0.71mg/dl (Gr-I) and 2.41±0.44mg/dl (gr-II) (p<0.05). Twelve (42.9%) patients required less amount of blood with or without prolong interval than baseline in group I and none in group II. Pre-HU therapy, mean RCC requirement were 1620.0±573.3 (ml) among 12 patients in Group-I, Post-HU therapy mean RCC requirements were 1111.7±458.8 ml and mean difference was 508.3±252.7 ml (p<0.05), and there was 31.2% reduction in their transfusion requirements. Two (7.1%) patients developed side effects in group I and none in group II. Conclusion: This study showed an improvement of Hb status and MCV, reduction of serum bilirubin as well as transfusion requirement were significantly more, among the children treated with HU.