Study of efficacy of hydroxyurea in transfusion dependent B-Haemoglobinopathies

Abstract

Introduction: Beta thalassaemia Major prevents the creation of a functioning β chain. The clinical manifestations of beta thalassaemia major include severe anaemia, stunted growth, and deformities of the skeleton within the first two years of life. The risk of transfusion-transmitted viral infections and the limited availability of blood have prompted the search for alternate methods of managing beta-thalassemia. Present study was undertaken to study effectiveness, safety profile, side effect of hydroxyurea in the treatment of children diagnosed with Beta thalassemia major

Materials and Methods: 50 beta thalassemia major patients were enrolled & divided randomly into two groups as Hydroxyurea (HU) n=25 & placebo group n=25. Hydroxyurea (HU) group patients were given hydroxyurea 10-20 mg/kg/day (To nearest 250 mg capsule). Complete hemogram including Haemoglobin (gm/dl), RBC (millions/dl). MCV (fl), MCH (pg) & Haematocrit (%) along with serum ferritin levels, urea & creatinine were evaluated at the time of enrolment and then repeated after 6 months.

Observations and Results: At Enrolment mean ± SD of Hb (gm/dl) was 8.11 ± 1.95 in HU group & 7.85 ± 1.13 in placebo group. After 6 Month it was 9.32 ±1.86 in HU group & 8.12 ± 0.94 in placebo group. Result showed statistically significant difference in all parameters (Hb, RBC, MCV, MCH, Hematocrit, Serum Ferritin, Urea & Creatinine) in HU group

Conclusion: In conclusion, certain transfusion-dependent β-thalassemia patients may feel better after taking hydroxyurea, which is prescribed to them to reduce their transfusion needs

Keywords:  HU, placebo, B-Haemoglobinopathies