European Journal of Molecular & Clinical Medicine

Objective: To study effectiveness, safety profile, side effect of hydroxyurea (HU) to decrease need of PRC transfusions in children with β-thalassemia major.
Study design: Single-Centre open-label randomized control trial
Setting: Thalassemia Clinic of GMCH, Aurangabad
Participants: 58 patients are enrolled in this study out of which 27 were included in HU
group and 28 were in control group, after exclusion and inclusion criteria.
Intervention: Randomization done by stratified block method with concealed allocation. Before starting hydroxyurea, all patient went through physical examination and routine biochemical  laboratory  tests.  The  intervention   group  receives  Hydroxyurea10  to  20
mg/kg/day as a single dose daily for12 months while the control group receives a placebo. All
were monitored for 12 months. All other standard treatment including iron chelation were continued. The statistical analysis was based on intention to treat.
Outcome: Reduction of PRC transfusion requirement during the treatment period was the primary outcome and to study the safety profile and side effect of hydroxyurea were considered as secondary outcome.
Result: Mean Hb ± 2SD was 7.29 ± 1.67 at enrolment and 9.25 ± 1.81 after 12 months in
study group (P=0.0001), It was 7.41 ± 1.71 at enrolment and 8.53 ± 1.84 after 12 months in Control group (P=0.02). Percentage drop in Mean Annual blood requirement (ABR) was (ml/kg/year) in 21% in study group as compare to control group (8%). Mild (Hb rise 0-5%) response was observed in 2 (7.4%) Cases, Moderate (Hb rise 5-10%) in 23 (85.18%) and marked (Hb rise >10%) in 2 (7.4%) cases was observed. Hydroxyurea was well tolerated in and we got no remarkable adverse effect.
Conclusion: Hydroxyurea can be safely prescribed to transfusion-dependent β-thalassemia major patients for reduction their transfusion requirements.