Effectiveness of hydroxyurea treatment compared to placebo in reducing transfusions in beta-thalassaemia patients.

INTERVENTION: Place of study will be Adolescent and Adult Thalassaemia Center of the Colombo North Teaching Hospital, Ragama, Sri Lanka. Method of randomization will be simple randomization using an online simple randomization tool. Subjects will be randomized to two arms Active arm treated with hydroxyurea Control arm treated with placebo (This is a matching capsule which contains ingredients identical to hydroxyurea except for the active ingredient). Investigational product [Active hydroxyurea] will be administered at a dose of 500mg (if body weight =<50kg) or 1000mg (if body weight >50kg) orally by subject once daily for six months. Placebo will also be administered once daily for six months. The following will be blinded; participants, data collectors, outcome adjudicators and data analysts CONDITION: Beta‐thalassaemia PRIMARY OUTCOME: Response to treatment: Complete response to treatment is defined as complete cessation of blood transfusions with a baseline haemoglobin >9.0g/dl during the treatment period.; Partial response is defined as over 50% reduction in transfusion requirement during treatment period (=<3 transfusions during treatment 6 months) with baseline haemoglobin >7.0g/dl.; ; Subjects will be evaluated monthly during the period of treatment by full blood count measurements.; ; ; ; SECONDARY OUTCOME: 1. Compliance to hydroxyurea treatment (measured as actual number of tablets taken divided by the estimated number of tablets to be taken). ; ; 2. Side effects of hydroxyurea ; ; Subjects will be evaluated monthly during the period of treatment. Evaluation includes an interview and physical examination by a trained medical officer to assess adverse effects of hydroxyurea. Following investigations will also be done to assess side effects of hydroxyurea; full blood count, serum creatinine and liver transaminases. ; ; INCLUSION CRITERIA: 1. Male and female patients with confirmed genotype of homozygous or compound heterozygous beta‐thalassaemia major or HbE beta‐thalassaemia 2. Age above 12 years 3. Patients who required more than 10 transfusions during the preceding year