Beta-thalassaemia
Conditions
Interventions
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
participants, data collectors, outcome adjudicators and data analysts
Sponsors
Faculty of Medicine - University of Kelaniya
Eligibility
Inclusion criteria
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
Exclusion criteria
Exclusion criteria: 1. Patients who have undergone bone marrow transplantation 2. Patients with very high serum ferritin (>5000ng/ml) 3. Sickle beta-thalassaemia 4. Chronic liver disease 5. Chronic kidney disease 6. Patients with contraindications for hydroxyurea therapy (eg: hypersensitivity, severe anaemia with haemoglobin less than 5g/dl at the beginning of treatment, bone marrow depression, pregnancy and lactation) 7. Patients who are expecting to get pregnant during next 12 months 8. White blood cell count less than 4000/uL. 9. Platelet count less than 150,000/uL. 10. Evidence of active viral infection, including viral hepatitis 11. Patients on immunosuppressant therapy
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 (=7.0g/dl. Subjects will be evaluated monthly during the period of treatment by full blood count measurements. [At the end of six months of initiating treatment.] | — |
Secondary
| Measure | Time frame |
|---|---|
| Compliance to hydroxyurea treatment (measured as actual number of tablets taken divided by the estimated number of tablets to be taken). [Compliance to hydroxyurea treatment will be assessed monthly for 6 months after initiating treatment ] 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. [ Side effects will be assessed monthly for 12 months after initiating treatment] | — |
Countries
Sri Lanka
Contacts
Public ContactDr Sachith Mettananda
Senior Lecturer and Consultant Paediatrician
Outcome results
None listed