Efficacy and safety of oral hydroxyurea on gene expression in Transfusion dependent β-thalassaemia major.
- Conditions
- Health Condition 1: D561- Beta thalassemia
- Registration Number
- CTRI/2023/01/048782
- Lead Sponsor
- Multidisciplinary Research Unit MR
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Open to Recruitment
- Sex
- Not specified
- Target Recruitment
- 0
Patients aged 6 years and above.
Patients with confirmed genotypes of homozygous/ compound heterozygous β-thalassaemia major or HbE β-thalassaemia.
Patients who required more than eight blood transfusions during the preceding 12 months.
Sickle β-thalassaemia
Coexisting chronic liver disease
Coexisting chronic kidney disease
Coexisting viral hepatitis
Patients with contraindications for hydroxyurea (e.g., hypersensitivity, bone marrow depression, pregnancy and lactation)
Patients who are expecting to get pregnant during the next 12 months
Patients who have undergone bone marrow transplantation
Patients on immunosuppressant therapy
Baseline serum ferritin >5000 ng/mL
Baseline white cell count <4000/μL
Baseline platelet count <1 50 000/μL
Patients who were started on regular transfusions for a predetermined limited period
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To evaluate the reduction in transfusion requirement as an outcome measure.Timepoint: Follow up monthly during the 6 months of treatment and post treatment follow up for another 6 months
- Secondary Outcome Measures
Name Time Method Reduction in iron overload as assessed by serial serum Ferritin valuesTimepoint: The test will be done every 3 monthly.