Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia
- Registration Number
- NCT03651102
- Lead Sponsor
- Blood Diseases Clinic
- Brief Summary
Thalidomide is known to have hypnosedative, immuno-modulatory and anti-angiogenic effects. The drug is widely used in several neoplastic disorders (e.g. multiple myeloma and malignant melanoma), inflammatory conditions (e.g. Crohn's disease) and skin disorders (e.g. leprosy). Thalidomide has been successfully used in adult thalassemia patients. The current study explores its role in transfusion-dependent thalassemia patients.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 654
Inclusion Criteria
- Transfusion dependent thalassemia patients refractory to Hydroxyurea
Exclusion Criteria
- Those with active systemic comorbidity, with past personal or family history of thrombophilia, recent fracture or recent major surgery
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Thalidomide in combination with hydroxyurea Thalidomide and Hydroxyurea All the study patients refractory to hydroxyurea will be given thalidomide at an average dose of 2mg/kg/day (range 1-4mg/kg/day). Participants will also be continued on hydroxyurea at a dose of 10-20 mg/kg/day.
- Primary Outcome Measures
Name Time Method Adverse Effects 6-24 months Haemoglobin Level 6-24 months mg/dL
Response Rate (Excellent, Good, Partial and No response) 6-24 months
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Blood Diseases Clinic
🇵🇰Peshawar, Khyber Pakhtukhwa, Pakistan