Beta Thalassemia
Conditions
Interventions
Participants in the single-arm study will receive combination therapy with Hydroxyurea plus Thalidomide.
Hydroxyurea will be given via oral route [capsule form], once daily every 24 hours at a dose of
Sponsors
Dow University of Health Sciences
Eligibility
Inclusion criteria
Inclusion criteria: Male and female patients with major thalassemia B type within the age range of 20 to 50 years.
Exclusion criteria
Exclusion criteria: 1. Patients with other types of thalassemia. 2. Pregnant or breastfeeding women. 3. Significant hepatic (ALT/AST >2× ULN) or renal dysfunction (eGFR <60 mL/min/1.73m²) 4. Uncontrolled infection, active malignancy 5. Prior thalidomide exposure 6. Peripheral neuropathy grade 2 or more, baseline platelet < 100 × 10^9/L
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in Hb gm/dl. [3 and 6 months after initiation of treatment] Change in Blood transfusion frequency. [ 3 and 6 months after initiation of treatment] Change in Hb F. [ 3 months after initiation of treatment] | — |
Secondary
| Measure | Time frame |
|---|---|
| Transfusion dependence will be assessed by calculating the total Packed Red Blood Cell volume (mL/kg) transfused during the 6-month period before treatment initiation and during the 6-month treatment period. Participants will be classified as good, partial, or non-responders based on the percentage reduction in transfusion volume, using previously published thresholds (>50%, 25–50%, <25%) [Before treatment initiation, 6 months after starting treatment. ] Quality of life will be assessed using the TranQoL (Transfusion-dependent Quality of Life Questionnaire), a validated disease-specific tool for transfusion-dependent thalassemia patients. It will be administered at baseline and after 6 months of therapy. The validated Urdu version will be used for participants who prefer it. [ baseline and at 6 months] | — |
Countries
Pakistan
Contacts
Public ContactInshal Jawed
General Physician
Outcome results
None listed