Thalassemia
Conditions
Brief summary
This study aimed to determine the safety and efficacy of thalidomide in multi-transfused thalassemic children.
Detailed description
Given the significant burden of transfusion dependency, an effective and safe inducer is needed that could improve quality of life and reduce the healthcare costs associated with frequent transfusions and iron chelation therapy. If this study could establish the safety of thalidomide in terms of teratogenicity along with its efficacy, it would be a great support to multi-transfused thalassemic children.
Interventions
DRUGThalidomide
Thalidomide was administered at a dose of 1.5 mg/kg/day, and all patients continued standard iron chelation therapy and other supportive treatments as per institutional protocols.
Sponsors
Muhammad Aamir Latif
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 5 Years
Healthy volunteers
No
Inclusion criteria
* Children of both genders * Aged 2-5 years * With transfusion-dependent thalassemia (TDT) * Whose parents/guardians showed willingness to adhere to follow-up visits
Exclusion criteria
* Children with uncontrolled infections * Significant hepatic or renal dysfunction * Malignancy * Known contraindications to thalidomide
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of treatment | 6 months | Efficacy was categorized as excellent response showing an increase in hemoglobin by 1-2 g/dL or freedom from blood transfusions, partial response as an increase in hemoglobin \<1 g/dL or a decrease in transfusion frequency, or no response if there was no increase in hemoglobin or an increase in transfusion requirement within three months. |
| Safety of treatment | 6 months | Treatment was considered safe if no adverse events were documented based on clinical assessment and laboratory findings. |
Countries
Pakistan
Outcome results
None listed