This study aimed to determine the safety and efficacy of thalidomide in multi-transfused thalassemic children.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
121
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.
The Children's Hospital & Institute of Child Health
Multan, Punjab Province, Pakistan
Efficacy of treatment
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.
Time frame: 6 months
Safety of treatment
Treatment was considered safe if no adverse events were documented based on clinical assessment and laboratory findings.
Time frame: 6 months
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