RecruitingPhase 2NCT06490627

Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia

Sponsor

National Institute of Blood and Marrow Transplant (NIBMT), Pakistan

Enrollment

54 participants

Start Date

Apr 22, 2024

Study Type

INTERVENTIONAL

Conditions

Genetic Disease Fetal Hemoglobin Thalassemia Major

Summary

The project "Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing anemia. Conducted over two years at NIBD hospital, the study involves 54 transfusion-dependent patients aged 8-35. The primary objective is to correlate thalidomide doses with disease severity, adverse effects, and treatment response, aiming to optimize treatment strategies and reduce side effects. Data will be collected through clinical interviews and medical record reviews and analyzed using SPSS. Key variables include hemoglobin levels, leukocyte and reticulocyte counts, platelets, liver and spleen size, genetic modifiers, and transfusion frequency. Inclusion criteria are specific to beta thalassemia patients, while exclusion criteria rule out those with liver dysfunction, married patients, lactating mothers, and those with a history of thrombosis or fits.

Eligibility

Min Age: 8 YearsMax Age: 35 Years

Inclusion Criteria2

Know case of beta thalassemia major/ intermediate ( transfusion dependent)
willing to give informed consent

Exclusion Criteria4

Patients with comorbidities such as liver dysfunction
Married patients
Lactating mother
H/O thrombosis and fits

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Interventions

DRUGThalidomide

Thalidomide is being investigated for its therapeutic efficacy and safety profile in transfusion-dependent beta thalassemia patients. Participants in this arm received thalidomide. Thalidomide was administered orally at a dosage of 5-6mg/kg/day. The medication was taken continuously or on a specified schedule (e.g., daily,) for the duration of the study period, which lasted 2 years. Participants were monitored for adherence to the medication regimen and for any adverse effects throughout the intervention period.

DRUGThalidomide

Thalidomide is being investigated for its therapeutic efficacy and safety profile in transfusion-dependent beta thalassemia patients. Participants in this arm received thalidomide. Thalidomide was administered orally at a dosage of 7-8mg/kg/day. The medication was taken continuously or on a specified schedule (e.g., daily,) for the duration of the study period, which lasted 2 years. Participants were monitored for adherence to the medication regimen and for any adverse effects throughout the intervention period.