Safety and Efficacy of Immunomodulator Drug Thalidomide in Multi-transfused Thalassemia Patients

Muhammad Aamir Latif

Status: Completed

Conditions: Thalassemia

Treatments: Drug: Thalidomide

Study type: Interventional

Funder types: Other

Identifiers

NCT06888232

DRASLAMCHMULTAN

Details and patient eligibility

About

This study aimed to determine the safety and efficacy of thalidomide in multi-transfused thalassemic children.

Full 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.

Enrollment: 121 patients

Sex: All

Ages: 2 to 5 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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121 participants in 1 patient group

Thalidomide group

Experimental group

Description:

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.

Treatment:

Drug: Thalidomide