Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia

Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation. Around 23,000 babies are affected by BTM each year, of which around 90% belong to low- or middle-income nations. In Pakistan, the carrier prevalence of thalassemia is 5-7% resulting in a significant population of approximately 10 million carriers in the general population. There are 50,000 thalassemia patients registered in treatment facilities around the country, one of the highest global prevalence rates for transfusion dependent BTM. The average life expectancy of BTM patients in Pakistan is around 10 years of age, while life expectancy in developed countries is around 50 to 60 years. This difference is due to poor transfusion support, transfusion-transmitted infections (TTIs) and inadequate iron chelation leading to hepatotoxicity and cardiac failure. The standard of care for BTM remains bone marrow transplantation or lifelong blood transfusions followed by iron chelation therapies. While standard care involves, challenges such as limited resources, lack of access to transplant services, and transfusion-related complications persist, particularly in low-and-middle-income countries.

NCT Number	NCT07292259
Lead Sponsor	Pakistan Blood and Marrow Transplant (PBMT) Group
Conditions	Transfusion Dependent Beta Thalassemia
Enrollment	100 participants
Start Date	2024-01-01
Primary Completion	2025-12-30 (estimated)
Study Completion	2025-12-30 (estimated)
Updated on ClinicalTrials.gov	2026-05-05