Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients
Status:
Completed
Trial end date:
2023-07-30
Target enrollment:
Participant gender:
Summary
Objectives
Primary objective:
• To determine the efficacy and safety of the combination therapy of Hydroxyurea and
thalidomide in beta-thalassemia patients.
Secondary objective:
• To determine the change in liver and spleen size of beta-thalassemia patients on the
combination therapy.
A single-arm non-randomized trial to evaluate the efficacy and safety of combination therapy
of hydroxyurea and thalidomide in beta-thalassemia patients. Participants were monitored for
six months on Hydroxyurea alone and then the combination therapy of hydroxyurea and
thalidomide was started. Findings of physical examination, vital signs, laboratory, and
ultrasound findings were recorded at baseline, during, and end of the study.
The assessment of treatment outcomes was conducted at the 1-year, 2-year, and 3-year
follow-up points during the combination therapy period, categorizing patients as either "good
responders," "responders," or "non-responders."