A Phase 2 Randomized Controlled Trial of Single-Agent Hydroxyurea Versus Thalidomide Among Adult Transfusion Dependent β Thalassemia Patients.

ABSTRACT: Hydroxyurea and low dose thalidomide are low-cost, easily accessible Hb F inducing agents that have been found to decrease transfusion dependency among transfusion-dependent thalassemia patients. However, these drugs have not much been explored in a randomized controlled setting. The objective of this study was to determine the efficacy and safety of hydroxyurea and low dose thalidomide in adult transfusion dependent β thalassemia. A total of 39 transfusion dependent β thalassemia patients were randomized into three arms: Arm A (Hydroxyurea 500 mg/day), Arm B (thalidomide 50 mg/day), and Control Arm. The primary outcome was rise in haemoglobin at 24-weeks from the baseline levels. The mean age of the cohort was 26.9 ± 4.7 years. Total 13 patients (33.3%) were splenectomised. The mean rise

SUBMITTER: Bhattacharjee U 

PROVIDER: S-EPMC9809516 | biostudies-literature | 2023 Apr

REPOSITORIES: biostudies-literature