Thalidomide for Patients with β-Thalassemia: A Multicenter Experience.

ABSTRACT:

Objective

This study focused on the efficacy and safety of thalidomide for patients with β-thalassemia in a multicenter trial.

Methods

Patients with non-transfusion-dependent thalassemia (NTDT) or transfusion-dependent thalassemia (TDT), who were unable to pursue conventional therapy with transfusion and chelation, were recruited over 3 years in three centers in southern China. We evaluated the efficacy and safety of thalidomide in the short-term (three months) and long-term follow-up (12 and 24 months). Response to thalidomide was defined as follows: Main Responder (MaR) showing an increase in hemoglobin (Hb) level of >2.0 g/dl or free from blood transfusion and Minor Responder (MiR) achieving elevated Hb level of 1.0-2.0 g/dl or ≥50% reduction in blood transfusion frequenc

SUBMITTER: Yang K 

PROVIDER: S-EPMC7202343 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature