Clinical patterns of thalidomide in the treatment of transfusion-dependent β-thalassaemia in children: a prospective single-arm study in China.

ABSTRACT:

Background

Previous studies in β-thalassaemia have shown that thalidomide is effective in reducing transfusion volume and is safe for short-term therapy of 6 months or less. The aim of this prospective study was to investigate long-term benefits and side effects of thalidomide in the treatment of transfusion-dependent β-thalassaemia (TDT) in children.

Methods

We analyzed the data from children and adolescents with TDT enrolled in a single-arm prospective study. The overall response rate, the incidence of discontinuation due to adverse events, the effect on height growth, and the effect on nerve conduction velocity were analyzed when patients received thalidomide for up to 1 year, or until unacceptable toxicity during the extended follow-up. The Kruskal-Wallis H test, one-way

SUBMITTER: Li X 

PROVIDER: S-EPMC12517424 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature