ArticleAnnals of medicine2025
Clinical patterns of thalidomide in the treatment of transfusion-dependent β-thalassaemia in children: a prospective single-arm study in China.
Article in Annals of medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
3 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Efficacy and safety of thalidomide in β-thalassaemia: a systematic review and meta-analysis.Scientific reports · 2026Pooled it
- Efficacy and Safety of Thalidomidein Patients with Transfusion-Dependent B-Thalassemia: A Systematic Review, Meta-Analysis and GRADE Evaluation.Mediterranean journal of hematology and infectious diseases · 2026Review
- Comprehensive Review of Genetic and Epigenetic Regulation of Fetal Hemoglobin in β-Hemoglobinopathies: From Molecular Mechanisms to Clinical Applications.Mediterranean journal of hematology and infectious diseases · 2026Review
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Authors and funding
11 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPrevious 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.
methodsWe 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 ANOVA, the Cochran-Mantel-Haenszel Chi-square test, and others were used for analysis.
resultsThe overall response rate was 82.9%. The most common grade 3-4 adverse events were abdominal pain, haemolysis, and leukocytopenia/neutropenia. Of the 73 patients who underwent nerve conduction velocity assessment during treatment for more than 12 months, 12 patients developed polyneuropathy and 26 developed motor neuropathy. The standard deviation scores (SDS) of height in major responders were significantly improved (-1.57, 95% CI (-1.95, -1.18)) at the end of 12 to 18-month treatment (
conclusionThalidomide is associated with long-term benefits in growth and transfusion burden. A minority of patients required prolonged therapy beyond 6 months to achieve a response. Neuropathy and unexpected adverse events prevent the long-term use of thalidomide in children with thalassaemia and close monitoring is needed. (
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