Trial reportSignal transduction and targeted therapy2021
Safety and efficacy of thalidomide in patients with transfusion-dependent β-thalassemia: a randomized clinical trial.
Trial report in Signal transduction and targeted therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 22 papers, 2 of them syntheses that pooled it.
What it found
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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.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Safety & Efficacy of Thalidomide in Children With Transfusion Dependent Thalassemia: a Quasi Randomized Control Trial in a Tertiary Care Hospital in Bangladesh
Efficacy of Combination of Hdroxyurea and Thalidomide Over Either Hydroxyurea or Thalidomide Alone in the Treatment of Transfusion Dependent Thalassemia in Children: A Quasi-Randomised Clinical Trial
Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia: a Prospective 12 Months Study
Who cites it
22 citing papers in PubMed, 2 syntheses or guidelines pooled it, 39 citations in OpenAlex.
- Efficacy and safety of thalidomide in β-thalassaemia: a systematic review and meta-analysis.Scientific reports · 2026Pooled it
- [Chinese guideline for diagnosis and treatment of transfusion dependent β-thalassemia (2022)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2022Guideline
- Long-term clinical efficacy and safety of thalidomide in patients with transfusion-dependent β-thalassemia: results from Thal-Thalido study.Scientific reports · 2023Trial
- Thalassaemia.Nature reviews. Disease primers · 2026Review
- Low- to moderate-dose thalidomide combination rescue after luspatercept failure in transfusion-dependent β-thalassemia: a real-world cohort study.Annals of hematology · 2026Article
- Roxadustat induced fetal hemoglobin in anemic CKD patients: a dual-center real-world observational cohort study.BMC nephrology · 2026Observational
- Expression of Immune CheckpointsTurkish journal of haematology : official journal of Turkish Society of Haematology · 2026Article
- 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
- Clinical patterns of thalidomide in the treatment of transfusion-dependent β-thalassaemia in children: a prospective single-arm study in China.Annals of medicine · 2025Article
- Thalidomide Treatment for Thrombocytopenia Secondary to Hypersplenism in Children with Transfusion-Dependent β-Thalassemia: A Case Series.Mediterranean journal of hematology and infectious diseases · 2025Article
- Cardiac injury caused by iron overload in thalassemia.Frontiers in pediatrics · 2025Review
- Hedy: a groundbreaking revelation of cartilage oxygen homeostasis.Acta biochimica et biophysica Sinica · 2024Article
- Long-Term Follow-Up of Patients Undergoing Thalidomide Therapy for Transfusion-Dependent β-Thalassaemia: A Single-Center Experience.International journal of general medicine · 2024Article
- Effects of Thalidomide on Erythropoiesis and Iron Homeostasis in Transfusion-Dependent β-Thalassemia.Mediterranean journal of hematology and infectious diseases · 2024Article
- Article
- Thalidomide-Induced Primary Amenorrhea in a Patient With HbE/Beta-Thalassemia.JCEM case reports · 2023Article
- Combined approaches for increasing fetal hemoglobin (HbF) andFrontiers in genome editing · 2023Review
- Measurement of Erythrocyte Lifespan Using a CO Breath Test in Patients with Thalassemia and the Impact of Treatment.Mediterranean journal of hematology and infectious diseases · 2023Article
- Evaluation of the combination therapy of hydroxyurea and thalidomide in β-thalassemia.Blood advances · 2022Article
- Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective.Pharmaceuticals (Basel, Switzerland) · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
31 authors at 13 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Thalidomide induces γ-globin expression in erythroid progenitor cells, but its efficacy on patients with transfusion-dependent β-thalassemia (TDT) remains unclear. In this phase 2, multi-center, randomized, double-blind clinical trial, we aimed to determine the safety and efficacy of thalidomide in TDT patients. A hundred patients of 14 years or older were randomly assigned to receive placebo or thalidomide for 12 weeks, followed by an extension phase of at least 36 weeks. The primary endpoint was the change of hemoglobin (Hb) level in the patients. The secondary endpoints included the red blood cell (RBC) units transfused and adverse effects. In the placebo-controlled period, Hb concentrations in patients treated with thalidomide achieved a median elevation of 14.0 (range, 2.5 to 37.5) g/L, whereas Hb in patients treated with placebo did not significantly change. Within the 12 weeks, the mean RBC transfusion volume for patients treated with thalidomide and placebo was 5.4 ± 5.0 U and 10.3 ± 6.4 U, respectively (P < 0.001). Adverse events of drowsiness, dizziness, fatigue, pyrexia, sore throat, and rash were more common with thalidomide than placebo. In the extension phase, treatment with thalidomide for 24 weeks resulted in a sustainable increase in Hb concentrations which reached 104.9 ± 19.0 g/L, without blood transfusion. Significant increase in Hb concentration and reduction in RBC transfusions were associated with non β0/β0 and HBS1L-MYB (rs9399137 C/T, C/C; rs4895441 A/G, G/G) genotypes. These results demonstrated that thalidomide is effective in patients with TDT.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.