ArticleJournal of cellular and molecular medicine2020
Smad2/3-pathway ligand trap luspatercept enhances erythroid differentiation in murine β-thalassaemia by increasing GATA-1 availability.
Article in Journal of cellular and molecular medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06146478 (Deciphering Effects of Thalidomide on Red Blood Cells in Transfusion Dependents Beta Thalassemia Patients), which is not on this map. Cited by 33 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Deciphering Effects of Thalidomide on Red Blood Cells in Transfusion Dependents Beta Thalassemia Patients: A Pharmacodynamics and Pharmacogenetics Analysis
Who cites it
33 citing papers in PubMed, 45 citations in OpenAlex.
- [Luspatercept combined with roxadustat in the treatment of refractory myelodysplastic neoplasms with ring sideroblasts: a prospective, randomized, single-center study].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Trial
- The use of luspatercept for thalassemia in adults.Blood advances · 2021Trial
- Luspatercept: From Bench to Bedside and Beyond in the Management of Ineffective Erythropoiesis.Journal of hematology · 2026Review
- Mesangial sclerosing glomerulopathy following luspatercept treatment - a case report.BMC nephrology · 2026Article
- UBE2O as a key regulator of drug-induced erythropoiesis in the context of myelodysplastic syndromes.Blood advances · 2026Article
- Luspatercept for the treatment of transfusion-dependent non-severe aplastic anemia.Annals of hematology · 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 Applications of Ligand Traps Targeting Activin Type II Receptors.Anti-inflammatory & anti-allergy agents in medicinal chemistry · 2026Review
- Genetic Engineering in Hematopoietic Stem Cells for β-Hemoglobinopathies Treatment: Advances, Challenges, and Clinical Translation.International journal of hematology-oncology and stem cell research · 2025Review
- Splicing of erythroid transcription factor is associated with therapeutic response in myelodysplastic syndromes.The Journal of clinical investigation · 2025Article
- TGF-β inhibition restores hematopoiesis and immune balance via bone marrow EPCs in aplastic anemia.Experimental & molecular medicine · 2025Article
- Targeting TGF-β: a promising strategy for cancer therapy.Medical oncology (Northwood, London, England) · 2025Review
- Population Pharmacokinetic/Pharmacodynamic and Exposure-Response Modeling Analyses of Sotatercept in Healthy Participants and Patients with Pulmonary Arterial Hypertension.Clinical pharmacology and therapeutics · 2025Article
- Malaria and dyserythropoiesis: a mini review.Frontiers in cellular and infection microbiology · 2025Review
- Therapeutic targeting of the hepcidin-ferroportin axis and erythropoietic modulators: a narrative review.Frontiers in medicine · 2025Review
- Iron-Loading Anemias.Advances in experimental medicine and biology · 2025Review
- Combination of a TGF-β ligand trap (RAP-GRL) and TMPRSS6-ASO is superior for correcting β-thalassemia.American journal of hematology · 2024Article
- A human anti-matriptase-2 antibody limits iron overload, α-globin aggregates, and splenomegaly in β-thalassemic mice.Blood advances · 2024Article
- The role of CoQ10 in embryonic development.Journal of assisted reproduction and genetics · 2024Review
- Follistatin is a crucial chemoattractant for mouse decidualized endometrial stromal cell migration by JNK signalling.Journal of cellular and molecular medicine · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In β-thalassaemia, anaemia results from ineffective erythropoiesis characterized by inhibition of late-stage erythroid differentiation. We earlier used luspatercept and RAP-536 protein traps for certain Smad2/3-pathway ligands to implicate Smad2/3-pathway overactivation in dysregulated erythroid differentiation associated with murine β-thalassaemia and myelodysplasia. Importantly, luspatercept alleviates anaemia and has been shown to reduce transfusion burden in patients with β-thalassaemia or myelodysplasia. Here, we investigated the molecular mechanisms underlying luspatercept action and pSmad2/3-mediated inhibition of erythroid differentiation. In murine erythroleukemic (MEL) cells in vitro, ligand-mediated overactivation of the Smad2/3 pathway reduced nuclear levels of GATA-1 (GATA-binding factor-1) and its transcriptional activator TIF1γ (transcription intermediary factor 1γ), increased levels of reactive oxygen species, reduced cell viability and haemoglobin levels, and inhibited erythroid differentiation. Co-treatment with luspatercept in MEL cells partially or completely restored each of these. In β-thalassaemic mice, RAP-536 up-regulated Gata1 and its target gene signature in erythroid precursors determined by transcriptional profiling and gene set enrichment analysis, restored nuclear levels of GATA-1 in erythroid precursors, and nuclear distribution of TIF1γ in erythroblasts. Bone marrow cells from β-thalassaemic mice treated with luspatercept also exhibited restored nuclear availability of GATA-1 ex vivo. Our results implicate GATA-1, and likely TIF1γ, as key mediators of luspatercept/RAP-536 action in alleviating ineffective erythropoiesis.
Indexed as
Identifiers
What OpenQuestion holds
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.