ReviewInternational journal of molecular sciences2021
Ineffective Erythropoiesis in β-Thalassaemia: Key Steps and Therapeutic Options by Drugs.
Review in International journal of molecular sciences, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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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.
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Who cites it
20 citing papers in PubMed, 44 citations in OpenAlex.
- Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent β-thalassemia: results from a randomized phase 2a study.Orphanet journal of rare diseases · 2025Trial
- Multiomics analysis of red blood cells reveals thalassemia severity beyond globin gene mutations.Blood advances · 2026Article
- Distribution of Abnormal Hemoglobins in Saudi Arabia: Analysis of Private Sector Laboratory Data.Journal of epidemiology and global health · 2026Article
- SNH-119014, a novel pyruvate kinase activator, enhances ATP production and reduces oxidative stress in erythroid cells from patients with β-thalassemia major.Frontiers in pharmacology · 2026Article
- Ineffective Erythropoiesis Markers in β-Thalassemia: A Systematic Review.Journal of clinical medicine · 2025Review
- Beta thalassemia syndromes: New insights.World journal of clinical cases · 2025Review
- Metabolic and Proteomic Divergence is Present in Spleens and Livers from Berkeley Sickle Cell Anemia and β-Thalassemia Mice.Journal of proteome research · 2025Article
- Investigation on the Anxiety and Depression Status of Thalassemia Carriers After Genetic Counseling.Neuropsychiatric disease and treatment · 2025Article
- Leptin and thyroid hormones in beta-thalassemia major: A cross-sectional study.Caspian journal of internal medicine · 2025Article
- A Review of Key Regulators of Steady-State and Ineffective Erythropoiesis.Journal of clinical medicine · 2024Review
- The interactions between ineffective erythropoiesis and ferroptosis in β-thalassemia.Frontiers in physiology · 2024Review
- Low Vitamin D Levels Are Associated with Increased Cardiac Iron Uptake in Beta-Thalassemia Major.Diagnostics (Basel, Switzerland) · 2023Article
- Pyruvate kinase activators: targeting red cell metabolism in thalassemia.Hematology. American Society of Hematology. Education Program · 2023Article
- Post-transcriptional regulation of erythropoiesis.Blood science (Baltimore, Md.) · 2023Review
- TGF-beta signal transduction: biology, function and therapy for diseases.Molecular biomedicine · 2022Review
- Redox Balance in β-Thalassemia and Sickle Cell Disease: A Love and Hate Relationship.Antioxidants (Basel, Switzerland) · 2022Review
- Prime Editor 3 Mediated Beta-Thalassemia Mutations of theInternational journal of molecular sciences · 2022Article
- Phosphatidylserine-exposed red blood cells and ineffective erythropoiesis biomarkers in patients with thalassemia.American journal of translational research · 2022Article
- Expression of γ-globin genes in β-thalassemia patients treated with sirolimus: results from a pilot clinical trial (Sirthalaclin).Therapeutic advances in hematology · 2022Article
- The Utility of HbA1c and Fructosamine in Evaluating the Glucose Tolerance in Adult Patients with Transfusion-Dependent Beta-Thalassemia.Indian journal of endocrinology and metabolismArticle
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
β-thalassaemia is a rare genetic condition caused by mutations in the β-globin gene that result in severe iron-loading anaemia, maintained by a detrimental state of ineffective erythropoiesis (IE). The role of multiple mechanisms involved in the pathophysiology of the disease has been recently unravelled. The unbalanced production of α-globin is a major source of oxidative stress and membrane damage in red blood cells (RBC). In addition, IE is tightly linked to iron metabolism dysregulation, and the relevance of new players of this pathway, i.e., hepcidin, erythroferrone, matriptase-2, among others, has emerged. Advances have been made in understanding the balance between proliferation and maturation of erythroid precursors and the role of specific factors in this process, such as members of the TGF-β superfamily, and their downstream effectors, or the transcription factor GATA1. The increasing understanding of IE allowed for the development of a broad set of potential therapeutic options beyond the current standard of care. Many candidates of disease-modifying drugs are currently under clinical investigation, targeting the regulation of iron metabolism, the production of foetal haemoglobin, the maturation process, or the energetic balance and membrane stability of RBC. Overall, they provide tools and evidence for multiple and synergistic approaches that are effectively moving clinical research in β-thalassaemia from bench to bedside.
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Registered trials
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