ReviewDrugs2020
Beta Thalassemia: New Therapeutic Options Beyond Transfusion and Iron Chelation.
Review in Drugs, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 51 papers.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
51 citing papers in PubMed, 94 citations in OpenAlex.
- The use of luspatercept for thalassemia in adults.Blood advances · 2021Trial
- The shifting global thalassemia burden and lessons from Chineseintegrated control strategy.iScience · 2026Article
- Contemporary Management of Thalassemia: A Perspective on Current Standards and the Emergence of FDA-Approved Oral Therapy.Health science reports · 2026Article
- Article
- The Effectiveness of a Resilience Enhancement Program on the Quality of Life Among Adolescents with Thalassemia in Nakhon Si Thammarat Province, Thailand.Healthcare (Basel, Switzerland) · 2026Article
- Utility of Native T1 Mapping for the Evaluation of Myocardial Iron Overload in Patients with Thalassemia Major.Tomography (Ann Arbor, Mich.) · 2026Article
- Glomerular Injury Findings in Patients with Thalassemia Minor.International journal of molecular sciences · 2026Article
- CCND3 Suppression Ameliorates β-Thalassaemia in a Murine Disease Model: A Potential Therapeutic Strategy.Cells · 2026Article
- Discovery of a Novel DNMT1 Inhibitor with Improved Efficacy in Treating β-Thalassemia.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Direct and indirect medical costs associated with transfusion-dependent beta-thalassemia in Brazil, Thailand, and India.Therapeutic advances in hematology · 2026Article
- Erythropoiesis in health and disease: Distinguishing defective and ineffective erythropoiesis.HemaSphere · 2026Review
- The Association between β-Thalassemia Major (β-TM) and Cardiac Complications: Recent Insights.Current cardiology reviews · 2026Review
- MAZ regulates fetal hemoglobin repression by activating MYB transcription.Scientific reports · 2025Article
- Impact of transferrin levels on iron accumulation in transfusion-dependent beta-thalassemia: A genotype-specific analysis.Journal of medical biochemistry · 2025Article
- Evaluating the effect of chicory on serum ferritin levels in transfusion-dependent Β-thalassemia patients: a randomized clinical trial.Annals of medicine and surgery (2012) · 2025Article
- Genetic analysis of thalassemia in putian: comparative insights into mutation spectra with other global regions.Annals of hematology · 2025Article
- Beta thalassemia syndromes: New insights.World journal of clinical cases · 2025Review
- iFGF23 Plasma Levels in Transfusion-Dependent β-Thalassemia: Insights into Bone and Iron Metabolism.Journal of clinical medicine · 2025Article
- Haplotype-Resolved Genotyping and Association Analysis of 1,020 β-Thalassemia Patients by Targeted Long-Read Sequencing.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Assessment of cardiac iron deposition and genotypic classification in pediatric beta-thalassemia major: the role of cardiac MRI.BMC medical imaging · 2025Article
Corrections and comments
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Authors and funding
4 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hemoglobinopathies are among the most common monogenic diseases worldwide. Approximately 1-5% of the global population are carriers for a genetic thalassemia mutation. The thalassemias are characterized by autosomal recessive inherited defects in the production of hemoglobin. They are highly prevalent in the Mediterranean, Middle East, Indian subcontinent, and East and Southeast Asia. Due to recent migrations, however, the thalassemias are now becoming more common in Europe and North America, making this disease a global health concern. Currently available conventional therapies in thalassemia have many challenges and limitations. A better understanding of the pathophysiology of β-thalassemia in addition to key developments in optimizing transfusion programs and iron-chelation therapy has led to an increase in the life span of thalassemia patients and paved the way for new therapeutic strategies. These can be classified into three categories based on their efforts to address different features of the underlying pathophysiology of β-thalassemia: correction of the globin chain imbalance, addressing ineffective erythropoiesis, and improving iron overload. In this review, we provide an overview of the novel therapeutic approaches that are currently in development for β-thalassemia.
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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.