ReviewDiseases (Basel, Switzerland)2022
Luspatercept: A New Tool for the Treatment of Anemia Related to β-Thalassemia, Myelodysplastic Syndromes and Primary Myelofibrosis.
Review in Diseases (Basel, Switzerland), 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 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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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
15 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Luspatercept for patients with lower-risk myelodysplastic syndromes/neoplasms: a systematic review and meta-analysis.Blood advances · 2025Pooled it
- Erythropoiesis-Targeted Doping in Sports: From Improved Oxygen Transport to Cardiovascular Risk.Pathophysiology : the official journal of the International Society for Pathophysiology · 2026Review
- Sex and gender differences in responses to antianemic therapies: biological mechanisms and clinical implications.Naunyn-Schmiedeberg's archives of pharmacology · 2026Review
- Review
- Luspatercept: From Bench to Bedside and Beyond in the Management of Ineffective Erythropoiesis.Journal of hematology · 2026Review
- miR-29a-3p and TGF-β Axis in Fanconi anemia: mechanisms driving metabolic dysfunction and genome stability.Cellular and molecular life sciences : CMLS · 2025Article
- Emerging Therapeutic Approaches for Anemia in Myelofibrosis.Current hematologic malignancy reports · 2025Review
- Mechanisms and treatment of pulmonary arterial hypertension.Nature reviews. Cardiology · 2025Review
- Therapeutic targeting of the hepcidin-ferroportin axis and erythropoietic modulators: a narrative review.Frontiers in medicine · 2025Review
- Momelotinib in myelofibrosis and beyond: a comprehensive review of therapeutic insights in hematologic malignancies.Discover oncology · 2024Review
- Luspatercept enhances hemoglobin levels in a Chinese boy with congenital sideroblastic anemia: A case report.World journal of clinical cases · 2024Article
- Luspatercept's use in a patient with transfusion-dependent beta-thalassemia and intrathoracic extramedullary hematopoiesis (EMH).Clinical case reports · 2024Article
- Review
- Predicting Thalassemia Using Feature Selection Techniques: A Comparative Analysis.Diagnostics (Basel, Switzerland) · 2023Article
- Association of Myelofibrosis Phenotypes with Clinical Manifestations, Molecular Profiles, and Treatments.Cancers · 2023Review
Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Anemia is a common feature of both benign and malignant hematologic diseases. Beta-thalassemia (β-thalassemia) syndromes are a group of hereditary disorders characterized by ineffective erythropoiesis, due to a genetic deficiency in the synthesis of the beta chains of hemoglobin, often accompanied by severe anemia and the need for red blood cell (RBC) transfusions. Myelodysplastic syndromes (MDS) are characterized by cytopenia(s) and ineffective hematopoiesis, despite a hypercellular bone marrow. Primary myelofibrosis (PMF) is a clonal myeloproliferative neoplasm characterized by reactive fibrosis of the bone marrow, accompanied by extramedullary hematopoiesis. Luspatercept, previously known as ACE-536, is a fusion protein that combines a modified activin receptor IIB (ActRIIB), a member of the transforming growth factor-β (TGF-β) superfamily, with the Fc domain of human immunoglobulin G (IgG1). It has shown efficacy in the treatment of anemia due to beta β-thalassemia, MDS and PMF and recently gained approval by the Federal Drug Agency (FDA) and the European Medicines Agency (EMA) for transfusion-dependent (TD) patients with β-thalassemia and very low to intermediate-risk patients with MDS with ringed sideroblasts who have failed to respond to, or are ineligible for, an erythropoiesis-stimulating agent. In this review, we describe the key pathways involved in normal hematopoiesis and the possible mechanism of action of luspatercept, present its development and data from the most recent clinical trials in β-thalassemia, MDS and PMF, and discuss its potential use in the treatment of these hematological disorders.
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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.