Trial reportBlood advances2021
The use of luspatercept for thalassemia in adults.
Trial report in Blood advances, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 25 papers, 1 of them a synthesis 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.
A Phase 2, Open-Label, Ascending Dose Study to Evaluate the Effects of ACE-536 in Patients With β-Thalassemia
A Phase 3, Double-Blind, Placebo Controlled Multicenter Study to Determine the Efficacy and Safety of Luspatercept (ACE-536) in Adults With Transfusion Dependent Beta (B)-Thalassemia
Who cites it
25 citing papers in PubMed, 1 synthesis or guideline pooled it, 51 citations in OpenAlex.
- Outcomes with luspatercept in patients with β-thalassemia: a systematic review and meta-analysis.Annals of hematology · 2026Pooled it
- Contemporary Management of Thalassemia: A Perspective on Current Standards and the Emergence of FDA-Approved Oral Therapy.Health science reports · 2026Article
- Navigating Anemia in Solid and Hematologic Malignancies: A Comprehensive Review of Classical and Emerging Therapies.European journal of haematology · 2026Review
- Recent Advances in Thalassemia Research: A Comprehensive Assessment From Diagnostic Technologies to Clinical Treatment.Journal of clinical laboratory analysis · 2026Review
- Luspatercept: From Bench to Bedside and Beyond in the Management of Ineffective Erythropoiesis.Journal of hematology · 2026Review
- UBE2O as a key regulator of drug-induced erythropoiesis in the context of myelodysplastic syndromes.Blood advances · 2026Article
- Erythropoiesis in health and disease: Distinguishing defective and ineffective erythropoiesis.HemaSphere · 2026Review
- Acute splenomegaly, haemolysis, and thrombocytopenia following luspatercept initiation in a patient with transfusion-dependent thalassaemia: A case report.British journal of haematology · 2025Article
- Long-Term Experience with Luspatercept in Relapsed/Refractory Myelodysplastic Neoplasms: A Chinese Real-World Study.Advances in therapy · 2025Article
- Chronic Inflammation in Primary Myelofibrosis: In-Depth Insights Into Pathogenesis and Promising Anti-Inflammatory Therapeutic Strategies.Mediators of inflammation · 2025Review
- Luspatercept in β-thalassemia: Who and when. Strengths and weaknesses points of a real-world evidence.EJHaem · 2024Article
- Beta Thalassemia in Children: Established Approaches, Old Issues, New Non-Curative Therapies, and Perspectives on Healing.Journal of clinical medicine · 2024Review
- Combination of a TGF-β ligand trap (RAP-GRL) and TMPRSS6-ASO is superior for correcting β-thalassemia.American journal of hematology · 2024Article
- TGF-β signaling: critical nexus of fibrogenesis and cancer.Journal of translational medicine · 2024Review
- Luspatercept's use in a patient with transfusion-dependent beta-thalassemia and intrathoracic extramedullary hematopoiesis (EMH).Clinical case reports · 2024Article
- Human cellular model systems of β-thalassemia enable in-depth analysis of disease phenotype.Nature communications · 2023Article
- Real-life experience with luspatercept in transfusion-dependent β-thalassemia.Annals of hematology · 2023Article
- Therapeutic perspective for children and young adults living with thalassemia and sickle cell disease.European journal of pediatrics · 2023Review
- Efficacy and Safety of Luspatercept in the Treatment of β-Thalassemia: A Systematic Review.Cureus · 2022Review
- Myostatin/Activin Receptor Ligands in Muscle and the Development Status of Attenuating Drugs.Endocrine reviews · 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
2 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Luspatercept is an activin receptor ligand trap that has been shown to enhance late-stage erythropoiesis in animal models of β-thalassemia. A multicenter, international, phase 2 dose-finding study was initiated in adult patients with β-thalassemia, either non-transfusion-dependent thalassemia (NTDT) or transfusion-dependent thalassemia (TDT). Positive results of the phase 2 study paved the way to a randomized phase 3 clinical trial (BELIEVE) to assess the efficacy and safety of luspatercept. The BELIEVE trial is a randomized, double-blind, placebo-controlled phase 3 trial. Three hundred thirty-six patients aged ≥18 years with TDT (regularly transfused, 6-20 red blood cell units within 24 weeks before randomization) were included in the trial. Patients received luspatercept or placebo subcutaneously every 21 days for ≥48 weeks and best supportive care. Forty-eight of 224 patients (21.4%) in the luspatercept group achieved the primary end points (≥33% reduction in transfusion burden) compared with those in the placebo group (4.5%; P < .001). Moreover, more patients had a ≥33% reduction in transfusion burden during any rolling 12-week interval (70.5% vs 29.5%) or any 24-week interval (41.1% vs 2.7%) with luspatercept than with the placebo. Transfusion independence was achieved by 11% of patients in the luspatercept group. Transient adverse events were more frequent with luspatercept than with placebo, but were manageable. Luspatercept was approved by the US Food and Drug Administration in 2019 and by the European Medicines Agency in 2020. The luspatercept trial is registered on www.clinicaltrials.gov at #NCT01749540 and the BELIEVE trial at #NCT02604433.
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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.