ReviewHematology. American Society of Hematology. Education Program2018
Beyond transfusion therapy: new therapies in thalassemia including drugs, alternate donor transplant, and gene therapy.
Review in Hematology. American Society of Hematology. Education Program, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 21 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.
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
A Phase 3, Double-blind, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo for the Treatment of Anemia Due to the IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in Subjects With Ring Sideroblasts Who Require Red Blood Cell Transfusions.
Closely Matched Unrelated Donor Peripheral Blood Stem Cell Transplantation With TCRαβ+ T Cell and B Cell Depletion For Patients With Sickle Cell Disease and Thalassemia Major
Who cites it
21 citing papers in PubMed, 38 citations in OpenAlex.
- Thalassemia in Guangxi: current situation and future prospects.Frontiers in public health · 2026Article
- From misconceptions to empowerment: assessing health and genetic literacy on thalassemia among Tunisian secondary school students.BMC public health · 2025Article
- 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
- Sickle cell β-thalassemia diagnosed at age 40: a case report.Annals of hematology · 2025Article
- Comparison of Asymptomatic Brain Lesions Between Thalassemia Major and Sickle Cell Anemia Patients.Medicina (Kaunas, Lithuania) · 2025Article
- Impact of hand grip strength reduce on health related quality of life in patients with transfusion-dependent thalassemia insights from the SF-36 survey: a cross-sectional analysis.Frontiers in medicine · 2025Article
- Beta Thalassemia in Children: Established Approaches, Old Issues, New Non-Curative Therapies, and Perspectives on Healing.Journal of clinical medicine · 2024Review
- Human cellular model systems of β-thalassemia enable in-depth analysis of disease phenotype.Nature communications · 2023Article
- Health-Related Quality-of-Life Profile of Pediatric Patients with β Thalassemia after Hematopoietic Stem Cell Transplantation.Journal of clinical medicine · 2023Article
- Interpreting Iron Homeostasis in Congenital and Acquired Disorders.Pharmaceuticals (Basel, Switzerland) · 2023Review
- Systematic Review and Meta-Analysis of Health-Related Quality of Life in Patients with β-Thalassemia that Underwent Hematopoietic Stem Cell Transplantation.Clinical practice and epidemiology in mental health : CP & EMH · 2023Article
- Conditioning Regimens in Patients with β-Thalassemia Who Underwent Hematopoietic Stem Cell Transplantation: A Scoping Review.Journal of clinical medicine · 2022Article
- HSCT remains the only cure for patients with transfusion-dependent thalassemia until gene therapy strategies are proven to be safe.Bone marrow transplantation · 2021Review
- Molecular genetics of β-thalassemia: A narrative review.Medicine · 2021Review
- Ineffective Erythropoiesis in β-Thalassaemia: Key Steps and Therapeutic Options by Drugs.International journal of molecular sciences · 2021Review
- Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts.Translational pediatrics · 2021Article
- How to Facilitate Decision-Making for Hematopoietic Stem Cell Transplantation in Patients With Hemoglobinopathies. The Perspectives of Healthcare Professionals.Frontiers in pediatrics · 2021Article
- Novel genetic therapeutic approaches for modulating the severity of β-thalassemia (Review).Biomedical reports · 2020Review
- Oral ferroportin inhibitor VIT-2763: First-in-human, phase 1 study in healthy volunteers.American journal of hematology · 2020Article
- CaM kinase II regulates cardiac hemoglobin expression through histone phosphorylation upon sympathetic activation.Proceedings of the National Academy of Sciences of the United States of America · 2019Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Transfusion combined with chelation therapy for severe β thalassemia syndromes (transfusion-dependent thalassemia [TDT]) has been successful in extending life expectancy, decreasing comorbidities and improving quality of life. However, this puts lifelong demands not only on the patients but also on the health care systems that are tasked with delivering long-term treatment and comprehensive support. Prevention programs and curative approaches are therefore an important part of overall strategy. Curative treatments alter the dynamic of a patient's health care costs, from financial commitment over 50 years, into a potential "one-off" investment. Since the 1980s, this has usually been available only to the 30% or so of young children with matched sibling donors. By improving the safety of matched related donors and haploidentical hematopoietic stem cell transplants, the potential size of the donor pool for curative therapies may be increased. Recent advances in gene therapy demonstrate that even patients lacking a matched donor can be rendered transfusion independent with an autograft of genetically modified autologous stem cells, with a low short-term risk. Noncurative treatments are also of potential value by decreasing use of blood and chelators and decreasing hospital visits. An example is luspatercept, an activin-receptor trap that modifies transforming growth factor-β signaling, thereby increasing the efficiency of erythropoiesis. This has entered phase 3 clinical trials for TDT and non-TDT and, usefully increases in both Hb and quality of life in non-TDT as well as decreasing transfusion requirements in TDT. Other novel noncurative treatments are entering clinical trials such improvement of erythropoiesis through pharmacological manipulation of hepcidin and iron metabolism.
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.