ReviewBone marrow transplantation2021
HSCT remains the only cure for patients with transfusion-dependent thalassemia until gene therapy strategies are proven to be safe.
Review in Bone marrow transplantation, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 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.
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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
10 citing papers in PubMed, 23 citations in OpenAlex.
- Neuroimaging in the diagnosis and treatment of cerebral toxoplasmosis in children with severe β-thalassemia after allo-HSCT.Biomolecules & biomedicine · 2025Review
- CRISPR/Cas-edited iPSCs and mesenchymal stem cells: a concise review of their potential in thalassemia therapy.Frontiers in cell and developmental biology · 2025Review
- Role of gut microbiota in thalassemia: a review of therapeutic prospects.Frontiers in physiology · 2025Review
- Article
- Treosulfan Exposure Predicts Thalassemia-Free Survival in Patients with Beta Thalassemia Major Undergoing Allogeneic Hematopoietic Cell Transplantation.Clinical pharmacology and therapeutics · 2024Article
- CRISPR/Cas9 Ablated BCL11A Unveils the Genes with Possible Role of Globin Switching.Advanced pharmaceutical bulletin · 2023Article
- Decision making on HSCT in patients with hemoglobinopathies; an EBMT Pediatric Diseases Working Party and Inborn Errors Working Party scenario-based survey on physicians' perspectives.Bone marrow transplantation · 2023Article
- Case report: Curing a rare, unstable hemoglobin variant Hb Bristol-Alesha using haploidentical hematopoietic stem cell transplantation.Frontiers in immunology · 2023Article
- Conditioning Regimens in Patients with β-Thalassemia Who Underwent Hematopoietic Stem Cell Transplantation: A Scoping Review.Journal of clinical medicine · 2022Article
- Advancing the care of β-thalassaemia patients with novel therapies.Blood transfusion = Trasfusione del sangue · 2022Review
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Patients with β-thalassemia suffer from severe anemia, iron overload and multiple complications, that affect their quality of life and well-being. Allogeneic hematopoietic stem cell transplantation (HSCT) from an HLA-matched sibling donor, performed in childhood, has been the gold standard for thalassemic patients for decades. Unfortunately, siblings are available only for the minority of patients. Fully matched unrelated donors have been the second choice for cure, with equal results as far as overall survival is concerned, having though the cost of frequent and serious complications. On the other hand, haploidentical transplantation is performed more frequently during the last decade, with promising results. Gene therapy represents a novel therapeutic approach, with impressive results from clinical trials, both from gene addition strategies, as well as from the emerging gene editing tools. After reviewing current critical points of HSCT using alternative donors and assessing recently reported safety issues of gene therapy methods, we conclude that, although a breakthrough, the safety of gene therapy remains to be established.
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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.