ArticleBone marrow transplantation2016
Hematopoietic stem cell transplantation for homozygous β-thalassemia and β-thalassemia/hemoglobin E patients from haploidentical donors.
Article in Bone marrow transplantation, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 39 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.
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
39 citing papers in PubMed, 1 synthesis or guideline pooled it, 104 citations in OpenAlex.
- [Chinese guideline for diagnosis and treatment of transfusion dependent β-thalassemia (2022)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2022Guideline
- Effect of increased dose of total body irradiation on graft failure associated with HLA-haploidentical transplantation in patients with severe haemoglobinopathies: a prospective clinical trial.The Lancet. Haematology · 2019Trial
- Haploidentical haematopoietic stem cell transplantation for thalassaemia major based on an FBCA conditioning regimen.British journal of haematology · 2018Trial
- Germ Cell Dysfunction is Universal in Adolescent Male Patients with β-thalassemia Following Earlier Successful Hematopoietic Stem Cell TransplantationJournal of clinical research in pediatric endocrinology · 2025Article
- αß T-cell depleted haploidentical stem cell transplantation for pediatric and young adult patients with transfusion-dependent thalassemia.Bone marrow transplantation · 2025Article
- Conditioning Regimen Options.Advances in experimental medicine and biology · 2025Review
- Risk factors and prognosis of poor graft function after allogeneic hematopoietic stem cell transplantation in pediatric: a retrospective study.Frontiers in cell and developmental biology · 2025Article
- A novel conditioning regimen with pre-transplantation immunosuppression reduces the complication rates in hematopoietic stem cell transplantation in transfusion-dependent β-thalassemia.Stem cell research & therapy · 2024Article
- An update on recent studies of extracellular vesicles and their role in hypercoagulability in thalassemia (Review).Biomedical reports · 2024Review
- Nutritional and Body Composition Changes in Paediatric β-Thalassemia Patients Undergoing Hematopoietic Stem Cell Transplantation: A Retrospective Study Using Bioelectrical Impedance Analysis.Journal of multidisciplinary healthcare · 2024Article
- Health state utilities for beta-thalassemia: a time trade-off study.The European journal of health economics : HEPAC : health economics in prevention and care · 2023Article
- Review
- Busulfan-fludarabine- or treosulfan-fludarabine-based myeloablative conditioning for children with thalassemia major.Annals of hematology · 2022Article
- Conditioning Regimens in Patients with β-Thalassemia Who Underwent Hematopoietic Stem Cell Transplantation: A Scoping Review.Journal of clinical medicine · 2022Article
- The Incidence, Outcomes, and Risk Factors of Secondary Poor Graft Function in Haploidentical Hematopoietic Stem Cell Transplantation for Acquired Aplastic Anemia.Frontiers in immunology · 2022Article
- KIR in Allogeneic Hematopoietic Stem Cell Transplantation: Need for a Unified Paradigm for Donor Selection.Frontiers in immunology · 2022Review
- Advancing the care of β-thalassaemia patients with novel therapies.Blood transfusion = Trasfusione del sangue · 2022Review
- HSCT remains the only cure for patients with transfusion-dependent thalassemia until gene therapy strategies are proven to be safe.Bone marrow transplantation · 2021Review
- Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts.Translational pediatrics · 2021Article
- T-cell replete Haplo-identical HSCT with Post transplant Cyclophosphamide for Hemoglobinopathies: A retrospective analysis from a single center.Blood cell therapy · 2021Article
Corrections and comments
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Authors and funding
21 authors at 13 institutions in 3 countries.
Funding
Abstract
Thalassemia-free survival after allogeneic stem cell transplantation (SCT) is about 80-90% with either matched-related or -unrelated donors. We explored the use of a mismatched-related ('haplo- ') donor. All patients received two courses of pretransplant immunosuppressive therapy (PTIS) with fludarabine (Flu) and dexamethasone (Dxm). After two courses of PTIS, a conditioning regimen of rabbit antithymocyte globulin, Flu and IV busulfan (Bu) was given followed by T-cell-replete peripheral blood progenitor cells. GvHD prophylaxis consisted of cyclophosphamide (Cy) on days SCT +3 and +4 (post-Cy), and on day SCT +5 tacrolimus or sirolimus was started together with a short course of mycophenolate mofetil. Thirty-one patients underwent haplo-SCT. Their median age was 10 years (range, 2-20 years). Twenty-nine patients engrafted with 100% donor chimerism. Two patients suffered primary graft failure. Median time to neutrophil engraftment was 14 days (range, 11-18 days). Five patients developed mild to moderate, reversible veno-occlusive disease, while nine patients developed acute GvHD grade II. Only five patients developed limited-chronic GvHD. Projected overall and event-free survival rates at 2 years are 95% and 94%, respectively. The median follow up time is 12 months (range, 7-33 months).
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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.