ArticleBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2014
Outcomes of thalassemia patients undergoing hematopoietic stem cell transplantation by using a standard myeloablative versus a novel reduced-toxicity conditioning regimen according to a new risk stratification.
Article in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 1 of them a synthesis that pooled it.
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Who cites it
28 citing papers in PubMed, 1 synthesis or guideline pooled it, 55 citations in OpenAlex.
- Diagnosis and Management of Transfusion-Dependent Thalassemia: Evidence-Based Guidelines From the Pediatric Hematology Oncology Chapter of the Indian Academy of Pediatrics.Indian pediatrics · 2026Guideline
- Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2018Trial
- Bringing the rare into sight: applications of haploidentical stem cell transplantation in the treatment of rare diseases.Bone marrow transplantation · 2026Review
- [Guideline for allogeneic hematopoietic stem cell transplantation in the treatment of transfusion-dependent thalassemia in China (2026)].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2026Article
- Outcomes of hematopoietic stem cell transplantation for pediatric patients with transfusion-dependent thalassemia in Thailand.Clinical and experimental pediatrics · 2026Article
- Curative Approach to the Treatment of Beta-Thalassemia and Sickle Cell Disease with Hematopoietic Stem Cell Transplantation.Journal of clinical medicine · 2026Review
- Abatacept improves posttransplant survival and reduces endothelial injury syndromes in β-thalassemia major.Blood advances · 2025Article
- Gene therapy in transfusion-dependent non-β0/β0 genotype β-thalassemia: first real-world experience of beti-cel.Blood advances · 2025Article
- Symptoms of depression and anxiety in patients with transfusion-dependent thalassemia after hematopoietic stem cell transplantation: a single-center study.Therapeutic advances in hematology · 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
- Article
- Differences in longitudinal growth patterns of children and adolescents with transfusion-dependent hemoglobin E/β-thalassemia and those achieving successful hematopoietic stem-cell transplantation.International journal of hematology · 2022Article
- 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
- Advances in the management of α-thalassemia major: reasons to be optimistic.Hematology. American Society of Hematology. Education Program · 2021Review
- Chronic graft-versus-host disease in children and adolescents with thalassemia after hematopoietic stem cell transplantation.International journal of hematology · 2021Article
- In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- No differences in hemostatic and endothelial activations between haploidentical and matched-donor hematopoietic stem cell transplantation in thalassemia disease.Thrombosis journal · 2020Article
- [Effect of cyclophosphamide on hematopoietic stem cells in mice with iron overload].Nan fang yi ke da xue xue bao = Journal of Southern Medical University · 2020Article
- Complication free survival long-term after hemopoietic cell transplantation in thalassemia.Haematologica · 2018Article
Corrections and comments
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Authors and funding
17 authors at 9 institutions in 2 countries.
Funding
Abstract
Improving outcomes among class 3 thalassemia patients receiving allogeneic hematopoietic stem cell transplantations (HSCT) remains a challenge. Before HSCT, patients who were ≥ 7 years old and had a liver size ≥ 5 cm constitute what the Center for International Blood and Marrow Transplant Research defined as a very high-risk subset of a conventional high-risk class 3 group (here referred to as class 3 HR). We performed HSCT in 98 patients with related and unrelated donor stem cells. Seventy-six of the patients with age < 10 years received the more conventional myeloablative conditioning (MAC) regimen (cyclophosphamide, busulfan, ± fludarabine); the remaining 22 patients with age ≥ 10 years and hepatomegaly (class 3 HR), and in several instances additional comorbidity problems, underwent HSCT with a novel reduced-toxicity conditioning (RTC) regimen (fludarabine and busulfan). We then compared the outcomes between these 2 groups (MAC versus RTC). Event-free survival (86% versus 90%) and overall survival (95% versus 90%) were not significantly different between the respective groups; however, there was a higher incidence of serious treatment-related complications in the MAC group, and although we experienced 6 graft failures in the MAC group (8%), there were none in the RTC group. Based on these results, we suggest that (1) class 3 HR thalassemia patients can safely receive HSCT with our novel RTC regimen and achieve the same excellent outcome as low/standard-risk thalassemia patients who received the standard MAC regimen, and further, (2) that this novel RTC approach should be tested in the low/standard-risk patient population.
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Registered trials
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