ArticleAnnals of hematology2022
Busulfan-fludarabine- or treosulfan-fludarabine-based myeloablative conditioning for children with thalassemia major.
Article in Annals of hematology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 1 of them a synthesis that pooled it.
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Who cites it
14 citing papers in PubMed, 1 synthesis or guideline pooled it, 29 citations in OpenAlex.
- Conditioning regimen for hematopoietic cell transplant in transfusion dependent thalassemia: Recommendations from the EBMT practice harmonisation and guidelines committee.Bone marrow transplantation · 2026Guideline
- Treosulfan vs busulfan conditioning for allogeneic bmt in children with nonmalignant disease: a randomized phase 2 trial.Bone marrow transplantation · 2024Trial
- Alternative conditioning regimens for hemoglobinopathy gene therapy: balancing efficacy, toxicity, and the next frontier.Blood advances · 2026Review
- Fludarabine combined with treosulfan or total body irradiation as a conditioning therapy before allogeneic hematopoietic stem cell transplantation in acute lymphoblastic leukemia. A retrospective matched-pair analysis from the ALWP of the EBMT.Bone marrow transplantation · 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
- Iron toxicity and hematopoietic cell transplantation: do we understand why iron affects transplant outcome?Hematology. American Society of Hematology. Education Program · 2025Review
- Low rates of endothelial cell dysfunction and transplant-related mortality in 537 children receiving fludarabine-treosulfan conditioning for all transplant indications: A retrospective multicentre study on behalf of the UK Paediatric BMT group.British journal of haematology · 2025Article
- αß T-cell depleted haploidentical stem cell transplantation for pediatric and young adult patients with transfusion-dependent thalassemia.Bone marrow transplantation · 2025Article
- High Glucose Promotes the Ferroptosis and Dysfunction of Endothelial Cells by Downregulating SLC3A2 and Promoting the Development of Nephropathy.International journal of endocrinology · 2025Article
- Beta Thalassemia in Children: Established Approaches, Old Issues, New Non-Curative Therapies, and Perspectives on Healing.Journal of clinical medicine · 2024Review
- Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression.Bone marrow transplantation · 2024Article
- Excellent outcome of stem cell transplantation for sickle cell disease.Annals of hematology · 2023Article
- Treatment of sickle cell disease - options and perspective.American journal of blood research · 2023Review
Corrections and comments
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Authors and funding
22 authors at 16 institutions in 8 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Significant advances in supportive care for patients with transfusion-dependent thalassemia major (TDT) have improved patients' life expectancy. However, transfusion-associated iron overload remains a significant barrier to long-term survival with good quality of life. Today, allogeneic hematopoietic stem cell transplantation (HSCT) is the current curative standard of care. Alongside selection of the best available donor, an optimized conditioning regimen is crucial to maximize outcomes for patients with TDT undergoing HSCT. The aim of this retrospective analysis was to investigate the role of busulfan-fludarabine-based and treosulfan-fludarabine-based conditioning in TDT patients undergoing HSCT. We included 772 patients registered in the European Society for Blood and Marrow Transplantation (EBMT) database who underwent first HSCT between 2010 and 2018. Four hundred ten patients received busulfan-fludarabine-based conditioning (median age 8.6 years) and 362 patients received treosulfan-fludarabine-based conditioning (median age 5.7 years). Patient outcomes were retrospectively compared by conditioning regimen. Two-year overall survival was 92.7% (95% confidence interval: 89.3-95.1%) after busulfan-fludarabine-based conditioning and 94.7% (95% confidence interval: 91.7-96.6%) after treosulfan-fludarabine-based conditioning. There was a very low incidence of second HSCT overall. The main causes of death were infections, graft-versus-host disease, and rejection. In conclusion, use of busulfan or treosulfan as the backbone of myeloablative conditioning for patients with TDT undergoing HSCT resulted in comparably high cure rates. Long-term follow-up studies are warranted to address the important issues of organ toxicities and gonadal function.
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