ArticleBone marrow transplantation2025
αß T-cell depleted haploidentical stem cell transplantation for pediatric and young adult patients with transfusion-dependent thalassemia.
Article in Bone marrow transplantation, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
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Who cites it
5 citing papers in PubMed.
- Alternative conditioning regimens for hemoglobinopathy gene therapy: balancing efficacy, toxicity, and the next frontier.Blood advances · 2026Review
- Bringing the rare into sight: applications of haploidentical stem cell transplantation in the treatment of rare diseases.Bone marrow transplantation · 2026Review
- Roxadustat induced fetal hemoglobin in anemic CKD patients: a dual-center real-world observational cohort study.BMC nephrology · 2026Observational
- Curative Approach to the Treatment of Beta-Thalassemia and Sickle Cell Disease with Hematopoietic Stem Cell Transplantation.Journal of clinical medicine · 2026Review
- Preclinical efficacy and safety evaluation of BD211 autologous CD34Frontiers in cell and developmental biology · 2025Article
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Authors and funding
16 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Life expectancy of patients with severe transfusion-dependent beta-thalassemia (TDT) remains below that of the general population. Allogenic hematopoietic stem cell transplantation (HSCT) is the standard curative treatment. Due to the paucity of matched donor (MD) availability, haploidentical HSCT (haplo-HSCT) is a reasonable alternative. Twenty patients with TDT (median age 10 years; range 2-23) received either a matched sibling donor (MSD; n = 7) or a haplo-HSCT (n = 13) in a single center (Regensburg, Germany) between 2016 and 2022, including two patients referred for a haplo-HSCT as rescue failing prior MD- and haplo-HSCT, respectively. The conditioning regimen consisted of anti-thymocyte globulin (ATG; Grafalon®), treosulfan, thiotepa, and fludarabine (FTT). Immunosuppression consisted of a calcineurin inhibitor and mycophenolate mofetil (MMF). At a median follow-up of 37 months (range 6-90), overall survival (OS) was 100% with a disease-free survival (DFS) of 100% in MSD and 92% in haplo-HSCT, respectively. Two patients in haplo-HSCT experienced graft failure, one achieving DFS after a second haplo-HSCT. No acute graft-versus-host disease (aGvHD) ≥ °III or severe chronic GvHD (cGvHD) were observed. No sinusoidal obstruction syndrome (SOS) was observed in this high-risk population. Treosulfan-based T-cell depleted haplo-HSCT can achieve comparable OS and DFS even in young adult TDT patients with no SOS/VOD.
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