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July 26, 2026Pediatric Blood & Cancer

Fludarabine, Busulfan, Thiotepa, and Melphalan: A Novel Low‐Toxicity Myeloablative Conditioning Regimen for Haploidentical HSCT in Children and Adolescents Aged ≥7 Years With Transfusion‐Dependent β‐Thalassemia

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Authors

GLGuiping LiaoDLDongmei LiuBYBeibei Yang

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Overview

Prospective trial demonstrates improved transplant outcomes in children with transfusion-dependent thalassemia, suggesting enhanced safety and efficacy.

Key Points

  • This research evaluates a new low-toxicity myeloablative conditioning regimen for haploidentical HSCT in children with transfusion-dependent thalassemia.
  • Prospective, single-center clinical trial (ChiCTR2300071890)
  • Evaluated conditioning regimen: fludarabine, busulfan, thiotepa, and melphalan in patients aged ≥7 years
  • Included GVHD prophylaxis with half-dose PTCY and in vivo T-cell depletion.
  • The 2-year thalassemia-free survival and overall survival rates were both 93.1%.
  • Neutrophil and platelet engraftment were achieved in all patients, with no graft failures reported.
  • Grade II-IV acute GVHD occurred in 17.2% of patients, chronic GVHD in 6.9%, and manageable regimen-related toxicities were observed.

Cite This Study

Liao et al. (2026) studied this question.

synapsesocial.com/papers/6a65a2e2d3aea3239cd763c1https://doi.org/10.1002/1545-5017.70563
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1A novel reduce toxity myeloablative conditioning regimen f-BMT improves efficacy and safety of allogeneic hematopoietic stem cell transplantation in patients with transfusion-dependent thalassemia: A multicenter clinical trial2025 · 1 citations
  2. 2The efficacy and safety of using low-dose cyclophosphamide, fludarabine and thymoglobulin in allogeneic hematopoietic stem cell transplantation for transfusion-dependent non-severe aplastic anemia：a retrospective study.2025
  3. 3Haploidentical haematopoietic stem cell transplantation for thalassaemia major: A phase IV , open‐label, single‐centre trial2026
  4. 4Analysis of efficacy and prognostic factors in transfusion-dependent thalassemia following allogeneic hematopoietic stem cell transplantation using the ‘gx-07-tm’ regimen2025
  5. 5Prospective Study of Targeted Busulfan–Fludarabine Conditioning for Hematopoietic Stem Cell Transplantation in Genetic Rare Diseases2026