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December 8, 2025BloodOpen Access

A novel thiotepa-containing conditioning regimen for haploidentical hematopoietic cell transplantation in pediatric aplastic anemia at high risk of cardiotoxicity

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Authors

FZFeng ZhangZXZhengli XuLXLanping Xu

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Overview

Prospective trial demonstrates a thiotepa regimen improves survival in pediatric aplastic anemia, suggesting cardiotoxicity reduction.

Key Points

  • To evaluate the safety and efficacy of a thiotepa-containing conditioning regimen in pediatric patients with aplastic anemia at high risk of cardiotoxicity.
  • Conducted a prospective, single-arm clinical trial
  • Enrolled 20 pediatric patients with ≥ 2 cyclophosphamide-related cardiotoxicity risk factors
  • Administered busulfan, thiotepa, cyclophosphamide, and rabbit anti-thymocyte globulin as conditioning regimen
  • 100% overall survival rate observed in 20 children after transplantation
  • All patients achieved hematopoietic engraftment within median time of 11 days
  • Mild pericardial effusion was noted in 5 patients, with no severe cardiotoxicity reported

Cite This Study

Zhang et al. (2025) studied this question.

synapsesocial.com/papers/69362f5a4fa91c937236db3bhttps://doi.org/10.1182/blood-2025-5977
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Also Consider

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

  1. 1The 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
  2. 2Combination of post-transplant cyclophosphamide and anti-thymocyte globulin for haploidentical stem cell transplantation in pediatric patients with acute leukemia2026
  3. 3Novel FABT-Based Conditioning Regimen with Haploidentical Transplantation for Severe Aplastic Anemia: A prospective, single-center, phase II clinical trial2026 · 4 citations
  4. 4A novel reduced-toxicity conditioning regimen with busulfan/fludarabine/cyclophosphamide/anti-thymocyte globulin for severe aplastic anemia2025
  5. 5Expanding access in pediatrics: Outcomes of haploidentical transplantation with reduced intensity and peripheral blood stem cells2025