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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 shows thiotepa conditioning reduces cardiotoxicity in pediatric aplastic anemia, implying its safety for transplantation.

Key Points

  • All patients achieved successful engraftment, with a median time to neutrophil recovery of 11 days, highlighting the regimen's efficacy.
  • No moderate or severe cardiotoxicity occurred, with only mild pericardial effusion seen in five patients, indicating safety during treatment.
  • Implemented conditioning regimen featured thiotepa and reduced cyclophosphamide, providing a novel approach to pediatric transplantation.
  • Study supports thiotepa use for patients at risk of cardiotoxicity, with all participants alive and a 100% overall survival rate.

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. 3A novel reduced-toxicity conditioning regimen with busulfan/fludarabine/cyclophosphamide/anti-thymocyte globulin for severe aplastic anemia2025
  4. 4Expanding access in pediatrics: Outcomes of haploidentical transplantation with reduced intensity and peripheral blood stem cells2025
  5. 5Thiotepa and busulfan combined with cyclophosphamide conditioning regimen plus maintenance therapy improved the disease-free survival of patients with relapsed/refractory haematologic malignancies after undergoing allogeneic transplantation2025