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December 8, 2025BloodOpen Access

Efficacy of thiotepa-containing conditioning regimens and mucositis burden in allogeneic HSCT for relapsed/refractory acute leukemia with extramedullary disease

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Authors

YYYixin YangJQJiang Qian

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Overview

Retrospective analysis shows enhanced survival in acute leukemia patients with extramedullary disease, suggesting promising efficacy of thiotepa-based conditioning regimens.

Key Points

  • This study aims to evaluate the efficacy and safety of thiotepa-containing conditioning regimens in patients with relapsed/refractory acute leukemia and extramedullary disease undergoing allogeneic HSCT.
  • Retrospective analysis of 101 patients with relapsed/refractory acute leukemia and extramedullary disease
  • Thiotepa regimen combined with other agents like cladribine, cytarabine, and granulocyte colony-stimulating factor
  • Assessment of primary endpoints like survival rates and complications such as mucositis and infections
  • 1-year overall survival rate of 53.1% and 2-year rate of 50.3% in the cohort
  • Mucositis occurred in 55.4% of patients, contributing to 12.9% of deaths
  • Cumulative incidence of relapse at 1-year was 33.3% and non-relapse mortality was 32.4%

Cite This Study

Yang et al. (2025) studied this question.

synapsesocial.com/papers/69362f4e4fa91c937236d87dhttps://doi.org/10.1182/blood-2025-4192
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Also Consider

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

  1. 1Addition of thiotepa to total body irradiation-based conditioning in allogeneic hematopoietic stem cell transplantation for relapsed/refractory acute leukemia and lymphoma: a propensity score-matched retrospective study2026
  2. 2Thiotepa 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
  3. 3Total marrow and lymphoid irradiation (2000 cGy) together with cyclophosphamide and etoposide prior to allogeneic hematopoietic cell transplantation is an effective regimen for Relapsed/Refractory leukemia, with low non-relapse mortality2025
  4. 4Salvage allogeneic hematopoietic stem cell transplantation conditioning regimens for relapsed/refractory acute leukemia: Efficacy of radiotherapy combined with CLAG vs FLAG2025
  5. 5Allogeneic hematopoietic stem cell transplantation for hematological malignancies using nonmyeloablative fludarabine, cyclophosphamide, 200 centigray total body irradiation, and post-transplant cyclophosphamide, sirolimus, and mycophenolate mofetil for graft-versus-host disease prophylaxis is associated with low non-relapse mortality.2025