Addition 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 study.
- 1Department of Hematology, Aerospace Center Hospital, Beijing, 100049, China.
- 2Department of Hematology, Aerospace Center Hospital, Beijing, 100049, China. [email protected].
Abstract
Background
Relapsed/refractory (R/R) acute leukemia (AL) carries a poor prognosis, with allogeneic hematopoietic stem cell transplantation (allo-HSCT) the primary curative option. Total body irradiation (TBI)-based conditioning is a cornerstone, but optimizing intensity remains challenging. Thiotepa, a bifunctional alkylating agent, may enhance disease control when added to TBI, but data in R/R AL are lacking.Methods
This single-center retrospective study (2020-2025) included R/R AL and lymphoma patients receiving TBI-based conditioning with (TBI + TT) or without (TBI) thiotepa. Propensity score matching (1:2, caliper = 0.2) used 9 covariates. Primary endpoints were overall survival (OS) and disease-free survival (DFS). Secondary endpoints included relapse, non-relapse mortality (NRM), engraftment, GVHD, CMV infection, and mucositis. Kaplan-Meier, Fine-Gray competing risk, and inverse probability of treatment weighting (IPTW) analyses were employed.Results
After matching, 48 TBI + TT and 86 TBI patients were analyzed. OS (HR = 0.901, 95% CI 0.569-1.425, P = 0.655) and DFS (HR = 0.809, 95% CI 0.504-1.299, P = 0.380) were comparable. The 1-year relapse cumulative incidence was lower with TBI + TT (6.7% vs. 17.8%, Gray's P = 0.035; multivariable sHR = 0.280, 95% CI 0.083-0.942, P = 0.040), though hypothesis-generating given only 3 relapse events. NRM was comparable (Gray's P = 0.374). Mucositis was significantly higher with TBI + TT (81.2% vs. 64.0%, P = 0.049). IPTW confirmed comparable OS (P = 0.690) and DFS (P = 0.975).Conclusion
Adding thiotepa to TBI-based conditioning for R/R AL is associated with lower relapse incidence without compromising OS, DFS, engraftment, or GVHD, at the cost of increased mucosal toxicity. The relapse finding requires cautious interpretation and prospective validation.
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BibTeX
@article{li2026addition,
title = {{Addition 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 study.}},
author = {Minglu Li and Shuqin Zhang and Xiaoyong Man and Hongxia Wen and Wei Wei and Yixin Yang and Jingbo Wang},
journal = {Annals of hematology},
year = {2026},
month = jul,
doi = {10.1007/s00277-026-07198-4},
url = {https://europepmc.org/article/MED/42897391},
note = {CC BY-NC-ND},
}
RIS
TY - JOUR
TI - Addition 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 study.
AU - Minglu Li
AU - Shuqin Zhang
AU - Xiaoyong Man
AU - Hongxia Wen
AU - Wei Wei
AU - Yixin Yang
AU - Jingbo Wang
PY - 2026
DA - 2026/07/01
JO - Annals of hematology
DO - 10.1007/s00277-026-07198-4
UR - https://europepmc.org/article/MED/42897391
AB - Background Relapsed/refractory (R/R) acute leukemia (AL) carries a poor prognosis, with allogeneic hematopoietic stem cell transplantation (allo-HSCT) the primary curative option. Total body irradiation (TBI)-based conditioning is a cornerstone, but optimizing intensity remains challenging. Thiotepa, a bifunctional alkylating agent, may enhance disease control when added to TBI, but data in R/R AL are lacking.Methods This single-center retrospective study (2020-2025) included R/R AL and lymphoma patients receiving TBI-based conditioning with (TBI + TT) or without (TBI) thiotepa. Propensity score matching (1:2, caliper = 0.2) used 9 covariates. Primary endpoints were overall survival (OS) and disease-free survival (DFS). Secondary endpoints included relapse, non-relapse mortality (NRM), engraftment, GVHD, CMV infection, and mucositis. Kaplan-Meier, Fine-Gray competing risk, and inverse probability of treatment weighting (IPTW) analyses were employed.Results After matching, 48 TBI + TT and 86 TBI patients were analyzed. OS (HR = 0.901, 95% CI 0.569-1.425, P = 0.655) and DFS (HR = 0.809, 95% CI 0.504-1.299, P = 0.380) were comparable. The 1-year relapse cumulative incidence was lower with TBI + TT (6.7% vs. 17.8%, Gray's P = 0.035; multivariable sHR = 0.280, 95% CI 0.083-0.942, P = 0.040), though hypothesis-generating given only 3 relapse events. NRM was comparable (Gray's P = 0.374). Mucositis was significantly higher with TBI + TT (81.2% vs. 64.0%, P = 0.049). IPTW confirmed comparable OS (P = 0.690) and DFS (P = 0.975).Conclusion Adding thiotepa to TBI-based conditioning for R/R AL is associated with lower relapse incidence without compromising OS, DFS, engraftment, or GVHD, at the cost of increased mucosal toxicity. The relapse finding requires cautious interpretation and prospective validation.
SN - 0939-5555
SN - 1432-0584
ER -