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Use of bone marrow cells is associated with improved outcomes when compared to peripheral blood stem cell after haplo-identical transplants with post transplant cyclophosphamide, a study from of the CTIWP-EBMT

Abstract

Few studies analyzed the impact of bone marrow (BM) or peripheral blood stem cells (PBSC) on outcomes after haploidentical transplantation using post-cyclophosphamide (Haplo-PTCY). We analyzed 8854 adults with malignant disorders, given a first Haplo-PTCY. BM cells was used in 2914 and PBSC in 5940 patients. Multivariate models were built adjusting for the statistical differences between the 2 groups. Median follow-up time for survivors in the BM and PBSC groups were 48 and 30 months, respectively. Neutrophil Engraftment was observed in 92.4% of BM and 93.7% of PBSC recipients (p = 0.01). In a multivariate analysis, use of PBSC compared to BM, was associated with higher incidence of acute (HR:1.53; p < 0.0001) and chronic GVHD (HR:1.34; p < 10-3), increased non-relapse mortality (HR:1.22; p = 0.002), similar risk of relapse (HR:1.02; p = 0.79), and decreased overall survival (OS)(HR:1.13; p = 0.008); progression-free survival (PFS)(HR:1.11; p = 0.024) and GVHD-Relapse free survival (GRFS) (HR:1.2; p < 10–3). In conclusion, use of BM cells is associated with better outcomes compared to PBSC after Haplo-PTCY. Future studies should investigate better GVHD prophylaxis in the PBSC-Haplo-PTCY setting and the association of measured T-cell or other subpopulations of lymphocyte content in the PBSC graft.

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Fig. 1: Main outcomes by graft source.

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Data availability

Data cannot be shared unless a specific request is sent to the EBMT. This was a multicenter retrospective study, conducted on behalf of the Cellular therapy and Immunobiology Working Party (CTIWP) of EBMT.

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VR and AR designed the study, VR and AR wrote the manuscript; ML performed the statistical analysis. JEG has reviewed the statistical analysis. AMR, RD, SB, FC, JLDM, YK, JT, SS, MM, ZG, AK, LLC, AS provided cases for the study. All authors edited and approved the manuscript. VR: honoraria of Takeda, Janssen, Amgen, Gilead Kite, Sanofi, Roche, Pierre Fabre. AS: Honoraria: Takeda, BMS/Celgene, MSD, Janssen, Amgen, Novartis, Gilead Kite, Sanofi, Roche, Alexion, Pierre Fabre, Jazz Pharmaceuticals, Verte. Consultancy: Takeda, BMS/Celgene, Novartis, Janssen, Gilead, Sanofi, GenMab, Abbvie, MSD, Incyte, Autolus, Regeneron, Pierre Fabre. Speaker’s Bureau: Takeda. Research Support: Takeda. Non-profit organizations: Presidency of the GETH-TC, Presidency of the EBMT. The remaining authors have no conflict of interest to disclose.

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Correspondence to Annalisa Ruggeri.

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The authors declare no competing interests.

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Data were collected according to EBMT rules. All patients or their legal guardians provided written informed consent for data collection and analysis in accordance with the Declaration of Helsinki, and with the centers’ ethical research guidelines. The scientific board of the CTIWP of the EBMT approved this study.

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Rocha, V., Labopin, M., Raiola, A.M. et al. Use of bone marrow cells is associated with improved outcomes when compared to peripheral blood stem cell after haplo-identical transplants with post transplant cyclophosphamide, a study from of the CTIWP-EBMT. Bone Marrow Transplant 61, 75–81 (2026). https://doi.org/10.1038/s41409-025-02735-7

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