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Outcomes of allogeneic hematopoietic stem cell transplantation versus intensive chemotherapy in patients with myeloid sarcoma: a nationwide representative multicenter study

Abstract

Myeloid sarcoma (MS) is a rare hematological neoplasm with poor prognosis, posing a significant clinical challenge due to the absence of effective and standardized treatments. We conducted a retrospective analysis of 162 MS patients treated at 12 centers to compare outcomes between intensive chemotherapy and allogeneic hematopoietic stem cell transplantation (allo-HSCT). Our analysis revealed that allo-HSCT demonstrated superior overall survival (OS) within the initial 36 months compared to intensive chemotherapy alone (p = 0.037). However, beyond 36 months (36–60 months), a reverse trend was observed (p = 0.056). Subgroup analysis revealed potential benefit for isolated MS patients with allo-HSCT, but not for those with leukemic MS. Additionally, in patients achieving first complete remission (CR1) after induction chemotherapy, allo-HSCT did not significantly improve 5-year OS compared with intensive chemotherapy alone (p = 0.25). Conversely, allo-HSCT significantly improved 5-year OS in non-CR1 patients (p < 0.001). Notably, HLA-matched HSCT and haploidentical HSCT showed comparable outcomes in terms of OS, disease-free survival, and cumulative incidence of relapse. In conclusion, allo-HSCT improved outcomes for MS patients within 36 months of disease onset, and haploidentical HSCT emerged as a viable treatment option for patients without matched donors.

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Fig. 1: Overall survival of MS patients comparing intensive chemotherapy and allo-HSCT groups.
Fig. 2: Overall survival of patients according to MS subtype in intensive chemotherapy and allo-HSCT groups.
Fig. 3: Overall survival of MS patients according to disease status after induction chemotherapy in intensive chemotherapy and allo-HSCT groups.
Fig. 4: Survival of MS patients comparing HLA-matched HSCT and Haplo-HSCT.
Fig. 5: Cumulative incidence of events in MS patients comparing HLA-matched HSCT and Haplo-HSCT.

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Data availability

The data that support the findings of this study are available on request from the corresponding author, Xiao-Hui Zhang (zhangxh@bjmu.edu.cn). The data are not publicly available due to their containing information that could compromise the privacy of research participants.

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Acknowledgements

We thank all patients and the data collection team who participated in this study.

Funding

This work was supported by the National Key Research and Development Program of China (No. 2023YFC2507803), the Key Program of National Natural Science Foundation of China (No. 82230004) and the Beijing Natural Science Foundation (No. 7242154).

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Contributions

XHZ, JS and YCZ contributed to the study conception and design. JS, JW, YZ, AQW, YZ, FQL, CL, JHQ, JW, RX, QG, XPL and LLS collected the data. YJX, YHL, LF, ZXJ, MJ, PCH, XBH, SJG, SBW and SJF reviewed the cases and provided the data. JS and YCZ analyzed the data and wrote the original manuscript. LPX, XJH and XHZ reviewed the structure and statistical section of the paper. All the authors critically reviewed and approved the final version of the manuscript.

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Correspondence to Xiao-Hui Zhang.

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Sun, J., Zhang, YC., Wei, J. et al. Outcomes of allogeneic hematopoietic stem cell transplantation versus intensive chemotherapy in patients with myeloid sarcoma: a nationwide representative multicenter study. Bone Marrow Transplant 60, 319–325 (2025). https://doi.org/10.1038/s41409-024-02485-y

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