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Validation of risk stratification and novel prognostic biomarkers in asian adult HSCT recipients with TA-TMA: A multicenter real-world study

Bone Marrow Transplantation (2026)Cite this article

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Abstract

Background: Transplantation-associated thrombotic microangiopathy (TA-TMA) confers high mortality after allogeneic hematopoietic stem cell transplantation (HSCT). While characteristics are established in pediatric populations, real-world data on adults—particularly regarding standardized diagnostics and risk stratification—remain scarce. Methods: This multicenter, retrospective cohort study analyzed 113 Asian adult patients diagnosed per international consensus criteria (median onset: 60 days post-HSCT). Outcomes were assessed using Jodele and harmonized risk tools, treatment responses, and biomarker profiles (cytokines, aGVHD, proteinuria, sC5b-9, D-dimer). Results: The 6-month post-TMA and 1-year post-HSCT survival rates were 30.5% and 31.6%, respectively. High-risk classification by Jodele criteria (33.6% of patients) predicted significantly inferior 6-month survival (10.5% vs. 38.3%, p = 0.001). All patients met harmonized high-risk criteria. Immune reconstitution was severely impaired, especially in higher Jodele risk categories. Proteinuria (HR = 3.55, p = 0.027), multi-organ dysfunction syndrome (MODS; HR = 0.06, p < 0.001), and elevated IL-10 (HR = 0.24, p = 0.025) were independent predictors of reduced survival. Eculizumab yielded higher response rates than plasma exchange (57.1% vs. 31.3%). Conclusion: This study validates harmonized diagnostic criteria and Jodele risk stratification in Asian adults with TA-TMA, and identifies proteinuria, MODS, and IL-10 as prognostic biomarkers. The universal high-risk classification underscores disease severity in this population. Early complement inhibition and cytokine-targeted therapies merit further investigation.

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Fig. 1: The overall survival of TA-TMA adult HSCT patients.
Fig. 2: The immune reconstitution of TA-TMA adult HSCT patients.
Fig. 3: The plasma cytokines of TA-TMA adult HSCT patients.
Fig. 4: The risk factors associated with poor survival of TA-TMA adult HSCT patients.

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Data availability

The data that support the findings of this study are available from the corresponding author on reasonable request.

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Acknowledgements

We extend our gratitude to the Zhejiang Province Bone Marrow Transplant Collaboration Group for their coordination and data sharing, which made this multicenter retrospective study possible. We also thank all the patients whose data were included in this research.

Funding

This work was supported by grants from the National Natural Science Foundation of China (82300240 and 82170205) and the Natural Science Foundation of Zhejiang Province (LMS25H080005).

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Author notes

  1. These authors contributed equally: Yibo Wu, Xiaolin Yuan, Lieguang Chen.

  2. These authors jointly supervised this work: He Huang, Ying Lu, Yi Luo

Authors and Affiliations

  1. Bone Marrow Transplantation Center of the First Affiliated Hospital & Liangzhu Laboratory, Zhejiang University School of Medicine, Hangzhou, China

    Yibo Wu, Lieguang Chen, Xiaoyu Lai, Lizhen Liu, Luxin Yang, Jimin Shi, Jian Yu, Yanmin Zhao, Panpan Zhu, Zhen Cai, He Huang & Yi Luo

  2. Institute of Hematology, Zhejiang University, Hangzhou, China

    Yibo Wu, Xiaoyu Lai, Lizhen Liu, Luxin Yang, Jimin Shi, Jian Yu, Yanmin Zhao, Panpan Zhu, Zhen Cai, He Huang & Yi Luo

  3. Zhejiang Province Engineering Research Center for Stem Cell and Immunity Therapy, Hangzhou, China

    Yibo Wu, Xiaoyu Lai, Lizhen Liu, Luxin Yang, Jimin Shi, Jian Yu, Yanmin Zhao, Panpan Zhu, Zhen Cai, He Huang & Yi Luo

  4. Department of Hematology, Zhejiang Cancer Hospital, Hangzhou Institute of Medicine (HIM), Chinese Academy of Sciences, Hangzhou, Zhejiang, China

    Xiaolin Yuan

  5. Department of Hematology, The First Affiliated Hospital of Ningbo University, Ningbo, China

    Lieguang Chen & Guifang Ouyang

  6. Department of Hematology, Affiliated Jinhua Hospital, Zhejiang University School of Medicine, Jinhua, China

    Lihong Ni

  7. Department of Hematology and Cell Therapy, Sir Run Run Shaw Hospital, Zhejiang University School of Medicine, Hangzhou, China

    Haowen Xiao

  8. Department of Hematology, The Second Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China

    Yang Xu

  9. Department of Hematology, Luoyang Central Hospital Affiliated to Zhengzhou University, Luoyang, China

    Li Tang

  10. Department of Hematology, The Affiliated Peoples’ Hospital of Ningbo University, Ningbo, China

    Ying Lu

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Contributions

Y.W. designed the study and wrote the paper. XL.Y. data collecting, analyzing data, revising paper. L.C., X.L., L.L. recruiting patients, data collecting. P.Z. analyzing data, format figures. L.Y., J.S., J.Y., Y.Z., G.O., L.N., H.X., Y.X., L.T. recruiting patients, data collecting. Z.C., H.H., YING.L., Y.L. funding acquisition, project administration, review, and validation.

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Correspondence to Yi Luo.

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Wu, Y., Yuan, X., Chen, L. et al. Validation of risk stratification and novel prognostic biomarkers in asian adult HSCT recipients with TA-TMA: A multicenter real-world study. Bone Marrow Transplant (2026). https://doi.org/10.1038/s41409-025-02777-x

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