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<p>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, <i>p</i> = 0.027), multi-organ dysfunction syndrome (MODS; HR = 0.06, <i>p</i> &lt; 0.001), and elevated IL-10 (HR = 0.24, <i>p</i> = 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.</p>

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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

  • 期刊论文

  • 发表日期：

    2026年1月9日

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  • Yibo Wu,
  • Xiaolin Yuan,
  • Lieguang Chen,
  • Xiaoyu Lai,
  • Lizhen Liu,
  • Luxin Yang,
  • Jimin Shi,
  • Jian Yu,
  • Yanmin Zhao,
  • Guifang Ouyang,
  • Lihong Ni,
  • Haowen Xiao,
  • Yang Xu,
  • Li Tang,
  • Panpan Zhu,
  • Zhen Cai,
  • He Huang,
  • Ying Lu,
  • Yi Luo

摘要

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.