Bone marrow transplantation · 2026
Validation of risk stratification and novel prognostic biomarkers in asian adult HSCT recipients with TA-TMA: A multicenter real-world study.
СтатьяМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
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Wu Yibo, Yuan Xiaolin, Chen Lieguang, Lai Xiaoyu, Liu Lizhen, Yang Luxin, Shi Jimin, Yu Jian, Zhao Yanmin, Ouyang Guifang, Ni Lihong, Xiao Haowen, Xu Yang, Tang Li, Zhu Panpan, Cai Zhen, Huang He, Lu Ying, Luo Yi
Аннотация
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.
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.