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

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.