Retrospective study on allogeneic hematopoietic stem cell transplantation for the treatment of Hemophagocytic lymphohistiocytosis.
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OBJECTIVE: Hemophagocytic lymphohistiocytosis (HLH) is an immune dysregulation syndrome with an extremely poor prognosis. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only potentially curative treatment for high-risk HLH. This study aimed to evaluate the efficacy, safety, prognostic factors, and bridging value of novel targeted therapies in adolescent and adult patients with HLH. METHODS: We retrospectively analyzed the clinical data of 35 adolescents and adults with HLH who underwent individualized myeloablative conditioning (MAC)-based allo-HSCT between April 2019 and November 2025. RESULTS: Sustained hematopoietic engraftment was achieved in 34 of 35 patients (97.1%). With a median follow-up of 17 months, the estimated 1- and 2-year overall survival (OS) rates were 78.2% and 74.1%, respectively, and the 1- and 2-year relapse-free survival (RFS) rates were 73.0% and 69.0%, respectively. Notably, patients in an inactive disease state (complete or partial remission) prior to transplantation demonstrated a significantly superior 1-year RFS rate compared with those in an active state (86.3% vs. 48.6%, P = 0.022). Furthermore, Cox regression analysis identified elevated pre-transplant serum creatinine as a significant adverse prognostic factor for OS (HR = 1.02, P = 0.004). CONCLUSION: Allo-HSCT achieved high engraftment rates and acceptable a safety profile across different HLH subtypes. Achieving at least partial remission (in an inactive state) before transplantation significantly improves survival. For critically ill patients with an extremely high inflammatory burden, emapalumab serves as an effective salvage strategy for bridging transplantation. Furthermore, the cellular tracing of EBV-R using advanced sorting technologies is of great clinical value for differentiating simple viremia from early tumor relapse.