An abnormality in the cerebrospinal fluid before transplantation is predictive of poor outcomes after allotransplantation in AML patients

Abstract Central nervous system leukemia (CNSL) remains a serious complication in patients with AML and an ambiguous prognostic factor for those receiving allo-HSCT. Thus, we aimed to retrospectively analyse the clinical outcomes of 1472 AML patients with or without CNS abnormalities before transplantation. Abnormal cerebrospinal fluid (CSF) was detected via conventional cytology (CC) and MFC in 44 patients (CNS+) at any time after diagnosis. A control group of 175 CSF-normal (CNS-) patients was generated via propensity score matching (PSM) analyses according to sex, age at transplant, and white blood cell count at diagnosis. Compared to those in the CNS-negative group, the CC + and MFC + groups had comparable 8-year non-relapse mortality (NRM) (4%, 4% and 6%, P = .82), higher cumulative incidence of relapse (CIR) (14%, 31% and 32%, P = .007), lower leukemia-free survival (LFS) (79%, 63% and 64%, P = .024) and overall survival (OS) (83%, 63% and 68%, P = .021), with no significant differences between the CC + and MFC + groups. Furthermore, multivariate analysis confirmed that CSF involvement was an independent factor affecting OS and LFS. In conclusion, our results indicate that pretransplant CSF abnormalities is an adverse factor independently affecting OS and LFS after allotransplantation in AML patients.