Cerebrospinal fluid profile associated with the incidence and outcomes of patients with chronic graft-versus-host disease after haploidentical hematopoietic stem cell transplantation.

Chronic graft-versus-host disease (cGVHD) is a major complication of haploidentical hematopoietic stem cell transplantation (haplo-HSCT). In our previous studies, we speculated that cGVHD might be closely related to immune-mediated encephalopathy lesions; however, its involvement in the central nervous system (CNS) is exceedingly rare. We performed a retrospective nested case-control study of patients who underwent haplo-HSCT between January 2013 and June 2022 at our institution. In total, 87 patients with cGVHD who had their cerebrospinal fluid (CSF) tested were enrolled, of whom 43 showed evidence of CNS-cGVHD. In these patients, acute GVHD, CSF proteins, and oligoclonal bands were identified as risk factors for cGVHD onset. We combined these variables to establish a nomogram for the cumulative incidence of cGVHD. We found no statistically significant differences in overall survival, leukemia-free survival, or cumulative incidence of relapse between the cGVHD and control groups. However, a significant difference in non-relapse mortality (NRM) was observed. Multivariate analysis of 43 patients diagnosed with CNS-cGVHD showed that underlying diseases and CSF proteins were independently associated with CNS-cGVHD occurrence. In conclusion, the CSF profile may be associated with the onset of cGVHD and higher NRM in cGVHD following haplo-HSCT.