Real-world analysis of primary central nervous system diffuse large B-cell lymphoma

This study aimed to comprehensively analyze the clinical characteristics, therapeutic outcomes, and prognostic factors of primary central nervous system diffuse large B-cell lymphoma (PCNS-DLBCL) in a real-world setting.

A retrospective analysis was conducted on 143 newly diagnosed PCNS-DLBCL patients treated at the Department of Hematology, First Hospital of Jilin University between May 2011 and May 2024.

The cohort (median onset age: 58 years) demonstrated a 5-year overall survival (OS) rate of 37 %. Patients receiving triple-agent or more intensive induction regimens achieved significantly higher response rates, with the R-MT regimen (rituximab, methotrexate, thiotepa) showing favorable safety. For patients achieving ≥ partial response (PR) post-induction, consolidative whole-brain radiotherapy (WBRT) and/or autologous stem cell transplantation (ASCT), followed by maintenance therapy, significantly prolonged progression-free survival (PFS; P < 0.0001) and OS (P = 0.0097). Sequential maintenance therapy notably improved survival in MSKCC intermediate-high risk groups and dual-expressor subtypes. Relapsed/refractory PCNS-DLBCL patients receiving ≥ 2 lines of sequential maintenance therapy still exhibited OS benefits. BTK inhibitors and immunomodulatory agents were well-tolerated during maintenance.

PCNS-DLBCL, an aggressive malignancy with suboptimal prognosis, demonstrates promising short-term efficacy and safety with the R-MT induction regimen. Consolidative WBRT and/or ASCT remains recommended for induction responders. Sequential maintenance therapy should be prioritized for consolidation-ineligible patients to extend survival. Even in relapsed/refractory settings, post-chemotherapy consolidation and/or maintenance therapies confer OS advantages.