Real-world analysis of autologous stem cell transplantation in primary central nervous system lymphoma using Taiwan Blood and Marrow Transplantation Registry.

BACKGROUND

Purpose: High-dose chemotherapy followed by autologous stem cell transplantation (HDC/ASCT) is an established consolidation for primary central nervous system lymphoma (PCNSL). However, real-world data in Asian populations remain limited. This study aimed to evaluate the clinical outcomes of PCNSL patients undergoing HDC/ASCT in Taiwan.

METHODS

We retrospectively analyzed 65 PCNSL patients from the Taiwan Blood and Marrow Transplantation Registry (TBMTR) who received HDC/ASCT between 2012 and 2022. Patients were categorized into frontline (n = 47) and salvage (n = 18) groups. Survival outcomes and the impact of thiotepa-based conditioning regimens were assessed.

RESULTS

The 2-year overall survival (OS) and relapse-free survival (RFS) rates were 67.4% and 56.5% for the frontline group, and 76.0% and 61.1% for the salvage group, respectively. Non-thiotepa-based regimens demonstrated comparable survival outcomes; the 2-year OS was 64.3% for thiotepa recipients versus 72.2% for non-thiotepa recipients (p = 0.64). Disease progression was the leading cause of mortality, while non-relapse mortality was infrequent.

CONCLUSION

HDC/ASCT is an effective consolidation strategy improving OS and RFS for PCNSL in both frontline and salvage settings within the Taiwanese population. Non-thiotepa conditioning regimens demonstrated comparable efficacy to thiotepa-based conditioning in this cohort, supporting their feasibility as an institutional alternative in settings with limited thiotepa access.