Outcomes in patients with refractory/relapsed CNS lymphoma treated in complete remission: autologous transplantation vs. CAR-T therapy.

[BACKGROUND] Autologous hematopoietic cell transplantation (ASCT) is a reasonable consolidation therapy for eligible patients with chemosensitive relapsed central nervous system lymphoma (CNSL) who have achieved complete remission (CR) and maintained the CR. Chimeric antigen receptor T-cell (CAR-T) therapy is an effective treatment option for patients with relapsed CNSL, although evidence on outcomes in patients who achieve CR is limited.

[AIM] To compare the efficacy of ASCT versus CAR-T therapy as consolidation therapy in patients with relapsed CNSL once CR had been re-achieved.

[METHODS] A retrospective observational study was conducted on patients who underwent ASCT or CAR-T therapy at the Department of Lymphoma, Beijing Gobroad Hospital, between 2021 and 2024. CAR-T therapy was part of the clinical trial "Different B-cell-targeted CAR-T cells for relapsed/refractory CNSL (ChiCTR2200058972)".

[RESULTS] Sixty patients, including 42 (70%) with primary CNSL and 18 (30%) with diffuse large B-cell lymphoma (DLBCL) with secondary CNS involvement, were enrolled. The median follow-up duration was 12.1 months (1.28-59.9 months). Compared with patients in the CAR-T group, patients who received ASCT while in CR had superior progression-free survival (PFS) [3-year PFS 80% (95% CI: 48.4-93.4) vs. 64.8% (95% CI: 38.9-81.9); P = 0.026] and a lower cumulative incidence of relapse/progression [3-year relapse rate 20% (95% CI: 4.12-44.39) vs. 30.2% (95% CI: 11.0-52.2); P = 0.038].

[CONCLUSION] Compared with CAR-T therapy, ASCT was associated with improved PFS in patients with relapsed CNSL who had achieved CR.