Safety and efficacy of teniposide-based therapy for glioma: a single-center retrospective study

Purpose : Glioma remains challenging despite standard therapy, necessitating novel treatment strategies. This study aimed to evaluated the efficacy and safety of teniposide (VM-26)-based therapy in glioma patients. Methods : A retrospective analysis was conducted on glioma patients treated with teniposide-based therapy between 2022 and 2025. Safety assessments were performed using Common Terminology Criteria for Adverse Events (CTCAE). Efficacy was evaluated according to the Response Assessment in Neuro-Oncology (RANO) criteria. Kaplan-Meier analysis was utilized to estimate the prognosis of patients. Results : Thirty-eight patients were enrolled. Regarding adverse events (AEs), 39.5% of patients experienced no AEs, 44.7% developed AEs below grade 3, whereas 15.8% presented with grade 3 and 4 AEs. No treatment-related deaths occurred. In tumor-present gliomas patients, the objective response rate (ORR) was 33.3% and the disease control rate (DCR) was 62.5%. Among tumor-present glioblastomas (GBM) patients, the median progression-free survival (PFS) was 4.15 months versus 12.20 months in tumor-absent GBM. The median overall survival after VM-26 (OS-VM-26) was 7.57 months in tumor-present GBM, whereas the median value was not reached in tumor-absent GBM. The median OS-VM26 was 17.70 months for primary GBM and 6.63 months for recurrent GBM, respectively. Molecular subtypes analysis of the tumor-present GBM revealed that the MGMT-methylated group had superior OS-VM26 and overall survival (OS) (both p < 0.05), with no significant difference in PFS. One representative case illustrated durable response with manageable toxicity. Conclusion : Teniposide-based therapy demonstrated acceptable safety. This therapy may serve as a candidate treatment option for patients with GBM.