Koriyama Shunichi, Muragaki Yoshihiro, Nitta Masayuki, Maruyama Takashi, Saito Taiichi, Tsuzuki Shunsuke, Kobayashi Tatsuya, Ro Buntou, Komori Takashi, Masui Kenta, Kawamata Takakazu
Department of Neurosurgery, Tokyo Women's Medical University, Tokyo, Japan.
Faculty of Advanced Techno-Surgery (FATS), Institute of Advanced Biomedical Engineering and Science, Graduate School of Medicine, Tokyo Women's Medical University, Tokyo, Japan.
Brain Tumor Pathol. 2025 Aug 7. doi: 10.1007/s10014-025-00507-1.
Glioblastoma (GBM) is a primary brain tumor, characterized by rapid progression, high recurrence rates, and resistance to standard therapies. Current treatment modalities provide limited survival benefits, highlighting the need for novel therapeutic strategies. This retrospective study evaluated the efficacy of autologous formalin-fixed tumor vaccine (AFTV) in 375 patients with newly diagnosed GBM. Patients receiving AFTV therapy (n = 164) showed significantly improved progression-free survival (PFS; 14.0 months vs. 8.7 months, p = 0.03) and overall survival (OS; 32.0 months vs. 21.9 months, p < 0.01) compared with the non-AFTV group (n = 211). Subgroup analyses revealed that AFTV therapy was particularly effective in patients with wild-type IDH tumors and those negative for PD-L1 and p53 expression. In contrast, patients whose tumors were positive for both PD-L1 and p53 exhibited significantly poorer outcomes. These findings suggest that the combination of PD-L1 and p53 status may serve as a useful biomarker for predicting AFTV responsiveness, reflecting the influence of the immunosuppressive tumor microenvironment on treatment efficacy. These findings establish AFTV as a promising treatment option for GBM and highlight the importance of molecular profiling in treatment selection. Future studies should explore combining AFTV with immune checkpoint inhibitors to enhance efficacy in PD-L1-positive cases.
胶质母细胞瘤(GBM)是一种原发性脑肿瘤,其特征为进展迅速、复发率高且对标准疗法耐药。当前的治疗方式提供的生存获益有限,这凸显了新型治疗策略的必要性。这项回顾性研究评估了自体福尔马林固定肿瘤疫苗(AFTV)对375例新诊断GBM患者的疗效。与未接受AFTV治疗的组(n = 211)相比,接受AFTV治疗的患者(n = 164)的无进展生存期(PFS;14.0个月对8.7个月,p = 0.03)和总生存期(OS;32.0个月对21.9个月,p < 0.01)显著改善。亚组分析显示,AFTV治疗在野生型异柠檬酸脱氢酶(IDH)肿瘤患者以及程序性死亡受体配体1(PD-L1)和p53表达阴性的患者中特别有效。相比之下,肿瘤PD-L1和p53均呈阳性的患者预后明显较差。这些发现表明,PD-L1和p53状态的组合可能作为预测AFTV反应性的有用生物标志物,反映了免疫抑制性肿瘤微环境对治疗疗效的影响。这些发现确立了AFTV作为GBM一种有前景的治疗选择,并突出了分子谱分析在治疗选择中的重要性。未来的研究应探索将AFTV与免疫检查点抑制剂联合使用,以提高PD-L1阳性病例的疗效。