Regorafenib in adult patients with recurrent glioblastoma: a single-arm meta-analysis

Glioblastoma is an aggressive brain tumor with median survival under 2 years despite standard therapy. At recurrence, treatment options are limited, and regorafenib has emerged as a promising option. A systematic search was conducted through Pubmed, Cochrane Library, Embase, and Web of Science for studies on regorafenib in adult recurrent glioblastoma, and a single-arm meta-analysis with random-effects model was performed to pool the data. Across ten studies (724 patients), median overall survival (OS) was 7.2 months and median progression-free survival (PFS) was 2.6 months, with a 12-month OS of 22.5% and a 6-month PFS of 14.9%. Disease control rate (DCR) was 36.1%, including stable disease (SD) of 26.6% and a partial response of 8.5%; progressive disease occurred in 60.9%, and grade 3-4 adverse events in 31.4%. Meta-regression suggested MGMT methylation was associated with improved OS, PFS, DCR, and SD, while male sex was associated with better OS and SD. Overall, regorafenib demonstrated a predictable safety profile in recurrent glioblastoma, with outcomes potentially improved in male patients with MGMT-methylated tumors.