Abstract 359P
Background
Regorafenib (REG), showed encouraging benefit in recurrent GBM patients in REGOMA trial. We investigated the clinical outcome and safety of REG in a real-life population of recurrent GBM patients treated at Veneto Institute of Oncology as off-label use.
Methods
Patients receiving REG were entered prospectively on a clinical database. Data were retrospectively analyzed. The primary endpoints were overall survival (OS) and safety. The major inclusion criteria were: histologically confirmed diagnosis of GBM, disease progression by RANO criteria after surgery and chemoradiotherapy, ECOG PS ≤ 2; PTS with ≥ 2 prior lines of therapy were excluded. Patients received REG 160 mg once daily for the first 3 weeks of each 4-week cycle until disease progression, death, unacceptable toxicity, or consent withdrawal. Kaplan-Meier method was used to estimate the survival curves, CTCAE v5.0 for drug related adverse events.
Results
From February2018 to September2020, 54 consecutive patients were treated with REG: median age was 56, ECOG PS 0-1 in 91%, MGMTmet in 53%, second surgery at relapse were performed in 30%, 41% underwent steroids at baseline. Median follow-up was 11.1 ms, 30 PTS (56%) had died and 50 PTS (93%) had progressed. Median OS was 10.2 ms (95%CI, 6.4-13.9), 12m-OS was 43%; median PFS was 2.3ms (95%CI, 1.3-3.3) and 6m-PFS was 18%. All patients were evaluable for response: disease control rate (DCR) was 46.3%; stable disease was reported in 38.8% and partial response in 7.4%. Age, MGMT and corticosteroid use at baseline were not statistically significant on multivariate analysis for OS. Grade 3 drug-related adverse events (AEs) occurred in 10 patients (18%) and the most frequent were hand-foot skin reaction, asthenia and increased lipase and transaminases; 1 PT (2%) reported a grade 4 AE (maculo-papular rash). AEs led to REG dose reductions in 37% of patients and, it was permanently discontinued in 5%. No death was considered to be drug-related.
Conclusions
We reported a large, mono-institutional “real-world” experience of REG in recurrent GBM patients. Overall, results are close to those reported in REGOMA trial although, we showed a longer OS. Toxicity was moderate and manageable. Encouraging clinical benefits of REG in recurrent GBM population were confirmed.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
The authors.
Funding
Has not received any funding.
Disclosure
All authors have declared no conflicts of interest.