462P - Regorafenib for relapsed glioblastoma: Retrospective real-world analysis of a single Institution experience

Background

Glioblastoma has a high rate of recurrence after standard first-line treatment. Following the phase II REGOMA trial, establishing Regorafenib as a valuable option for the second-line treatment, the Italian Medicines Agency has provided reimbursement for the use of Regorafenib in this setting. Notably, the REGOMA-OSS, a multicenter, prospective, observational trial, has recently confirmed the REGOMA efficacy and safety data. We conducted a monocentric, retrospective analysis to compare the REGOMA-OSS data in our real-world setting.

Methods

We retrospectively analyzed the clinical data of relapsed glioblastoma patients treated with Regorafenib as second-line treatment or, following individual local authorization, as subsequent line of therapy at Modena Cancer Center. Patients had a histologically confirmed diagnosis of glioblastoma according to WHO 2016 or WHO 2021. We specifically analyzed survival endpoints of OS and PFS and we assessed safety according to CTCAE v. 5.

Results

36 patients were treated with Regorafenib from January 2020 to April 2024 in our cancer center: median age was 57 years (95% CI 51-63.7), males were 52.8%, ECOG PS was 0 in 5 (13.9%), 1 in 19 (52.8%), 2 in 11 patients (33.3%). 29 (80.5%) patients were undertaking steroids at baseline. MGMT was methylated in 30.5% of cases, IDH was wt in 88.9%. Regarding radiological response, both PR and SD were observed in 5.5% of patients. Median OS was 3.6 months (95% CI 2.4-4.5), median PFS was 2 months (95% CI 1.7-2.7). The median number of Regorafenib cycles per patient was 3.8. Mean daily dose was 160 mg/die in 55.6% and <160 mg/die in 44.4% of patients, respectively. Eight (22.2%) patients experienced at least one G3 adverse event (AE), no G4 AE were observed. Dose reduction/delay and permanent discontinuation due to AE were observed in 10 (27.8%) and 4 (11.1%) patients, respectively. No treatment-related deaths were detected.

Conclusions

Compared to the large, prospective, observational REGOMA-OSS trial, our monocentric, retrospective, real-world analysis showed a similar result in terms of PFS and a shorter OS, likely because of the inclusion of patients with a poorer PS or treated at subsequent lines of therapy. Moreover, it confirmed a favorable safety profile.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

R. Depenni: Non-Financial Interests, Personal, Speaker’s Bureau, lectures, presentations, manuscript writing, educational events: Novartis, MSD, BMS, Roche, Pierre Fabre, Merck, Sanofi, Sun Pharma. All other authors have declared no conflicts of interest.