Data from a phase II trial involving patients with relapsed glioblastoma following chemoradiotherapy, a setting in which few evidence-based therapies are currently available, demonstrate the superiority of the anti-angiogenic tyrosine-kinase inhibitor regorafenib over lomustine chemotherapy. Patients receiving regorafenib had a median overall survival duration of 7.6 months versus 5.6 months in the lomustine group (HR 0.50, 95% CI 0.33–0.75; log-rank P = 0.0009). Grade 3–4 adverse events were more common in the regorafenib group (occurring in 56% versus 40% of patients) and the majority were haematological: decreased platelet count, decreased lymphocyte count and neutropenia were observed in 13%, 13% and 12% of patients, respectively. No treatment-related deaths were reported in either arm. These findings merit further investigation in a randomized phase III trial.
Lombardi, G. et al. Regorafenib compared with lomustine in patients with relapsed glioblastoma (REGOMA): a multicentre, open-label, randomised, controlled, phase 2 trial. Lancet https://doi.org/10.1016/S1470-2045(18)30675-2 (2018)
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Sidaway, P. Relapsed glioblastomas respond to regorafenib. Nat Rev Clin Oncol 16, 144 (2019). https://doi.org/10.1038/s41571-018-0161-5