Lombardi G, et al. - Regorafenib's efficacy and safety as a treatment option for recurrent glioblastoma were tested in the REGOMA, which was a randomised, multicentre, open-label phase 2 trial done in ten centres in Italy. Regorafenib is an oral multikinase inhibitor of angiogenic, stromal, and oncogenic receptor tyrosine kinases. According to findings, the overall survival benefit with regorafenib in the treatment of recurrent glioblastoma was encouraging. This drug can possibly afford a new potential treatment for these patients.

Methods

• Researchers performed a randomised, multicentre, open-label phase 2 trial, named REGOMA, in ten centres in Italy.
• Patients who were considered eligible were those (aged ≥18 years) with histologically confirmed glioblastoma, Eastern Cooperative Oncology Group performance status 0 or 1, and documented disease progression after surgery followed by radiotherapy and temozolomide chemoradiotherapy.
• Using a web-based system, stratified by centre and surgery at recurrence (yes vs no), the participants were randomly administered (1:1) regorafenib 160 mg once daily for the first 3 weeks of each 4-week cycle or lomustine 110 mg/m² once every 6 weeks until disease progression, death, unacceptable toxicity, or consent withdrawal.
• Overall survival (primary endpoint) was assessed in the intention-to-treat population.

Results

• Screening in 124 patients and randomization of 119 eligible patients to receive regorafenib (n=59) or lomustine (n=60) was done between Nov 27, 2015, and Feb 23, 2017.
• For median 15·4 months (IQR 13·8–18·1), participants were followed-up.
• Death was reported in 99 (83%) of 119 patients at the analysis cutoff date: 42 (71%) of 59 in the regorafenib group and 57 (95%) of 60 in the lomustine group.
• The regorafenib group vs the lomustine group demonstrated significantly improved overall survival, with a median overall survival of 7·4 months (95% CI 5·8–12·0) and 5·6 months (4·7–7·3) seen in the regorafenib group and in the lomustine group, respectively (hazard ratio 0·50, 95% CI 0·33–0·75; log-rank p=0·0009).
• A total of 33 (56%) of 59 patients treated with regorafenib and 24 (40%) of 60 with lomustine experienced grade 3–4 treatment-related adverse events.
• Hand–foot skin reaction, increased lipase, and blood bilirubin increased (in six [10%] of 59 patients each) were documented as the most frequently occurred grade 3 or 4 adverse events related to regorafenib.
• Patients treated with lomustine most commonly experienced decreased platelet count (eight [13%] of 60 patients), decreased lymphocyte count (eight [13%]), and neutropenia (seven [12%]) as grade 3 or 4 adverse events.
• No death was considered to be related to drug.