In the Italian phase II REGOMA trial reported in The Lancet Oncology, Lombardi et al found that regorafenib was associated with improved overall survival vs lomustine in patients with relapsed glioblastoma.
In the open-label trial, 119 patients from 10 Italian sites were randomly assigned between November 2015 and February 2017 to receive regorafenib (n = 59) or lomustine (n = 60). Patients had to have disease progression after surgery followed by radiotherapy and temozolomide chemoradiotherapy. Regorafenib was given at 160 mg once daily for the first 3 weeks of 4-week cycles, and lomustine was given at 110 mg/m² once every 6 weeks, both until disease progression, death, or unacceptable toxicity.
The primary endpoint was overall survival in the intention-to-treat population. Median follow-up was 15.4 months.
Median overall survival was 7.4 months in the regorafenib group vs 5.6 months in the lomustine group (hazard ratio [HR] = 0.50, P = .0009). Overall survival at 12 months was 38.9% vs 15.0%. Median progression-free survival was 2.0 vs 1.9 months (HR = 0.65, P = .022), with 6-month rates of 16.9% vs 8.3%.
Salvage therapy at disease progression was received by 64% of the regorafenib group (mainly fotemustine) and by 43% of the lomustine group (mainly fotemustine). No lomustine-treated patients received regorafenib after disease progression.
Grade 3 or 4 treatment-related adverse events occurred in 56% of the regorafenib group and 40% of the lomustine group. The most common events in the regorafenib group were hand-foot skin reaction, increased lipase, and increased blood bilirubin (10% each); the most common events in the lomustine group were decreased platelet count (13%), decreased lymphocyte count (13%), and neutropenia (12%). No treatment-related deaths were observed.
The investigators concluded, “REGOMA showed an encouraging overall survival benefit of regorafenib in recurrent glioblastoma. This drug might be a new potential treatment for these patients and should be investigated in an adequately powered phase III study.”
Vittorina Zagonel, MD, of the Veneto Institute of Oncology, Padua, Italy, is the corresponding author for The Lancet Oncology article.
Disclosure: The study was funded by Veneto Institute of Oncology and Bayer Italy. The study authors’ full disclosures can be found at thelancet.com.
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