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Regorafenib in patients with relapsed advanced or metastatic chordoma: results of a non-comparative, randomised,
A Le Cesne1, C Chevreau2, C Perrin3
1Medical Oncology Department, Gustave Roussy, Villejuif.
Background:
REGOBONE multicohort study explored the efficacy and safety of regorafenib for patients with advanced bone sarcomas; this report details the cohort of patients with relapsed advanced or metastatic chordoma.
Methods:
Patients with relapsed chordoma progressing despite 0-2 prior lines of systemic therapy, were randomised (2 : 1) to receive regorafenib (160 mg/day, 21/28 days) or placebo. Patients on placebo could cross over to receive regorafenib after centrally-confirmed progression. The primary endpoint was the progression-free rate at 6 months (PFR-6) (by RECIST 1.1). With one-sided α of 0.05, and 80% power, at least 10/24 progression-free patients at 6 months (PFR-6) were needed for success.
Results:
From March 2016 to February 2020, 27 patients were enrolled. A total of 23 patients were assessable for efficacy: 7 on placebo, 16 on regorafenib, 16 were men, median age was 66 (32-85) years. At 6 months, in the regorafenib arm, 1 patient was not assessable, 6/14 were non-progressive (PFR-6: 42.9%; one-sided 95% CI = 20.6) 3/14 discontinued regorafenib due to toxicity; and in the placebo arm, 2/5 patients were non-progressive (PFR-6: 40.0%; one-sided 95% CI = 7.6), 2 were non-assessable. Median progression-free survival was 8.2 months (95% CI 4.5-12.9 months) on regorafenib and 10.1 months (95% CI 0.8 months-non evaluable [NE]) on placebo. Median overall survival rates were 28.3 months (95% CI 14.8 months-NE) on regorafenib but not reached in placebo arm. Four placebo patients crossed over to receive regorafenib after centrally-confirmed progression. The most common grade ≥3 regorafenib-related adverse events were hand-foot skin reaction (22%), hypertension (22%), pain (22%), and diarrhoea (17%), with no toxic death.
Conclusion:
This study failed to show any signal of benefit for regorafenib in patients with advanced/metastatic recurrent chordoma.
Insights
Regorafenib did not show a benefit for patients with advanced chordoma. The study found no significant improvement in progression-free survival for regorafenib compared to placebo in this patient group.
Area of Science:
- Oncology
- Medical Research
Background:
- The REGOBONE study investigated regorafenib in advanced bone sarcomas.
- This report focuses on patients with relapsed, advanced, or metastatic chordoma.
Purpose of the Study:
- To evaluate the efficacy and safety of regorafenib in patients with relapsed chordoma.
- To determine if regorafenib improves progression-free survival at 6 months (PFR-6).
Main Methods:
- Randomized trial comparing regorafenib (160 mg/day) to placebo in a 2:1 ratio.
- Patients had chordoma progressing after 0-2 prior systemic therapies.
- Primary endpoint was PFR-6; placebo patients could cross over to regorafenib.
Main Results:
- 27 patients enrolled; 23 were efficacy-assessable (16 regorafenib, 7 placebo).
- PFR-6 was 42.9% for regorafenib vs. 40.0% for placebo.
- Median progression-free survival was 8.2 months (regorafenib) vs. 10.1 months (placebo).
Conclusions:
- Regorafenib did not demonstrate a significant benefit in patients with advanced/metastatic recurrent chordoma.
- The study failed to show any signal of efficacy for regorafenib in this population.
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