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The following is a summary of “Efficacy and safety of regorafenib in the treatment of bone sarcomas: systematic review and meta-analysis,” published in the February 2025 issue of the BMC Cancer by Han et al.

Metastatic or recurrent bone sarcomas present a significant clinical challenge due to their poor prognosis and limited treatment options. Regorafenib, a multikinase inhibitor, has demonstrated therapeutic potential in these patients, yet its efficacy and safety profile remain subjects of ongoing debate. This systematic review and meta-analysis aim to synthesize available evidence from randomized controlled trials to provide a comprehensive assessment of regorafenib’s impact on progression-free survival (PFS), OS, and adverse events in patients with metastatic or recurrent bone sarcomas.

Studies were included if they investigated regorafenib monotherapy of metastatic or recurrent bone sarcomas. The primary endpoints were PFS and OS, while the frequency and severity of AEs were analyzed as secondary outcomes. A total of 335 studies were screened, of which five met the inclusion criteria and were subjected to quantitative analysis.

The pooled data revealed that regorafenib significantly prolonged PFS at both three months (OR: 2.04, 95% CI: 1.21–2.86, P < 0.01) and six months (OR: 1.03, 95% CI: 0.08–1.99, P < 0.05) when compared to the control group. However, despite these improvements in disease control, regorafenib did not confer a survival advantage, as OS remained statistically comparable between treatment and control groups (P > 0.05). Additionally, regorafenib was associated with a higher incidence of AEs, with an odds ratio of 1.35 (95% CI: 0.63–2.07, P < 0.01), underscoring the need for vigilant management of treatment-related toxicities.

In conclusion, regorafenib demonstrates potential as a therapeutic option for patients with metastatic or recurrent bone sarcomas, offering meaningful improvements in disease stabilization. However, the absence of a survival benefit and the increased risk of AEs highlight the necessity for further research to optimize treatment strategies. Future studies should focus on refining the dosing regimen, identifying biomarkers for patient selection, and exploring combination therapies to enhance regorafenib’s clinical utility while mitigating its adverse effects.

Source: bmccancer.biomedcentral.com/articles/10.1186/s12885-025-13722-y