Latest clinical guidelines: The role of regorafenib in the treatment of osteosarcoma
Metastatic or recurrent osteosarcoma presents significant clinical challenges due to its poor prognosis and limited treatment options. Regorafenib is a multikinase inhibitor that has demonstrated therapeutic potential in these patients, but its efficacy and safety remain the subject of current debate. This systematic review and meta-analysis aimed to synthesize the available evidence from randomized controlled trials to comprehensively evaluate the impact of regorafenib on progression-free survival (PFS), OS, and adverse events in patients with metastatic or recurrent osteosarcoma.
Studies were included if they investigated regorafenib monotherapy for metastatic or recurrent osteosarcoma. The primary endpoints were PFS and OS, while frequency and severity of AEs were analyzed as secondary outcomes. A total of 335 studies were screened, of which 5 met the inclusion criteria and were quantitatively analyzed.

Summary data showed that compared with the control group, regorafenib significantly prolonged PFS at 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). However, despite improvements in disease control, regorafenib did not confer a survival advantage as OS remained statistically comparable between treatment and control groups (P>0.05). In addition, 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), emphasizing the need for vigilant management of treatment-related toxicities.
In conclusion, regorafenib shows potential as a treatment option for patients with metastatic or recurrent osteosarcoma, providing meaningful improvements in disease stabilization. However, the lack of survival benefit and increased risk of AEs highlight the need for further studies to optimize treatment strategies. Future studies should focus on refining dosing regimens, identifying biomarkers for patient selection, and exploring combination treatments to enhance the clinical utility of regorafenib while mitigating its adverse effects.
References:https://www.physiciansweekly.com/impact-of-regorafenib-in-bone-sarcomas-treatment/
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