Efficacy and safety of regorafenib in the treatment of bone sarcomas: systematic review and meta-analysis

Background Metastatic or recurrent bone sarcomas are often associated with an unfavorable prognosis, posing a formidable challenge in extending patients' survival. Currently, regorafenib has shown promise in treating metastatic and recurrent bone sarcomas. However, there is a lack of consensus on its efficacy and safety. This systematic review and meta-analysis aims to consolidate existing data to assess the efficacy and safety of regorafenib in bone sarcomas. Methods A comprehensive search strategy utilizing MeSH terms and free-text keywords was employed to systematically search the Embase, PubMed, Web of Science, and Cochrane databases up to May 26, 2024. Randomized controlled trials investigating regorafenib monotherapy for metastatic or recurrent bone sarcomas were included. The primary outcomes of interest were progression-free survival (PFS), overall survival(OS) and adverse events (AEs). Results We retrieved 335 articles and included 5 of them. Regorafenib significantly extended PFS-3 months and PFS-6 months in patients with metastatic or recurrent bone sarcomas compared to the control group, exhibiting a favorable odds ratio (OR) of 2.04 (95% CI: 1.21-2.86, P < 0.01) and 1.03 (95% CI: 0.08-1.99, P < 0.05), respectively. However, regorafenib did not improve OS at any observation point compared with the control group(P > 0.05), and the frequency of AEs was higher, with an odds ratio of 1.35 (95% CI: 0.63-2.07, P < 0.01). Conclusion Regorafenib emerges as a promising therapeutic option for metastatic or recurrent bone sarcomas, demonstrating certain clinical benefits alongside manageable adverse reactions. Nevertheless, further research is warranted to refine the efficacy and safety profile of regorafenib, particularly in exploring safe dosage ranges or alternative treatment modalities.