Efficacy and safety of IDH inhibitors in IDH-mutated cancers: a systematic review and meta-analysis of 4 randomized controlled trials

Isocitrate dehydrogenase (IDH) inhibitors have shown great promise in the treatment of cancers with IDH mutations. There have been numerous clinical trials conducted on IDH inhibitors, and to evaluate their efficacy and safety, we aim to perform a meta-analysis.

To gather data on the efficacy and safety of IDH inhibitors for IDH-mutated cancers, we systematically searched through databases including PubMed, EMBASE, and Cochrane Library. Using RevMan5.4, we performed a meta-analysis and calculated the odds ratio (OR) or weighted mean difference (WMD) with a 95% confidence interval (95%CI). The parameters considered were overall survival (OS), progression-free survival (PFS), objective response rate (ORR), disease control rate (DCR), treatment-related adverse events (TRAEs), and TRAEs ≥ 3.

This meta-analysis included four studies, involving a total of 751 patients. According to the analysis, there was no significant difference in overall survival, treatment-related adverse events, and severe treatment-related adverse events between the experimental group (receiving IDH inhibitors) and the control group. However, the progression-free survival, objective response rate, and disease control rate in the experimental group were significantly higher than those in the control group.

The overall efficacy of IDH inhibitors in treating cancers with IDH mutations is superior to that of conventional medical therapy, potentially providing more clinical benefits to patients. The incidence of adverse events was not significantly different from conventional medical therapy. Therefore, IDH inhibitors should be considered as the preferred choice for treating cancers with IDH mutations. However, further randomized controlled clinical trials are still required for verification.