AbstractBackgroundPatients with alveolar rhabdomyosarcoma (ARMS) have inferior outcomes compared to patients with embryonal rhabdomyosarcoma (ERMS) and more effective chemotherapy options are needed for these patients. Vinorelbine is a semisynthetic vinca alkaloid that has clinical activity in relapsed rhabdomyosarcoma (RMS) when used alone or in combination with cyclophosphamide.AimsThe goal of our study was to evaluate whether RMS histology subtype influences response rate to vinorelbine alone or in combination.Materials & MethodsFive Phase 2 trials that enrolled RMS patients were included in the meta‐analysis. Two studies evaluated vinorelbine alone, two studies evaluated vinorelbine in combination with low dose oral cyclophosphamide, and one study evaluated vinorelbine and intravenous cyclophosphamide in combination with temsirolimus or bevacizumab. All RMS patients had relapsed or refractory disease and had received at least one prior therapy. Response was reported according to RECIST1.1 and was defined as a complete or partial response. Response data was obtained from published results or from trial principal investigator. RMS NOS patients were grouped with ERMS patients for this analysis. Summary estimates comparing differences between ARMS and ERMS response rates were generated using a random‐effects model to account for heterogeneity among the studies.ResultsOne hundred fifty‐six enrolled patients evaluable for response were included in the meta‐analysis, 85 ARMS, 64 ERMS and 7 RMS‐NOS. The combined effect generated from the random‐effects model demonstrated a 41% increase (p= 0.001, 95% CI; 0.21–0.60) in response to vinorelbine as a single agent or in combination in patients with ARMS compared to patients with ERMS. There was no significant difference in the rate of progressive disease between patients with ARMS compared to ERMS (p= 0.1, 95%CI; −0.26–0.02).DiscussionVinorelbine is an active agent for the treatment of relapsed or refractory RMS and a meta‐analysis of Phase 2 studies shows that radiographic responses in patients with ARMS were significantly higher than ERMS or RMS‐NOS.ConclusionThese data support further investigation of vinorelbine in newly diagnosed patients with RMS particularly those with alveolar histology.