Efficacy and Safety of Vemurafenib in Acral and Mucosal Melanoma Patients with BRAF Gene Mutation

Objective To explore the efficacy and safety of Vemurafenib in acral and mucosal melanoma patients with BRAF gene mutation.

Methods We retrospectively analyzed the clinical data of 24 patients diagnosed as BRAF gene-mutant acral melanoma (AM) or mucosal melanoma (MM) hospitalized in Peking University Cancer Hospital from January 2011 to January 2016. All patients had died by the follow-up cutoff date of January 2017. Fisher exact test was used to detect the difference of baseline characteristics between AM and PM patients. Kaplan-Meier method was used for the estimation of overall survival (OS) and progression-free survival (PFS). We compared patients' survival data between two melanoma types using Log rank test.

Results There was no statistical difference in baseline characteristics between AM and MM patients. Overall median OS and PFS were 8.0 and 4.9 months. No statistical difference was found in median OS, median PFS or disease control rate. The severity of most adverse events was less than grade 3. Vemurafenib was well tolerated during patients' treatment.

Conclusion Vemurafenib has acceptable efficacy and safety in BRAF-gene-mutant AM and MM patients, which is similar to that in cutaneous melanoma patients.