Efficacy of Vemurafenib Treatment in 43 Metastatic Melanoma Patients with BRAF Mutation. Single-Institute Retrospective Analysis, Early Real-Life Survival Data

Abstract

BRAF inhibitor vemurafenib achieved improved overall survival over chemotherapy and have been approved by the FDA and EMA for the treatment of BRAF-mutated metastatic melanoma. The aim of our retrospective analysis was to determine the efficacy and safety of vemurafenib therapy for BRAF mutated metastatic melanoma and subsequently to prove the clinical benefit for the studied 43 patients, based on real-life data. From November 2012 to October 2015 we have selected 43 BRAF mutated, metastatic melanoma patients, treated with vemurafenib. The median follow-up time was 15.9 months. We evaluated progression free survival (PFS), overall survival (OS) and toxicities. According to the AJCC staging system 70% of the patients had stage M1c metastasis, including 6 with stable brain metastasis. Objective responses were noted in 51.1%, the disease control rate was achieved in 79% of the patients. Complete responses were attained by 5 patients (11.6%). Median PFS was 6.48 (95% CI:4.8-15.0) months, median OS was 11.47 (95% CI:8.08-NA) months. We found significant association between LDH level and OS in univariate (p = 0.000613) and multivariate analysis (p = 0.0168). The most common adverse events (AEs) included follicular hyperkeratosis, rash, arthralgia and photosensitivity. Grade 3 AEs, such as cutaneous squamous-cell carcinoma, QTcB interval prolongation, rash, arthralgia were reported in 7 patients (17%). We had no Grade 4 side effects. Similar to the previously published data our analysis confirms the improved survival with vemurafenib treatment (11.47 months) in patients with BRAF V600 mutation. Vemurafenib therapy was well tolerated, the AE profile was almost consistent with the previously reported data of randomised clinical trials.

Survival in adult patients with BRAFV600 mutation-positive advanced melanoma: a noninterventional ambispective study of patients with cobimetinib combined with vemurafenib during the French early access program: MELANIS study

“…This usual clinical practice would explain that only a slightly longer PFS was estimated in coBRIM-like compared with non-coBRIM-like patients. In addition, the median PFS estimated in the MELANIS study (stage IV in 88% of the patients) was slightly longer than in a real-world retrospective study conducted in 43 patients with metastatic melanoma treated with vemurafenib only (6.48; 95% CI, 4.8–15.0) [7].…”

Efficacy of vemurafenib in a heavy smoker with BRAF-mutated lung adenocarcinoma: A case report and literature review

“…Approximately 55% of NSCLC patients with a BRAF mutation have been found to harbor the V600E mutation (8). Overall survival (OS) with chemotherapy has improved by the use of vemurafenib (a BRAF inhibitor), which was approved by the Food and Drug Administration and European Medicines Agency for the treatment of metastatic melanoma with a BRAF mutation (9). There are studies and clinical trials showing that BRAF inhibitors are effective against BRAF-mutated NSCLC (10,11).…”

Human Metastatic Melanoma Cell Lines Panel for In Vitro and In Vivo Investigations

“…In 2021, 11,412 new cases of melanoma and 3217 fatal cases were registered, which accounted for 69.2% of all deaths from malignant skin neoplasms [2]. A number of new agents for melanoma treatment have been registered by the FDA and EMA, and a number of promising targets have been proposed recently [3][4][5][6][7]. Patient-derived tumor models, including melanoma cell lines and tumor xenografts, are invaluable tools for developing and conducting highly reproducible studies under certain conditions that provide insight into drug sensitivity, the basics of cell biology, and elucidation of signaling pathways.…”