In a nutshell
This study examined the effectiveness and safety of vemurafenib (Zelboraf) as treatment for BRAF-mutant metastatic melanoma. Researchers concluded that vemurafenib is an effective treatment option for patients with BRAF-mutated melanoma and brain metastasis (BM; spread to the brain).
Over 40% of melanoma patients experience BM, with an overall survival of only 3.8-5 months. Some melanoma patients have a mutation (permanent change) in the BRAF gene which increases tumor aggressiveness. Treatment with BRAF inhibitors (such as vemurafenib) can improve the overall survival of these patients. However, the outcomes of patients with BM treated with vemurafenib are less well studied.
Methods & findings
The objective of this study was to study the effectiveness and safety of vemurafenib in melanoma patients with BRAF mutation and BM.
146 patients were enrolled and divided into two groups, previously untreated BM (group 1; 90 patients) and previously treated BM (group 2; 56 patients). All patients were treated with vemurafenib. The average follow-up period was 4.1 months.
The overall response rate was 33% in group 1 and 23% in group 2. The average brain-related PFS (progression-free survival; time from treatment to disease progression) was 3.7 months in group 1 and 4 months in group 2. The average overall survival (time from treatment until death from any cause) for group 1 was 8.9 months and was 9.6 months for group 2.
Treatment with vemurafenib was tolerable and manageable. Moderate side effects were present in 66% of the patients in group 1 and in 64% of group 2. Severe effects were reported in 41% of patients in group 1 and 48% in group 2. The most common severe effect was squamous skin cancer (11%-12%).
The bottom line
This study suggested that vemurafenib was effective in the treatment of BRAF-mutated melanoma patients with BM, with manageable side effects.
Published By :
Annals of oncology : official journal of the European Society for Medical Oncology
Dec 19, 2016