Activating mutations in the BRAF gene occur in approximately 50% of melanomas. 
More than 70% of BRAF mutations are V600E and 10-30% are V600K. Potent and 
selective BRAF inhibitors have demonstrated significant clinical benefits in 
patients with V600E and V600K BRAF-mutated melanoma. V600R mutations constitute 
approximately 3-7% of all BRAF mutations and the activity of BRAF inhibitors in 
patients with this mutation is unknown. We have treated 45 patients with V600 
mutated melanoma including patients with V600R mutation between July 2011 and 
October 2012 with the selective BRAF inhibitor dabrafenib (n=43) or vemurafenib 
(n=2) via a compassionate access programme. The overall response rate was 50% 
for the whole population with a progression-free survival of 5.5 months. Five 
objective responses were seen in six assessable patients with V600R BRAF 
mutation (n=9). Our experience suggests that patients with V600R BRAF mutations 
can be treated successfully with oral BRAF inhibitors, and molecular diagnostic 
assays should include detection of this type of mutation.