CONTEXT: Vemurafenib, a selective BRAF inhibitor, appears to have promising 
clinical activity in patients with papillary thyroid cancer (PTC) harboring the 
BRAF(V600E) mutation.
OBJECTIVE: To determine the efficacy and safety of vemurafenib when used outside 
of a clinical trial.
DESIGN: A retrospective review at MD Anderson Cancer Center.
METHODS: The best responses were evaluated using RECIST v1.1. A single 
radiologist reviewed all images. Adverse events (AEs) were evaluated using CTCAE 
v.4.0.
RESULTS: We identified 17 patients with advanced PTC harboring the BRAF(V600E) 
mutation who were treated with vemurafenib outside of a clinical trial. Median 
age at diagnosis was 63 years, and 53% were male. At vemurafenib start, 3 (18%) 
patients had disease confined to the neck, and 14 (72%) had distant metastases. 
Tyrosine kinase inhibitors had been previously administered to 4 (24%) patients. 
Two (12%) patients discontinued vemurafenib because of AEs before restaging. 
Best response: partial response (PR) in 7/15 (47%) and stable disease (SD) in 
8/15(53%) patients. The rate of durable response (PR plus SD ≥ 6 months) was 
67%. Median time to treatment failure was 13 months. There was no association 
between change in thyroglobulin and tumor size. Drug discontinuation, drug 
interruptions, and dose reductions were needed in 5 (29%), 13 (76%), and 10 
(59%) patients, respectively. Most common AEs were fatigue (71%), weight loss 
(71%), anorexia (65%), arthralgias (59%), hair loss (59%), rash (59%), hand-foot 
syndrome (53%), calluses (47%), diarrhea (47%), fever (41%), dry mouth (35%), 
nausea (35%), and verrucous keratosis (35%). Grade ≥ 3 AEs were present in 8 
(47%) patients.
CONCLUSIONS: Vemurafenib is a potentially effective and well-tolerated treatment 
strategy in patients with advanced PTC harboring the BRAF(V600E) mutation. Our 
results are similar to those reported in a phase II clinical trial and support 
the potential role of vemurafenib in this patient population.