TY - JOUR
T1 - Efficacy and tolerability of vemurafenib in patients with BRAFV600E positive papillary thyroid cancer
T2 - M.D. Anderson Cancer center off label experience
AU - Dadu, Ramona
AU - Shah, Komal
AU - Busaidy, Naifa L.
AU - Waguespack, Steven G.
AU - Habra, Mouhammad A.
AU - Ying, Anita K.
AU - Hu, Mimi I.
AU - Bassett, Roland
AU - Jimenez, Camilo
AU - Sherman, Steven I.
AU - Cabanillas, Maria E.
N1 - Publisher Copyright:
© 2015 by the Endocrine Society.
PY - 2015/1/1
Y1 - 2015/1/1
N2 - Context: Vemurafenib, a selective BRAF inhibitor, appears to have promising clinical activity in patients with papillary thyroid cancer (PTC) harboring the BRAFV600E mutation. Objective: To determine the efficacyandsafety of vemurafenibwhenused 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:Weidentified 17 patients with advanced PTC harboring the BRAFV600E mutationwhowere treated with vemurafenib outside of a clinical trial. Median age at diagnosis was 63 years, and53% 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 BRAFV600E 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.
AB - Context: Vemurafenib, a selective BRAF inhibitor, appears to have promising clinical activity in patients with papillary thyroid cancer (PTC) harboring the BRAFV600E mutation. Objective: To determine the efficacyandsafety of vemurafenibwhenused 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:Weidentified 17 patients with advanced PTC harboring the BRAFV600E mutationwhowere treated with vemurafenib outside of a clinical trial. Median age at diagnosis was 63 years, and53% 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 BRAFV600E 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.
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U2 - 10.1210/jc.2014-2246
DO - 10.1210/jc.2014-2246
M3 - Article
C2 - 25353071
AN - SCOPUS:84920598146
SN - 0021-972X
VL - 100
SP - E77-E81
JO - Journal of Clinical Endocrinology and Metabolism
JF - Journal of Clinical Endocrinology and Metabolism
IS - 1
ER -