{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,28]],"date-time":"2026-07-28T03:26:00Z","timestamp":1785209160433,"version":"3.55.0"},"reference-count":19,"publisher":"The Endocrine Society","issue":"1","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2015,1,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:sec>\n                  <jats:title>Context:<\/jats:title>\n                  <jats:p>Vemurafenib, a selective BRAF inhibitor, appears to have promising clinical activity in patients with papillary thyroid cancer (PTC) harboring the BRAFV600E mutation.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Objective:<\/jats:title>\n                  <jats:p>To determine the efficacy and safety of vemurafenib when used outside of a clinical trial.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Design:<\/jats:title>\n                  <jats:p>A retrospective review at MD Anderson Cancer Center.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Methods:<\/jats:title>\n                  <jats:p>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.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results:<\/jats:title>\n                  <jats:p>We identified 17 patients with advanced PTC harboring the BRAFV600E 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 \u2265 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 \u2265 3 AEs were present in 8 (47%) patients.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusions:<\/jats:title>\n                  <jats:p>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.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1210\/jc.2014-2246","type":"journal-article","created":{"date-parts":[[2014,10,29]],"date-time":"2014-10-29T21:53:47Z","timestamp":1414619627000},"page":"E77-E81","source":"Crossref","is-referenced-by-count":100,"title":["Efficacy and Tolerability of Vemurafenib in Patients with BRAFV600E -Positive Papillary Thyroid Cancer: M.D. Anderson Cancer Center Off Label Experience"],"prefix":"10.1210","volume":"100","author":[{"given":"Ramona","family":"Dadu","sequence":"first","affiliation":[{"name":"Departments of Endocrine Neoplasia and Hormonal Disorders (R.D., N.L.B., S.G.W., M.A.H., A.K.Y., M.I.H., C.J., S.I.S., M.E.C.), Houston, Texas 77030"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Komal","family":"Shah","sequence":"additional","affiliation":[{"name":"Diagnostic Radiology (K.S.), Houston, Texas 77030"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Naifa L.","family":"Busaidy","sequence":"additional","affiliation":[{"name":"Departments of Endocrine Neoplasia and Hormonal Disorders (R.D., N.L.B., S.G.W., M.A.H., A.K.Y., M.I.H., C.J., S.I.S., M.E.C.), Houston, Texas 77030"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven G.","family":"Waguespack","sequence":"additional","affiliation":[{"name":"Departments of Endocrine Neoplasia and Hormonal Disorders (R.D., N.L.B., S.G.W., M.A.H., A.K.Y., M.I.H., C.J., S.I.S., M.E.C.), Houston, Texas 77030"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mouhammad A.","family":"Habra","sequence":"additional","affiliation":[{"name":"Departments of Endocrine Neoplasia and Hormonal Disorders (R.D., N.L.B., S.G.W., M.A.H., A.K.Y., M.I.H., C.J., S.I.S., M.E.C.), Houston, Texas 77030"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anita K.","family":"Ying","sequence":"additional","affiliation":[{"name":"Departments of Endocrine Neoplasia and Hormonal Disorders (R.D., N.L.B., S.G.W., M.A.H., A.K.Y., M.I.H., C.J., S.I.S., M.E.C.), Houston, Texas 77030"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mimi I.","family":"Hu","sequence":"additional","affiliation":[{"name":"Departments of Endocrine Neoplasia and Hormonal Disorders (R.D., N.L.B., S.G.W., M.A.H., A.K.Y., M.I.H., C.J., S.I.S., M.E.C.), Houston, Texas 77030"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roland","family":"Bassett","sequence":"additional","affiliation":[{"name":"Biostatistics (R.B.), The University of Texas M.D. Anderson Cancer Center, Houston, Texas 77030"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Camilo","family":"Jimenez","sequence":"additional","affiliation":[{"name":"Departments of Endocrine Neoplasia and Hormonal Disorders (R.D., N.L.B., S.G.W., M.A.H., A.K.Y., M.I.H., C.J., S.I.S., 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