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Efficacy and Tolerability of Vemurafenib in Patients with BRAFV600E -Positive Papillary Thyroid Cancer: M.D. Anderson Cancer Center Off Label Experience

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 efficacy and safety of vemurafenib when used outside of a clinical trial. Design: A retrospectiv... Full description

Journal Title: The journal of clinical endocrinology and metabolism 2015, Vol.100 (1), p.E77-E81
Main Author: Dadu, Ramona
Other Authors: Shah, Komal , Busaidy, Naifa L , Waguespack, Steven G , Habra, Mouhammad A , Ying, Anita K , Hu, Mimi I , Bassett, Roland , Jimenez, Camilo , Sherman, Steven I , Cabanillas, Maria E
Format: Electronic Article Electronic Article
Language: English
Subjects:
Brief Reports
JCEM Online
Publisher: Chevy Chase, MD: Endocrine Society
ID: ISSN: 0021-972X
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title: Efficacy and Tolerability of Vemurafenib in Patients with BRAFV600E -Positive Papillary Thyroid Cancer: M.D. Anderson Cancer Center Off Label Experience
format: Article
creator:
  • Dadu, Ramona
  • Shah, Komal
  • Busaidy, Naifa L
  • Waguespack, Steven G
  • Habra, Mouhammad A
  • Ying, Anita K
  • Hu, Mimi I
  • Bassett, Roland
  • Jimenez, Camilo
  • Sherman, Steven I
  • Cabanillas, Maria E
subjects:
  • Brief Reports
  • JCEM Online
ispartof: The journal of clinical endocrinology and metabolism, 2015, Vol.100 (1), p.E77-E81
description: 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 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 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 ≥ 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.
language: eng
source:
identifier: ISSN: 0021-972X
fulltext: no_fulltext
issn:
  • 0021-972X
  • 1945-7197
url: Link


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titleEfficacy and Tolerability of Vemurafenib in Patients with BRAFV600E -Positive Papillary Thyroid Cancer: M.D. Anderson Cancer Center Off Label Experience
creatorDadu, Ramona ; Shah, Komal ; Busaidy, Naifa L ; Waguespack, Steven G ; Habra, Mouhammad A ; Ying, Anita K ; Hu, Mimi I ; Bassett, Roland ; Jimenez, Camilo ; Sherman, Steven I ; Cabanillas, Maria E
creatorcontribDadu, Ramona ; Shah, Komal ; Busaidy, Naifa L ; Waguespack, Steven G ; Habra, Mouhammad A ; Ying, Anita K ; Hu, Mimi I ; Bassett, Roland ; Jimenez, Camilo ; Sherman, Steven I ; Cabanillas, Maria E
descriptionContext: 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 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 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 ≥ 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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descriptionContext: 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 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 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 ≥ 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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titleEfficacy and Tolerability of Vemurafenib in Patients with BRAFV600E -Positive Papillary Thyroid Cancer: M.D. Anderson Cancer Center Off Label Experience
authorDadu, Ramona ; Shah, Komal ; Busaidy, Naifa L ; Waguespack, Steven G ; Habra, Mouhammad A ; Ying, Anita K ; Hu, Mimi I ; Bassett, Roland ; Jimenez, Camilo ; Sherman, Steven I ; Cabanillas, Maria E
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atitleEfficacy and Tolerability of Vemurafenib in Patients with BRAFV600E -Positive Papillary Thyroid Cancer: M.D. Anderson Cancer Center Off Label Experience
jtitleThe journal of clinical endocrinology and metabolism
date2015-01
risdate2015
volume100
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pagesE77-E81
issn0021-972X
eissn1945-7197
notesThis work was supported by the NIH/NCI under Award No. P30CA016672.
abstractContext: 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 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 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 ≥ 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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pmid25353071
doi10.1210/jc.2014-2246
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