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Dabrafenib plus trametinib treatment in patients with anaplastic thyroid carcinoma: an Argentinian experience

PURPOSE: To present our real-life experience with dabrafenib and trametinib (D-T) treatment in patients with BRAF V600E-mutated ATC in Argentina. PATIENTS Y METHODS: We included five patients from four different hospitals. The median age was 70 years, and 60% were male. The performance status at dia...

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Autores principales: Bueno, Fernanda, Smulever, Anabella, Califano, Inés, Guerra, Jorgelina, Del Grecco, Andrés, Carrera, Juan Manuel, Giglio, Raúl, Rizzo, Manglio, Lingua, Alejo, Voogd, Ana, Negueruela, María del Carmen, Abelleira, Erika, Pitoia, Fabian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer US 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9838471/
https://www.ncbi.nlm.nih.gov/pubmed/36617605
http://dx.doi.org/10.1007/s12020-022-03295-2
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author Bueno, Fernanda
Smulever, Anabella
Califano, Inés
Guerra, Jorgelina
Del Grecco, Andrés
Carrera, Juan Manuel
Giglio, Raúl
Rizzo, Manglio
Lingua, Alejo
Voogd, Ana
Negueruela, María del Carmen
Abelleira, Erika
Pitoia, Fabian
author_facet Bueno, Fernanda
Smulever, Anabella
Califano, Inés
Guerra, Jorgelina
Del Grecco, Andrés
Carrera, Juan Manuel
Giglio, Raúl
Rizzo, Manglio
Lingua, Alejo
Voogd, Ana
Negueruela, María del Carmen
Abelleira, Erika
Pitoia, Fabian
author_sort Bueno, Fernanda
collection PubMed
description PURPOSE: To present our real-life experience with dabrafenib and trametinib (D-T) treatment in patients with BRAF V600E-mutated ATC in Argentina. PATIENTS Y METHODS: We included five patients from four different hospitals. The median age was 70 years, and 60% were male. The performance status at diagnosis was grade 0 in 60% and grade 2 in 40% of patients. Four patients could undergo total thyroidectomy; in one of them, surgical treatment was amenable due to the indication of D-T as neoadjuvant therapy. From the total cohort, the best response to treatment was complete response in 40%, partial response in 20%, and stable disease in 20%. The median duration of response was 20 weeks, ranging from 16 to 92 weeks. All patients experienced at least one adverse event (AE). Grade ≥3 AEs were observed in two (40%) patients. They were upper gastrointestinal bleeding and subclavian vein thrombosis. The median follow-up was 20 weeks (range: 16 to 92). CONCLUSION: This report contributes to illustrate the feasibility and effectiveness of D-T treatment in five patients with loco-regionally advanced and metastatic BRAF V600E-mutated ATC in a real-life setting. A multidisciplinary approach and rapid molecular-tailored testing are essential to begin this therapeutic option.
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spelling pubmed-98384712023-01-17 Dabrafenib plus trametinib treatment in patients with anaplastic thyroid carcinoma: an Argentinian experience Bueno, Fernanda Smulever, Anabella Califano, Inés Guerra, Jorgelina Del Grecco, Andrés Carrera, Juan Manuel Giglio, Raúl Rizzo, Manglio Lingua, Alejo Voogd, Ana Negueruela, María del Carmen Abelleira, Erika Pitoia, Fabian Endocrine Original Paper PURPOSE: To present our real-life experience with dabrafenib and trametinib (D-T) treatment in patients with BRAF V600E-mutated ATC in Argentina. PATIENTS Y METHODS: We included five patients from four different hospitals. The median age was 70 years, and 60% were male. The performance status at diagnosis was grade 0 in 60% and grade 2 in 40% of patients. Four patients could undergo total thyroidectomy; in one of them, surgical treatment was amenable due to the indication of D-T as neoadjuvant therapy. From the total cohort, the best response to treatment was complete response in 40%, partial response in 20%, and stable disease in 20%. The median duration of response was 20 weeks, ranging from 16 to 92 weeks. All patients experienced at least one adverse event (AE). Grade ≥3 AEs were observed in two (40%) patients. They were upper gastrointestinal bleeding and subclavian vein thrombosis. The median follow-up was 20 weeks (range: 16 to 92). CONCLUSION: This report contributes to illustrate the feasibility and effectiveness of D-T treatment in five patients with loco-regionally advanced and metastatic BRAF V600E-mutated ATC in a real-life setting. A multidisciplinary approach and rapid molecular-tailored testing are essential to begin this therapeutic option. Springer US 2023-01-09 2023 /pmc/articles/PMC9838471/ /pubmed/36617605 http://dx.doi.org/10.1007/s12020-022-03295-2 Text en © The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature 2023, Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law. This article is made available via the PMC Open Access Subset for unrestricted research re-use and secondary analysis in any form or by any means with acknowledgement of the original source. These permissions are granted for the duration of the World Health Organization (WHO) declaration of COVID-19 as a global pandemic.
spellingShingle Original Paper
Bueno, Fernanda
Smulever, Anabella
Califano, Inés
Guerra, Jorgelina
Del Grecco, Andrés
Carrera, Juan Manuel
Giglio, Raúl
Rizzo, Manglio
Lingua, Alejo
Voogd, Ana
Negueruela, María del Carmen
Abelleira, Erika
Pitoia, Fabian
Dabrafenib plus trametinib treatment in patients with anaplastic thyroid carcinoma: an Argentinian experience
title Dabrafenib plus trametinib treatment in patients with anaplastic thyroid carcinoma: an Argentinian experience
title_full Dabrafenib plus trametinib treatment in patients with anaplastic thyroid carcinoma: an Argentinian experience
title_fullStr Dabrafenib plus trametinib treatment in patients with anaplastic thyroid carcinoma: an Argentinian experience
title_full_unstemmed Dabrafenib plus trametinib treatment in patients with anaplastic thyroid carcinoma: an Argentinian experience
title_short Dabrafenib plus trametinib treatment in patients with anaplastic thyroid carcinoma: an Argentinian experience
title_sort dabrafenib plus trametinib treatment in patients with anaplastic thyroid carcinoma: an argentinian experience
topic Original Paper
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9838471/
https://www.ncbi.nlm.nih.gov/pubmed/36617605
http://dx.doi.org/10.1007/s12020-022-03295-2
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