Real word outcomes of cabozantinib therapy in poorly differentiated thyroid carcinoma

Objective: Poorly differentiated thyroid carcinomas (PDTCs) are rare and aggressive head and neck malignancies with a poor prognosis. Systemic treatment for incurable PDTC consists of multi-kinase inhibitors (MKIs) based on extrapolation from the experience with radioiodine refractory differentiated...

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Main Authors: Omar Elghawy, Adam Barsouk, Jessica Xu, Simon Chen, Roger B Cohen, Lova Sun
Format: Article
Language:English
Published: Bioscientifica 2024-11-01
Series:European Thyroid Journal
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Online Access:https://etj.bioscientifica.com/view/journals/etj/13/6/ETJ-24-0225.xml
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author Omar Elghawy
Adam Barsouk
Jessica Xu
Simon Chen
Roger B Cohen
Lova Sun
author_facet Omar Elghawy
Adam Barsouk
Jessica Xu
Simon Chen
Roger B Cohen
Lova Sun
author_sort Omar Elghawy
collection DOAJ
description Objective: Poorly differentiated thyroid carcinomas (PDTCs) are rare and aggressive head and neck malignancies with a poor prognosis. Systemic treatment for incurable PDTC consists of multi-kinase inhibitors (MKIs) based on extrapolation from the experience with radioiodine refractory differentiated thyroid cancer (DTC). Cabozantinib is an approved second-line MKI therapy for DTC, but there are limited data regarding the safety and efficacy of cabozantinib for PDTC. Methods: We conducted a single-institution, retrospective analysis of patients with PDTC who received cabozantinib in any line of therapy. Baseline demographics, disease characteristics, treatment history, toxicity, and clinical outcomes were abstracted from the electronic medical record. Median progression-free survival (PFS) and overall survival (OS) were primary endpoints and estimated using Kaplan–Meier methodology. Results: Seven patients with PDTC who received cabozantinib were included. 4/7 (57%) patients had a partial response to cabozantinib, while 2/7 (29%) had stable disease (SD) as their best response. The median time on treatment for cabozantinib was 10.53 months. The median PFS from the start of cabozantinib was 12.9 months, and median OS was 14.21 months. Most adverse events to treatment (5/6) were low grade. Two (29%) patients were alive at the date of the last follow-up. Conclusion: Cabozantinib is an effective and reasonably well-tolerated treatment option for patients with PDTC. Prospective studies are needed to further investigate the role of cabozantinib in the treatment of PDTC, alone and in combination with other agents, including checkpoint inhibitors.
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spelling doaj-art-c9efc544eace433cb1d56d670fcf23a32024-11-26T10:00:23ZengBioscientificaEuropean Thyroid Journal2235-08022024-11-0113617https://doi.org/10.1530/ETJ-24-0225Real word outcomes of cabozantinib therapy in poorly differentiated thyroid carcinomaOmar Elghawy0Adam Barsouk1Jessica Xu2Simon Chen3Roger B Cohen4Lova Sun5Division of Hematology/Oncology, Department of Medicine, Abramson Cancer Center, University of Pennsylvania, Philadelphia, Pennsylvania, USADivision of Hematology/Oncology, Department of Medicine, Abramson Cancer Center, University of Pennsylvania, Philadelphia, Pennsylvania, USADivision of Hematology/Oncology, Department of Medicine, Abramson Cancer Center, University of Pennsylvania, Philadelphia, Pennsylvania, USADepartment of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USADivision of Hematology/Oncology, Department of Medicine, Abramson Cancer Center, University of Pennsylvania, Philadelphia, Pennsylvania, USADivision of Hematology/Oncology, Department of Medicine, Abramson Cancer Center, University of Pennsylvania, Philadelphia, Pennsylvania, USAObjective: Poorly differentiated thyroid carcinomas (PDTCs) are rare and aggressive head and neck malignancies with a poor prognosis. Systemic treatment for incurable PDTC consists of multi-kinase inhibitors (MKIs) based on extrapolation from the experience with radioiodine refractory differentiated thyroid cancer (DTC). Cabozantinib is an approved second-line MKI therapy for DTC, but there are limited data regarding the safety and efficacy of cabozantinib for PDTC. Methods: We conducted a single-institution, retrospective analysis of patients with PDTC who received cabozantinib in any line of therapy. Baseline demographics, disease characteristics, treatment history, toxicity, and clinical outcomes were abstracted from the electronic medical record. Median progression-free survival (PFS) and overall survival (OS) were primary endpoints and estimated using Kaplan–Meier methodology. Results: Seven patients with PDTC who received cabozantinib were included. 4/7 (57%) patients had a partial response to cabozantinib, while 2/7 (29%) had stable disease (SD) as their best response. The median time on treatment for cabozantinib was 10.53 months. The median PFS from the start of cabozantinib was 12.9 months, and median OS was 14.21 months. Most adverse events to treatment (5/6) were low grade. Two (29%) patients were alive at the date of the last follow-up. Conclusion: Cabozantinib is an effective and reasonably well-tolerated treatment option for patients with PDTC. Prospective studies are needed to further investigate the role of cabozantinib in the treatment of PDTC, alone and in combination with other agents, including checkpoint inhibitors.https://etj.bioscientifica.com/view/journals/etj/13/6/ETJ-24-0225.xmlpoorly differentiated thyroid cancerthyroid cancercabozantiniblenvatinibsorafenib
spellingShingle Omar Elghawy
Adam Barsouk
Jessica Xu
Simon Chen
Roger B Cohen
Lova Sun
Real word outcomes of cabozantinib therapy in poorly differentiated thyroid carcinoma
European Thyroid Journal
poorly differentiated thyroid cancer
thyroid cancer
cabozantinib
lenvatinib
sorafenib
title Real word outcomes of cabozantinib therapy in poorly differentiated thyroid carcinoma
title_full Real word outcomes of cabozantinib therapy in poorly differentiated thyroid carcinoma
title_fullStr Real word outcomes of cabozantinib therapy in poorly differentiated thyroid carcinoma
title_full_unstemmed Real word outcomes of cabozantinib therapy in poorly differentiated thyroid carcinoma
title_short Real word outcomes of cabozantinib therapy in poorly differentiated thyroid carcinoma
title_sort real word outcomes of cabozantinib therapy in poorly differentiated thyroid carcinoma
topic poorly differentiated thyroid cancer
thyroid cancer
cabozantinib
lenvatinib
sorafenib
url https://etj.bioscientifica.com/view/journals/etj/13/6/ETJ-24-0225.xml
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