ETA2023 Poster Presentations Treatment 2 (9 abstracts)
Dabrafenib and trametinib treatment in patients with braf-mutated advanced thyroid cancer
Lea Contartese 1 , Alessandro Prete 2 , Carla Gambale 2 , Liborio Torregrossa 3 , Piermarco Papini 4 , Luigi De Napoli 4 , Elisa Minaldi 2 , Gabriele Materazzi 4 , Eleonora Molinaro 2 , Rossella Elisei 2 & Antonio Matrone 2
1University of Pisa, Department of Clinical and Experimental Medicine, Unit of Endocrinology, Pisa, Italy; 2Unit of Endocrinology, Department of Clinical and Experimental Medicine, University Hospital of Pisa; 3Anatomic Pathology Section, Department of Surgical Medical and Molecular Pathology and Critical Area, University Hospital of Pisa; 4Unit of Endocrine Surgery, Department of Surgical Medical and Molecular Pathology and of Critical Area, University Hospital of Pisa
Introduction: The combination of dabrafenib (BRAF inhibitor) and trametinib (MEK inhibitor) was approved by the Food and Drug Administration in USA as first line treatment for BRAF-positive anaplastic thyroid cancer. Moreover, it was also available as second line treatment in BRAF-positive advanced thyroid cancers, previously treated with other systemic treatments.
Purpose: We evaluated the efficacy and safety of the combination therapy with dabrafenib and trametinib (D+T) in BRAF-mutated iodorefractory thyroid cancers (TC/BRAF+) and anaplastic thyroid cancers (ATC/BRAF+), followed at the Unit of Endocrinology of Pisa University Hospital.
Patients and Methods: We assessed the Best Overall Response (BOR) and the clinical outcome of 5 TC/BRAF+ patients and 2 ATC/BRAF+ patients, treated with D+T. All patients with TC/BRAF+ had an advanced, metastatic and progressive disease: 4 of them were already treated with total thyroidectomy and radioiodine, and all performed at least one MKI (lenvatinib=3, sorafenib+lenvatinib=1, Lenvatinib+cabozantinib=1). Patients with ATC/BRAF+ were inoperable: both received prior radiation therapy and one of them also MKI (lenvatinib).
Results: Mean follow-up time was 5.6 months. Total body CT scan with i.v. contrast was used for the imaging assessment over time. Six patients performed at least one imaging evaluation after the beginning of D+T, while 1 patient died before the first imaging evaluation. One patient with ATC/BRAF+, showed a partial response during D+T, without relevant adverse events over time, and is still alive after 18 months of treatment. The other patient with ATC/BRAF+, showed a significant shrinkage of the neck mass but at the same time a progression on the metastatic lung lesions, and died because of disease progression. Four TC/BRAF+ patients showed progression of the disease on D+T and only one of them experienced a stable disease, but all of them died.
Conclusions: In our clinical experience, the combination therapy with D+T showed poor efficacy in controlling the disease of TC/BRAF+ patients. Conversely, better results were obtained in patients with ATC/BRAF+, particularly in the control of the disease in the neck. Larger studies are needed to better clarify the real efficacy of this treatment.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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