ETA2024 Oral Presentations Oral Session 1: Topic Highlights (6 abstracts)
Dabrafenib and trametinib in BRAFV600E anaplastic thyroid cancers: an italian real-word series
Rossella Elisei 1 , Durante Cosimo 2 , Colombo Elena 3 , Leli Claudia 4 , Salvatore Domenico 5 , Fugazzola Laura 6 , Bossi Paolo 7 , Zovato Stefania 8 , Magri Flavia 4 , Malovini Alberto 4 & Locati Laura Deborah 4
1Oncology Section of the Endocrine Unit, Department of Clin and Exp Medicine, University Pisa, Pisa, Italy; 2Sapienza University of Rome, Department of Translational and Precision Medicine, Roma, Italy; 3Head and Neck Medical Oncology Unit, Fondazione Irccs Istituto Nazionale Dei Tumori di Milano, Milano, Italy; 4Irccs Fondazione Salvatore Maugeri, Pavia, Italy; 5University of Naples "Federico Ii", Department of Molecular and Clinical Endocrinology and Oncology, Napoli, Italy; 6Irccs Istituto Auxologico Italiano, Department of Endocrine and Metabolic Diseases, Milano, Italy; 7Humanitas University, Rozzano, Italy; 8Uosd Hereditary Tumors, Istituto Oncologico Veneto, Padova, Italy
Background: Anaplastic thyroid cancer (ATC) is a lethal tumor with a median overall survival of 6 months. In about 30-40% of cases, a BRAFV600E mutation is present. In 2018 dabrafenib and trametinib (D/T) have been approved in USA for BRAFV600E-mutated ATC, while no approval has been obtained so far in Europe. Up to December 2022, D/T were provided by the company for BRAF-altered cancers within a compassionate named program. Here we report the results from an Italian cohort of ATC patients.
Methods: We retrospectively collected data from 8 Italian centers. Primary endpoint was the overall response rate (ORR) per RECIST 1.1 according to investigator judgment; secondary aims were progression-free survival (PFS); overall survival (OS) and treatment safety. D (150 mg twice daily) and T (2 mg daily) were administered until disease progression, toxicity, or patient's withdrawal of consent. PFS and OS were calculated by Kaplan-Meier analysis.
Results: Between May 2018 and December 2022, 19 ATC patients received D/T. M/F were 10/9; median age at diagnosis was 69 years (range: 40-80 years). 16 cases (84%) were pure ATC, while 3 were characterized by a mixed histology (papillary thyroid cancer + ATC). BRAF V600E has been assessed either through RT-PCR (42%) or NGS (58%). Stage IVC was present in 63% of patients at diagnosis. When D/T was started, 14 patients (74%) were treatment naïve; 5 patients had only locoregional disease, whereas 14 had distant metastases in lungs, liver, bone, lymph nodes or other sites. ORR was 74% (14 PR), whereas disease stability (SD) was achieved in 3 patients and 2 experienced disease progression (PD) at the first evaluation. Treatment withdrawn occurred due to PD in 13 patients (65%), death (10%), toxicity (5%), or treatment-unrelated complications (5%). Treatment was well tolerated, with the most common grade ≥ 3 adverse events being fever (15%), diarrhea (5%), and pneumonia (5%). Median (m) OS was 21 months (9.9 months-NA); mPFS for naïve patients and pretreated was 8.3 (7.2 months-NA) and 3.6 (2.3 months-NA), respectively. At the time of cut-off analysis (April 2023), 5 patients were still alive, with 3 under D+T.
Conclusions: This is the largest Italian series of BRAFV600E ATC patients treated with D/T. The drug combination confirms its activity, especially in ATC naïve patients, suggesting the use of D/T in the early phase of disease. Since March 2023, D/T has been approved for BRAFV600E ATC patients in Italy (law 648/96).
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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