BRAF V600E-mutated anaplastic thyroid carcinoma (ATC) and treatment with BRAF-inhibitors: Real-world data from a single-institution, still far from the cure.

Medientyp: E-Artikel

Titel: BRAF V600E-mutated anaplastic thyroid carcinoma (ATC) and treatment with BRAF-inhibitors: Real-world data from a single-institution, still far from the cure

Beteiligte: Platini, Francesca; Ortolan, Elisa; Cavalieri, Stefano; Massa, Giacomo; Filippini, Daria Maria; Alfieri, Salvatore; Bergamini, Cristiana; Resteghini, Carlo; Orlandi, Ester; Paolini, Biagio; Piazza, Cesare; Perrone, Federica; Tamborini, Elena; Ferrarini, Linda; Serafini, Mara Serena; Cappelletti, Vera; Daidone, Maria Grazia; Licitra, Lisa F.; Locati, Laura Deborah

Erschienen: American Society of Clinical Oncology (ASCO), 2020

Sprache: Englisch

DOI: 10.1200/jco.2020.38.15_suppl.e18577

ISSN: 0732-183X; 1527-7755

Schlagwörter: Cancer Research ; Oncology

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Beschreibung: e18577 Background: ATC is a very aggressive disease. In the last years new insights on molecular profile of ATC have led to novel therapeutic options, primarily BRAF-targeted agents. Methods: From May 2018 to December 2019, data on recurrent/metastatic ATC patients treated at our Institution have been reviewed. BRAF V600E was assessed in each patient. BRAF mutation was tested both on tumor sections and on cell-free DNA (cfDNA). Circulating BRAF V600E was assessed by using Droplet Digital PCR (ddPCR) Technology. BRAF wild-type (WT) patients were treated with standard chemotherapy (adriamicin or taxans or platinum-based regimens). BRAF V600E patients were treated with dabrafenib 300 mg per day and trametinib 2 mg per day (combo regimen) until progression or unacceptable toxicities. Response was assessed by RECIST 1.1; progression-free survival (PFS) and overall survival (OS) were estimated with Kaplan-Meier method and compared with log-rank test. Safety was evaluated according to CTCAE version 5.0. Results: 16 ATC patients were included. Male/female 5/11, median age was 68 years (range 43-80). Eight patients (50%) carried BRAF mutation. Best response in BRAF V600E was 4 partial response (PR), 3 stable disease (SD) and response in 1 patient has not been assessed yet. Combo was well tolerated, the most common adverse events (G2) were fatigue (14%), pyrexia (14%), nausea and vomiting (9%). Only one patient experienced drugs interruptions. Best response in BRAF WT patients was SD for 1, progressive disease for 4 and 3 patients received supportive care. Concordance between circulating BRAF status with tumor tissue was 80%. Assessment of circulating BRAF performed at baseline and at best response was available for 3 patients achieving a PR: mean reduction was -99.48% from baseline (range -99.37 – -99.66%). Median follow up was 22.8 weeks (95% CI 12.8-22.8). Median PFS was 13 weeks in BRAF V600E and 7.4 weeks in BRAF wt patients (p=0.1); in the same groups median OS was 18.4 and 7.8 weeks, respectively (p=0.051). Conclusions: Real-life data with combo regimen showed an ORR of 50%, with short PFS and OS. Reduction of circulating BRAF observed during treatment might be an indirect marker of tumor shrinkage, even if more data are needed to correlate circulating BRAF and disease response.

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