Details

Autor(en) / Beteiligte

• Lorimer, C.
• Cheng, L.
• Chandler, R.
• Garcez, K.
• Gill, V.
• Graham, K.
• Grant, W.
• Sardo Infirri, S.
• Wadsley, J.
• Wall, L.
• Webber, N.
• Wong, K.H.
• Newbold, K.

Titel

Dabrafenib and Trametinib Therapy for Advanced Anaplastic Thyroid Cancer – Real-World Outcomes From UK Centres

Ist Teil von

• Clinical oncology (Royal College of Radiologists (Great Britain)), 2023-01, Vol.35 (1), p.e60-e66

Ort / Verlag

England: Elsevier Ltd

Erscheinungsjahr

2023

Link zum Volltext

Quelle

MEDLINE

Beschreibungen/Notizen

• Anaplastic thyroid cancer (ATC) is a rare but aggressive form of thyroid cancer with a median survival of 4 months. Recent advances in molecular profiling have shown that up to half of ATCs harbour the BRAF-V600E mutation. The aim of this study was to provide real-world data and experience on the use of combination therapy dabrafenib and trametinib in patients with BRAF-V600E-mutated advanced ATC. We retrospectively evaluated patients with confirmed BRAF-V600E-mutated ATC, defined as patients with locally advanced or metastatic ATC with no locoregional, radical treatment options. Outcomes measured were overall survival, progression-free survival, response rate, discontinuation rate, dose reduction rate and toxicity data. Seventeen patients were evaluated and the mean age was 68 years. Ten patients died by the time of censoring. The median duration of follow-up was 12 months (3–43 months). The estimated median overall survival was 6.9 months (95% confidence interval 2.46 months – upper confidence interval not reached) and the median progression-free survival was 4.7 months (95% confidence interval 1.4–7.8 months). Dose interruptions and/or reductions were common, but none of the patients had to permanently discontinue treatment because of toxicities. Severe toxicities (grades 3 and 4) were uncommon. This study supports the indication of dabrafenib and trametinib in BRAF-V600E-mutated ATC as an effective and well-tolerated treatment in an historically difficult to treat cancer. •Dabrafenib and trametinib increase survival of BRAF-mutant anaplastic thyroid cancer.•Real-world data plug gaps in evidence in rare disease group.•Largest cohort of real-world patient outcomes reported in the literature.•Dabrafenib and trametinib combination is well-tolerated.