Dabrafenib plus trametinib treatment in patients with anaplastic thyroid carcinoma: an Argentinian experience

Endocrine. 2023 Apr;80(1):134-141. doi: 10.1007/s12020-022-03295-2. Epub 2023 Jan 9.

Abstract

Purpose: To present our real-life experience with dabrafenib and trametinib (D-T) treatment in patients with BRAF V600E-mutated ATC in Argentina.

Patients y methods: We included five patients from four different hospitals. The median age was 70 years, and 60% were male. The performance status at diagnosis was grade 0 in 60% and grade 2 in 40% of patients. Four patients could undergo total thyroidectomy; in one of them, surgical treatment was amenable due to the indication of D-T as neoadjuvant therapy. From the total cohort, the best response to treatment was complete response in 40%, partial response in 20%, and stable disease in 20%. The median duration of response was 20 weeks, ranging from 16 to 92 weeks. All patients experienced at least one adverse event (AE). Grade ≥3 AEs were observed in two (40%) patients. They were upper gastrointestinal bleeding and subclavian vein thrombosis. The median follow-up was 20 weeks (range: 16 to 92).

Conclusion: This report contributes to illustrate the feasibility and effectiveness of D-T treatment in five patients with loco-regionally advanced and metastatic BRAF V600E-mutated ATC in a real-life setting. A multidisciplinary approach and rapid molecular-tailored testing are essential to begin this therapeutic option.

Keywords: Anaplastic thyroid carcinoma; Dabrafenib; Real-life experience; Targeted therapy; Trametinib.

MeSH terms

  • Aged
  • Argentina
  • Female
  • Humans
  • Male
  • Mutation
  • Proto-Oncogene Proteins B-raf / genetics
  • Pyridones / adverse effects
  • Pyridones / therapeutic use
  • Thyroid Carcinoma, Anaplastic* / drug therapy
  • Thyroid Carcinoma, Anaplastic* / pathology
  • Thyroid Neoplasms* / drug therapy

Substances

  • dabrafenib
  • trametinib
  • Proto-Oncogene Proteins B-raf
  • Pyridones