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BRAF Inhibition in BRAFV600E-Positive Anaplastic Thyroid Carcinoma

Annette M Lim1, Graham R Taylor2, Andrew Fellowes3

  • 1Department of Medical Oncology, Sir Charles Gairdner Hospital, Nedlands, Western Australia

Journal of the National Comprehensive Cancer Network : JNCCN
|March 10, 2016
PubMed

Insights

Targeted BRAF inhibitor monotherapy for BRAF(V600E)-positive anaplastic thyroid carcinoma (ATC) showed short-term clinical benefits in two cases. Further research into upfront combination therapy is recommended for this patient subgroup.

Area of Science:

  • Oncology
  • Molecular Biology
  • Genetics

Background:

  • Anaplastic thyroid carcinoma (ATC) is an aggressive thyroid cancer.
  • The efficacy of targeted monotherapy for BRAF(V600E)-positive ATC remains unclear.
  • BRAF mutations are present in a subset of ATC patients.

Observation:

  • Two patients with BRAF(V600E)-positive ATC were treated with BRAF inhibitor monotherapy.
  • One patient experienced a complete symptomatic and radiologic response within one month.
  • The second patient showed tumor size reduction and pain stabilization within ten days.

Findings:

  • BRAF inhibitor monotherapy provided temporary clinical benefit in both ATC cases.
  • Disease progression occurred after 3 months in the first patient and stable disease lasted 11 weeks in the second.
  • Both patients ultimately succumbed to the disease within 11 months of diagnosis.

Implications:

  • BRAF inhibitor monotherapy may offer transient benefits for BRAF(V600E)-positive ATC.
  • Upfront combination therapy strategies should be explored for this patient population.
  • This highlights the need for novel therapeutic approaches in advanced ATC.

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