Highlights
- •Anaplastic thyroid carcinoma is poorly responsive to chemotherapy and highly lethal.
- •BRAF/MEKi and immunotherapy have shown promising results in certain subsets of ATC.
- •International guidelines recommend molecular testing to address appropriate therapy.
- •In Europe, no innovative treatments have been approved recently for advanced ATC.
- •This survey captures the diversified scenario of ATC diagnosis/therapy across E.U.
Abstract
Anaplastic thyroid carcinoma (ATC) is a rare cancer accounting for 40% of thyroid
cancer-specific deaths. In the last 5 years, improved insights into molecular pathways
led the Food and Drug Administration to license BRAF/MEK inhibitors (B/Mi) in BRAFV600E-mutant ATC, and pembrolizumab in solid cancer with high tumour mutational burden
(TMB-H) (≥10 mutations/megabase) (mut/Mb). In Europe, clinicians face challenges in
prescribing novel treatments, as the European Medical Association (EMA) has not licensed
B/Mi nor immunotherapy (IO) for ATC so far. Some patients manage to receive these
drugs through alternative ways. We investigated the extent of this phenomenon launching
an online survey from March 12th to 19th 2021 open to 239 Institutions in the EORTC
Endocrine and Head & Neck Cancer Groups. Questions enquired about the number of ATC
patients evaluated/year, feasibility of BRAF assessment, accessibility to B/Mi-IO, availability of clinical trials and interest
in new studies. Colleagues from 94 Institutions (20 Countries) joined: 30 centres
evaluated ≥5 ATC patients/year, with an overall incidence >200 patients/year. 80.8%
tested BRAF status, 43.6% by next-generation sequencing. 62.7% and 70% of responders reported
limitations in prescribing B/Mi and IO, respectively: either the impossibility of
offering them, or drugs accessibility exclusively under certain conditions (e.g. health
insurance, clinical trials, compassionate use, off-label). Only 13.8% had clinical
trials ongoing while 91.5% of sites claimed ATC-dedicated trials. Disparities in access
to novel treatments are diffuse. Access to cutting-edge therapies is an urgent issue
in this setting, and clinical trials seem feasible within an appropriate network.
Keywords
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Article info
Publication history
Published online: January 02, 2023
Accepted:
December 1,
2022
Received in revised form:
December 1,
2022
Received:
November 12,
2022
Identification
Copyright
© 2022 Elsevier Ltd. All rights reserved.
