Anaplastic Thyroid Cancer (THAP)
Overview
Anaplastic thyroid carcinoma (ATC/THAP) is the most aggressive thyroid malignancy, with a median survival of 3–6 months and near-universal lethality. On OncoTree it is a child of THYROID. ATC typically arises from dedifferentiation of a pre-existing differentiated thyroid cancer (DTC), particularly papillary thyroid carcinoma (THPA). It is characterized by a high mutation burden compared to PTC but fewer mutations than most other adult cancers.
Cohorts in the corpus
- 213 ATC regions from 179 primary ATCs, plus 34 co-occurring DTC components; 115 papillary thyroid cancer regions; 13 ATC cell lines; from 292 patients across 15 institutions (GATCI consortium). WES (n=132), WGS (n=9), SNP arrays (n=110), RNA-seq (n=24). Dataset: thyroid_gatci_2024. PMID:38412093
Recurrent alterations
- TP53 — mutations in 36.8% of ATCs vs. 0.9% of PTCs; frequency increases progressively from PTC to co-DTC (21.4%) to ATC; associated with elevated TP53 mRNA abundance (P=0.0067). PMID:38412093
- BRAF — V600E in 21.3% of ATCs vs. 50.9% of PTCs; clonal/early subclonal timing; mutually exclusive with RAS; co-occurs with PIK3CA mutations (FDR=0.034). PMID:38412093
- NRAS — recurrently mutated in ATC (top 5 by FDR); mutually exclusive with BRAF V600E. PMID:38412093
- PIK3CA — co-occurs with BRAF V600E (FDR=0.034); preferentially mutated in ATCs and co-DTCs. PMID:38412093
- CDKN2A — deleted in 42% of ATCs; recurrent in co-DTCs, rare in PTCs (~5%). PMID:38412093
- BRCA2 — deleted in 33.6% of ATCs; deletion associated with better survival (HR=0.48, P=0.005). PMID:38412093
- TERT — promoter mutations correlated with TP53 mutations (FDR<0.01). PMID:38412093
- ATM, BRCA1 — recurrent somatic and germline alterations at both SNV and CNA level. PMID:38412093
- RB1 — recurrent deletions in ATCs. PMID:38412093
- USH2A — recurrently mutated (top 5 by FDR). PMID:38412093
- Mutational burden: 3.8 +/- 1.2 SNVs/Mb and 120 +/- 44 CNAs per tumor; 42 recurrently mutated genes by SeqSig (FDR<0.05). PMID:38412093
- MSK-IMPACT deep-sequencing of 33 ATC tumors found TP53 mutation in 73%, BRAF V600E in 45%, TERT promoter mutations in 73%, and enrichment of PI3K/AKT/mTOR (39%), SWI/SNF (36%), and histone-methyltransferase (24%) alterations; TERT-mutant ATCs had markedly shorter survival (median 147 vs 732 days, P=0.03) PMID:26878173
Subtypes
- Five CNA subtypes (A-E) by consensus clustering, with characteristic genomic and clinical features: subtype A enriched for older patients with better survival; subtype D enriched for male patients with metastatic disease. PMID:38412093
- Multi-region WGS (9 patients) confirmed that ATC and co-occurring DTC share a common clonal origin, with the common ancestor harboring ~95% of CNAs but only 19.1% of SNVs. PMID:38412093
- Patients with lower mutation rate (>10 SNVs/Mb) had significantly better survival (HR=0.51, P=0.002). PMID:38412093
Therapeutic landscape
- BRAF V600E-targeted therapy (dabrafenib + trametinib) FDA-approved for BRAF V600E-mutant ATC; present study mostly pre-approval, limiting survival analysis. PMID:38412093
- Recurrent BRCA1, BRCA2, and ATM alterations rationalize investigation of PARP inhibitors in ATC. PMID:38412093
- Surgery (FDR=0.0089) and radiotherapy (FDR=1.7e-5) associated with improved OS. PMID:38412093
- Germline variants in cancer predisposition genes: RECQL4 (5%), BRCA2 (n=3), FANCF (n=3). PMID:38412093
Sources
PMID:38412093 — The genomic and evolutionary landscapes of anaplastic thyroid carcinoma (Cell Reports, 2024)
PMID:26878173 — Landa et al., MSK-IMPACT sequencing of 84 PDTC and 33 ATC; ATC characterized by TP53 (73%), BRAF V600E (45%), TERT promoter (73%), and SWI/SNF (36%) alterations
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