Verrucous esophageal carcinoma is a unique indolent subtype of squamous cell carcinoma: a systematic review and individual patient regression analysis

J Gastroenterol. 2021 Jan;56(1):12-24. doi: 10.1007/s00535-020-01736-1. Epub 2020 Oct 20.

Abstract

Background and aims: Verrucous esophageal carcinoma (VEC) is a rare malignancy that presents a diagnostic challenge. We aim to characterize the clinical and genomic features, tumor behavior, and treatment outcomes of VEC to guide clinical practice.

Methods: We performed a systematic review of the literature and identified additional cases from Massachusetts General Hospital records and The Cancer Genome Atlas (TCGA). We obtained individual VEC patient data and analyzed publicly available clinicogenomic data from TCGA. We performed a regression analysis comparing cases of VEC to esophageal squamous cell carcinoma (ESCC) to identify factors influencing survival.

Results: A total of 135 patients were reported in 82 publications, and four unpublished cases from Massachusetts General Hospital (median age 65 years, 69% males, 48% smokers, 33% consumed alcohol). Symptoms were present at diagnosis in 95% of patients, most commonly dysphagia and weight loss. Median symptom onset to diagnosis time was 11.5 months with frequent misdiagnosis as Candida esophagitis. Among VEC cases with pathologic staging, lymph node metastases were rare (5%) compared to ESCC (40%). VEC was genomically characterized by enrichment of SMARCA4 missense mutations and a lack of pathogenic TP53 mutations. Despite its diagnostic elusiveness, in a multivariate regression analysis, VEC was detected at earlier stages (p = < 0.001) compared to ESCC, and advanced stage was the only significant factor affecting survival (p = 0.013).

Conclusions: VEC is a rare, clinically and genomically distinct subtype of ESCC. Recognition and diagnosis of this lesion may allow the pursuit of curative and less morbid treatment strategies.

Keywords: Endoscopic submucosal dissection; Esophageal squamous cell carcinoma; Verrucous esophageal carcinoma.

Publication types

  • Systematic Review

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Biomarkers, Tumor / genetics
  • Carcinoma, Verrucous* / diagnosis
  • Carcinoma, Verrucous* / genetics
  • Carcinoma, Verrucous* / mortality
  • Carcinoma, Verrucous* / therapy
  • Combined Modality Therapy
  • DNA Helicases / genetics
  • Early Detection of Cancer
  • Esophageal Neoplasms* / diagnosis
  • Esophageal Neoplasms* / genetics
  • Esophageal Neoplasms* / mortality
  • Esophageal Neoplasms* / therapy
  • Esophageal Squamous Cell Carcinoma* / diagnosis
  • Esophageal Squamous Cell Carcinoma* / genetics
  • Esophageal Squamous Cell Carcinoma* / mortality
  • Esophageal Squamous Cell Carcinoma* / therapy
  • Female
  • Follow-Up Studies
  • Humans
  • Male
  • Middle Aged
  • Mutation, Missense
  • Nuclear Proteins / genetics
  • Regression Analysis
  • Survival Analysis
  • Transcription Factors / genetics
  • Treatment Outcome
  • Tumor Suppressor Protein p53 / genetics

Substances

  • Biomarkers, Tumor
  • Nuclear Proteins
  • TP53 protein, human
  • Transcription Factors
  • Tumor Suppressor Protein p53
  • SMARCA4 protein, human
  • DNA Helicases