TP53 mutational status and cetuximab benefit in rectal cancer: 5-year results of the EXPERT-C trial

J Natl Cancer Inst. 2014 Jun 23;106(7):dju121. doi: 10.1093/jnci/dju121. Print 2014 Jul.

Abstract

In this updated analysis of the EXPERT-C trial we show that, in magnetic resonance imaging-defined, high-risk, locally advanced rectal cancer, adding cetuximab to a treatment strategy with neoadjuvant CAPOX followed by chemoradiotherapy, surgery, and adjuvant CAPOX is not associated with a statistically significant improvement in progression-free survival (PFS) and overall survival (OS) in both KRAS/BRAF wild-type and unselected patients. In a retrospective biomarker analysis, TP53 was not prognostic but emerged as an independent predictive biomarker for cetuximab benefit. After a median follow-up of 65.0 months, TP53 wild-type patients (n = 69) who received cetuximab had a statistically significant better PFS (89.3% vs 65.0% at 5 years; hazard ratio [HR] = 0.23; 95% confidence interval [CI] = 0.07 to 0.78; two-sided P = .02 by Cox regression) and OS (92.7% vs 67.5% at 5 years; HR = 0.16; 95% CI = 0.04 to 0.70; two-sided P = .02 by Cox regression) than TP53 wild-type patients who were treated in the control arm. An interaction between TP53 status and cetuximab effect was found (P < .05) and remained statistically significant after adjusting for statistically significant prognostic factors and KRAS.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Cetuximab
  • Chemotherapy, Adjuvant
  • Clinical Trials, Phase II as Topic
  • Disease-Free Survival
  • Female
  • Follow-Up Studies
  • Humans
  • Kaplan-Meier Estimate
  • Magnetic Resonance Imaging
  • Male
  • Middle Aged
  • Mutation*
  • Neoadjuvant Therapy / methods*
  • Odds Ratio
  • Radiotherapy, Adjuvant
  • Randomized Controlled Trials as Topic
  • Rectal Neoplasms / drug therapy*
  • Rectal Neoplasms / genetics*
  • Rectal Neoplasms / pathology
  • Rectal Neoplasms / surgery
  • Retrospective Studies
  • Risk Factors
  • Sample Size
  • Tumor Suppressor Protein p53 / genetics*

Substances

  • Antibodies, Monoclonal, Humanized
  • TP53 protein, human
  • Tumor Suppressor Protein p53
  • Cetuximab