Phase II Trial of Target-guided Personalized Chemotherapy in First-line Metastatic Colorectal Cancer

Am J Clin Oncol. 2016 Jun;39(3):236-42. doi: 10.1097/COC.0000000000000045.

Abstract

Purpose: The aim of this study was to investigate the feasibility and efficacy of personalizing treatment of patients with advanced untreated colorectal cancer (CRC).

Patients and methods: Patients with untreated metastatic CRC, performance status 0-1, and candidates for systemic chemotherapy were eligible. Tumor tissues were analyzed for KRAS, BRAF, and PI3K mutations and expression of topoisomerase-1 (Topo-1), excision repair cross-complementing gene 1 (ERCC1), thymidylate synthase (TS), and thymidine phosphorylase (TP). Patients with Topo-1 expression received irinotecan, whereas patients with negative Topo-1 and ERCC1 expression received oxaliplatin. Otherwise, patients received physician's choice of treatment. If TS was positive, no fluoropyrimidine was administered and if negative, 5-flurorouracil if TP was negative, or capecitabine if TP was positive. KRAS-mutated patients were treated with bevacizumab, whereas KRAS-native received cetuximab. The primary endpoint of the study was progression-free survival (PFS).

Results: A total of 74 patients were enrolled and 67 received personalized treatment including irinotecan (n=27), oxaliplatin (n=16), FOLFIRI (n=12), and FOLFOX (n=12). Thirty-eight patients received cetuximab and 29 bevacizumab. With a median follow-up time of 18.3 months (95% confidence interval [CI], 4-36), the overall median PFS was 8.3 months (95% CI, 6.9-9.7), representing a 12-month PFS rate of 36.5% (95% CI, 25-48). Overall clinical benefit, including response rate and disease stabilization, was 86% (95% CI, 73%-97%). The overall median survival was 21 months (95% CI, 11-40).

Conclusions: Real-time target-guided personalized first-line treatment of patients with advanced CRC is feasible but, with the approached used, did not result in a clear improvement in PFS to warrant phase III testing.

Publication types

  • Clinical Trial, Phase II

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Bevacizumab / administration & dosage
  • Camptothecin / administration & dosage
  • Camptothecin / analogs & derivatives
  • Capecitabine / administration & dosage
  • Cetuximab / administration & dosage
  • Clinical Decision-Making
  • Colorectal Neoplasms / chemistry
  • Colorectal Neoplasms / drug therapy*
  • Colorectal Neoplasms / genetics
  • Colorectal Neoplasms / pathology*
  • DNA Mutational Analysis
  • DNA Topoisomerases, Type I / analysis
  • DNA-Binding Proteins / analysis
  • Decision Trees
  • Disease-Free Survival
  • Endonucleases / analysis
  • Female
  • Fluorouracil / administration & dosage
  • Humans
  • Irinotecan
  • Male
  • Middle Aged
  • Molecular Targeted Therapy*
  • Organoplatinum Compounds / administration & dosage
  • Oxaliplatin
  • Phosphatidylinositol 3-Kinase / genetics
  • Precision Medicine*
  • Proto-Oncogene Proteins B-raf / genetics
  • Proto-Oncogene Proteins p21(ras) / genetics
  • Response Evaluation Criteria in Solid Tumors
  • Thymidine Phosphorylase / analysis
  • Thymidylate Synthase / analysis

Substances

  • DNA-Binding Proteins
  • KRAS protein, human
  • Organoplatinum Compounds
  • Oxaliplatin
  • Bevacizumab
  • Capecitabine
  • Irinotecan
  • Thymidylate Synthase
  • Thymidine Phosphorylase
  • Phosphatidylinositol 3-Kinase
  • BRAF protein, human
  • Proto-Oncogene Proteins B-raf
  • ERCC1 protein, human
  • Endonucleases
  • Proto-Oncogene Proteins p21(ras)
  • DNA Topoisomerases, Type I
  • Cetuximab
  • Fluorouracil
  • Camptothecin