Larynx Preservation Clinical Trial Design: Key Issues and Recommendations-A Consensus Panel Summary

Jean Louis Lefebvre, Kie-Kian Ang

Research output: Contribution to journalArticle

97 Citations (Scopus)

Abstract

Purpose: To develop guidelines for the conduct of Phase III clinical trials of larynx preservation in patients with locally advanced laryngeal and hypopharyngeal cancer. Methods and Materials: A multidisciplinary international consensus panel developed recommendations after reviewing results from completed Phase III randomized trials, meta-analyses, and published clinical reports with updates available through November, 2007. The guidelines were reviewed and approved by the panel. Results: According to the recommendations, the trial population should include patients with T2 or T3 laryngeal or hypopharyngeal squamous cell carcinoma not considered for partial laryngectomy and exclude those with laryngeal dysfunction or age greater than 70 years. Functional assessments should include speech and swallowing. Voice should be routinely assessed with a simple, validated instrument. The primary endpoint should capture survival and function. The panel created a new endpoint: laryngo-esophageal dysfunction-free survival. Events are death, local relapse, total or partial laryngectomy, tracheotomy at 2 years or later, or feeding tube at 2 years or later. Recommended secondary endpoints are overall survival, progression-free survival, locoregional control, time to tracheotomy, time to laryngectomy, time to discontinuation of feeding tube, and quality of life/patient-reported outcomes. Correlative biomarker studies for near-term trials should include estimated glomerular filtration rate, excision repair cross-complementary-1 gene, E-cadherin and β-catenin, epiregulin and amphiregulin, and TP53 mutation. Conclusions: Revised trial designs in several key areas are needed to advance the study of larynx preservation. With consistent methodologies, clinical trials can more effectively evaluate and quantify the therapeutic benefit of novel treatment options for patients with locally advanced laryngeal and hypopharyngeal cancer.

Original languageEnglish (US)
Pages (from-to)1293-1303
Number of pages11
JournalInternational Journal of Radiation Oncology Biology Physics
Volume73
Issue number5
DOIs
StatePublished - Apr 1 2009

Fingerprint

larynx
Laryngectomy
Larynx
Hypopharyngeal Neoplasms
recommendations
Consensus
Tracheotomy
Laryngeal Neoplasms
Clinical Trials
Enteral Nutrition
Survival
cancer
Guidelines
Catenins
Phase III Clinical Trials
Cadherins
Deglutition
tubes
Glomerular Filtration Rate
DNA Repair

Keywords

  • Guidelines
  • Head and neck carcinoma
  • Hypopharyngeal carcinoma
  • Laryngeal carcinoma
  • Larynx preservation

ASJC Scopus subject areas

  • Radiation
  • Oncology
  • Radiology Nuclear Medicine and imaging
  • Cancer Research

Cite this

Larynx Preservation Clinical Trial Design : Key Issues and Recommendations-A Consensus Panel Summary. / Lefebvre, Jean Louis; Ang, Kie-Kian.

In: International Journal of Radiation Oncology Biology Physics, Vol. 73, No. 5, 01.04.2009, p. 1293-1303.

Research output: Contribution to journalArticle

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