Related Experiment Video

Updated: May 11, 2026

A Mouse Model of Incompletely Resected Soft Tissue Sarcoma for Testing (Neo)adjuvant Therapies
07:15

A Mouse Model of Incompletely Resected Soft Tissue Sarcoma for Testing (Neo)adjuvant Therapies

Published on: July 28, 2020

Activity endpoints reported in soft tissue sarcoma phase II trials: quality of reported endpoints and correlation

Nicolas Penel1, Sophie Cousin, Alain Duhamel

  • 1General Oncology Department, Centre Oscar Lambret, Lille, France; Unit Research (EA 2694), Medical School, Lille-Nord-de-France University, Lille, France.

Critical Reviews in Oncology/Hematology
|May 28, 2013
PubMed
Abstract

Insights

Investigational drugs for advanced soft tissue sarcoma often fail to improve survival due to flawed trial designs and unclear endpoints. Refining these elements is crucial for future drug development success.

Area of Science:

  • Oncology
  • Clinical Trials
  • Drug Development

Background:

  • Extensive research over 3 decades has yielded few promising investigational drugs for advanced soft tissue sarcoma.
  • Many investigational drugs have failed to demonstrate significant improvements in overall survival.

Purpose of the Study:

  • To systematically review literature and understand reasons for trial failures in advanced soft tissue sarcoma.
  • To critically evaluate primary endpoints used in phase II trials.

Main Methods:

  • Systematic review of 53 phase II trial reports for advanced soft tissue sarcoma (1999-2011).
  • Critical analysis of selected primary endpoints in these trials.

Main Results:

  • 40% of trials were uninterpretable due to methodological flaws.
  • Only progression-free survival metrics (3- and 6-month rates, median PFS) correlated with overall survival.
  • Median overall survival was not significantly higher for active drugs.

Conclusions:

  • Improvement needed in defining primary active endpoints for clinical trials.
  • Development of better trial designs is essential for future research.
  • The definition of "promising drugs" requires refinement.
Keywords:
3-month progression free rate6-month progression free rateActivity endpointAdvanced soft tissue sarcomaBORRBTCRCorrelationEORTCEuropean Organization on Research and Treatment of CancerPFR3PFR6Phase II trialsbest objective response ratebest tumor control ratemOSmPFSmedian overall survivalmedian progression free survival

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