BMC Medicine (Mar 2023)

Association between progression-free survival and overall survival in women receiving first-line treatment for metastatic breast cancer: evidence from the ESME real-world database

  • Coralie Courtinard,
  • Sophie Gourgou,
  • William Jacot,
  • Matthieu Carton,
  • Olivier Guérin,
  • Laure Vacher,
  • Aurélie Bertaut,
  • Marie-Cécile Le Deley,
  • David Pérol,
  • Patricia Marino,
  • Christelle Levy,
  • Lionel Uwer,
  • Geneviève Perrocheau,
  • Renaud Schiappa,
  • Florence Bachelot,
  • Damien Parent,
  • Mathias Breton,
  • Thierry Petit,
  • Thomas Filleron,
  • Agnès Loeb,
  • Simone Mathoulin-Pélissier,
  • Mathieu Robain,
  • Suzette Delaloge,
  • Carine Bellera

DOI
https://doi.org/10.1186/s12916-023-02754-5
Journal volume & issue
Vol. 21, no. 1
pp. 1 – 13

Abstract

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Abstract Background Overall survival (OS) is the gold standard endpoint to assess treatment efficacy in cancer clinical trials. In metastatic breast cancer (mBC), progression-free survival (PFS) is commonly used as an intermediate endpoint. Evidence remains scarce regarding the degree of association between PFS and OS. Our study aimed to describe the individual-level association between real-world PFS (rwPFS) and OS according to first-line treatment in female patients with mBC managed in real-world setting for each BC subtype (defined by status for both hormone-receptor [HR] expression and HER2 protein expression/gene amplification). Methods We extracted data from the ESME mBC database (NCT03275311) which gathers deidentified data from consecutive patients managed in 18 French Comprehensive Cancer Centers. Adult women diagnosed with mBC between 2008 and 2017 were included. Endpoints (PFS, OS) were described using the Kaplan–Meier method. Individual-level associations between rwPFS and OS were estimated using the Spearman’s correlation coefficient. Analyses were conducted by tumor subtype. Results 20,033 women were eligible. Median age was 60.0 years. Median follow-up duration was 62.3 months. Median rwPFS ranged from 6.0 months (95% CI 5.8–6.2) for HR-/HER2 − subtype to 13.3 months (36% CI 12.7–14.3) for HR + /HER2 + subtype. Correlation coefficients were highly variable across subtypes and first-line (L1) treatments. Among patients with HR − /HER2 − mBC, correlation coefficients ranged from 0.73 to 0.81, suggesting a strong rwPFS/OS association. For HR + /HER2 + mBC patients, the individual-level associations were weak to strong with coefficients ranging from 0.33 to 0.43 for monotherapy and from 0.67 to 0.78 for combined therapies. Conclusions Our study provides comprehensive information on individual-level association between rwPFS and OS for L1 treatments in mBC women managed in real-life practice. Our results could be used as a basis for future research dedicated to surrogate endpoint candidates.

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