Survival Endpoints in Women Treated for Metastatic Breast Cancer: Contribution of Real-life Databases

Survival Endpoints for Treatment Evaluation in Subjects Treated for Metastatic Breast Cancer: Contribution of Real-life Databases

Overall survival (OS) is considered the most reliable cancer endpoint and used by the Health Rregulatory authorities (HRA). OS presents multiple advantages in cancer randomized controlled trials (RCT): it is universally accepted as a measure of clinical benefit for the patient; it is objectively defined, both in terms of events and date of incidence; it is easily and precisely measured and thus reproducible; it can be exhaustively collected. As such, OS has been validated by HRAs. On the other hand, OS presents some limitations. Observing a benefit on OS may require a large number of patients and/or considerable time for patient follow-up. Costs for trials may be increased, and there might be delays in the introduction of possible beneficial treatments for patients. The development of alternative endpoints that could capture treatment benefit appropriately and be measurable earlier, is central for the evolution of clinical research in oncology.

Real world data (RWD) are defined as other sources than clinical trials such as: electronic medical records, registries, insurance claims, pharmacy records, death certificates and other patient-generated data.

This research is aimed at (i) describing the existing endpoints of survival in real-life setting, (ii) comparing the correlation at individual level with data to clinical trials for related to anti-HER2 targeted therapies and endocrine therapies in MBC. We will investigate the individual correlation between candidate surrogate endpoints and overall survival in a population-based record-computerized database centralizing data on about 20,000 patients from 2008 to 2017 in France.

This work should lead to the estimation of various time-to event endpoints (e.g. OS, PFS, etc), in the real-life setting, for mBC patients. In addition, we will estimate their individual correlation with OS, which should help us highlight potential surrogate endpoints in this setting. We will focuss on three distinct population, accounting for a large population of mBS patients: : patients treated with anti-HER2 targeted agents, patients treated with endocrine therapies and elderly population.

Study Overview

• Status: Completed
• Conditions: Metastatic Breast Cancer
• Intervention / Treatment: Drug: Chemotherapy (exclusive); Drug: Endocrine therapy (exclusive); Drug: Combination of endocrine therapy and chemotherapy; Drug: Chemotherapy and targeted treatment; Drug: Combination of endocrine therapy and targeted treatment; Drug: Combination of chemotherapy, endocrine therapy and targeted treatment

• Study Type: Observational
• Enrollment (Actual): 20033

Contacts and Locations

Study Locations

• France
  • Bordeaux, France, 33076
    • Insitut Bergonié

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Data source : 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. The Epidemio-Strategy and Medical Economic (ESME) Research Program is a French academic initiative supporting the centralization of structured and non- structured data documented in the electronic health records (EHR) (clinical notes, pathology reports and radiology reports) of patients treated for malignant conditions in a unique secured web-based data platform available for researchers. The ESME mBC data platform is an EHR-derived database that gathers exhaustive data on consecutive patients who initiated a L1 treatment for mBC between 01 January 2008 and 31 December 2017 in one of 18 French Comprehensive Cancer Centers.

Description

ELIGIBILITY

• female patients older than 18 years
• diagnosis of metastatic breast cancer (de novo disease or first metastatic recurrence) between January 1, 2008, and December 31, 2017
• received a fist-line systemic treatment such as chemotherapy, endocrine therapy or targeted therapy, whatever the sequence (monotherapy or combination of therapies using distinct mechanisms of actions, i.e., polytherapy).

Study Plan

How is the study designed?

Number of groups / cohorts

4

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Women with a diagnosis of HR+ /HER2- metastatic breast cancer (mBC)	Drug: Chemotherapy (exclusive); Administration of any chemotherapeutic agent(s); Drug: Endocrine therapy (exclusive); Administration of any type of endocrine therapy; Drug: Combination of endocrine therapy and chemotherapy; Any combination of endocrine therapy and chemotherapy; Drug: Chemotherapy and targeted treatment; Any combination of chemotherapy and targeted treatment(s); Drug: Combination of endocrine therapy and targeted treatment; Any combination of endocrine therapy and targeted treatment; Drug: Combination of chemotherapy, endocrine therapy and targeted treatment; Any combination of chemotherapy, endocrine therapy and targeted treatment(s)
Women with a diagnosis of HR- /HER2- metastatic breast cancer (mBC)	Drug: Chemotherapy (exclusive); Administration of any chemotherapeutic agent(s); Drug: Chemotherapy and targeted treatment; Any combination of chemotherapy and targeted treatment(s)
Women with a diagnosis of HR+ /HER2+ metastatic breast cancer (mBC)	Drug: Chemotherapy (exclusive); Administration of any chemotherapeutic agent(s); Drug: Endocrine therapy (exclusive); Administration of any type of endocrine therapy; Drug: Combination of endocrine therapy and chemotherapy; Any combination of endocrine therapy and chemotherapy; Drug: Chemotherapy and targeted treatment; Any combination of chemotherapy and targeted treatment(s); Drug: Combination of endocrine therapy and targeted treatment; Any combination of endocrine therapy and targeted treatment; Drug: Combination of chemotherapy, endocrine therapy and targeted treatment; Any combination of chemotherapy, endocrine therapy and targeted treatment(s)
Women with a diagnosis of HR- /HER2+ metastatic breast cancer (mBC)	Drug: Chemotherapy (exclusive); Administration of any chemotherapeutic agent(s); Drug: Chemotherapy and targeted treatment; Any combination of chemotherapy and targeted treatment(s)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival (OS) for Commonly Prescribed First-line Treatment Strategies	OS was defined as the time from diagnosis of mBC to the date of death from any cause.	10 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Real-world Progression-free Survival (rwPFS) for Commonly Prescribed First-line Treatment Strategies	rwPFS was defined as the delay between time from initial diagnosis of mBC to the date of disease progression (regional recurrence, progression, appearance/occurrence of metastases and distant recurrence) or death (any cause), whichever came first. Disease progression was assessed by the treating physician based on observed clinical events, as per routine practice (regional recurrence, progression, appearance/occurrence of metastases and distant recurrence). These events were recorded in the patient medical record. As this study relates to real-world data, no specific procedure was imposed to assess these events.	10 years
Association Between Overall Survival and Real-world Progression-free Survival for Commonly Prescribed First-line Treatment Strategies	Individual-level association between rwPFS and OS estimated using a Spearman rank correlation coefficient.	10 years

Collaborators and Investigators

• Sponsor: Institut Bergonié
• Collaborators: Institut du Cancer de Montpellier - Val d'Aurelle; UNICANCER

Publications and helpful links

General Publications

• Courtinard C, Gourgou S, Jacot W, Carton M, Guerin O, Vacher L, Bertaut A, Le Deley MC, Perol D, Marino P, Levy C, Uwer L, Perrocheau G, Schiappa R, Bachelot F, Parent D, Breton M, Petit T, Filleron T, Loeb A, Mathoulin-Pelissier S, Robain M, Delaloge S, Bellera C. 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. BMC Med. 2023 Mar 8;21(1):87. doi: 10.1186/s12916-023-02754-5.

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2008
• Primary Completion (Actual): December 1, 2023
• Study Completion (Actual): December 1, 2023

Study Registration Dates

• First Submitted: September 17, 2018
• First Submitted That Met QC Criteria: September 17, 2018
• First Posted (Actual): September 18, 2018

Study Record Updates

• Last Update Posted (Estimated): December 10, 2025
• Last Update Submitted That Met QC Criteria: November 24, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Keywords: Real world data; Metastatic breast cancer; Surrogate
• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Skin Diseases; Breast Diseases; Skin and Connective Tissue Diseases; Breast Neoplasms; Therapeutics; Drug Therapy
• Other Study ID Numbers: IB-2017DATECAN-ESME

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Availability of data and materials : The data that support the findings of this study are available from UNICANCER but restrictions apply to the availability of these data, which were used under license for the current study, and so are not publicly available. Data are however available from the authors upon reasonable request and with permission of UNICANCER.