Clinical features associated with exceptional response on first-line anti-HER2-based therapy for metastatic breast cancer: real-world data from the ESME national cohort.
18 affiliations
- 1Medical Oncology Department, Institut Gustave Roussy, Villejuif, France; Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA, USA. Electronic address: [email protected].
- 2Department of Biostatistics, Institut Curie, Saint-Cloud, France.
- 3Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA, USA.
- 4Medical Oncology Department, Centre Oscar Lambret, Lille, France.
- 5Medical Oncology Department, Centre Francois Baclesse, Caen, France.
- 6Medical Oncology Department, Institut de Cancérologie Strasbourg Europe ICANS, Strasbourg, France.
- 7Medical Oncology Department, Centre Léon Bérard, Lyon, France.
- 8Medical Oncology Department, Institute Bergonié, Bordeaux, France.
- 9Medical Oncology Department, Centre Eugene Marquis, Rennes, France.
- 10Medical Oncology Department, Institut de cancérologie de Lorraine, Vandoeuvre Les Nancy, France.
- 11Medical Oncology Department, Oncopole Claudius Regaud, IUCT, Toulouse, France.
- 12Medical Oncology Department, Centre Georges François Leclerc, Dijon, France.
- 13Department of Medical Oncology, Institut Curie, Paris, France.
- 14Medical Oncology Department, Institut Paoli Calmettes, Marseille, France.
- 15Medical Oncology Department, Centre Antoine Lacassagne, Nice, France.
- 16Medical Oncology Department, Institut de Cancerologie de l'Ouest René Gauducheau, Saint-Herblain, France.
- 17Health Data and Partnerships Department, Unicancer, Paris, France.
- 18Medical Oncology Department, Institut du cancer de Montpellier, INSERM U1194, Montpellier University, Montpellier, France.
Abstract
Some patients with HER2-positive metastatic breast cancer (MBC) achieve prolonged disease control with first-line anti-HER2-based therapy, but associated clinical features remain poorly characterized in real-world populations. We conducted a retrospective study using the Epidemiological Strategy and Medical Economics (ESME) MBC cohort, including 18 French comprehensive cancer centers. Women with HER2-positive MBC who initiated first-line anti-HER2-based therapy between January 2008 and December 2021 and had at least 3 years of follow-up were eligible. Exceptional responders had no documented progression within 3 years. A secondary analysis evaluated factors associated with remaining progression-free at 5 years. Multivariable logistic regression with bootstrap cross-validation was used. Among 4307 eligible patients, 1016 (23.6%) were exceptional responders. Of 765 evaluable at 5 years, 523 (68.4%) remained progression-free. Factors associated with a lower likelihood of exceptional response included hormone receptor-negative (HR-) disease, a disease-free interval of 6-24 months, ≥3 metastatic sites, visceral metastases, and central nervous system involvement. First-line treatment with chemotherapy, trastuzumab, and pertuzumab was associated with exceptional response. Among exceptional responders, HR-disease, grade III tumors and first-line chemotherapy plus trastuzumab and pertuzumab were associated with progression-free at 5-year, whereas visceral metastases were associated with lower odds. Model discrimination was moderate (AUC 0.72 and 0.64, respectively). Nearly one quarter of patients remained progression-free for at least 3 years, and more than two thirds of those evaluable remained progression-free at 5 years. Clinical variables alone had limited predictive value, supporting the need for molecular and immune biomarkers to better identify this subgroup.
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BibTeX
@article{grinda2026clinical,
title = {{Clinical features associated with exceptional response on first-line anti-HER2-based therapy for metastatic breast cancer: real-world data from the ESME national cohort.}},
author = {Thomas Grinda and Matthieu Carton and Stefania Morganti and Audrey Mailliez and Christelle Levy and Thierry Petit and Thomas Bachelot and Monica Arnedos and Véronique Diéras and Vincent Massard and Florence Dalenc and Isabelle Desmoulins and Jean-Yves Pierga and Anthony Gonçalves and Caroline Bailleux and Jean-Sebastien Frenel and Lise Bosquet and Heather A Parsons and William Jacot and Nancy U Lin},
journal = {Breast (Edinburgh, Scotland)},
year = {2026},
month = oct,
doi = {10.1016/j.breast.2026.104943},
url = {https://europepmc.org/article/MED/42880095},
note = {CC BY-NC-ND},
}
RIS
TY - JOUR
TI - Clinical features associated with exceptional response on first-line anti-HER2-based therapy for metastatic breast cancer: real-world data from the ESME national cohort.
AU - Thomas Grinda
AU - Matthieu Carton
AU - Stefania Morganti
AU - Audrey Mailliez
AU - Christelle Levy
AU - Thierry Petit
AU - Thomas Bachelot
AU - Monica Arnedos
AU - Véronique Diéras
AU - Vincent Massard
AU - Florence Dalenc
AU - Isabelle Desmoulins
AU - Jean-Yves Pierga
AU - Anthony Gonçalves
AU - Caroline Bailleux
AU - Jean-Sebastien Frenel
AU - Lise Bosquet
AU - Heather A Parsons
AU - William Jacot
AU - Nancy U Lin
PY - 2026
DA - 2026/10/13
JO - Breast (Edinburgh, Scotland)
DO - 10.1016/j.breast.2026.104943
UR - https://europepmc.org/article/MED/42880095
AB - Some patients with HER2-positive metastatic breast cancer (MBC) achieve prolonged disease control with first-line anti-HER2-based therapy, but associated clinical features remain poorly characterized in real-world populations. We conducted a retrospective study using the Epidemiological Strategy and Medical Economics (ESME) MBC cohort, including 18 French comprehensive cancer centers. Women with HER2-positive MBC who initiated first-line anti-HER2-based therapy between January 2008 and December 2021 and had at least 3 years of follow-up were eligible. Exceptional responders had no documented progression within 3 years. A secondary analysis evaluated factors associated with remaining progression-free at 5 years. Multivariable logistic regression with bootstrap cross-validation was used. Among 4307 eligible patients, 1016 (23.6%) were exceptional responders. Of 765 evaluable at 5 years, 523 (68.4%) remained progression-free. Factors associated with a lower likelihood of exceptional response included hormone receptor-negative (HR-) disease, a disease-free interval of 6-24 months, ≥3 metastatic sites, visceral metastases, and central nervous system involvement. First-line treatment with chemotherapy, trastuzumab, and pertuzumab was associated with exceptional response. Among exceptional responders, HR-disease, grade III tumors and first-line chemotherapy plus trastuzumab and pertuzumab were associated with progression-free at 5-year, whereas visceral metastases were associated with lower odds. Model discrimination was moderate (AUC 0.72 and 0.64, respectively). Nearly one quarter of patients remained progression-free for at least 3 years, and more than two thirds of those evaluable remained progression-free at 5 years. Clinical variables alone had limited predictive value, supporting the need for molecular and immune biomarkers to better identify this subgroup.
SN - 0960-9776
SN - 1532-3080
ER -