Long-term responders to trastuzumab monotherapy in first-line HER-2+ advanced breast cancer: Characteristics and survival data

Sabine Schmid, Dirk Klingbiel, Stefan Aebi, Aron Goldhirsch, Christoph Mamot, Elisabetta Munzone, Franco Nolè, Christian Oehlschlegel, Olivia Pagani, Bernhard Pestalozzi, Christoph Rochlitz, Beat Thürlimann, Roger Von Moos, Patrik Weder, Khalil Zaman, Thomas Ruhstaller

Research output: Contribution to journalArticlepeer-review


Background: The impact of HER2-targeted therapy alone followed by the addition of chemotherapy at disease progression (PD) versus upfront combination was investigated by the SAKK 22/99 trial. The aim of this exploratory analysis of the SAKK 22/99 trial was to characterize the specific subset of patients deriving long-term benefit from trastuzumab monotherapy alone and to identify potential predictive factors of long-term response. Methods: This is an unplanned post-hoc analysis of patients randomized to Arm A (trastuzumab monotherapy). Patients were divided in two groups: patients with durable clinical benefit from trastuzumab monotherapy and short-term responders without durable clinical benefit from trastuzumab monotherapy Univariate and multivariate analyses of clinical characteristics correlating with response duration was performed. Results: Eighty six patients were randomized in arm A, 24 patients (28%) were long-term responders and 62 (72%) were short-term responders with a 5y-overall survival (OS) of 54% (95% CI 31-72) and of 18% (95%CI 10-30), respectively. Absence of ER expression, absence of PgR expression and presence of visceral disease emerged as possible negative predictive factors for durable clinical benefit. Conclusion: Durable clinical benefit can be achieved with trastuzumab monotherapy in a subgroup of HER2-positive patients with advanced disease and it is predictive for longer OS. Further investigations of predictive biomarkers are necessary to better characterize this subgroup of patients and develop further de-escalating strategies. Trial registration: NCT00004935; first posted 27.01.2003, retrospectively registered.

Original languageEnglish
Article number902
JournalBMC Cancer
Issue number1
Publication statusPublished - Sep 10 2019


  • HER-positive breast cancer
  • Long-term responders
  • Trastuzumab monotherapy

ASJC Scopus subject areas

  • Genetics
  • Oncology
  • Cancer Research


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