Data for: Neoadjuvant palbociclib and endocrine therapy versus chemotherapy in ER+/HER2- breast cancer: a randomized phase II trial
Documentation files
Documentation files
Citation and access
Citation and access
Data access level:
Creator/Principal investigator(s):
Research principal:
Data contains personal data:
Yes
Type of personal data:
The dataset does not contain direct identifiers such as names, social security numbers, or contact details. Each patient is represented by a trial-specific ID. The trial-specific IDs are pseudonymized and cannot be used to identify individual patients. Linkage to individual identities is only possible by the local principal investigator and designated site staff through site-specific keys, which are not shared. The metadata include gender and treatment arm, which are indirect identifiers. The sequencing data (FASTQ files from WES and RNA-Seq) represent molecular profiles, which qualify as sensitive health-related data. Although pseudonymized, genomic data are inherently identifiable and are protected under regulations for genetic and health data.
Code key exists:
Yes
Sensitive personal data:
Yes
Citation:
Language:
Method and outcome
Method and outcome
Population:
The study was conducted at three hospitals in Stockholm, Sweden (Karolinska University Hospital, Södersjukhuset, and Capio S:t Göran Hospital). Between March 18, 2016, and July 29, 2021, 181 patients were enrolled. Two patients were excluded from all analyses, leaving 179 patients in the intention-to-treat (ITT) population. The study population consisted of women and men aged ≥35 years with ER-positive (≥10%) and HER2-negative breast cancer, Eastern Cooperative Oncology Group (ECOG) performance status 0–1, tumor size >2 cm and/or node-positive disease. Patients with up to two distant metastases treatable with curative intent were eligible. All participants had adequate cardiac, renal, and hepatic function and no other malignancy within the past five years. Tissue biospecimens were collected from the patients prior to the initiation of the neoadjuvant therapy, as specified in the study protocol. Up to five 14-gauge ultrasound-guided core needle tissue biopsies were collected. Three fresh-frozen (FF) tissue samples were stored directly at −80 °C after collection. The remaining two samples were directly immersed into 10% neutral-buffered formalin for preparation of formalin-fixed paraffin-embedded (FFPE) tissue blocks. Biopsies at baseline and on-treatment were obtained after the PET/CT scan. Whole peripheral blood was also collected before the initiation of neoadjuvant therapy in 4ml EDTA tubes and stored at –80 °C. Plasma and serum samples were collected at baseline and every six weeks during treatment, as well as during follow-up. After all quality control and preprocessing steps, biopsy samples available for translational analysis comprised 166 for RNA sequencing (RNA-Seq) and 166 for whole exome sequencing (WES).
Study design:
- Experimental study
- Randomised controlled trial (RCT)
- Cross-over
Geographic coverage
Geographic coverage
Geographic location:
Administrative information
Administrative information
Responsible department/unit:
Department of Oncology-Pathology [K7]
Ethics Review:
Stockholm - 2014/1492-31/4
Funding
Funding
Funding agency:
- Swedish Research Council
Opens a new window at ror.org.
ROROpens in a new tab
Award number:
2020-00636_VR
Award title:
Randomized Trials on Biology-Driven De-escalation of Neoadjuvant Treatment for HER2-positive Breast Cancer: ARIADNE master protocol
Funding information:
Abstract (1446/1500 chars)ARIADNE is a multicenter, open-label, randomized, comparative phase IIB trial which consists of two separate sub-studies. The main aim is to evaluate whether neoadjuvant therapy for HER2-positive breast cancer can be safely de-escalated while maintaining efficacy, based on biology-driven management decisions. Eligible patients will have early or locally advanced breast cancer (cT2-4, N0-3, M0) with confirmed HER2-positive status. Patients with ER-positive disease will be randomized 1:1 to receive standard of care therapy (docetaxel, carboplatin, trastuzumab, pertuzumab (TCHP) for six cycles) or T-DM1 in combination with ribociclib and letrozole for six cycles. Patients with ER-negative disease will be randomized 1:1 to receive TCHP x6 or treatment according to PAM50 molecular intrinsic subtype: HER2-enriched patients will be treated with T-DM1 x6; tumors in other subtype groups will be treated with dose-dense epirubicin and cyclophosphamide x4, followed by TCHP x4. In both studies, patients progressing after two cycles with de-escalated therapy will crossover to standard of care. Further adjuvant therapy will be given depending on assigned arm and residual disease. The primary endpoint is rates of pathological complete response (pCR), defined as ypT0/Tis, ypN0, as determined at the surgical specimen. Fewer adverse events, improved quality of life and similar outcomes compared to standard therapy are the expected benefits for the study participants.
