Multicenter Trial for Eliminating Breast Cancer Surgery or Radiotherapy in Exceptional Responders to Neoadjuvant Systemic Therapy
Summary
This clinical trial studies eliminating surgery and/or radiation therapy in treating breast cancer patients. In HER2 positive and triple negative breast cancers, after systemic therapy, when image-guided biopsy shows no residual cancer, patients then receive standard breast radiotherapy with no surgery. In ER positive/HER2 negative breast cancers, after endocrine therapy and ablative radiotherapy, when image-guided biopsy shows no residual cancer, patients then receive standard endocrine therapy with no surgery. For patients who have HER2 positive and triple negative breast cancers and standard surgery, after systemic therapy, breast radiotherapy is being eliminated when the pathology from surgery shows no residual cancer.
Detailed description
PRIMARY OBJECTIVES: I. Cohort A1 and A2: To determine the 6 mo, 1, 2, 3, 5, 7, and 10 year biopsy confirmed ipsilateral breast tumor recurrence rate (IBTR, invasive and/or in situ) among patients who do not undergo surgery. II. Cohort B: To determine the pCR rate 6 or 12 months after radiation therapy based on image-guided biopsy. III. Cohort C: To determine the 6 mo, 1, 2, 3, 5, 7 and 10-year ipsilateral breast tumor recurrence rate among patients who undergo surgery alone without radiation. SECONDARY OBJECTIVES: I. Cohort B: To determine the 6 mo, 1, 2, 3, 5, 7 and 10--year biopsy confirmed ipsilateral breast tumor recurrence rate (IBTR, invasive and/or in situ) among patients who do not undergo surgery. II. To determine the number (%) of patients where final biopsy reveals residual disease and quantify the residual disease (residual cancer burden, RCB) determined by routine pathologic examination of surgery specimens. III. To assess baseline, 6 months, 1, 3, 5, 7, and 10 years decisional comfort of clinical trial participation using the Decisional Regret Scale (DRS). IV. To determine patient-reported cosmetic outcome, breast pain, and functional status using the Breast Cancer Treatment Outcomes Scale (BCTOS) at baseline, 6 months, 1, 3, 5, 7, and 10 years. V. To determine the 6 mo, 1, 2, 3, 5, 7, and 10 years incidence of ipsilateral breast and nodal recommendation and performance of biopsy based on breast imaging follow-up. VI. Correlate "liquid biopsy" analyses (after standard routine NST, 6 months and one year post-radiotherapy or surgery) among protocol participants with pCR, utilizing circulating tumor cells (CTCs) and circulating tumor-DNA (ctDNA). VII. Among patients who decide to proceed with routine surgery, record the results of final biopsy compared with routine pathologic examination of surgery specimens. VIII. To determine patient-reported quality of life using the FACT B+4 instrument at baseline, 6 months, 1, 3, 5, 7, and 10 years after treatment. IX. To explore if radiation genomic sensitivity scores and Oncotype performed on the initial diagnostic core biopsy specimen correlate with pCR rates in Cohort B. X. To determine if changes in blood-based RNA Sequencing are elicited with radiation in Cohort B, measured at baseline, at the first 4-6 week follow-up after radiation, and at the 6 month post-radiation follow-up. XI. In Cohort B to determine the 3-year rate of tumor control/ progression free survival (PFS). XII. In Cohort C/D to determine whether nanomechanical biomarkers or quantification of stromal and tumor TILS can predict for low risk of local recurrence in exceptional responders who omit radiation therapy. XIII. In Cohorts A/B/C/D: To record 6 mo, 1, 2, 3, 5, 7, and 10 year breast cancer disease-free and overall survival. XIV. In Cohort D: Correlate nanomechanical biomarker analyses with pCR and/or local recurrence in patients with triple negative or her-2 positive breast cancer treated with lumpectomy +/- radiation. OUTLINE: For Cohorts A, within 12 weeks of completing neoadjuvant systemic therapy, patients undergo whole breast irradiation over 15-25 fractions on consecutive days. Patients then undergo external beam radiation therapy (EBRT) boost over 7 fractions on consecutive days beginning the day following completion of whole breast irradiation. For Cohort B, patients will receive 3 months of neoadjuvant endocrine therapy, after which they will receive ablative partial breast irradiation over 5 fractions. Patients will then continue with endocrine therapy. After completion of study treatment, patients are followed up every 6 months for 10 years.
Arms & interventions
- RadiationUndergo EBRT
Undergo EBRT
- OtherLaboratory Biomarker Analysis
Correlative studies
- OtherQuality-of-Life Assessment
Ancillary studies
- OtherQuestionnaire Administration
Ancillary studies
- RadiationPartial Breast Irradiation
Undergo partial breast irradiation
Outcome measures
Primary
Ipsilateral breast tumor recurrence-free survival (IBT-RFS)
Will monitor IBT-RFS using the method of Thall et al. Will be estimated using the Kaplan-Meier method log-rank test will be performed to test the difference in time-to-event distributions between patient groups. Cox proportional hazards model will be used to include multiple covariates in the time-to-event analysis.
Time frame: From confirmation of pathologic complete response (pCR) to the time of ipsilateral breast tumor recurrence or death, whichever occurs first or the time of last contact, assessed for up to 10 years
Overall survival
Will be estimated using the Kaplan-Meier method log-rank test will be performed to test the difference in time-to-event distributions between patient groups. Cox proportional hazards model will be used to include multiple covariates in the time-to-event analysis.
Time frame: Up to 10 years
Eligibility criteria
Study locations (8)
MD Anderson Cancer Center - Banner
Gilbert, Arizona, 85234
Baptist MD Anderson Cancer Center
Jacksonville, Florida, 32207
Queen's Medical Center
Honolulu, Hawaii, 96813
Mayo Clinic
Rochester, Minnesota, 55905
Cooper Hospital Univ Med Ctr, MD Anderson at Cooper Voorhees
Voorhees Township, New Jersey, 08103
Carolinas Medical Center/Levine Cancer Institute
Charlotte, North Carolina, 28203
University of Pittsburgh Cancer Institute (UPCI)
Pittsburgh, Pennsylvania, 15232
M D Anderson Cancer Center
Houston, Texas, 77030
References
- Kuerer HM, Valero V, Smith BD, Krishnamurthy S, Diego EJ, Johnson HM, Lin H, Shen Y, Lucci A, Shaitelman SF, Mitchell MP, Boughey JC, White RL, Hunt KK, Yang WT, Rauch GM; Exceptional Responders Study Group. Selective Elimination of Breast Surgery for Invasive Breast Cancer: A Nonrandomized Clinical Trial. JAMA Oncol. 2025 May 1;11(5):529-534. doi: 10.1001/jamaoncol.2025.0207.(PubMed)
- Johnson HM, Lin H, Shen Y, Diego EJ, Krishnamurthy S, Yang WT, Smith BD, Valero V, Lucci A, Sun SX, Shaitelman SF, Mitchell MP, Boughey JC, White RL, Rauch GM, Kuerer HM; Exceptional Responders Study Group. Patient-Reported Outcomes of Omission of Breast Surgery Following Neoadjuvant Systemic Therapy: A Nonrandomized Clinical Trial. JAMA Netw Open. 2023 Sep 5;6(9):e2333933. doi: 10.1001/jamanetworkopen.2023.33933.(PubMed)
- Kuerer HM, Smith BD, Krishnamurthy S, Yang WT, Valero V, Shen Y, Lin H, Lucci A, Boughey JC, White RL, Diego EJ, Rauch GM; Exceptional Responders Clinical Trials Group. Eliminating breast surgery for invasive breast cancer in exceptional responders to neoadjuvant systemic therapy: a multicentre, single-arm, phase 2 trial. Lancet Oncol. 2022 Dec;23(12):1517-1524. doi: 10.1016/S1470-2045(22)00613-1. Epub 2022 Oct 25.(PubMed)
- Hariharan N, Rao TS, Rajappa S, Naidu CCK. Precision medicine - A new era in multidisciplinary care. Cancer Treat Res Commun. 2022;32:100577. doi: 10.1016/j.ctarc.2022.100577. Epub 2022 May 17. No abstract available.(PubMed)