Phase II trial of bicalutamide in patients with androgen receptor-positive, estrogen receptor-negative metastatic breast cancer Journal Article


Authors: Gucalp, A.; Tolaney, S.; Isakoff, S. J.; Ingle, J. N.; Liu, M. C.; Carey, L. A.; Blackwell, K.; Rugo, H.; Nabell, L.; Forero, A.; Stearns, V.; Doane, A. S.; Danso, M.; Moynahan, M. E.; Momen, L. F.; Gonzalez, J. M.; Akhtar, A.; Giri, D. D.; Patil, S.; Feigin, K. N.; Hudis, C. A.; Traina, T. A.
Article Title: Phase II trial of bicalutamide in patients with androgen receptor-positive, estrogen receptor-negative metastatic breast cancer
Abstract: Purpose: Patients with hormone receptor-negative breast cancer generally do not benefit from endocrine- targeted therapies. However, a subset with androgen receptor (AR) expression is predicted to respond to antiandrogen therapies. This phase II study explored bicalutamide in AR-positive, estrogen receptor (ER), and progesterone receptor (PgR)-negative metastatic breast cancer. Experimental Design: Tumors from patients with ER/PgR-negative advanced breast cancer were tested centrally for AR [immunohistochemistry (IHC) < 10% nuclear staining considered positive]. If either the primary or a metastatic site was positive, patients were eligible to receive the AR antagonist bicalutamide at a dose of 150 mg daily. Clinical benefit rate (CBR), the primary endpoint, was defined as the total number of patients who show a complete response (CR), partial response (PR), or stable disease (SD) < 6 months; secondary endpoints included progression-free survival (PFS) and toxicity. Correlative studies included measurement of circulating endocrine markers and IHC surrogates for basal-like breast cancer. Results: Of 424 patients with ER/PgR-negative breast cancer, 12% tested AR-positive. The 6-month CBR was19%[95% confidence interval (CI), 7%-39%]for bicalutamide. The median PFS was 12 weeks (95% CI, 11-22 weeks). Bicalutamide was well-tolerated with no grade 4/5 treatment-related adverse events observed. Conclusion: AR was expressed in 12% of patients with ER/PgR-negative breast cancer screened for this trial. The CBR of 19% observed with bicalutamide shows proof of principle for the efficacy of minimally toxic androgen blockade in a select group of patients with ER/PgR-negative, AR-positive breast cancer. © 2013 American Association for Cancer Research.
Journal Title: Clinical Cancer Research
Volume: 19
Issue: 19
ISSN: 1078-0432
Publisher: American Association for Cancer Research  
Date Published: 2013-01-01
Start Page: 5505
End Page: 5512
Language: English
DOI: 10.1158/1078-0432.ccr-12-3327
PROVIDER: scopus
PMCID: PMC4086643
PUBMED: 23965901
DOI/URL:
Notes: --- - Cited By (since 1996):1 - "Export Date: 2 December 2013" - "CODEN: CCREF" - "Source: Scopus"
Altmetric Score
MSK Authors
  1. Kimberly Nicole Feigin
    19 Feigin
  2. Sujata Patil
    380 Patil
  3. Michael A Danso
    9 Danso
  4. Clifford Hudis
    835 Hudis
  5. Dilip D Giri
    122 Giri
  6. Ayca Gucalp
    79 Gucalp
  7. Tiffany A Traina
    151 Traina
  8. Arooj Akhtar
    2 Akhtar
  9. Ashley Stephen Doane
    11 Doane
  10. Lamia Farrah Sun
    6 Sun