Neoadjuvant palbociclib in women with operable, hormone receptor-positive breast cancer

The addition of a cyclin-dependent kinase 4/6 (CDK4/6) inhibitor to endocrine therapy augments biological response in breast cancer. This phase III randomized, double-blind study evaluated the efficacy of adding palbociclib to neoadjuvant endocrine therapy (NET) for operable, hormone receptor-positive human epidermal growth factor receptor 2 (HER2)-negative breast cancer. Patients randomly received 16 weeks of endocrine therapy (letrozole for postmenopausal and tamoxifen plus ovarian function suppression for pre-/perimenopausal patients) plus palbociclib or placebo. The co-primary endpoints included preoperative endocrine prognostic index (PEPI) score and EndoPredict (EPclin) risk score according to the gatekeeping procedure. Of 141 randomized patients, 130 completed the treatment with surgical samples evaluable for endpoints in 126 patients. The proportion of patients with a low, moderate, and high PEPI score was 15.2, 50.0, and 34.8% in the palbociclib arm and 13.3, 55.0, and 31.7% in the placebo arm, respectively, with no statistical difference (one-sided P = 0.563). Statistical analysis was not performed on EPclin risk score. No new safety signals were reported. Permanent treatment discontinuation by adverse events was reported for seven (9.7%) and zero patients in the palbociclib and placebo arms, respectively. In conclusion, the addition of palbociclib to NET did not improve the efficacy. ClinicalTrials.gov NCT03969121.

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Publication Details

Journal
Endocrine Related Cancer
Published
2025-09-01
DOI
https://doi.org/10.1530/erc-24-0353
Primary Topic
Advanced Breast Cancer Therapies
Type
article
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article

Neoadjuvant palbociclib in women with operable, hormone receptor-positive breast cancer

Wonshik Han, Ming‐Feng Hou, Sachiko Takahara, Michelle White et al.
Endocrine Related Cancer
Advanced Breast Cancer Therapies
article

Neoadjuvant palbociclib in women with operable, hormone receptor-positive breast cancer

Wonshik Han, Ming‐Feng Hou, Sachiko Takahara, Michelle White, Eriko Tokunaga, Tomoyuki Aruga, Shigeru Imoto, Yosuke Yamada, Yuko Tanabe, Richard H DeBoer, Yasuaki Sagara, Chiun‐Sheng Huang, Hironori Haga, Hirofumi Suwa, Toshinari Yamashita, M. Tokiwa, Masahiro Takada, Hiroko Bando, Chi-Feng Chung, Sachiko Minamiguchi, Takayuki Ueno, Elham Fakhrejahani, G. Bruce Mann, Hiroji Iwata, Yoshiko Umeyama, Masakazu Toi, Takayuki Kobayashi, Jee Hyun Kim, Hyun‐Ah Kim, Kenichi Inoue, Eun Sook Lee, Satoshi Morita, Louis W.C. Chow, Hung Wen Lai
article en

Abstract

The addition of a cyclin-dependent kinase 4/6 (CDK4/6) inhibitor to endocrine therapy augments biological response in breast cancer. This phase III randomized, double-blind study evaluated the efficacy of adding palbociclib to neoadjuvant endocrine therapy (NET) for operable, hormone receptor-positive human epidermal growth factor receptor 2 (HER2)-negative breast cancer. Patients randomly received 16 weeks of endocrine therapy (letrozole for postmenopausal and tamoxifen plus ovarian function suppression for pre-/perimenopausal patients) plus palbociclib or placebo. The co-primary endpoints included preoperative endocrine prognostic index (PEPI) score and EndoPredict (EPclin) risk score according to the gatekeeping procedure. Of 141 randomized patients, 130 completed the treatment with surgical samples evaluable for endpoints in 126 patients. The proportion of patients with a low, moderate, and high PEPI score was 15.2, 50.0, and 34.8% in the palbociclib arm and 13.3, 55.0, and 31.7% in the placebo arm, respectively, with no statistical difference (one-sided P = 0.563). Statistical analysis was not performed on EPclin risk score. No new safety signals were reported. Permanent treatment discontinuation by adverse events was reported for seven (9.7%) and zero patients in the palbociclib and placebo arms, respectively. In conclusion, the addition of palbociclib to NET did not improve the efficacy. ClinicalTrials.gov NCT03969121.

Endocrine Related CancerVol. 32(9)
The Royal Melbourne Hospital (AU), Kyoto University (JP), Peter MacCallum Cancer Centre (AU), Monash Medical Centre (AU), Breast Cancer Network Australia (AU), Kaohsiung Medical University Chung-Ho Memorial Hospital (TW), Seoul National University Hospital (KR), Seoul National University Bundang Hospital (KR), Kanagawa Prefectural Hospital Organization (JP), Tokyo Metropolitan Komagome Hospital (JP), National Cancer Center (US), National Hospital Organization Kyushu Cancer Center (JP), Unimed Medical Institute (CN), University of Tsukuba Hospital (JP), National Cancer Center (KR), Saitama Cancer Center (JP), National Taiwan University Hospital (TW), Aichi Cancer Center (JP), Hyogo Prefectural Amagasaki General Medical Center (JP), Koo Foundation Sun Yat-Sen Cancer Center (TW), The Cancer Institute Hospital (JP), Kyorin University Hospital (JP), Pfizer (Japan) (JP), Kyoto University Hospital (JP), Changhua Christian Hospital (TW), Kitano Hospital (JP), Kobe City Medical Center General Hospital (JP), Toranomon Hospital (JP), Japanese Foundation For Cancer Research (JP), Kanagawa Cancer Center
Openalex Percentile: Top 66%
Advanced Breast Cancer Therapies
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