Pexidartinib and standard neoadjuvant therapy in the adaptively randomized I-SPY2 trial for early breast cancer

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2024年12月3日

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Hope S. Rugo,
Mike Campbell,
Christina Yau,
A. Jo Chien,
Anne M. Wallace,
Claudine Isaacs,
Judy C. Boughey,
Hyo S. Han,
Meredith Buxton,
Julia L. Clennell,
Smita M. Asare,
Katherine Steeg,
Amy Wilson,
Ruby Singhrao,
Jeffrey B. Matthews,
Jane Perlmutter,
W. Fraser Symmans,
Nola M. Hylton,
Angela M. DeMichele,
Douglas Yee,
Laura J. Van’t Veer,
Donald A. Berry,
Laura J. Esserman

摘要

Purpose

We investigated the small-molecule receptor tyrosine kinase-inhibitor of colony-stimulating factor-1 receptor pexidartinib in the stage II/III breast cancer in the I-SPY2 platform trial.

Methods

I-SPY2 is an adaptive platform trial that features multiple arms of experimental agents administered on a background of standard neoadjuvant therapy with paclitaxel and adriamycin/cyclophosphamide, followed by definitive surgery. The adaptive randomization engine preferentially assigns patients based upon cumulative performance of each agent in a given breast cancer subtype based on hormone receptor and HER2 receptor status. The study endpoint is pathologic complete response.

Results

A total of 9 participants were randomized to receive pexidartinib with neoadjuvant paclitaxel before enrollment was halted due to a serious adverse event of vanishing bile duct syndrome. No participants received a full course of the study drug.

Conclusion

Although there remains interest in agents targeting CSF-1, hepatic toxicity appears to be a limiting factor for their use in early breast cancer.

Trial registration

NCT01042379 (www.clinicaltrials.gov/ct2/show/NCT01042379).