Palbociclib in patients with ovarian cancer with CDKN2A alterations: Results from the targeted agent and profiling utilization registry study

Authors

• Dan S. Veljovich, Swedish Cancer Institute, Seattle, WA.
• Michael Rothe, American Society of Clinical Oncology, Alexandria, VA.
• Elizabeth Garrett-Mayer, American Society of Clinical Oncology, Alexandria, VA.
• Hussein M. Ali-Ahmad, Michigan Cancer Research Consortium, Lansing, MI.
• R Wendel Naumann, Atrium Health Levine Cancer Institute, Wake Forest University School of Medicine, Charlotte, NC.Follow
• Jean Siedel, University of Michigan, Ann Arbor, MI.
• John K. Chan, Sutter Cancer Research Consortium, San Francisco, CA.
• Andrew Gregory, Advocate Health - MidwestFollow
• Evan Pisick, City of Hope Chicago, Zion, IL.
• Bamidele Adesunloye, City of Hope Atlanta, Atlanta, GA.
• Rebecca C. Arend, Division of Gynecologic Oncology, O'Neal Comprehensive Cancer Center at the University of Alabama at Birmingham, Birmingham, AL.
• Maria Bell, Sanford Health, Sioux Falls, SD.
• Marina Frimer, Division of Gynecologic Oncology, Department of Obstetrics and Gynecology, Northwell Health, Northwell Health Cancer Institute, New Hyde Park, NY.
• Bernard Tawfik, University of New Mexico Comprehensive Cancer Center, Albuquerque, NM.
• Ramya Thota, Intermountain Healthcare, Murray, UT.
• Apostolia M. Tsimberidou, The University of Texas MD Anderson Cancer Center, Houston, TX.
• Pam K. Mangat, American Society of Clinical Oncology, Alexandria, VA.
• Dominique C. Hinshaw, American Society of Clinical Oncology, Alexandria, VA.
• Abigail Gregory, American Society of Clinical Oncology, Alexandria, VA.
• Gina N. Grantham, American Society of Clinical Oncology, Alexandria, VA.
• Susan Halabi, Duke University Medical Center, Durham, NC.
• Richard L. Schilsky, American Society of Clinical Oncology, Alexandria, VA.

Recommended Citation

Veljovich DS, Rothe M, Garrett-Mayer E, et al. Palbociclib in Patients With Ovarian Cancer With CDKN2A Alterations: Results From the Targeted Agent and Profiling Utilization Registry Study. JCO Precis Oncol. 2026;10(7):e2600456. doi:10.1200/PO-26-00456

Affiliations

Wake Forest University School of Medicine

Abstract

Purpose: The Targeted Agent and Profiling Utilization Registry Study is a phase II basket trial evaluating the antitumor activity of commercially available targeted agents in patients with advanced cancer and genomic alterations. Results of a cohort of patients with ovarian cancer (OC) with CDKN2A alterations treated with palbociclib are reported.

Methods: Eligible patients had advanced OC, measurable disease (RECIST), Eastern Cooperative Oncology Group performance status 0-2, adequate organ function, tumors with CDKN2A alterations, and no remaining standard treatment options. The primary end point was disease control (DC), defined as objective response (OR) or stable disease (SD) of at least 16+ weeks duration (SD16+). Simon two-stage design with a null DC rate of 15% versus 35% (power = 0.85; α = .10) was used. Secondary end points included OR, progression-free survival (PFS), overall survival (OS), duration of response, duration of SD, and safety.

Results: Twenty-eight patients were enrolled from February 2017 to April 2023, and all had OC with a CDKN2A alteration (loss [n = 21], mutation [n = 5], and loss and mutation [n = 2]). One patient was not evaluable for efficacy because of an excluded genomic finding. Three patients had a partial response and seven had SD16+ for DC and OR rates of 38% (90% CI, 25 to 100) and 11% (95% CI, 2 to 29), respectively. The null hypothesis of 15% DC rate was rejected (P = .004). The median PFS was 8 weeks (95% CI, 8 to 18) and the median OS was 33 weeks (95% CI, 24 to 73). Nineteen patients (68%) had at least one treatment-related grade 3-4 adverse event (AE) or serious AE including anemia, dyspnea, fatigue, leukopenia, neutropenia, and thrombocytopenia.

Conclusion: Palbociclib met the prespecified criteria to declare a signal of activity in patients with OC and CDKN2A alterations.

Type

Article

PubMed ID

42456088