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Palbociclib in Patients With Pancreatic and Biliary Cancer With CDKN2A Alterations: Results From the Targeted Agent
Tareq Al Baghdadi1, Susan Halabi2, Elizabeth Garrett-Mayer3
1Michigan Cancer Research Consortium, Ypsilanti, MI.
Purpose:
The Targeted Agent and Profiling Utilization Registry (TAPUR) Study identifies signals of antitumor activity of commercially available targeted agents in patients with advanced cancers that harbor genomic alterations known as drug targets. In this article, data from two cohorts of patients with pancreatic and biliary cancers with CDKN2A loss or mutation treated with palbociclib are reported.
Methods:
Eligible patients age 12 years and older with advanced measurable or evaluable solid tumors are provided treatment according to protocol-specified genomic matching rules. The primary study end point is objective response or stable disease of at least 16 weeks duration. For each cohort, a Simon two-stage design was used with a futility evaluation after 10 patients. Secondary end points include safety, progression-free survival (PFS), and overall survival (OS).
Results:
Between July 2016 and November 2017, 12 and 10 patients with pancreatic and biliary cancer, respectively, with CDKN2A loss or mutation were treated with palbociclib. Twenty evaluable patients (10 per cohort) were included in the analysis. No patients had objective response or stable disease at 16 weeks, and both cohorts were closed. Two patients, neither with response, were determined to be ineligible. All patients were evaluated for safety, PFS, and OS. A median PFS of 7.2 weeks (90% CI, 4.0 to 8.0 weeks) and median OS of 12.4 weeks (90% CI, 4.7 to 23.1 weeks) were observed in the pancreatic cohort. A median PFS of 7.3 weeks (90% CI, 3.9 to 7.9 weeks) and median OS of 11.1 weeks (90% CI, 5.1 to 14.0 weeks) were observed in the biliary cohort. No unexpected toxicities were observed.
Conclusion:
Palbociclib monotherapy does not have clinical activity in patients with advanced pancreatic or biliary cancers with CDKN2A loss or mutation. Toxicity is similar to reported experience with palbociclib in other tumor types.
Insights
Palbociclib did not show clinical activity in patients with advanced pancreatic or biliary cancers with CDKN2A loss or mutation. Toxicity was similar to previous studies.
Area of Science:
- Oncology
- Genomics
- Clinical Trials
Background:
- The Targeted Agent and Profiling Utilization Registry (TAPUR) study investigates targeted agents for advanced cancers with specific genomic alterations.
- CDKN2A loss or mutation is a genomic alteration targeted by certain therapies.
Purpose of the Study:
- To report data from two cohorts of the TAPUR study evaluating palbociclib in patients with pancreatic and biliary cancers harboring CDKN2A loss or mutation.
- To assess the antitumor activity and safety of palbociclib in this patient population.
Main Methods:
- Patients aged 12+ with advanced solid tumors and CDKN2A loss/mutation received palbociclib.
- The primary endpoint was objective response or stable disease of at least 16 weeks.
- A Simon two-stage design with futility evaluation was used; secondary endpoints included safety, progression-free survival (PFS), and overall survival (OS).
Main Results:
- Twelve patients with pancreatic cancer and 10 with biliary cancer with CDKN2A alterations were treated.
- No patients achieved objective response or stable disease at 16 weeks, leading to cohort closure.
- Median PFS was 7.2 weeks for pancreatic and 7.3 weeks for biliary cancer cohorts; median OS was 12.4 and 11.1 weeks, respectively. No unexpected toxicities were observed.
Conclusions:
- Palbociclib monotherapy demonstrated no clinical activity in advanced pancreatic or biliary cancers with CDKN2A loss or mutation.
- The observed toxicity profile of palbociclib was consistent with prior experience in other cancer types.
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