Phase II study of pembrolizumab and capecitabine for triple negative and hormone receptor-positive, HER2−negative endocrine-refractory metastatic breast cancer

Background Response rates to single agent immune checkpoint blockade in unselected pretreated HER2−negative metastatic breast cancer (MBC) are low. However, they may be augmented when combined with chemotherapy.Methods We conducted a single-arm, phase II study of patients with triple negative (TN) o...

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Main Authors: Ami N Shah, Lisa Flaum, Irene Helenowski, Cesar A Santa-Maria, Sarika Jain, Alfred Rademaker, Valerie Nelson, Dean Tsarwhas, Massimo Cristofanilli, William Gradishar
Format: Article
Language:English
Published: BMJ Publishing Group 2020-05-01
Series:Journal for ImmunoTherapy of Cancer
Online Access:https://jitc.bmj.com/content/8/1/e000173.full
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author Ami N Shah
Lisa Flaum
Irene Helenowski
Cesar A Santa-Maria
Sarika Jain
Alfred Rademaker
Valerie Nelson
Dean Tsarwhas
Massimo Cristofanilli
William Gradishar
author_facet Ami N Shah
Lisa Flaum
Irene Helenowski
Cesar A Santa-Maria
Sarika Jain
Alfred Rademaker
Valerie Nelson
Dean Tsarwhas
Massimo Cristofanilli
William Gradishar
author_sort Ami N Shah
collection DOAJ
description Background Response rates to single agent immune checkpoint blockade in unselected pretreated HER2−negative metastatic breast cancer (MBC) are low. However, they may be augmented when combined with chemotherapy.Methods We conducted a single-arm, phase II study of patients with triple negative (TN) or hormone receptor-positive endocrine-refractory (HR+) MBC who were candidates for capecitabine. Patients were treated with pembrolizumab 200 mg intravenously day 1 and capecitabine 1000 mg/m2 by mouth twice daily on days 1–14 of a 21-day cycle. The primary end point was median progression-free survival (mPFS) compared with historic controls and secondary end points were overall response rate (ORR), safety and tolerability. The study had 80% power to detect a 2-month improvement in mPFS with the addition of pembrolizumab over historic controls treated with capecitabine alone.Results Thirty patients, 16 TN and 14 HR+ MBC, were enrolled from 2017 to 2018. Patients had a median age of 51 years and received a median of 1 (range 0–6) prior lines of therapy for MBC. Of 29 evaluable patients, the mPFS was 4.0 (95% CI 2.0 to 6.4) months and was not significantly longer than historic controls of 3 months. The median overall survival was 15.4 (95% CI 8.2 to 20.3) months. The ORR was 14% (n=4), stable disease (SD) was 41% (n=12) and clinical benefit rate (CBR=partial response+SD>6 months) was 28% (n=8). The ORR and CBR were not significantly different between disease subtypes (ORR 13% and 14%, CBR 25% and 29% for TN and HR+, respectively). The 1-year PFS rate was 20.7% and three patients have ongoing responses. The most common adverse events were low grade and consistent with those seen in MBC patients receiving capecitabine, including hand-foot syndrome, gastrointestinal symptoms, fatigue and cytopenias. Toxicities at least possibly from pembrolizumab included grade 3 or 4 liver test abnormalities (7%), rash (7%) and diarrhea (3%), as well as grade 5 hepatic failure in a patient with liver metastases.Conclusions Compared with historical controls, pembrolizumab with capecitabine did not improve PFS in this biomarker unselected, pretreated cohort. However, some patients had prolonged disease control.Trial registration number NCT03044730.
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spelling doaj-art-8f13f23e68dd4f8e940351b6de1ccf172024-11-08T18:15:10ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262020-05-018110.1136/jitc-2019-000173Phase II study of pembrolizumab and capecitabine for triple negative and hormone receptor-positive, HER2−negative endocrine-refractory metastatic breast cancerAmi N Shah0Lisa Flaum1Irene Helenowski2Cesar A Santa-Maria3Sarika Jain4Alfred Rademaker5Valerie Nelson6Dean Tsarwhas7Massimo Cristofanilli8William Gradishar91 Robert H Lurie Comprehensive Cancer Center of Northwestern University, Chicago, Illinois, USA1 Robert H Lurie Comprehensive Cancer Center of Northwestern University, Chicago, Illinois, USA1 Robert H Lurie Comprehensive Cancer Center of Northwestern University, Chicago, Illinois, USADepartment of Immunology, The Bloomberg–Kimmel Institute for Cancer Immunotherapy, Johns Hopkins University, Baltimore, Maryland, USA1 Robert H Lurie Comprehensive Cancer Center of Northwestern University, Chicago, Illinois, USA1 Robert H Lurie Comprehensive Cancer Center of Northwestern University, Chicago, Illinois, USA1 Robert H Lurie Comprehensive Cancer Center of Northwestern University, Chicago, Illinois, USA1 Robert H Lurie Comprehensive Cancer Center of Northwestern University, Chicago, Illinois, USA1 Robert H Lurie Comprehensive Cancer Center of Northwestern University, Chicago, Illinois, USA1 Robert H Lurie Comprehensive Cancer Center of Northwestern University, Chicago, Illinois, USABackground Response rates to single agent immune checkpoint blockade in unselected pretreated HER2−negative metastatic breast cancer (MBC) are low. However, they may be augmented when combined with chemotherapy.Methods We conducted a single-arm, phase II study of patients with triple negative (TN) or hormone receptor-positive endocrine-refractory (HR+) MBC who were candidates for capecitabine. Patients were treated with pembrolizumab 200 mg intravenously day 1 and capecitabine 1000 mg/m2 by mouth twice daily on days 1–14 of a 21-day cycle. The primary end point was median progression-free survival (mPFS) compared with historic controls and secondary end points were overall response rate (ORR), safety and tolerability. The study had 80% power to detect a 2-month improvement in mPFS with the addition of pembrolizumab over historic controls treated with capecitabine alone.Results Thirty patients, 16 TN and 14 HR+ MBC, were enrolled from 2017 to 2018. Patients had a median age of 51 years and received a median of 1 (range 0–6) prior lines of therapy for MBC. Of 29 evaluable patients, the mPFS was 4.0 (95% CI 2.0 to 6.4) months and was not significantly longer than historic controls of 3 months. The median overall survival was 15.4 (95% CI 8.2 to 20.3) months. The ORR was 14% (n=4), stable disease (SD) was 41% (n=12) and clinical benefit rate (CBR=partial response+SD>6 months) was 28% (n=8). The ORR and CBR were not significantly different between disease subtypes (ORR 13% and 14%, CBR 25% and 29% for TN and HR+, respectively). The 1-year PFS rate was 20.7% and three patients have ongoing responses. The most common adverse events were low grade and consistent with those seen in MBC patients receiving capecitabine, including hand-foot syndrome, gastrointestinal symptoms, fatigue and cytopenias. Toxicities at least possibly from pembrolizumab included grade 3 or 4 liver test abnormalities (7%), rash (7%) and diarrhea (3%), as well as grade 5 hepatic failure in a patient with liver metastases.Conclusions Compared with historical controls, pembrolizumab with capecitabine did not improve PFS in this biomarker unselected, pretreated cohort. However, some patients had prolonged disease control.Trial registration number NCT03044730.https://jitc.bmj.com/content/8/1/e000173.full
spellingShingle Ami N Shah
Lisa Flaum
Irene Helenowski
Cesar A Santa-Maria
Sarika Jain
Alfred Rademaker
Valerie Nelson
Dean Tsarwhas
Massimo Cristofanilli
William Gradishar
Phase II study of pembrolizumab and capecitabine for triple negative and hormone receptor-positive, HER2−negative endocrine-refractory metastatic breast cancer
Journal for ImmunoTherapy of Cancer
title Phase II study of pembrolizumab and capecitabine for triple negative and hormone receptor-positive, HER2−negative endocrine-refractory metastatic breast cancer
title_full Phase II study of pembrolizumab and capecitabine for triple negative and hormone receptor-positive, HER2−negative endocrine-refractory metastatic breast cancer
title_fullStr Phase II study of pembrolizumab and capecitabine for triple negative and hormone receptor-positive, HER2−negative endocrine-refractory metastatic breast cancer
title_full_unstemmed Phase II study of pembrolizumab and capecitabine for triple negative and hormone receptor-positive, HER2−negative endocrine-refractory metastatic breast cancer
title_short Phase II study of pembrolizumab and capecitabine for triple negative and hormone receptor-positive, HER2−negative endocrine-refractory metastatic breast cancer
title_sort phase ii study of pembrolizumab and capecitabine for triple negative and hormone receptor positive her2 negative endocrine refractory metastatic breast cancer
url https://jitc.bmj.com/content/8/1/e000173.full
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