ARID1A alterations function as a biomarker for longer progression-free survival after anti-PD-1/PD-L1 immunotherapy

Background Several cancer types harbor alterations in the gene encoding AT-Rich Interactive Domain-containing protein 1A (ARID1A), but there are no approved therapies to address these alterations. Recent studies have shown that ARID1A deficiency compromises mismatch repair proteins. Herein, we analy...

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Main Authors: Razelle Kurzrock, Ryosuke Okamura, Shumei Kato, Suzanna Lee, Rebecca E Jimenez, Jason K Sicklick
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/e000438.full
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author Razelle Kurzrock
Ryosuke Okamura
Shumei Kato
Suzanna Lee
Rebecca E Jimenez
Jason K Sicklick
author_facet Razelle Kurzrock
Ryosuke Okamura
Shumei Kato
Suzanna Lee
Rebecca E Jimenez
Jason K Sicklick
author_sort Razelle Kurzrock
collection DOAJ
description Background Several cancer types harbor alterations in the gene encoding AT-Rich Interactive Domain-containing protein 1A (ARID1A), but there are no approved therapies to address these alterations. Recent studies have shown that ARID1A deficiency compromises mismatch repair proteins. Herein, we analyzed 3403 patients who had tumor tissue next-generation sequencing.Findings Among nine cancer subtypes with >5% prevalence of ARID1A alterations, microsatellite instability-high as well as high tumor mutational burden was significantly more frequent in ARID1A-altered versus ARID1A wild-type tumors (20% vs 0.9%, p<0.001; and 26% vs 8.4%, p<0.001, respectively). Median progression-free survival (PFS) after checkpoint blockade immunotherapy was significantly longer in the patients with ARID1A-altered tumors (n=46) than in those with ARID1A wild-type tumors (n=329) (11 months vs 4 months, p=0.006). Also, multivariate analysis showed that ARID1A alterations predicted longer PFS after checkpoint blockade (HR (95% CI), 0.61 (0.39 to 0.94), p=0.02) and this result was independent of microsatellite instability or mutational burden; median overall survival time was also longer in ARID1A-altered versus wild-type tumors (31 months vs 20 months), but did not reach statistical significance (p=0.13).Conclusions Our findings suggest that ARID1A alterations merit further exploration as a novel biomarker correlating with better outcomes after checkpoint blockade immunotherapy.
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spelling doaj-art-a3e99685d1364a2da58731a4e9e5b73d2024-11-08T16:00:08ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262020-05-018110.1136/jitc-2019-000438ARID1A alterations function as a biomarker for longer progression-free survival after anti-PD-1/PD-L1 immunotherapyRazelle Kurzrock0Ryosuke Okamura1Shumei Kato2Suzanna Lee3Rebecca E Jimenez4Jason K Sicklick5Medical College of Wisconsin, Milwaukee, Wisconsin, USA1 Center for Personalized Cancer Therapy, UC San Diego Moores Cancer Center, La Jolla, California, USA5Moores UCSD Cancer Center, La Jolla, CA, USA2University of California, San Diego, San Diego, CA, USA1 Center for Personalized Cancer Therapy, UC San Diego Moores Cancer Center, La Jolla, California, USA1 Center for Personalized Cancer Therapy, UC San Diego Moores Cancer Center, La Jolla, California, USABackground Several cancer types harbor alterations in the gene encoding AT-Rich Interactive Domain-containing protein 1A (ARID1A), but there are no approved therapies to address these alterations. Recent studies have shown that ARID1A deficiency compromises mismatch repair proteins. Herein, we analyzed 3403 patients who had tumor tissue next-generation sequencing.Findings Among nine cancer subtypes with >5% prevalence of ARID1A alterations, microsatellite instability-high as well as high tumor mutational burden was significantly more frequent in ARID1A-altered versus ARID1A wild-type tumors (20% vs 0.9%, p<0.001; and 26% vs 8.4%, p<0.001, respectively). Median progression-free survival (PFS) after checkpoint blockade immunotherapy was significantly longer in the patients with ARID1A-altered tumors (n=46) than in those with ARID1A wild-type tumors (n=329) (11 months vs 4 months, p=0.006). Also, multivariate analysis showed that ARID1A alterations predicted longer PFS after checkpoint blockade (HR (95% CI), 0.61 (0.39 to 0.94), p=0.02) and this result was independent of microsatellite instability or mutational burden; median overall survival time was also longer in ARID1A-altered versus wild-type tumors (31 months vs 20 months), but did not reach statistical significance (p=0.13).Conclusions Our findings suggest that ARID1A alterations merit further exploration as a novel biomarker correlating with better outcomes after checkpoint blockade immunotherapy.https://jitc.bmj.com/content/8/1/e000438.full
spellingShingle Razelle Kurzrock
Ryosuke Okamura
Shumei Kato
Suzanna Lee
Rebecca E Jimenez
Jason K Sicklick
ARID1A alterations function as a biomarker for longer progression-free survival after anti-PD-1/PD-L1 immunotherapy
Journal for ImmunoTherapy of Cancer
title ARID1A alterations function as a biomarker for longer progression-free survival after anti-PD-1/PD-L1 immunotherapy
title_full ARID1A alterations function as a biomarker for longer progression-free survival after anti-PD-1/PD-L1 immunotherapy
title_fullStr ARID1A alterations function as a biomarker for longer progression-free survival after anti-PD-1/PD-L1 immunotherapy
title_full_unstemmed ARID1A alterations function as a biomarker for longer progression-free survival after anti-PD-1/PD-L1 immunotherapy
title_short ARID1A alterations function as a biomarker for longer progression-free survival after anti-PD-1/PD-L1 immunotherapy
title_sort arid1a alterations function as a biomarker for longer progression free survival after anti pd 1 pd l1 immunotherapy
url https://jitc.bmj.com/content/8/1/e000438.full
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