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Research Letter
September 5, 2019

Clinical Validation of PBRM1 Alterations as a Marker of Immune Checkpoint Inhibitor Response in Renal Cell Carcinoma

Author Affiliations
  • 1Dana-Farber Cancer Institute, Boston, Massachusetts
  • 2Bristol-Myers Squibb, Lawrenceville, New Jersey
JAMA Oncol. Published online September 5, 2019. doi:10.1001/jamaoncol.2019.3158

Nivolumab, an immune checkpoint inhibitor (ICI) targeting the programmed death-1 (PD-1) pathway, is approved for metastatic clear cell renal cell carcinoma (ccRCC).1 Loss-of-function (truncating) mutations in PBRM1, a PBAF-complex gene commonly mutated in ccRCC, were previously associated with clinical benefit from anti–PD-1 therapy in a smaller study,2 Herein, this association was examined in an independent cohort from a randomized clinical trial1 to determine whether PBRM1 alterations are a marker of response to ICI treatment.