biostudies-literatureNaidoo JNational Cancer InstituteNCI NIH HHSMerck Sharp and Dohmee002473https://www.ebi.ac.uk/biostudies/studies/S-EPMC8359453biostudies-literatureUnknown9(8)<h4>Background</h4>The benefit of immune checkpoint inhibitors (ICIs) in patients with leptomeningeal metastases (LMM) is unknown.<h4>Methods</h4>We undertook a phase II trial of pembrolizumab in patients with LMM from solid tumors. Eligible patients had radiologic/cytologic LMM and Eastern Cooperative Oncology Group performance status 0-1. Pembrolizumab was administered intravenously at 200 mg q3W until disease progression/unacceptable toxicity. The primary endpoint was central nervous system (CNS) response after four cycles, defined radiologically/cytologically/clinically. Serial cerebrospinal fluid (CSF) was assessed for tumor-derived DNA (t-DNA) aneuploidy and cytokines.<h4>Results</h4>Thirteen of a planned 16 patients were treated between April 2017 and December 2019. The study closedJournal for immunotherapy of cancerPembrolizumab for patients with leptomeningeal metastasis from solid tumors: efficacy, safety, and cerebrospinal fluid biomarkers.PMC8359453U01 CA230691NIH RA37CA230400U01CA230691R37 CA230400Papadopoulos NSchreck KCConnolly RMRiemer JNaidoo JCarvajal-Gonzalez ABettegowda CDouville CKinzler KWBrahmer JRFu WAygun NKleinberg LVenkatesan AForde PMHu CGrossman SALipson EJHoldhoff MSanta-Maria CABarnes APage BRedmond KJfalsePembrolizumab for patients with leptomeningeal metastasis from solid tumors: efficacy, safety, and cerebrospinal fluid biomarkers.<h4>Background</h4>The benefit of immune checkpoint inhibitors (ICIs) in patients with leptomeningeal metastases (LMM) is unknown.<h4>Methods</h4>We undertook a phase II trial of pembrolizumab in patients with LMM from solid tumors. Eligible patients had radiologic/cytologic LMM and Eastern Cooperative Oncology Group performance status 0-1. Pembrolizumab was administered intravenously at 200 mg q3W until disease progression/unacceptable toxicity. The primary endpoint was central nervous system (CNS) response after four cycles, defined radiologically/cytologically/clinically. Serial cerebrospinal fluid (CSF) was assessed for tumor-derived DNA (t-DNA) aneuploidy and cytokines.<h4>Results</h4>Thirteen of a planned 16 patients were treated between April 2017 and December 2019. The study closed2021-01-01T00:00:00Z2021 Aug2026-06-16T03:11:56.224Z2026-06-16T03:07:04.065ZS-EPMC83594533438066210.1136/jitc-2021-002473