ArticleJournal for immunotherapy of cancer2021
Pembrolizumab for patients with leptomeningeal metastasis from solid tumors: efficacy, safety, and cerebrospinal fluid biomarkers.
Article in Journal for immunotherapy of cancer, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03091478 (Pembrolizumab in Patients With Leptomeningeal Disease), which is not on this map. Cited by 54 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Pembrolizumab in Patients With Leptomeningeal Disease
Who cites it
54 citing papers in PubMed, 63 citations in OpenAlex.
- Exploring uncertainties in melanoma management: a Spanish multidisciplinary Delphi consensus.Clinical & translational oncology : official publication of the Federation of Spanish Oncology Societies and of the National Cancer Institute of Mexico · 2026Article
- Review
- Review
- Intrathecal nivolumab in metastatic solid tumors with leptomeningeal disease: dose escalation part of the multicenter IT-PD1/NOA-26 phase 1 trial.Nature cancer · 2026Article
- Tocilizumab for severe immune checkpoint inhibitor myopathy-myocarditis: acute and long-term outcomes.Journal of neurology · 2026Article
- CSF proteomics identifies ADGRG1 as a predictive biomarker of intrathecal immune checkpoint inhibitor response in leptomeningeal metastasis.Journal for immunotherapy of cancer · 2026Article
- Leptomeningeal metastasis from large-cell neuroendocrine carcinoma of the cervix: a case report and literature review.Frontiers in oncology · 2026Article
- Overview of ocular toxicities associated with breast cancer therapies.Breast cancer research and treatment · 2025Review
- Advances in the research of leptomeningeal metastases in non-small cell lung cancer: a narrative review.Translational lung cancer research · 2025Review
- Article
- Chinese Expert Consensus on Leptomeningeal Metastases of Lung Cancer.Thoracic cancer · 2025Article
- Leptomeningeal Disease: Current Approaches and Future Directions.Current neurology and neuroscience reports · 2025Review
- The research progress on meningeal metastasis in solid tumors.Discover oncology · 2025Review
- NANO-LM: An updated scorecard for the clinical assessment of patients with leptomeningeal metastases.Neuro-oncology · 2025Article
- Leptomeningeal metastatic disease: new frontiers and future directions.Nature reviews. Clinical oncology · 2025Review
- Treatment options for EGFR mutation-positive NSCLC patients with leptomeningeal metastases after third-generation TKI resistance: a retrospective real-world study.Therapeutic advances in medical oncology · 2025Article
- A rare case of vision loss caused by leptomeningeal metastasis of lung adenocarcinoma: a case report and literature review.Frontiers in oncology · 2025Article
- A review on intrathecal administration of medications for leptomeningeal metastases in solid tumors.Frontiers in pharmacology · 2025Review
- Leptomeningeal metastases from solid tumors: A Society for Neuro-Oncology and American Society of Clinical Oncology consensus review on clinical management and future directions.Neuro-oncology · 2024Review
- Radiotherapy and Systemic Treatment for Leptomeningeal Disease.Biomedicines · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
23 authors at 4 institutions in 2 countries.
Funding
Abstract
backgroundThe benefit of immune checkpoint inhibitors (ICIs) in patients with leptomeningeal metastases (LMM) is unknown.
methodsWe 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.
resultsThirteen of a planned 16 patients were treated between April 2017 and December 2019. The study closed early for poor accrual. Median age was 57 years (range: 22-79). Sixty-two percent of patients had tumors not traditionally ICI-responsive (hormone-receptor (HR)-positive breast carcinoma=39%; high-grade glioma=23%), while 38% had ICI-responsive tumors (non-small cell lung cancer (NSCLC)=23%, head and neck carcinoma=8%, cutaneous squamous carcinoma (CSC)=8%). CNS response was observed in 38% of patients at 12 weeks (95% CI 13.9% to 68.4%) by pre-defined criteria and LM-RANO, and 2 achieved durable complete responses (CSC=1, overall survival (OS) 3+ years; NSCLC=1, OS 9 months). Median CNS progression-free survival and OS was 2.9 months (95% CI 1.3 to NR) and 4.9 months (95% CI 3.7 to NR), respectively. Grade 3+ treatment-related adverse events occurred in 15% of patients. Sensitivity for LMM detection by t-DNA and cytopathology was 84.6% (95% CI 54.6% to 98.1%) and 53.9% (95% CI 25.1% to 80.8%), respectively. Pre-therapy and on-therapy CSF cytokine analysis demonstrated complete responders clustered together.
conclusionsPembrolizumab conferred a 38% CNS response rate in patients with LMM, a tolerable safety profile, and deep responses in selected patients with ICI-responsive tumors. CSF t-DNA may be sensitive for LMM detection, and immunologic subsets of CNS response warrant further study. TRIAL REGISTRATION NUMBER: NCT03091478.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.