biostudies-literatureFan CWu Jieping Medical FoundationBeijing Association for Cancer Control and Prevention1559-1566https://www.ebi.ac.uk/biostudies/studies/S-EPMC12309713biostudies-literatureUnknown27(6)<h4>Background</h4>Leptomeningeal metastases (LMs) are serious complications of non-small cell lung cancer (NSCLC). This study aimed to investigate the safety and efficacy of intrathecal immune checkpoint inhibitors (ICIs) in treating NSCLC-LM.<h4>Methods</h4>We conducted this prospective phase 1 study (ChiCTR2200062245) using a traditional "3+3" design with intrathecal sintilimab (dose escalation 10, 20, 30, and 40 mg) for NSCLC-LM patients who had progressed from targeted therapy and intrathecal pemetrexed. The primary study endpoints were safety and recommended dose, and the secondary endpoints included clinical response rate, progression-free survival (PFS), intracranial progression-free survival (iPFS), and overall survival (OS).<h4>Results</h4>No dose-limiting toxicity was found at 1Neuro-oncologyIntrathecal sintilimab for leptomeningeal metastases of non-small cell lung cancer failed from targeted therapy and intrathecal chemotherapy (LMIS study).PMC12309713320.6750.2022-16-3CAPTRALung2021008Huang DHu YZhang JLv YMan SJiang QWang GDu YLi LTeng CZhang ZXin TSong XDu LFan CShen WfalseIntrathecal sintilimab for leptomeningeal metastases of non-small cell lung cancer failed from targeted therapy and intrathecal chemotherapy (LMIS study).<h4>Background</h4>Leptomeningeal metastases (LMs) are serious complications of non-small cell lung cancer (NSCLC). This study aimed to investigate the safety and efficacy of intrathecal immune checkpoint inhibitors (ICIs) in treating NSCLC-LM.<h4>Methods</h4>We conducted this prospective phase 1 study (ChiCTR2200062245) using a traditional "3+3" design with intrathecal sintilimab (dose escalation 10, 20, 30, and 40 mg) for NSCLC-LM patients who had progressed from targeted therapy and intrathecal pemetrexed. The primary study endpoints were safety and recommended dose, and the secondary endpoints included clinical response rate, progression-free survival (PFS), intracranial progression-free survival (iPFS), and overall survival (OS).<h4>Results</h4>No dose-limiting toxicity was found at 12025-01-01T00:00:00Z2025 Jul2026-07-14T21:27:00.555Z2026-06-25T03:07:54.626ZS-EPMC123097133989902910.1093/neuonc/noaf025