Case report| Volume 37, 101447, January 2020

Neuromyelitis optica spectrum disorder after treatment with pembrolizumab

Published:October 14, 2019DOI:


      • Pembrolizumab induced an anti-AQP4 antibody-positive NMOSD.
      • The results from flow cytometry of the CSF cells revealed increased plasmablasts.
      • Discontinuation of pembrolizumab with immune therapy led to a complete remission.


      While immune checkpoint inhibitors (ICIs) have contributed to the development of therapeutic treatments for previously incurable advanced malignancies, they may induce immune-related adverse events (irAEs) in many organs including the CNS [1]. Because immune checkpoint molecules are predominantly expressed on T cells, irAEs are largely not B cell-mediated. Here, we report a patient who was treated with pembrolizumab (a PD-1 monoclonal antibody) for lung adenocarcinoma with brain metastasis, and who developed anti-aquaporin-4 antibody (AQP4-Ab) positive neuromyelitis optica spectrum disorder (NMOSD). We hypothesized that PD-1 immune checkpoint blockage might induce a B cell-mediated immune response in CNS resulting in this complication, which was further supported by the observation of a transient increase in plasmablasts in their CSF.


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