• Ramona Cordani, Concetta Micalizzi, Thea Giacomini, Matteo Gastaldi, Diego Franciotta, Francesca Fioredda, Silvia Buratti, Giovanni Morana, Daniela Pirlo, Salvatore Renna, Elio Castagnola, Marco Risso, Paola Lanteri, Maria Stella Vari, and Maria Margherita Mancardi.
• Unit of Child Neuropsichiatry, Department of Neuroscience, Ophthalmology Genetics and Maternal Infantile Sciences (DINOGMI), University of Genoa, IRCCS Giannina Gaslini, Genova, Italy.
• Pediatr. Neurol. 2020 Feb 1; 103: 61-64.

BackgroundAnti-N-methyl-d-aspartate receptor encephalitis is a central nervous system inflammatory autoimmune disease affecting adults and children. The use of first- and second-line immunotherapies is supported. Recent reports suggest the efficacy of bortezomib in severe anti-N-methyl-d-aspartate encephalitis in adult patients not responsive to second-line treatment; there are no data about pediatric patients.Patient DescriptionWe describe an eight-year-old child with anti-N-methyl-d-aspartate encephalitis not responsive to first- and second-line treatments who experienced marked clinical improvement after bortezomib administration.DiscussionBortezomib is a selective and reversible inhibitor of the 26S proteasome, which is used to treat oncologic and rare autoimmune disorders in pediatric patients. As observed in adult patients, bortezomib administration induced anti-N-methyl-d-aspartate antibody titer decline and clinical improvement with an acceptable risk profile.ConclusionThis is the first report of the use of bortezomib in children with anti-N-methyl-d-aspartate encephalitis; it could be a useful therapeutic option in children with refractory anti-N-methyl-d-aspartate encephalitis.Copyright © 2019 Elsevier Inc. All rights reserved.

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