Bortezomib-Responsive Refractory Anti-N-Methyl-d-Aspartate Receptor Encephalitis

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Abstract

BACKGROUND: Anti-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 DESCRIPTION: We 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.

DISCUSSION: Bortezomib 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.

CONCLUSION: This 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.

Original languageEnglish
JournalPediatric Neurology
DOIs
Publication statusE-pub ahead of print - Oct 19 2019

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Encephalitis
Aspartic Acid
Autoimmune Diseases of the Nervous System
Pediatrics
Immunotherapy
Therapeutics
Central Nervous System
aspartic acid receptor
Bortezomib
Antibodies

Cite this

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title = "Bortezomib-Responsive Refractory Anti-N-Methyl-d-Aspartate Receptor Encephalitis",
abstract = "BACKGROUND: Anti-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 DESCRIPTION: We 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.DISCUSSION: Bortezomib 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.CONCLUSION: This 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.",
author = "Ramona Cordani and Concetta Micalizzi and Thea Giacomini and Matteo Gastaldi and Diego Franciotta and Francesca Fioredda and Silvia Buratti and Giovanni Morana and Daniela Pirlo and Salvatore Renna and Elio Castagnola and Marco Risso and Paola Lanteri and Vari, {Maria Stella} and Mancardi, {Maria Margherita}",
note = "Copyright {\circledC} 2019 Elsevier Inc. All rights reserved.",
year = "2019",
month = "10",
day = "19",
doi = "10.1016/j.pediatrneurol.2019.09.004",
language = "English",
journal = "Pediatric Neurology",
issn = "0887-8994",
publisher = "Elsevier Inc.",

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TY - JOUR

T1 - Bortezomib-Responsive Refractory Anti-N-Methyl-d-Aspartate Receptor Encephalitis

AU - Cordani, Ramona

AU - Micalizzi, Concetta

AU - Giacomini, Thea

AU - Gastaldi, Matteo

AU - Franciotta, Diego

AU - Fioredda, Francesca

AU - Buratti, Silvia

AU - Morana, Giovanni

AU - Pirlo, Daniela

AU - Renna, Salvatore

AU - Castagnola, Elio

AU - Risso, Marco

AU - Lanteri, Paola

AU - Vari, Maria Stella

AU - Mancardi, Maria Margherita

N1 - Copyright © 2019 Elsevier Inc. All rights reserved.

PY - 2019/10/19

Y1 - 2019/10/19

N2 - BACKGROUND: Anti-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 DESCRIPTION: We 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.DISCUSSION: Bortezomib 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.CONCLUSION: This 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.

AB - BACKGROUND: Anti-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 DESCRIPTION: We 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.DISCUSSION: Bortezomib 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.CONCLUSION: This 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.

U2 - 10.1016/j.pediatrneurol.2019.09.004

DO - 10.1016/j.pediatrneurol.2019.09.004

M3 - Article

C2 - 31759783

JO - Pediatric Neurology

JF - Pediatric Neurology

SN - 0887-8994

ER -