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Bortezomib-Responsive Refractory Anti-N-Methyl-d-Aspartate Receptor Encephalitis.
Pediatr Neurol. 2020 02; 103:61-64.PN

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.

Authors+Show Affiliations

Unit of Child Neuropsichiatry, Department of Neuroscience, Ophthalmology Genetics and Maternal Infantile Sciences (DINOGMI), University of Genoa, IRCCS Giannina Gaslini, Genova, Italy.Unit of Hematology, IRCCS Giannina Gaslini, Genova, Italy.Unit of Child Neuropsichiatry, Department of Neuroscience, Ophthalmology Genetics and Maternal Infantile Sciences (DINOGMI), University of Genoa, IRCCS Giannina Gaslini, Genova, Italy.Neuroimmunology Laboratory, IRCCS Mondino Foundation, Pavia, Italy.Neuroimmunology Laboratory, IRCCS Mondino Foundation, Pavia, Italy.Unit of Hematology, IRCCS Giannina Gaslini, Genova, Italy.Neonatal and Pediatric Intensive Care Unit, IRCCS Giannina Gaslini, Genova, Italy.Unit of Neuroradiology, IRCCS Giannina Gaslini, Genova, Italy.Department of Emergency, IRCCS Giannina Gaslini, Genova, Italy.Department of Emergency, IRCCS Giannina Gaslini, Genova, Italy.Unit of Infectious Diseases, IRCCS Giannina Gaslini, Genova, Italy.Unit of Immunology and Transfusional Medicine, IRCCS Giannina Gaslini, Genova, Italy.Unit of Neurophysiology, IRCCS Giannina Gaslini, Genova, Italy.Unit of Pediatric Neurology and Neurogenerative Diseases, IRCCS Giannina Gaslini, Genova, Italy.Unit of Child Neuropsichiatry, Department of Clinical and Surgical Neurosciences and Rehabilitation, IRCCS Giannina Gaslini, Genova, Italy. Electronic address: margheritamancardi@gaslini.org.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

31759783

Citation

Cordani, Ramona, et al. "Bortezomib-Responsive Refractory Anti-N-Methyl-d-Aspartate Receptor Encephalitis." Pediatric Neurology, vol. 103, 2020, pp. 61-64.
Cordani R, Micalizzi C, Giacomini T, et al. Bortezomib-Responsive Refractory Anti-N-Methyl-d-Aspartate Receptor Encephalitis. Pediatr Neurol. 2020;103:61-64.
Cordani, R., Micalizzi, C., Giacomini, T., Gastaldi, M., Franciotta, D., Fioredda, F., Buratti, S., Morana, G., Pirlo, D., Renna, S., Castagnola, E., Risso, M., Lanteri, P., Vari, M. S., & Mancardi, M. M. (2020). Bortezomib-Responsive Refractory Anti-N-Methyl-d-Aspartate Receptor Encephalitis. Pediatric Neurology, 103, 61-64. https://doi.org/10.1016/j.pediatrneurol.2019.09.004
Cordani R, et al. Bortezomib-Responsive Refractory Anti-N-Methyl-d-Aspartate Receptor Encephalitis. Pediatr Neurol. 2020;103:61-64. PubMed PMID: 31759783.
* Article titles in AMA citation format should be in sentence-case
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, Y1 - 2019/10/19/ PY - 2019/06/03/received PY - 2019/08/23/revised PY - 2019/09/09/accepted PY - 2019/11/25/pubmed PY - 2019/11/25/medline PY - 2019/11/25/entrez KW - Anti-N-methyl-d-aspartate receptor (NMDAR) antibodies KW - Arterial spin labeling (ASL) KW - Autoimmune encephalitis KW - Cell-based assay KW - Immunosuppressive therapy KW - Proteasome inhibitor KW - Refractory status epilepticus SP - 61 EP - 64 JF - Pediatric neurology JO - Pediatr. Neurol. VL - 103 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. SN - 1873-5150 UR - https://www.unboundmedicine.com/medline/citation/31759783/Bortezomib_Responsive_Refractory_Anti_N_Methyl_d_Aspartate_Receptor_Encephalitis_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0887-8994(19)30879-3 DB - PRIME DP - Unbound Medicine ER -
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