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.

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

Micalizzi C.;Morana G.;Pirlo D.;Renna S.;
2020

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.
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Anti-N-methyl-D-aspartate receptor (NMDAR) antibodies; Arterial spin labeling (ASL); Autoimmune encephalitis; Cell-based assay; Immunosuppressive therapy; Proteasome inhibitor; Refractory status epilepticus
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.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2318/1752981
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