Bortezomib for treatment of anti-NMDA receptor encephalitis in a pediatric patient refractory to conventional therapy.

PURPOSE

The case of a pediatric patient with treatment refractory anti-N-methyl-d-aspartate (NMDA) receptor encephalitis treated with the plasma cell-depleting therapy bortezomib is reported.

SUMMARY

A 5-year-old female presented to the hospital with a 1-week history of altered mental status, agitation, and possible seizure-like activity. She was admitted to the hospital for suspected meningitis or meningoencephalitis and an extensive workup was completed, including sending blood and cerebrospinal fluid (CSF) for testing for NMDA receptor antibodies. While test results were pending, the patient was treated initially with intravenous immunoglobulin (IVIG) for 4 days followed by high-dose methylprednisolone for 5 days. The patient's serum and CSF studies were positive for NMDA receptor antibodies, confirming the diagnosis of anti-NMDA receptor encephalitis. She was then treated with plasmapheresis therapy every other day for 5 treatments, without any clinical improvement. The patient then received rituximab once weekly for 6 weeks. Three weeks after completion of rituximab therapy, the patient was started on her first cycle of bortezomib therapy. She received a total of 6 cycles, with improvement in her clinical status beginning with the third cycle. Upon completion of 6 cycles, the patient's mental status and level of functioning had greatly improved. She was discharged to an inpatient rehabilitation facility and ultimately able to return home to her family.

CONCLUSION

A 5-year-old female with anti-NMDA receptor encephalitis was successfully treated with bortezomib after having shown no clinical improvement during treatment with IVIG, high-dose methylprednisolone, plasmapheresis, and rituximab.