Treatment of Anti-NMDA-Receptor Encephalitis in Children Under 18 When Initial Second-Line Immunotherapy Has Not Achieved Adequate Improvement
This protocol applies to children under 18 years with standard (non-severe) NMDA receptor antibody encephalitis whose first course of second-line immunotherapy has not resulted in adequate improvement within 1–3 months.
Patient Population
Children aged under 18 years with NMDA receptor antibody encephalitis of standard (non-severe) severity — meaning none of the severe safety and function markers are present. Severe disease is defined by the presence of any one or more severity markers affecting safety and function; their absence places the patient in the standard category addressed here.
Prior Treatment Has Not Met Its Goal
The preceding step — second-line immunotherapy with IV rituximab, or cyclophosphamide where rituximab was contraindicated or not available — is considered to have fallen short when adequate improvement has not been achieved within 1–3 months of starting that therapy. This protocol describes the escalation step taken in response to that shortfall.
Escalation Approach (partial overview)
When the first second-line agent has not produced adequate improvement, escalation involves a further immunotherapy option — and in the most refractory patients, an additional escalation approach beyond that. The specific agents, sequencing, and the criteria that determine each step are set out fully in the structured regimen.
Clinical goal: adequate improvement, assessed approximately 1–3 months after the next intervention.
References
DOI: 10.1212/NXI.0000000000001052
- Severe is defined by issues affecting SAFETY and FUNCTION.
- Any (≥1) of the severity markers present below (items a-i) scoring "severe" rather than "standard" would classify as severe disease.
- Another second-line therapy (i.e., CYC, if RTX was used first, or vice versa) can be used in any patient with severe disease who fails to improve adequately 1–3 mo following initiation of the first second-line immunotherapy.
- Escalation to IV tocilizumab should be considered only in the most refractory patients who fail to improve adequately after about 1–3 mo of treatment with RTX and/or CYC.
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