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Mycophenolate mofetil, azathioprine and methotrexate usage in paediatric anti-NMDAR encephalitis: A systematic
Margherita Nosadini1, Shekeeb S Mohammad2, Irene Toldo3
1Neuroimmunology Group, Institute for Neuroscience and Muscle Research, Kids Research Institute at the Children's Hospital at Westmead, University of Sydney, Australia; Paediatric Neurology and Neurophysiology Unit, Department of Women's and Children's Health, University Hospital of Padua, Italy.
Background:
Available data on mycophenolate mofetil (MMF), azathioprine (AZA) and methotrexate (MTX) for paediatric-onset anti-N-methyl-d-aspartate receptor encephalitis (anti-NMDARE) is limited.
Methods:
Systematic literature review on patients treated with MMF/AZA/MTX for paediatric-onset anti-NMDARE, with focus on modes of use, efficacy and safety.
Results:
87 patients were included (age at onset median 11 years, range 0.8-18 years; 69% females). 46% had a relapsing course. 52% received MMF, 27% AZA, 15% MTX, and 6% a combination of MMF/AZA/MTX (7 patients received intrathecal MTX). Before MMF/AZA/MTX, 100% patients received steroids, 83% intravenous immunoglobulin and 45% plasma exchange, and 50% received second-line treatments (rituximab/cyclophosphamide). MMF/AZA/MTX were administered >6 months from onset in 51%, and only after relapse in 40%. Worst mRS before MMF/AZA/MTX was median 4.5 (range 3-5). At last follow-up (median 2 years, range 0.2-8.6), median mRS was 1 (range 0-6). Median annualised relapse rate was 0.4 (range 0-6.7) pre-MMF/AZA/MTX (excluding first events), and 0 on MMF/AZA/MTX (mean 0.03, range 0-0.8). 7% patients relapsed on MMF/AZA/MTX. These relapsing patients had low rate of second-line treatments before MMF/AZA/MTX (25%), long median time between onset and MMF/AZA/MTX usage (18 months), and frequently they were started on MMF/AZA/MTX only after relapse (75%). Relapse rate was lower among patients who received first immune therapy ≤30 days (25%) than later (64%), who received second-line treatments at first event (14%) rather than not (64%), who were started on MMF/AZA/MTX after the first (12%) rather than subsequent events (17%), and who were started on MMF/AZA/MTX ≤3 months from onset (33%) rather than later (53%). Adverse reactions to MMF/AZA/MTX occurred in 2 cases (cytomegalovirus colitis and respiratory infection), of grade 3 Common Terminology Criteria for Adverse Events v4.0.
Discussion:
Our literature review disclosed heterogeneity in the use of MMF/AZA/MTX in paediatric-onset anti-NMDARE. MMF/AZA/MTX usage is mostly restricted to retrospective cohort descriptions. These agents may reduce risk of relapse, and have a reasonable safety profile, however data on larger cohorts are required to definitively determine effect.
Insights
Mycophenolate mofetil (MMF), azathioprine (AZA), and methotrexate (MTX) show potential in reducing relapses for pediatric anti-N-methyl-D-aspartate receptor encephalitis (anti-NMDARE). Further research is needed to confirm their efficacy and safety in larger patient cohorts.
Area of Science:
- Neurology
- Immunology
- Pediatrics
Background:
- Limited data exists on the use of mycophenolate mofetil (MMF), azathioprine (AZA), and methotrexate (MTX) for pediatric-onset anti-N-methyl-D-aspartate receptor encephalitis (anti-NMDARE).
- Anti-NMDARE is a severe autoimmune condition affecting the central nervous system, often requiring immunosuppressive therapies.
Purpose of the Study:
- To systematically review the available literature on the use of MMF, AZA, and MTX in pediatric-onset anti-NMDARE.
- To analyze the modes of administration, efficacy, and safety profiles of these immunosuppressive agents in this patient population.
Main Methods:
- A systematic literature review was conducted.
- Included patients were treated with MMF, AZA, or MTX for pediatric-onset anti-NMDARE.
- Data on treatment protocols, clinical outcomes, and adverse events were extracted and analyzed.
Main Results:
- Eighty-seven patients were included, with a median age of 11 years at onset; 46% experienced relapses.
- MMF, AZA, and MTX were administered to 52%, 27%, and 15% of patients, respectively, often after other treatments.
- Treatment with MMF/AZA/MTX was associated with a reduced annual relapse rate (0.03) and only 7% of patients relapsed while on these medications.
- Adverse reactions were infrequent and generally low-grade.
Conclusions:
- There is significant heterogeneity in the application of MMF, AZA, and MTX for pediatric anti-NMDARE.
- These agents demonstrate a potential to reduce relapse rates and possess a reasonable safety profile.
- Larger, prospective studies are necessary to definitively establish the efficacy and optimal use of MMF, AZA, and MTX in pediatric anti-NMDARE.
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