Long-term maintenance of mycophenolate mofetil in anti-NMDA receptor encephalitis (LEARN): a multicentre, open-label,...

Area of Science:

• Neurology
• Immunology
• Clinical Trials

Background:

• Anti-N-methyl-D-aspartate receptor encephalitis (NMDARE) is a severe autoimmune neurological disorder associated with significant morbidity and mortality.
• Current treatments for NMDARE face challenges with patient relapse, necessitating the development of effective long-term maintenance therapies.

Purpose of the Study:

• To evaluate the efficacy and safety of mycophenolate mofetil (MMF) as a long-term adjunctive therapy in newly diagnosed NMDARE patients.
• To assess MMF's impact on relapse rates, treatment response, cognitive function, and adverse events in NMDARE management.

Main Methods:

• A prospective, randomized, open-label trial was conducted across four academic centers in China.
• 100 patients aged 14+ with acute NMDARE, treated within 2 weeks of presentation and with mRS score ≥2, were randomized to receive standard treatment with or without MMF for 24 months.
• Primary outcomes included relapse rates and time to relapse; secondary outcomes covered cognitive deficits, treatment response, and adverse events.

Main Results:

• Patients receiving MMF exhibited significantly lower relapse rates (5.9% vs. 26.5%, p=0.006) and improved treatment response (84.3% vs. 65.3%, p=0.03).
• MMF group reported reduced fatigue, cognitive impairment, depression, and seizures, with no significant difference in long-term functional prognosis at 12 and 24 months.
• Adverse events associated with MMF were mild to moderate, with no reported deaths or anaphylactic reactions.

Conclusions:

• Long-term adjunctive MMF treatment in NMDARE significantly lowers the risk of relapse.
• MMF demonstrates good tolerability and is a viable maintenance therapy option for NMDARE patients beyond 24 months.
• The study provides Class II evidence supporting MMF's role in NMDARE management.