Mycophenolate Mofetil Treatment Reduces Relapse Risk in NMDARE Patients
Adjunctive treatment with mycophenolate mofetil (MMF) reduced the risk of relapse in patients with anti-N-methyl-D-aspartate receptor encephalitis (NMDARE), according to study results published in the Journal of Neurology, Neurosurgery & Psychiatry.
“Our findings indicate that long-term add-on therapy with MMF was safe, well-tolerated and showed efficacy in preventing relapse and reducing neurological deficits compared with first-line treatments alone in individuals with new-onset NMDARE,” wrote lead author Xue Gong, Department of Neurology, West China Hospital of Sichuan University, Chengdu, Sichuan, China, and study coauthors.
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The multicenter, open-label, blinded end point, randomized controlled LEARN trial involved 100 patients aged 14 or older with acute NMDARE. To be eligible, participants must have received first-line treatments within 2 weeks of presentation to the hospital and have a modified Rankin scale (mRS) score of 2 or more. Participants were randomly assigned to receive either first-line treatment for NMDARE (n=49) or a first-line treatment along with an adjunctive MMF treatment (n=51) for 24 months. Researchers sought to compare relapse rates and treatment response (at least a 1-point improvement in mRS score within 4 weeks of baseline) between the 2 groups. Safety was also assessed based on the rate and severity of adverse events (AE).
The study found that participants in the MMF group had a relapse rate of 5.9%, while those receiving only first-line treatment had a relapse rate of 26.5% (p=0.006). Results also showed that those in the MMF group had a better treatment response (84.3% vs 65.3%; p=0.03), and that the safety of the MMF group was consistent with the first-line treatment group. Several secondary outcomes, including fatigue, cognitive impairment, depression, and seizure, were lower in the MMF group at 6 months (76.5% vs 53.1%; p = 0.02).
“This supports the use of MMF as a safe and effective immunosequential therapy for patients with NMDARE at high risk of relapse,” the authors wrote.
The researchers noted several limitations of their study, including its open-label nature, which may have introduced observer bias or placebo effects. The study also excluded certain patient populations, including those under 14 and those with severe NMDARE, which might limit its representation of the full population of NMDARE patients. Furthermore, the study recruited participants from 4 academic hospital centers in China, meaning its results may not be generalizable to other populations.
The authors also acknowledged that NMDARE has a significantly lower relapse risk than multiple sclerosis (MS) and neuromyelitis optica spectrum disorder (NMOSD), suggesting that future research should explore MMF efficacy on higher risk patients and those who are most likely to benefit from long-term treatment.
