Mycophenolate mofetil: An alternative disease-modifying agent for MOG-IgG-associated disorders in childhood: A single-center bidirectional cohort study.

OBJECTIVE

To evaluate the efficacy of mycophenolate mofetil (MMF) in the treatment of childhood MOG-IgG-associated disorder (MOGAD).

METHODS

Thirty patients diagnosed with relapsing MOGAD and treated with MMF for >1 year from a childhood MOGAD ambispective cohort were included in the study. The clinical characteristics, therapeutic regimen, side effects, annualized relapse rate (ARR), and Expanded Disability Status Scale (EDSS) scores of these patients were evaluated.

RESULTS

The median age of disease onset was 7.05 (2.50-12.75) years. The male to female ratio was 1:1.31. All patients used MMF as first-line maintenance treatment. The median time to add MMF from disease onset was 1.08 (0.25-5.00) year. The median number of attacks before MMF initiation was 2 (2 - 8). The median duration of MMF therapy was 2.13 (1.00-3.58) years. Twenty (66.67%) patients did not experience further attacks during MMF therapy. The Kaplan-Meier curves showed a 3-year relapse-free rate of 59.8% (95% CI, 36.62-76.88%). ARR decreased during MMF therapy (0 (0 - 1.72) vs. 1.25 (0.60-4.00); P < 0.05). EDSS stabilized during MMF therapy (1.0 (0 - 2.0) vs. 0 (0 - 2.0); P = 0.206). None of the patients stopped the use of MMF due to intolerable side effects. Onset age, sex, phenotype of the first attack, ARR before MMF, MOG-IgG titers, and combined long-term prednisone (prednisone <10 mg daily for patients >40 kg or <5 mg daily for patients ≤40 kg longer than 6 months) did not predict recurrence during MMF therapy in univariate analysis.

CONCLUSIONS

MMF was effective and safe for treating childhood MOGAD. No clinical feature that could predict efficacy of MMF was found in pediatric patients with MOGAD.