Comparison of efficacy and tolerability of azathioprine, mycophenolate mofetil, and cyclophosphamide among patients with neuromyelitis optica spectrum disorder: A prospective cohort study

Abstract

Aims: To compare the efficacy and tolerability of azathioprine (AZA), mycophenolate mofetil (MMF), and cyclophosphamide (CTX) in patients with neuromyelitis optica spectrum disorder (NMOSD).

Methods: We performed a prospective cohort analysis of relapses, disability, and adverse events in NMOSD patients treated with AZA, MMF, or CTX (n=119, 38, and 41, respectively). All the patients were co-treated with oral prednisone.

Results: A significant reduction in relapse rate was found in patients taking AZA (p<0.001), MMF (p<0.001) or CTX (p=0.01). MMF was associated with a lower risk of relapse than AZA, but this difference was not statistically significant (p=0.08). AZA and MMF decreased the mean Expanded Disability Status Scale (EDSS) scores significantly (AZA: p=0.02; MMF: p=0.01), whereas CTX did not. Compared with AZA, MMF had a significantly lower risk of treatment discontinuation due to drug-related adverse events (p=0.02), whereas CTX had a comparable risk (p=0.35).

Conclusions: MMF is a good first-line treatment option for NMOSD and AZA remains a valuable first-line drug if its side effects are tolerable while CTX can be a treatment option for patients who cannot tolerate AZA and MMF.

Keywords: Azathioprine; Cyclophosphamide; Mycophenolate mofetil; Neuromyelitis optica spectrum disorder (NMOSD); Treatment.