Comparisons of the efficacy and tolerability of mycophenolate mofetil and azathioprine as treatments for neuromyelitis optica and neuromyelitis optica spectrum disorder

Abstract

Both MMF and AZA were effective in patients with NMO/NMOSD. Fewer and more mild adverse events were attributed to MMF than AZA. The probability of maintaining a relapse-free state was not significantly different between the MMF and AZA groups. However, more effective treatments with more acceptable safety profiles are still needed.

Mycophenolate mofetil in paediatric autoimmune or immune‐mediated diseases of the central nervous system: clinical experience and recommendations

“…MMF utility is generally supported by the data in some inflammatory demyelinating CNS diseases, and our results confirm MMF efficacy in the paediatric age group. MMF is beneficial in adult NMOSD, as often as or more effective than azathioprine, which is often poorly tolerated at higher doses required for immunotherapeutic effect. Data also suggest possible MMF efficacy in MOG‐associated disease …”

“…Although studies specifically designed to evaluate MMF safety in paediatric neurology are not available, our patients and other children treated with MMF both in neurological and in other paediatric conditions seem to support a favourable safety profile. In recent studies on adult NMOSD, MMF had significantly better tolerability than azathioprine …”

“…Clinical indications for MMF: literature data on specific conditions MMF seems to be an effective and safe option in paediatric primary angiitis of the CNS 18 and in NMOSD. 4,[10][11][12][13][14] Evidence is more limited, although encouraging, in MOG-associated demyelinating CNS syndromes. 13,15,16 MMF role is still to be clarified in autoimmune/immune-mediated encephalitis, 17 uncertain in multiple sclerosis, [19][20][21][22] and seems to be limited in opsoclonus myoclonus ataxia syndrome.…”

“…2 MMF is also largely used as a steroid-sparing agent for chronic immunosuppression in rheumatology and in neurological autoimmune and immune-mediated conditions, with data suggesting a relatively safe profile. [3][4][5][6] MMF has been used for nearly two decades in myasthenia gravis, 7,8 although its role is less clear in chronic inflammatory demyelinating polyradiculoneuropathy. 9 In central nervous system (CNS) autoimmune and immune-mediated conditions, MMF has been increasingly used in relapsing demyelinating diseases such as neuromyelitis optica spectrum disorders (NMOSD) 4,10-14 and myelin oligodendrocyte glycoprotein (MOG)-associated disease.…”

Progress in neurology 2017–2018

“…Treatment modalities are limited, with the exception of the immune‐mediated diseases and those with a defined biochemical cause that lends itself to specific therapy such as Pompe disease . Immune‐mediated diseases of the central nervous system are a heterogenous group of disorders characterized by specific antibodies to central nervous system proteins such as the autoimmune encephalitides and neuromyelitis optica. Significant progress has been made in the diagnosis and treatment of these .…”