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Neuromyelitis optica (NMO) and its spectrum disorder (NMOSD) is a severe autoimmune inflammatory disorder of the central nervous system, with recurrent attacks mainly focused on the optic nerves and spinal cord. Untreated, it has a high morbidity and mortality. Serum aquaporin-4 (AQP4) antibodies are present in the majority of patients, are recognised as pathogenic and are a predictor of further relapses.1
Most specialists treat AQP4 antibody-positive patients early and aggressively with immunosuppression to prevent further attacks. There have been no randomised trials in NMO, but prednisolone, azathioprine, mycophenolate mofetil, methotrexate and rituximab are among the therapies currently used and appear to have comparable efficacy.2 ,3 Because of the potentially devastating nature of NMO relapses, many clinicians would consider a single relapse on treatment to represent treatment failure and an indication to alter therapy, particularly if the relapse is severe and the therapy has had adequate time to work. This alteration can take the form of an increase in dosage of current treatment or a switch to an alternative agent.
There is an urgent need to identify the most effective treatment regimens, particularly because there are many novel therapeutic candidates being developed, including those which act on the antibody-producing or complement pathways, and an AQP4 monoclonal antibody blocker.4 Current NMO study designs under discussion vary, with some regulatory authorities requesting placebo comparator arms but others accepting standard treatments (azathioprine or alternative plus low dose oral steroids)3 as comparators either as add-on or …
Contributors Conception and design of study: Drs Palace and Kitley; Data collection: Drs Kitley, Leite and Elsone; Data analysis: Drs Kitley and Palace; Drafting of manuscript: Drs Kitley and Palace; Critical appraisal and revision of manuscript: all authors.
Competing interests JK is supported by the NHS National Specialised Commissioning Group for Neuromyelitis Optica and has received travel grants from Biogen Idec, Novartis and Teva and speaker honoraria from Novartis. MIL is involved in AQP4 and MOG antibody testing, is supported by the NHS National Specialised Commissioning Group for Neuromyelitis Optica and by the NIHR Oxford Biomedical Research Centre and has received speaking honoraria from Biogen Idec and travel grants from Novartis. LE is supported by the NHS National Specialised Commissioning Group for Neuromyelitis Optica and has received travel grants from Novartis and Teva. AJ is supported by the NHS National Specialised Commissioning Group for Neuromyelitis Optica. JP has received unrestricted grants and support for scientific meetings and scientific advisory honorariums from Merk Serono, TEVA, Biogen, Bayer Schering and Novartis, has held MS society grants, is a clinical lead for the UK DOH RSS and is supported by the NHS National Specialised Commissioning Group for Neuromyelitis Optica.
Ethics approval Oxfordshire REC.
Provenance and peer review Not commissioned; externally peer reviewed.