AAN 2026 | Treatment of pediatric neuromyelitis optica spectrum disorder
Kelsey Poisson, MD, Nationwide Children’s Hospital, Columbus, OH, discusses the treatment of pediatric neuromyelitis optica spectrum disorder (NMOSD). Dr Poisson notes that rituximab is the most commonly utilized agent and that tocilizumab may be considered in certain cases. This interview took place at the 78th American Academy of Neurology (AAN) Annual Meeting in Chicago, IL.
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Transcript
Yeah, so as I mentioned, there are no FDA-approved treatments, but we definitely do have experience in treating pediatric NMOSD and really focusing on aquaporin-4-positive disease rather than kind of seronegative disease, which is a whole separate question and has a lot more complexities. With aquaporin-4-positive pediatric NMOSD, the most commonly utilized agent is rituximab. And this is a B-cell depleting medication that is dosed typically every six months...
Yeah, so as I mentioned, there are no FDA-approved treatments, but we definitely do have experience in treating pediatric NMOSD and really focusing on aquaporin-4-positive disease rather than kind of seronegative disease, which is a whole separate question and has a lot more complexities. With aquaporin-4-positive pediatric NMOSD, the most commonly utilized agent is rituximab. And this is a B-cell depleting medication that is dosed typically every six months. But especially in children, they are at risk for B-cell repopulation. And so we do have to monitor their B-cells even earlier than we typically might in adults to make sure that they’re not repopulating because then they’re at higher risk for relapse. There are some areas that don’t have good access to rituximab. And in that case, there are still oral therapies that are used. So mycophenolate and azathioprine are two oral therapies that were used more frequently previously in pediatric NMOSD. And so our multicenter study really showed a shift around 2010 in terms of prescribing practices from the oral agents to rituximab just based on higher efficacy perceived with rituximab and a lot of patients that had failed those oral therapies and so the Brazilian consensus guidelines for pediatric NMOSD actually explicitly stated that it’s no longer recommended to use those oral therapies first line, except when there are certain circumstances, such as, you know, family discomfort with other agents or other immune suppression concerns with other agents. The other agent that’s been used in case series is tocilizumab, so an IL-6 inhibitor. And so that is something that is kind of center-dependent that can be used, but may have a faster effect than rituximab. And so that could be a case where if we aren’t able to wait for a couple of months to make sure rituximab is fully kicked in, could we consider tocilizumab? And then, of course, these FDA-approved treatments, you know, we do have some experience using them in children, but of course, getting access to these medications can be very difficult.
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