Brenda L. Banwell, MD: I want to talk about guidelines for therapy for pediatric onset multiple sclerosis. The first overall theme is that multiple sclerosis is a relapsing/remitting disease in children with a high relapse frequency. As a result of that observation, it’s important to consider early therapy. Studies in adults have clearly demonstrated that treating multiple sclerosis early in the disease course has a longer-term benefit. Patients who are offered effective therapy early do better in the long run. We have, of course, not necessarily the same length of observation in studies of children, but it makes intuitive sense that they should be treated promptly after the diagnosis is confirmed. So that is the most important, single guideline that I want to emphasize.
In terms of what medications to use, this gets a little bit more complicated in the pediatric age range because very few of the medications that are approved for adults have been formally studied in pediatrics. In fact, to date, really only 1 large study has led to FDA approval in pediatric multiple sclerosis. That was the PARADIGMS trial of a medication called fingolimod.
Other than fingolimod, the remaining medications at this point are used off-label, which means that although they are approved for adult disease, they were not formally yet studied, or may not be studied formally in the pediatric multiple sclerosis age group. So the International Pediatric Multiple Sclerosis Study Group has provided prior manuscripts and a very recent manuscript discussing how we suggest or advocate for access to therapies for pediatric patients. We advocate that children be offered access to what were conventionally referred to as frontline therapies—so the interferon preparations, or glatiramer acetate, which are treatments that are injected at different frequencies in slightly different ways depending on the preparation, but all of which have been available for multiple sclerosis since about 1985 to 1990, depending on the preparation. We have quite a bit of experience, as a community, in using these therapies for children.
Newer therapies have been studied in pediatric multiple sclerosis groups of patients. Although not the same as a formal trial, they still have shown benefit, and more importantly in this type of study, safety, along with some serious considerations for monitoring. For example, natalizumab, which is a treatment that is highly effective in multiple sclerosis, has been used for children and teenagers with very active disease. There are also studies published out of the Italian national cohort that have shown this therapy to be relatively safe and well tolerated. Therefore, some patients are offered that particular therapy.
The use of some of the more recently approved medications, notably ocrelizumab, for example, are about to be studied in formal trials in pediatric multiple sclerosis. So we’re in a new space. We’re at a time where medications that are approved for adults are either being studied, have just been studied, or are about to be studied in the pediatric multiple sclerosis population, which will give us the type of evidence to guide our therapy and will lead to regulatory approval if the studies are positive.