Navigating Myasthenia Gravis in Adolescents and Young Adults

Announcer:
You’re listening to NeuroFrontiers on ReachMD. On this episode, we’ll hear about treatment gaps in adolescents with generalized myasthenia gravis, or gMG, from Dr. Jonathan Strober. He’s a Professor of Neurology at the University of California, San Francisco and the Director of the Neuromuscular Clinic at UCSF Benihoff Children’s Hospital. Let’s hear from Dr. Stober now.

Dr. Strober:
Some of the unique challenges we face when diagnosing generalized myasthenia in adolescents and adults include that many times, their symptoms aren’t taken seriously, and so they’re often labeled as functional neurologic disorders. For instance, a big presenting complaint in myasthenia is ptosis, or the drooping of the eyelids. I’ve had patients who have been chastised by the school for doing drugs. Sometimes they can’t smile very well, and the parents get upset because they’re not smiling for the camera, and they’re not smiling in their pictures, and parents just don’t understand what’s going on. Because the symptoms can be confused with other things, a lot of times, these patients are not diagnosed very quickly, and therefore, there’s a long time from symptom onset to diagnosis.

One of the biggest gaps we have in treating adolescents and young adults with myasthenia is that a lot of the medications are not necessarily approved down below the age of, say, 18 or 16. In pediatrics, we’ve always used medications that are not necessarily approved in the pediatric age group. That’s been something we’ve been doing for decades. As drugs get more and more expensive, insurance companies really stick to whatever they can do to help decrease the number of patients that they have to pay out for these drugs. And especially since the newer medications are given through an IV and often either every other week or month-to-month or something like that, it can really add up to how much the payers have to pay out. So the patients themselves have a hard time getting the drugs that are newly approved. And there’s been many that have been recently approved that could be really beneficial for our patients and are much better for the overall patient given the focal nature of what they’re treating instead of some of the older medications that really had a lot of potential risks and side effects. So the newer ones are definitely much better tolerated and also safer for many of our patients.

Some of the steps that can be taken at the policy level to improve the treatment of adolescents and young adults with myasthenia would be allowing patients to get access to treatments that are available in adults. I know that companies are definitely doing clinical trials in the pediatric age group. The big problem is that it’s really hard to get patients enrolled in these trials because it’s such a rare disease to begin with, and then they have to be antibody positive, which is often not something that we see in a lot of our adolescents. So a lot of testing can be negative in our adolescent population, which is also unique to the adolescent patient group over adults, who tend to be more antibody positive.

And better educating providers out there as to the signs and symptoms of myasthenia and how they present in the adolescent and young adult age group could be really helpful in getting a lot of these patients diagnosed sooner and getting them into treatment sooner.

Announcer:
That was Dr. Jonathan Strober talking about treatment gaps in adolescents with generalized myasthenia gravis. To access this and other episodes in our series, visit NeuroFrontiers on ReachMD.com, where you can Be Part of the Knowledge. Thanks for listening!