There Is Great Need to Better Understand Pediatric Myasthenia Gravis: Jonathan Strober, MD

In this conclusion of a recent interview with Jonathan Strober, MD, a pediatric neurologist, he highlights current gaps in the understanding of pediatric myasthenia gravis, potential areas of future investigation, and the importance of treatments that specifically target these patients.

“I really want us to get a better understanding of pediatric myasthenia gravis,” he says, “and how we can follow these patients and best take care of them safely.” Strober specializes in neuromuscular disorders at the University of California, San Francisco, and Benioff Children's Hospital, where he is director both of clinical services for child neurology and of the muscular dystrophy clinic.

Rewatch part 1 for insight on nipocalimab use in these younger patients and part 2 to learn more about treatment-related challenges in the group.

This transcript was lightly edited for clarity; captions were auto-generated.

Transcript

What are some key unmet needs in the myasthenia gravis treatment landscape?

I think one is, in pediatrics, again, trying to come up with better tests would be really nice. I think what we're trying to do in our pediatric myasthenia gravis consortium, which has 6 centers currently, is to develop better tools to follow these patients but also develop a standard of care. There really is no standard of care for these patients. We're just so used to using drugs that have only been approved for adults that people just try the different ones on kids and hope that they're safe and hope we have the right dosing, and so kind of getting a better sense of what are people doing out there and what do we actually see in real life, in real time, what is actually working for the patients, so we can put together and say, “Hey, you know what, this is what we recommend as people who take care of these patients and take care of a good number of these patients, this is really the treatment options that you should go [with] and what route you should go in, what's the safest for the patients, what's the most effective for the patients.” That’s really what I want to see; I really want us to get a better understanding of pediatric myasthenia gravis and how we can follow these patients and best take care of them safely.

I think also what I've learned is that the reason that there are so many studies is because now it's become a requirement that in order to get approval for adult drugs, the companies have to have a pediatric arm. That's been a wonderful thing.

Just to be given the opportunity to talk about pediatric myasthenia gravis, that people are actually caring about kids with this condition, has been a huge step forward for those of us in the community who take care of it. I've had so many patients who have been told by providers, “Oh, myasthenia doesn't happen in kids. They can't have myasthenia.” We're kind of used to that in pediatrics for these rarer diseases, so the fact that word's getting out that, yes, kids can get myasthenia gravis and that we can treat them and we can treat them effectively and safely, it's just wonderful to have that opportunity, so I appreciate that and thank you.