番茄社区

At the American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) annual meeting, Michael Hehir, MD, of the University of Vermont in Burlington, participated in a debate on integrating novel and traditional therapies in the management of myasthenia gravis.

In this video courtesy of , Hehir discusses various considerations -- including efficacy, side effects, and costs -- that help guide optimal treatment.

Following is a transcript of his remarks:

2024 is an exciting time with regard to treatment development for myasthenia. Since 2017, we've had five new therapies approved for the treatment of our patients across two major classes of therapy. Some of those treatments are IVs, some of them are subcutaneously administered. And the two big classes are FcRn inhibitors, which cause a reduction in overall antibody circulation, and complements inhibition, which impacts the complement system, which is one of the major pathways of muscle damage in myasthenia.

And these medicines have been effective for patients in clinical trials and in clinical practice. One of the main uses for them has been to treat patients who are not responding to more traditional immunotherapies that we've used in the past.

While here at the AANEM meeting, we ran a first session of a debate discussing where we should place some of these newer medications in the treatment paradigm for patients with myasthenia with regard to other therapies, which also work. Some of the data that we were looking at is the likelihood that older generation and newer generation therapies can cause patients with myasthenia to have minimal or no symptoms, whether or not newer generation or more traditional therapies have a better side effect profile than the other. And then a big concern -- and something that I talked about in my discussion -- is cost of therapy, particularly the newer generation therapies.

So I think the major conclusion is that we have a lot more work to do. We have therapies that have been around for many years that are very good at helping patients achieve little or no symptoms over time. But some of those therapies can take a long time for that to happen. The newer therapies may be better at achieving that better clinical status, and these other therapies may be better at maintaining that good clinical status.

And really what we need to do is start thinking about how to compare these different treatments head to head. Thinking about efficacy, but also thinking about treatment burden and whether or not newer medicines or older medicines are better with regard to treatment burden. So things like side effects or how hard it is to get your therapy. Do you have to drive to an infusion center? Do you have to sit in an infusion chair? Is it a self-administered medication? There's a lot of questions that I think we need to grapple with as a community and hopefully in a research setting to understand what are the best strategies for particular patients.

I think that the take-home message is that there are many options to treat patients with myasthenia in 2024, and it's really important to engage your patient in a discussion of the types of things that are important to them. Is this a patient who needs to be on an oral therapy because they travel a lot? Is it a patient who would be OK with coming in for periodic infusions? Is it a patient who's failed therapy in the past and needs a newer medicine because they're just not doing well? And then all with the hats thinking about distributive justice as physicians, we need to consider the cost, and our healthcare system over time may not be able to sustain those costs, so we may not be able to use them for every patient. We might want to be more selective.

Comment