Cynthia Qi, Health Economics Outcomes Researcher at Argenx discusses her poster presentation focused on generalized myasthenia gravis.
Transcription:
The poster I have is about the real-world outcomes of myasthenia gravis patients receiving efgartigimod treatment. I would just talk about a few highlights of this poster.
I think the first highlight of this poster is, it represents the largest real-world cohort to date about efgartigimod or about any novel treatments in the real-world settings, as we have reported data on 705 patients. We can have such a large sample and report on the data in such a timely manner because we have a lot of support from our patient support program. We basically have most of the data gathered as our nurses are helping patients navigating their treatment decisions.
We have some of these, which is called MG-ADL is a common patient outcome for myasthenia gravis that was gathered during those conversations to help patients navigate the program. We also have a little bit of data from the outside patient support program, but the majority of data comes from this data source.
The second part I want to highlight is more on conclusions on the key findings that I find very interesting about this analysis. I think the first element we look at is whether the type of outcome we see in the real world is comparable to the clinical trial, as that’s what most people are interested in. In this analysis, we find that the reduction or the improvements that patient experience are very similar to what we have seen in the clinical trial data of efgartigimod. We see comparable magnitude of reduction in the MG-ADL score, and we also see similar proportion of patients experiencing clinically meaningful improvements.
Then the third highlight I wanted to talk about in the findings this week. In addition to generally comparable findings with trials, we also dive a little bit deeper to look at what is the best state that patients are able to achieve after they receive efgartigimod treatment and compare that with what they were previously. We do see a significant improvement as demonstrated by these patient-reported outcome measures.
MG-ADL, we see majority, more than 70% of patients. They have moderate to high disease activity prior to initiation. But then after at the best state, we see 80% of patients. They were basically in very low disease activity. At the same time, we also see about 35.5% of patients achieving minimum symptom expression, which means they have very minimal level of myasthenia gravis symptoms as manifested by this particular scale.
We’ve also been trying to understand whether there are consistent changes across different body parts, subdomains of the scale, and we find that similar improvements for the ocular, for the eyes, for their breathing, for their bulbar, for their limb strength. Generally, we’ve seen substantial benefits as experienced by the patients.
The last piece I want to emphasize a little bit is some considerations with interpreting the data. I think that’s more related to your previous question about the limitation of real-world data. While I do feel proud that we are able to represent one of the largest real-world cohorts for myasthenia gravis patients, there are limitations as is more representative patient enrolled in the patient support program. We also don’t always have the consistent timing in the capture of the outcomes. That’s the main limitations to keep in mind.
Think the take-home message is we think from our studies we find that efgartigimod is effective and it’s driving drastic change and improvements for patients who are suffering from the burden of the disease.
To learn more about generalized myasthenia gravis and other rare neurology diseases, click here: https://checkrare.com/diseases/neurology-nervous-system-diseases/
