Myasthenia Gravis Learning Zone
Transcript: Optimising gMG patient assessment
Professor Francesco Saccà
Interview recorded June 2024. All transcripts are created from interview footage and directly reflect the content of the interview at the time. The content is that of the speaker and is not adjusted by Medthority.
Thank you, Andreas. Thank you so much for the introduction. And a special thanks to argenx for inviting me and for organising this very nice symposium. So I will talk about optimising patient assessment to achieve treatment goals. How should we do that in clinical practise, and what is new in the field? This is the first question that I would like to ask you that is pertinent to our presentation. So which measure is the most useful to assess treatment success for your patients with generalised myasthenia gravis? And we have few answers here. Production in MG-ADL and QMG, in MGC, or achievement of minimal symptom expression, meaning an MG-ADL of zero or one or improvement in quality of life scales, or other that you might have in mind. So you can now start voting. Great, so the majority went for MG-ADL, and some went for achievement of minimal symptom expression, which is basically, I would say, the same scale but a different endpoint. So I agree with this, but I'd like to say that there's no wrong answer here because all of these are important for our patients for MG, and we'll see that later in the slides. So we all know that generalised myasthenia gravis is a highly debilitating disease. It has a major impact on, I would say, all aspects of patient's life, and some symptoms are quite easy to rate because they have difficulties with movement, they have difficulties with speech, with swallowing. They might have impaired vision, impaired respiratory function, and extreme fatigue.
Well fatigue is probably quite difficult to measure, we know, and this is one of the symptoms that might mislead at the diagnosis of MS. But we can, you know, fairly rate these, all of these symptoms using clinical scales. The problem is that it also impairs quality of life and the ability of patients to maintain an employment, and it also causes anxiety. You know, 46% of patients with myasthenia gravis have been diagnosed with an anxiety disorder, which is quite a lot. Means, you know, half of our patients. And 39% also have depression. And you can imagine how both of these can impact on quality of life and on a patient's daily routine. Also, 50% are unemployed. Now this is very interesting. Why are they unemployed? It could be because of the disease because they're unable to cope with, you know, the requests of a normal paid job. Or even because they didn't have a chance to study, to go to university, to college, whatever, because of the disease. And we see that very frequently in our patients. And 32% require help with daily activities. And this need, and I would say many of these factors, increase with an increasing MG-ADL score.
So one of the problems that we face with a myasthenia gravis patient is that the disease fluctuates. One day they're feeling better, the next day they're feeling worse, and even during the same day. So there is a need to kind of summarise all of these symptoms, and there's no other way than for the patient itself to summarise that. That's why the primary endpoint of many clinical trials is a patient-reported outcome. Not just because the FDA said so, but because we need someone to summarise that. And also there is an inconsistent use of myasthenia gravis burden assessment tools across clinical settings. This is true, at least it was true until a couple of years ago before innovative treatments were available, because everyone was kind of using their own evaluation. And another problem that adds on top of that is that there's no validated scale for depression, anxiety, and fatigue that is specific for myasthenia gravis. We have many scales, but none of these are really specific for the disease, and rate some very specific items that come with myasthenia gravis. So we thought a couple of years ago, why not create an expert panel, and then based on the evidence that was available at that time to come up with some recommendations for a patient assessment that could optimise our care in generalised myasthenia gravis. And I'm happy that three out of four authors are here, including Jennifer and Andreas. And, of course, what we did was to generate statements, and these statements were then voted by the panel.
So a classic way to build up guidelines too. And what we came up with is that the MG-ADL scale should be used consistently across all clinical settings. Meaning both regular visits, clinical trials, or even telemedicine. And fluctuations in the MG-ADL scores can swiftly highlight the need for a QMG, or quality of life assessment. Meaning that we realise that we cannot do the QMG to every single patient, but we need that desperately when patients are fluctuating. In certain circumstances, we cannot have the entire valuation depend on the MG-ADL alone. Then a very interesting thing is the PASS, or patient acceptable symptom state, could be, and should be, a typical question during a clinical evaluation to determine if the patient is satisfied or not because of the treatment. And also timing and frequency should reflect the patient's symptoms state, and should not happen in a very fixed scheme. So this is the general output of our publication and of after the voting process, and we agreed that the MG-ADL should take place as frequently as possible, virtually every single week, for all of our patients.
So to have a continuum in the way we assess patients. The QMG, if the MG-ADL score indicates a worsening in the QMG, and all of these measures here should be used to determine patient satisfaction in not just the PASS, but also the EQ-5D. Also the MG-QoL15R. And further assessment should take place if patients are still dissatisfied with their symptoms status. And again, as we said before, this should be adapted to the single patient, and if the disease is fluctuating. We also know that frequent in-person visits are almost impossible. Many of our MG centres are crowded with patients visits. We have a lot going on in our clinical practise. So what should we do in order to capture data on the MG-ADL? Well, we should use a digital assessment like a phone app in order to capture that, and have a remote monitoring take place. So the idea is that you could record symptoms experienced by patients between appointments so that you're not asking patients what happened during the past two months or three months. You can have patients record that weekly with our MG-ADL on their phone. And an app, which is not just an app, but it's an entire system, the way we communicate with patients can also enhance communication for patient outcomes, it can facilitate decision-making, and it can determine when we really need a face-to-face consultation. It can also deliver educational resources to patients and can create a repository for real world data. Because remember that at the end this is what we desperately need in our clinical practise.
So this is how the entire system could look like, a patient filling in data on their app, having a sort of report where they can see on their own how they're doing. Like today your MG-ADL is 6, 8, 12, 15, 24, hopefully not 24. But the most important thing is that we need a link with our patients. We don't want our patients to, you know, download a report and bring it to us. We want a portal that we can access on our side and where, you know, by just looking at the colours, if a patient is doing well, if there's not worsening, it's green. If a patient is worsening, has a very high MG-ADL score, it's red or orange, or whatever. So that that triggers our intervention only when needed. And this is, for instance, when we asked patients, we tested this app in patients, we asked patients, "Are you satisfied with the results of this?" And this is how it went in Italy and Spain. And as you can see, the vast majority of our patients were very positive about that, quite positive, and very few were actually not positive with it. And it's very interesting to note that most of the not positive were in Italy and not in Spain. And since Elena took part, it's interesting to know why are your patients more satisfied than ours? It's really troubling. - I guess the food is better in Spain, and that, for sure, it was. No, I think we should compare the characteristics of the patients that have been using the app. Maybe, I dunno, the age, which if they have had an exacerbation very recently, something that motivate them to use the app more.
- So, thank you for this answer. And just to summarise, so patients with generalised myasthenia gravis experience a variety of symptoms that impact brutally on their life, and their wellbeing and quality of life. It is a fluctuating disease. We need proper scales to assess that and to capture that. And in this panel, in this consensus that we reach the MG-ADL is the right scale. But please don't forget the QMG in critical points. It is necessary to have an objective measure as possible. And digital assessment tools will help us create this link with patients, and create a continuum in the reports that we have from patients. Thank you so much.
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