Myasthenia Gravis Learning Zone
Transcript: Efgartigimod in clinical practice
Dr Jennifer Spillane
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.
There have been a number of real world studies looking at efgartigimod in routine neurology practise. So this study from Italy, looking from November 21 to January 23, looking at ADL, looking at the very important outcome of minimal symptom expression, looking at safety and also looking at the burden of treatment, looking at steroid dose reduction should efficacy across all those factors. We got a study from Israel published from April 22 to September 23, again looking at ADL scores, significant reductions, looking at a rate of minimal symptom expression which achieved 54.5% throughout the study. And then looking at safety again, generally well tolerated and looking at steroid dose reduction, quite marked steroid dose reduction. And remember that these patients were often refractory, quite severely affected patients. And then looking at the UK Early Access to Medicine Scheme, we did an observation on multi-center study on 48 patients once again looking at ADL, once again looking at minimal symptom expression. Once again looking at safety. And I think what's reassuring for me and one of the benefits of coming to a meeting like this is the concordance in our data across different centres, across different communities and across different countries. And that gives me some confidence in the data.
We now have our steroid data available. So I was totting up the figures over the week and we found in the UK that 70% of patients were able to reduce their prednisolone dose once they had been treated with efgartigimod. So there's good concordance in the real world, and it reflects what was found in the clinical trials, which I think is extremely reassuring. And just to try and bring things to life a little bit, I want to talk about a patient case study about one of my patients who I've looked after for the last couple of years. So he had later onset myasthenia. He had myasthenia onset in his fifties, he's now 60. And when I first met him, he had myasthenia for about 10 years. I think he's 62 now actually. He's AChR positive. As with many patients, his symptoms started with double vision, followed by a fairly rapid onset of generalised bulbar respiratory and limb symptoms. He had brittle bones at the beginning and that got worse during the treatment. And you'll see why in a second. So this is his treatment timeline from 2014. I first met him in 2021, which is a quite long time into his illness. And I think this also talks about another question when may patients with myasthenia should be referred to specialist centres. Every red arrow you see here is a crisis, and it resulted in him being admitted to hospital often for a long period of time requiring NG feeding, respiratory support, high dependency unit admission, et cetera. So he'd quite frequent, quite a lot of unplanned acute exacerbations. He had one myasthenic crisis treated with plasma exchange and he was treated during all this time. So he remained on steroids at quite a high dose for many years. So his maximum dose was 70 milligrammes. And this is a gentleman in his fifties with osteoporosis.
He was initially treated with methotrexate, but it didn't improve his symptoms and he didn't tolerate it. And then he went to azathioprine. When I met him at first, we had a discussion about what to do next. It was the early part of the early access scheme. So we didn't have ready access to efgartigimod. So we treated him with rituximab at the standard dose, and he had two infusions of rituximab, but it didn't help his symptoms at all. And in fact he had multiple crises after that. While we were awaiting for the washout period after rituximab, we put him on regular IVIG such was the severity of his symptoms. And then he entered the efgartigimod scheme onto the Early Access Scheme. And you know, quality of life is important. These are the data we see in the hospital, but what happened to him? So he had to give up work as a cardiothoracic nurse, going to church was a massive part of his life and he couldn't go there anymore. He had bulbar symptoms, he couldn't talk to his friends there. He had 11 admissions often were quite prolonged. He required IV access and PICC lines, et cetera. He put on lots of weight, he had osteoporosis, he had a fracture and he had frequent infections. So this is our poor chap with severe myasthenia and he comes to see you. What's your highest priority treatment goal? Do you want to improve his ADL score? Do you want to achieve minimal symptom expression, improve his quality of life, reduce his steroid dose, bearing in mind he's on 70 milligrammes of prednisolone daily or is there something else?
And you know, we want all of these things but yeah, what's your highest priority? Okay, so fairly evenly spread. Wow, we couldn't have planned that if we tried. And I think that speaks to all of the above is the correct answer really there. We want to do all these things. Okay, let's move on. So these are his ADL scores. So looking on the left hand of your screen before efgartigimod, and this is despite rituximab, so climbed up to an MG-ADL score of 17 just prior to rituximab. And we started on the regular IVIG and just before he started efgartigimod, gone down to 15 and then you can see a reduction from 15 to seven by week three of his efgartigimod, and that was maintained at 35. And subsequent cycles have shown a similar pattern and actually his baseline MG-ADL score now is about four. He still has persistent diplopia and intermittent ptosis, but much, much, much better. And he gets efgartigimod every four weeks. And what's happened to him? So really importantly, we've reduced his steroid dose. So he was on 60 when he started efgartigimod, he's now on 17.5. He's gone from IV to subcutaneous treatment at home. He had to come into hospital for his IV access cause of poor venous axis. So now he can get his treatment at home. His treatment has been individualised to him, so he's getting treatment every four weeks. He's not at any emergency admission since starting the efgartigimodud, having had 11 in the years prior to starting. And importantly for him and his quality of life, he's been able to go to church and attend social occasions. So he is very happy and I'm popular in his book, which makes me happy.
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