Dedee Murrell, Dermatology Meeting News 2023: Pemphigus – prevalence, clinical presentations & impact on quality of life
Pemphigus is a rare autoimmune blistering disease, which results in painful blisters on the skin or mucous membranes, severely impacting patient quality of life. It was a pleasure to talk with Prof. Dedee F. Murrell (St George Hospital Campus, Kogarah, Sydney, Australia) around the prevalence, clinical presentations and impact of pemphigus on patient quality of life.
The presentation ‘New treatments in the pipeline for pemphigus‘ was presented at AAD 2023, 17-21 March, 2023.
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Questions
- Could you give us a brief overview of pemphigus, its prevalence and clinical presentations? (1:09)
- What is the impact of pemphigus on patient quality of life? (4:29)
Disclosures: Dedee Murrell discloses serving on advisory boards for Argenx, Sanofi, Principia, Janssen, Lilly, Almirall, Roche, Cocreator PDAI, BPDAI, MMPDAI, Abbvie, Novartis, Kineska, Amryt, Castle Creek, Rheacell, and Shire.
Support: Interview and filming supported by Touch Medical Media Ltd. Interview conducted by Victoria Jones.
Filmed in coverage of the 2023 AAD Annual Meeting.
Click here for more content on dermatological conditions.
Transcript
Could you give us a brief overview of pemphigus, its prevalence and clinical presentations? (1:09)
Pemphigus is a relatively rare autoimmune blistering disease, which is more common in certain countries where patients have the right genetic mix to become predisposed to developing it. Those countries include Israel, Iran, India and some other countries that are along the Silk Road. As people have migrated all over the world, these patients pop up in Western Europe and the United States, in fact, all over the world. And you can see here in these pictures how severe it is, how disfiguring it is. And it’s a disease, which typically, in most cases begins in the mouth or other mucous membranes and often spreads to the face, first, the scalp and then elsewhere on the body. And the patients are very distressed, as you can imagine, looking like this. And most people who were not aware of the condition would think it was an infection and would avoid going near these patients. It means bubbling skin in Greek and vulgaris means common and this is the commonest form of the disease, which starts in the mouth. And it’s both a mucous membrane and skin disease, whereas the less common variant is called foliaceus or leafy or superficial peeling, as in this case here. And whilst that doesn’t affect mucous membranes, it can spread all over the body and be very difficult to control. And then there’s two rarer subtypes of the disease, which are shown here IgA pemphigus and paraneoplastic pemphigus. As far as the incidence of the disease, it varies, as I said, according to the countries. It ranges from 0.75 patients to 5 million patients, new onset patients per million per year, typically in adults, most prevalent in these Middle Eastern populations. We’re not sure exactly why. And it’s typically a disease of working age adults which results in them losing their jobs because people don’t want them around when they look like this. And they’re usually quite sick because they can’t eventually eat or drink properly. The testing shows not only the blistering level from a skin biopsy, but also the green light is the autoantibodies, which are attacking the skin, which you can detect with this technique. And if you want to know more specifics about it, this is a textbook that I edited a few years ago, which 70 of the world’s experts contributed to, which can provide a lot more information. I just wanted to touch on another disease, which a lot of doctors have heard of and get confused with pemphigus. It’s called bullous pemphigoid, this is a disease, completely different disease, which causes much deeper blisters, mainly on the skin rather than the mucous membranes. It’s much more prevalent in Western countries and it’s increasing in incidence, especially among the elderly population where it has a very high mortality and morbidity.
What is the impact of pemphigus on patient quality of life? (4:29)
Quality of life in medicine is something subjective, which comprises a number of components in this visible disease, something which you don’t get the same with heart disease or other internal diseases that people can’t see. The disfigurement causes a lot of stigmatization of these patients. The blistering gives rise to pain, especially when they pop open because it’s like having open ulcers and they can’t function properly because they can’t go out when it’s visible. They can’t necessarily eat properly, drink properly. But then on top of all that, even if you treat the patient, the drugs as we’ll come to in a minute, while even if they can control this element of it, have many side effects. The newer drugs are not approved for this rare disease and therefore tend to cost a lot of money and the patients end up on a cocktail of medications to control the side effects of the primary active medication. So, quality of life is a comprising of both of these symptomatology and treatment components. I’ll just highlight a couple of studies which illustrated the impact of the disease. This is quite a huge study considering the rarity of the diseases, 380 pemphigus patients. It’s quite common in Japan and you can see that 40 odd percent of them lost income due to the disease. 60% of those with severe disease lost time at work. They describe their financial status as working adults being in poverty. And even if the disease got under control, it didn’t necessarily improve because by then they’d taken several months off work or lost their jobs and then had to explain that they had a lifelong condition which could relapse. And we found this in our own studies. So, as you can see, the quality of a patient’s life is greatly affected by having this condition. And up until group developed some way of measuring it, there were many non-specific ways that applied to other diseases which were being applied to this disease, which weren’t asking the right questions. So, over a decade ago, our group interviewed a lot of patients and came up with two different ways of measuring it. One way you look at questions that relate to having the disease itself, which is called the ABQOL. And another one, which, for the first time ever in medicine, is a questionnaire that’s asking how the treatments themselves are affecting the patient’s quality of life, which is called the TABQOL or treatment of autoimmune blistering disease quality of life. Each of these questionnaires is 17 questions with four response options: no it isn’t, a little, a little bit more and a lot, essentially adding up to a score out of 51. The higher the score, the worse the quality of life. And apart from publishing this in some good journals in Australia and the United States and the British Journal of Dermatology for the TABQOL, we’ve had this translated and tested in numerous maybe 20 different countries now and languages. And the results are coming back fairly similar in each of these countries. This is just a study, a big study done in China. This is one where it was done in Poland, in Polish. And they found that the strongest effects in quality of life for these patients was when the disease relapses and flares in patients working age under the age of 70. And then that one I showed you that was in the elderly in their study, had the highest quality of life impairment compared to the other autoimmune blistering diseases. This was a study we did in Arabic populations in Egypt and Tunisia, where they have a lot of younger patients with the foliaceus type. And they found that younger and less educated patients had worse quality of life when it came to the treatments.
Subtitles and transcript are autogenerated. All patients provided informed consent for the reuse of their images in medical education.
