Ep 18. GUIDELINES - BSR systemic sclerosis guideline 2024

BSR has published an update to the systemic sclerosis guideline, expanded to include paediatric and adolescent patients for the first time.
 
Join guideline working group Chair, Prof Chris Denton, members John Pauling, Bridget Griffiths and Clare Pain, and expert by experience member, Sue Farrington, in a roundtable discussion hosted by Prof Ernest Choy.

Read the full guideline and download the summary pdf here. 

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Transcript

Rheumatology Roundtable 2024

Systemic sclerosis guideline update

Transcript

Ernest Choy   
 Welcome to this episode of the BSR’s Rheumatology Roundtable to discuss what is new in the updated and expanded BSR, Systemic sclerosis guideline. I'm Ernest Choy. I'm a rheumatologist at Cardiff and I'm Editor in Chief of the Society's journal, Rheumatology. So, in this discussion, we'll aim to provide a summary of what is new in this guideline and how it can help clinicians to use the guideline to benefit patients. So, joining me today are a number of very esteemed panellists and I'm going to ask them to introduce themselves to you, starting off with Chris Denton.

Chris Denton
 So hello. I'm Chris Denton. I'm a rheumatologist at the Royal Free Hospital in University College London. I was the lead for this guideline working group, and it's fantastic after, in fact, two years of effort, I think, to be at this stage of launching the updated Systemic sclerosis guideline.

Ernest Choy   
 John.

John Pauling 
 Thank you. My name's John Pauling. I'm a rheumatologist in Bristol and I was delighted to support Chris Denton and the team to devise these new guidelines and help to support the task force, looking at vascular endpoints and some of the other manifestations. So, great to be here today.

Clare Pain 
 Hi everybody. I'm Clare Pain. I'm a paediatric rheumatologist at Alder Hey, in Liverpool. I've got a clinical and research interest in scleroderma in children. I was actually delighted to be asked to be part of this group. And for the first time, these BSR guidelines will include children and young people as well as adults.

Ernest Choy
 Bridget

Bridget Griffiths   
 Bridget Griffiths, I'm a rheumatologist working up in Newcastle and also for the last almost six years I was Chair of the specialised rheumatology clinical reference group, so obviously it's been really important to be able to try and cascade what's new for patients with systemic sclerosis and being able to access high-tariff drugs.

Ernest Choy 
 Last but not least, Sue.

Sue Farrington   
 Hi, I'm Sue Farrington. I'm Chief Exec of the patient organisation, Scleroderma and Raynaud’s UK and I was delighted to join the group to provide representation from a patient perspective.

Ernest Choy 
 Thank you. Thank you for joining us today. So we start off with Chris, could you give us a brief summary of the guideline and why is it important and what does it include and what is new?

Chris Denton
 So thank you, Ernest. So, you know, the BSR guidelines are incredibly important, I think, for practising rheumatologists, clinicians, patients and all stakeholders really with relevance to rheumatic disease. But in terms of systemic sclerosis, because it's such a severe multi system complex disease, and quite uncommon, I think the guideline is particularly important because it really just provides a comprehensive road map for evidence based expert management of systemic sclerosis.
 Within the UK, and specifically within the NHS, and I think that's another strength of the BSR guidelines because they're developed in the UK, they very much have a  focus on management within the structures of the NHS.
 We needed to update the guidelines which were previously published in 2016, in line with the BSR  protocol for updates, but also because there has been major progress in systemic sclerosis. Particularly, I would say, in the areas of interstitial lung disease, pulmonary hypertension, hypertension. For digital ulcer management, there are better opportunities to diagnose the disease early, which I think are now relevant and, most importantly, as Clare already introduced, you know this was an opportunity to have a focus on systemic sclerosis at all ages really including children and young people. So I think this is really exciting, but very, very timely.

Ernest Choy 
 Thank you. So how was the guideline developed?

Chris Denton 
 So, you know, we are very much guided by the BSR protocol, and I think the guideline protocol has recently been updated, but we followed the latest version of the protocol and really the first step was to develop and get the approval from the Guidelines Steering Group of the multidisciplinary group to develop the guidelines, the Guideline Working Group. And the important thing is that this was a mixture of experts in systemic sclerosis, rheumatologists from district general hospitals, working in a some more traditional secondary care settings. We had primary care representation, allied healthcare professionals. Most importantly, we had people with systemic sclerosis. We had two patients in the group and also we had close involvement of the main patient organisation SRUK and obviously Sue was very important in representing, along with Emma Blamont, her colleague. 

So we followed that guideline. We first of all developed the scope of the guideline and that was important work to  identify the questions we were going to address as a group and do our systematic literature review.

 Having published the scope in Rheumatology Advances in Practice, which, again, is part of the new BSR process, we then , really over about 18 months, had a series of virtual and in-person meetings to refine, develop and really review the evidence. We were very grateful to have three clinical fellows, three junior colleagues, who joined the group, and they were really important, not only because they did a lot of the work related to literature review, but also because they were very digitally literate and I thought it was really helpful to us in  maximising the use of phone apps and software to do our literature review,  in, I think, a much easier way than we've done previously. And also when it came to getting the consensus agreement and levels of agreement for the recommendations, we could do that all electronically, I think very efficiently. So it was an exciting time and I think we used modern technologies as well.

Ernest Choy 
 So thank you for that, Chris. You said that the guideline is based on the broad engagement of different stakeholders. Importantly, patients were involved in driving the important questions and it is based on a very thorough evidence review in the literature. So (how can) what is the big overview of the key recommendations that people can take away in the guideline? Maybe I can turn to John to answer that question.

John Pauling 
 Yeah. Well, as Chris says. It's a very timely period to embark on a new guideline development. Like the 2016 guidelines we took this multi-system disease and broke it up into the major organ manifestations as the key domains of the disease. We also dedicated a section, as previously, to early diffuse disease because there's a very specific period in a group of systemic sclerosis patients where management really is key, and where clinicians from across the UK and worldwide really encounter challenges in managing their patients. I think that really, you know, I suppose unsurprising, but important, point was how much had changed in the short period since the last guidelines, so as Chris has alluded to, the interstitial lung disease section, there was significant changes on the basis of large phase two and three trials. And I think what the BSR guideline has hopefully been successful in achieving is tackling the thorny issue of where some of these new targeted treatments are positioned in the modern day management of scleroderma-related lung disease.
 And so we've attempted to give clinicians a road map for where we might position some of these high-cost drugs in the management of scleroderma-related lung disease alongside background therapies and existing treatments.

 Other sections have changed for different reasons, so, peripheral vascular disease, really we're using many of the same classes of treatment to manage digital vascular disease and systemic sclerosis. But again, the positioning of some of those treatments have changed as drugs like phosphodiesterase inhibitors have reached the end of their natural patent, they've become much cheaper and easier to use and get commissioning approval for. So some of these drugs have been positioned further up the treatment pathway and treatments like sildenafil, which were third line treatments for management of Raynaud's and digital ulcers, we're increasingly using at a much earlier stage and we really wanted to get that message across to clinicians up and down the country. And then, finally, I think we've added new sections to the guidelines. So we've recognised the importance of cancer and systemic sclerosis, cardiovascular disease, neurological disease and these certain aspects that didn't feature too strongly in the original iteration of the guidelines, but we've attempted to assimilate and understand the evidence and again give clinicians a practical based approach to the management of their patients because it can be very difficult for all of us as clinicians to manage this truly multi-system and heterogenous disease.

Ernest Choy   
 Thank you for that, John. Maybe I can come to Clare. As a paediatric rheumatologist being involved in these guidelines, what recommendations are made and how are they relevant for young people with systemic sclerosis?

Clare Pain   
 Yeah. Thank you. Thank you, Ernest. So these recommendations are really key for children and young people and there's a couple of reasons why. First of all, juvenile systemic sclerosis is extremely rare. So even the larger specialist paediatric rheumatology centres in the UK will only have a handful of patients under their care at any one point.

 Secondly, the amount of research there is in juvenile systemic sclerosis is really lacking. So how do we position these drugs and apply the evidence we have from adults for the paediatric population? These guidelines are really needed to help standardise care across the UK. 

When we looked at the recommendations they fell into three broad areas. One were those that would apply both to adults and children and you'll see actually that most of the recommendations are both for both children and adults. There are some that don't apply to children and to adults only, for example cancer malignancy screening, but also glucocorticoid - minimising glucocorticoid use in renal crisis - because we really don't see renal crisis in children. And then there are those that needed a bit of a paediatric tweak and consideration to be used in paediatrics. So, for example, monitoring, screening and management of pulmonary hypertension and indications for autologous stem cell transplant. What's additionally new for children and I think new to the guidelines was that there are good practice points around transition. So that's the first time that's been mentioned in a BSR guideline on systemic sclerosis And also, in the research section, it was highlighted this big unmet need and the consideration for including children in future clinical trials in systemic sclerosis, so that we can get evidence for treatment in this group.

Ernest Choy   
 Wonderful, thank you. So maybe then I can come to Bridget. What sort of practical help can the guideline provide to healthcare professionals in the clinical setting and, particularly, how is the BSR guideline different?

Bridget Griffiths
 Yeah. So, Ernest, I think with the onset of all the recently published evidence, I think it highlights even more the importance of making an early diagnosis, so that then you can start treatment early. And, as John has alluded to, some of these drugs now are off-patent. So, for example, the PDE 5 inhibitors, so sildenafil, tadalafil are now only 30p a day. So it does make a huge difference to patients and their Raynaud’s. So it's being able to identify which patients may be eligible for these drugs and starting them at an early phase.

 And I think also the important thing, it highlights the importance of screening. So particularly for common end-organ problems. So, looking for interstitial lung disease, performing lung function tests and a baseline HRCT chest scan at the beginning. So in the past, we probably wouldn't have done that, but now it's a good tool that's very sensitive, that will pick up changes. And so then we could do it at appropriate times at intervals. Just looking to see if a patient is responding to treatment or if they're developing new interstitial lung disease. And then that would also be the same for patients that are going to develop pulmonary hypertension. Are you doing screening? So perhaps doing it a baseline echo and then using the NT-proBNP blood test to look to see if people are developing coronary hypertension and doing echos at the right time. So that, again, you can pick up patients that are likely to have developed pulmonary hypertension and then refer them to the local specialised regional centre to make the diagnosis. 

And so we've quite a few more drugs, but high cost drugs that are available. So we've got more treatment options for interstitial lung disease. So it's been aware that there's good evidence for mycophenylate. We've got the use of nintedanib that has to be ratified for patients that have got progressive pulmonary fibrosis, we're probably going to be able to access more easily rituximab in interstitial lung disease. We've got new treatments for pulmonary hypertension and they're easier for patients to take. So it highlights the importance of actually doing the screening earlier on and identifying these patients and considering them for early treatment. 

And then I think just overall with my  CRG background, I think the importance of actually having networks in the regions and having the regional MDT meetings so that any patient attending any hospital can have their case discussed and then the treatment can be ratified and obviously that does vary. It's slightly different in Wales and it's slightly different in Scotland, but we're probably all fairly aligned for the high-cost drugs that are criteria and guidelines as to when they could be used.

Ernest Choy 
 Thank you for that. So it sounds like these updated recommendations are fairly comprehensive and detailed and sound very important. So, Sue, do you feel that this guideline will help people with systemic sclerosis when having conversations with their healthcare professionals?

Sue Farrington   15:18
 So I think there are a couple of things. Firstly, I think it's going to be really reassuring for the Community to know that the most effective way to treat and care for patients with systemic sclerosis has had such rigorous attention from key experts and specialists in the field. And also that, as part of the process, the patient perspective has been taken into account. So one of the things I think is really important as well as making the best practice guidelines available to all relevant healthcare professionals,
 we must ensure, and this is probably a role also for SRUK, to make sure that patients know where they can find the guidelines and the lay summary. I think it's fair to say that for many patients with rare diseases like systemic sclerosis, they often become experts themselves through their lived experience. So having the best practice guidelines I think is really going to help provide a framework for those conversations with their clinicians. Those are the people with the learned experience.
 And I think, as we've heard today, the fact that systemic sclerosis is a rare multisystem disease affecting people in different ways just adds to the complexity of both treating and living with this condition. There's no clear pathway as there are with some diseases, which is another reason why I think these guidelines are so important. For example, if somebody starts to develop new symptoms, which may be unexpected to them, I think it's really helpful for people to know what tests are available, how often they should be done, and then what the treatment options are. So knowing what is best practice is really important information, not just for the healthcare professionals, but also for the patients and all of this will help support shared decision making, enabling patients to be more involved in their healthcare, which is what we all want.

Ernest Choy 
 Wonderful, Sue. Thank you for that. Maybe I'll come back to all the panellists and ask are there any other points that anybody want to make?

Chris Denton   
 So, Ernest, I just wanted to say, that, I think we've got a very good feel for the sort of breadth of approaches that we had in developing the guideline. And I hope you'll get a feel for the good discussions we had as we worked together. But I want to really perhaps make a note that people really should look at the full guideline. I think there is a tendency, of course, you know the executive summary will be published in Rheumatology, but it's the full guideline that will be accessible via the BSR website and linked in the publication, that really provides the depth of management guidance, I think, and also includes important sections like service organisation and delivery and what our standards would be, what we think patients should have available, and also it includes some audit tools and suggestions both for service audit but also for patient-specific audit and we very much hope it’s something, for instance, maybe the BSR or SRUK will be involved with in the future as we, not just publish the guideline, but also audit it in the future.

Ernest Choy 
 Right. Anything else? So, if I'm someone who is interested and excited about the guideline and I want to implement it in my local care pathway, and there are points that I'm not clear about, is there any support and help I can get from the guideline committee?

Chris Denton   
 So I would just also highlight that we've carefully constructed two figures in the full guideline. One will be in the executive summary. One provides a schematic for how we think patients in 2024 should be managed in terms of their journey through the disease. And the other is literally a schematic for each of the organs with the top line, take-home messages and drugs with the evidence based summaries. So I think perhaps a good starting point, for anyone looking at the guideline, will be to look at those two figures in the full guideline.

Ernest Choy   
 Yeah, I must say, I actually have read the guideline and really like that figure, the organ specific management recommendations, I find that being very helpful. I find systemic sclerosis being a rather heterogeneous disease in many ways. And I think that organ-specific way of looking at how best to manage patients is really wonderful and a fantastic help.

Chris Denton
Thank you.

Ernest Choy   
 So if there's no other comments, I want to thank everybody for joining me and giving the specific input and also the time and effort in developing and writing the guideline, which is a substantial document and I look forward to seeing the guideline in publication and, most importantly, being brought into clinical practice. Thank you.

Chris Denton   
 Thank you.

Bridget Griffiths   
 Thank you.

Clare Pain   
 Thank you.