Paediatric orthopaedics: the modern quest for standardized, evidence-based care

Listen to Andrew Duckworth, Yael Gelfer, Liz Ashby and Dan Perry discuss the paper 'Paediatric orthopaedics: the modern quest for standardized, evidence-based care' published in the January 2025 issue of The Bone & Joint Journal.

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[00:00:00] Welcome back everyone to our BJJ podcast series. I'm Andrew Duckworth and a warm welcome from your team here at The Bone and Joint Journal. As always, we'd like to thank you all for your continued comments and support as well. There's a big gratitude to our many authors and colleagues who take part in the series that highlight just some of the great work published by our authors each month.

So today for our monthly podcast, I have the pleasure of being joined by three authors from an editorial published in the January edition of the BJJ entitled 'Paediatric orthopaedics: the modern quest for standardized evidence-based care'. So firstly, I'm very pleased to be joined by my editorial board colleague here at the Journal, Yael Gelfer, who is a consultant paediatric orthopaedic surgeon at St.

George's in London. Thanks for joining us, Yael. It's great to have you with us. Thank you. Good to be here. Secondly, joining us is another of our editorial board colleagues here at the Journal, Liz Ashby, who is a consultant, paediatric orthopaedic surgeon in Cambridge. Liz, great to have you with us. Thank you for having me.

And finally, we are delighted to welcome back our awesome Speciality Editor for paediatrics here at the BJJ. Professor Dan Perry. Dan, great to have you back [00:01:00] with us on the podcast series. Always a pleasure. Thanks Dan. So Yael, I think I'll kick off with yourself if that's all right. And just about the editorial itself, starting by discussing about the evolution of paediatric research in, in your specialty over the past few years.

And can you maybe give us a brief overview of how things have just rapidly evolved and changed over the past five to ten years? It's really is impressive. Yes, thank you. To start with, the paediatric orthopaedic differs from adult orthopaedics in in several ways. There's low volume of pathology. There are no registries, there's no treatment benchmark.

There was, there used to be low level of evidence until recently to guide practice in decision making. So this created a large variation in treatment. In the last few years, things have moved forward, I think in three fronts. One is creating consensus statements. Mm-hmm. So allowing benchmarks and reducing variation in treatment in several pathologies.

Two is creating core outcome sets that define outcomes of the pathologies that we [00:02:00] treat, and we're able to conduct better research that way. And the third is defining research priorities and addressing them one by one with national and, and international RCTs. I think that's really nice. That actually sets us up really nicely for the rest of the, the podcast about the things we're gonna talk about, so that's really helpful.

Liz, maybe if I could move to yourself, you know, building what Yael has just said, what are your own experiences of this and how this has changed in, in your, your clinical practice and during your, your time? I. Yeah, well at the Journal we've really seen a shift in the research papers coming in and they, you know, they tended to be retrospective case series from single surgeons or single centres, and now that has really changed.

There's a much more of a collaborative approach. And back in 2018, the BJJ published the paper identifying the priorities of research topics amongst children's orthopaedic surgeons in the UK and that was really the start. Then, as Yael was saying, we then. Saw a flurry of consensus papers club feet looking at muscular skeletal [00:03:00] infection in children and looking at the treatment of DDH in young children, all published in the BJJ.

And then what we're now moving on to is, sort of setting up randomized controlled trials to really answer how best to manage these common paediatric conditions. But in order to get good results from those trials, we need to know what we're going to measure. And that's why we've been defining core outcome sets.

And again, the BJJ has published many of these Dan's paper looking at core outcomes set for Perthes. Then also a core outcomes set for childhood limb fractures and then clubfoot. But looking to the future, what we're all really looking forward to at the Journal is seeing some of the results of the randomized controlled trials, which I'm sure we'll be coming onto, and that Dan has set up with both his Big BOSS Study and his Perthes studies, and of course they yet come, but we wait with baited breath.

Absolutely Liz, and I think that that really sets us up nicely again for other topics we're gonna cover this evening. But Dan, maybe just before I move on, you know, [00:04:00] yourself as a specialty editor for paediatrics, is that very much what you've seen that sort of change in evolution over the past few years and that sort of, as Liz has said, that transition to sort of much higher levels, levels of evidence, but also setting up for bigger studies to come?

Yeah, absolutely. So, so we've, we've completely changed the, the research focus at the journal. So it, it's, it's all very much to gear towards the high level evidence and, and not everything is high level evidence, of course. But, but a lot of it is, is building the pathway to, to the evidence of the generation of bigger evidence.

And I think that's really important as well. So. So, so there's different frameworks you can use. I like to look at a framework called the IDEAL framework. Which looks at where we are in surgical innovation. So it really takes us through the whole kind of, in medicine is always phase one, phase two, phase three studies, and it doesn't really apply to us in surgery. But that IDEAL framework is really ideal for us in surgery. So as an, as a specialty editor, so I'm always looking to say, okay, where. Where does this particular paper fit in the IDEAL framework and how's it gonna take us to the next level? And that's what ultimately I'm looking for. Yeah, no, absolutely.

That, that [00:05:00] makes total sense. And I think just to highlight for our listeners, for all three of what have you said, I mean obviously we've got the BJJ, but also the BJO has a lot of this content as well and actually has been a really good outlet for things like protocols and some of the core outcomes that works across, across the specialty, not just in paediatrics.

And it's, I think it's important to highlight that. So Yael, maybe if, if we could. Move back to yourself and, you know, we've, we've mentioned briefly about this idea of core outcome sets. Can you give us a bit more detail about how that's developed and, and maybe an example that you've, I know you've been involved with yourself.

Yes. So the concept of core outcome set is basically capturing the most important outcomes. For a particular condition or disease from multiple stakeholders with including the patients and the families, or putting them at the centre of, of that. So these have been developed for, as Liz mentioned, for common paediatric conditions.

We have them for fractures, for clubfoot, for cerebral palsy, for Perthes disease. And the idea is that it, it, it all generates meaningful research and, and, [00:06:00] and all the audits and registries adopt the relevant core outcome sets. So. So that's the, and my example would be in clubfoot and that, that's the close to my heart.

So the Ponseti has been the gold standard for the primary correction. And the challenge is always maintaining the primary correction that was achieved. And there was a very large variability and inconsistency in outcome reporting, and the core outcome set was developed in order to address that.

But the core outcome set for clubfoot includes both the clear definition clinical outcomes, as well as. Outcomes that are more important to families and patients like quality of life measures. And, and all, all of these outcomes are relevant to different stakeholders and the, and combine the, they form the, the minimum set of outcomes that we can use.

This was already used in a multicentre observational study, the CoCo study that was published last year. And our next step would be the, the first clubfoot RCT, which we're looking forward to. That's, that's [00:07:00] awesome. Yeah, and I think it's a really nice way that that has developed, you've developed that over time.

It's also been used in the, in the the prospective, the observational study and then moving on that. I think that's a really nice example. And actually, like I, I highlights the, the progress in clubfoot, so if we move on, maybe to you, Liz, talk about Perthes, you know, you know, really important paediatric orthopaedic condition as well, and just what, how that the, the knowledge around that has developed.

Over the past few years and, and leading to an RCT, we'll actually also mention as well. Sure. So, I mean, there's huge variation in the treatment of children with Perthes, and it varies between surgeons, between centres, and hugely between countries. Non-operative management varies widely. Some surgeons advocate strict non-weightbearing with children in wheelchairs, and some surgeons allow children to run around as normal.

Also the frequency, the timing, the type of surgery varies widely. Some surgeons will only perform proximal femoral osteotomy. Some will also add in a pelvic osteotomy. So to further evaluate these different treatments of [00:08:00] Perthes and also look at the epidemiology, Dan set up his BOSS study which was published about three years ago now, and it looked at almost 400 hips just over a third of which were treated surgically.

And interestingly, the study showed no improvement in the radiographic outcome of treat of children treated with surgery versus those who were treated conservatively. And that makes you ask why is it that the surgery's no good, or is it actually that the surgery was good? And if the surgery hadn't been done, these children would've had a worse outcome?

So. In order to answer that Dan has set up his randomized controlled trial. And this has now started the Op NON-STOP Study. And I know, I'm sure Dan will want to say a lot more about it, but they started recruiting in November last year and they aiming to recruit 216 children with Perthes. And these children will be randomized into two groups, one of which will undergo surgical.

Containment and it's a [00:09:00] pragmatic study, so the surgeon can choose the type of surgery, either femoral plus minus the pelvic osteotomy, or they'll go into a group of active containment where they will have a physiotherapy protocol where the children, first of all will have lots of stretching and activity modification, followed by a strengthening programmemememe.

Dan has established a core outcome set, which he will use to evaluate these children, but the primary outcome will be the PROMIS mobility at three years, and then also looking at the rest of the core outcome set. And yes, so that has just started. But there's been a huge shift from literally you could choose any treatment you like and there would be some evidence somewhere to support it, but hopefully we're gonna get some really good data and we'll for the first time, be able to hopefully say which treatment is better.

That's such a lovely, I've, I've recruited my first patient for this trial today, actually. Oh, that's great. That's wonderful. And that's a great overview, Liz. Actually, I think in terms of, I do, I thought it was [00:10:00] really, I really liked the sentence you had in your editorial guys of the, you know, you know, in, in days gone by, the, the definition of success was basically osteoarthritis, the hip.

I mean, and, and that was just, you know that, and then you debate. Everybody has a debate about that. And actually you think. Is that important and actually have, that has evolved and the importance of the other outcomes there I think was just so, it's just so important that we actually take a step back sometimes and actually what do people need to look at is, Dan, is there anything, obviously it's your trial, anything you'd add, add to that from Liz and, Yael, so I think it's one of those really nice trials that's come through the Journal in terms of the, the whole process has come through the Journal and in this month, so in, in March.

There's an editorial about the process to setting up that randomized controlled trial. And, and, and, and we've gone through every stage systematically. So developing the core outcome set, working with families. Kind of looking at which, which outcome is best. So once you've got a core outcome set, you still don't quite know how you're gonna measure it.

So we looked at what the best way of measuring it is. Then Adam Galloway, who's a physiotherapist, has done all of the work to try and standardize physiotherapy care. He developed an app to make it, make it more attractive to families, and it's, it's really [00:11:00] gone through the whole process. We did a BOSS study, which really, really set out what the, what the evidence was at the moment.

I. So, so we did everything we could to work up to that, that ultimate trial. And I hope it's gonna deliver and as Yael say, she recruited, I think our 14th patient today of the trial, everyone said was impossible and it's recruiting really well. So we've only been over a few months. That is awesome Dan. And I think, like you said, I think it's a really good insight that into, to get these really, these studies.

Really, really good. And to give you the answer, like you say, the amount of work that goes in beforehand, before you actually just can't just set up a trial is, is awesome, but actually that's the right way to do it, isn't it? And actually that will like most likely give you the, the, the answer that everybody wants.

Yeah, no, absolutely. It's yeah, it's a way forward, but, but it is a lot of work together. Absolutely. And maybe I've come, come back, come to you now just maybe to finish, finish off some of the big four or five conditions, you know, you're seeing in paeds obviously management of DDH and SCFE. What have been the big sort of changes there?

So, so we're doing, so in terms of DDH so we'll start there. So we're, we're very much doing the same sort of process, so, so [00:12:00] getting to, to where we are with Perthes disease. And it's, there's lots of questions in DDH and particularly lots of like really, really super simple questions about, you know, how best to screen, you know, when we do screen and identify DDH, when do we even start treating it and how do we treat it and when do we stop treatment and, you know, what sort of harness do we use?

Like really little things. But you think we should have just got nailed by now? 'cause it's super common, but we don't know the answer to any of them. And, and so we, we've done what we're doing. So we're, we're, we're developing a core outcome set. So Joanna Craven, a PhD student doing that. And alongside that, we're, we're looking at what the, the current state of the evidence is through systematic reviews.

And we've got, and there's, there's a national database called SMaRT4NIPE which everyone in England. Puts all of the babies into automatically. So a midwife will enter all the babies into a computer and basically we're, we're making that a big national database with within which hopefully we can embed trials and if we can embed trials in that database that collects routinely every single baby in England, that's 50,000 babies a month.

And that means we can do really cool [00:13:00] screening trials. Really, really quickly. And that would be awesome. That, so that's the kind of, especially 'cause NHR, the funder have just asked for big data enabled trials to do with children. I'm like, this is awesome. Yeah. So so that's the plan. Great. And then in and then in, in slipped epiphysis, I mean, that's been another one through the BOSS study.

So again, we've been developing and working up trials in that area. So the Big BOSS trial is the, the one that we've been working on in that area comparing severe severe SCFE. So, so acute correction versus pinning inside like tube plus or minus select correction if we need it. So, so we really are tackling everything from all angles.

And yeah, so it's, it's really cool and, and. And we've just got the first result, which I'm not allowed to tell you yet, but the first result for the Science Study and it's so cool. So excellent. It's really good. That's when, when can we, when will we find out now, just so the listeners know to look out for it.

So it's probably gonna be in summer, I. Okay, awesome. But awesome. Yeah, it's, it's really cool. Something to be excited about, about medial epicondyle fractures. Just that, that is awesome. And I think, like you say, it's great for the community to hear these [00:14:00] results. I think you, you feel like you're progressing, don't you?

And I think that's a really important part of it. I think that's, that's awesome. And d Dan, just before we, we wrap up with everybody it's slight sidebar, but I just wanted to highlight, you know, another paper in the January edition that you've been involved with. And I think just looking at a slightly different part of.

Of, of, of research in, in, in, in paediatric orthopaedics, but it is coming through in other parts, parts of our specialty. And that was looking at automating radiological measurements of the hip in children with CP and just sort of highlights the ever-growing role and investigation maybe of of machine learning technology and how it can be potentially really helpful, both clinically and, and maybe as well in, in research as well.

Yeah, sure. So, so we did a paper where we were looking at children with cerebral palsy, and basically we're trying to, so, so kids with cerebral palsy have got a national surveillance programmemememe. And that national surveillance programmemememe is recommended by NICE or recommended by the, the, kind of the, the, the authorities which monitors kids, depending on how severely they're affected by CP, monitors kids to see their, their, the degree of hip migration, so how much their hip's escaping [00:15:00] from their acetabulum. 

And there's a, there's a whole programme of, of, of kind of when they should be measured. But that relies on people to actually remember to do it. It reminds on people to make the measurements. It relies on people then to enter the measurements into the national computer system and keep an eye on the measurements.

So there's kind of lots of little bits where it can fail. So, so developing an an AI tool seemed like a, a really good way to, to kind of simplify a lot of this. So something that can actually do the measurements then, then can automatically populate the national database and then everything can be automated.

Even the, the kind of the, the sending out the information to the parents and, and invites for, for radiographs and stuff. At least that's the theory. So, so we developed a system, so the, the, the super clever engineers at the University of Manchester. Developed a system with about one and a half thousand pelvic radiographs.

Each looked by many different observers that could essentially identify these kids with hip migration super early. And and we've been working over the last few years and developing and refining it in kind of a load of different hips. 'cause these hips are often really hard. 

Like the hips are all over the place. [00:16:00] It's not like. You know, it's not like just an arthritic hip, which the hip's always centred and it's always in the right place. These hips are wildly they outta place, so it's quite hard for a computer. But basically we, we've developed a system that works that's as good as experts.

And, and now and now we're at the stage of saying to, to the big national funding bodies, look, this is really cool. Let's, you know, give us some money to implement this. And that. That's basically what the study, the study shows it, it kind of goes through that process and. Yeah. And it's really helpful and kids', orthopaedic surgeons really excited about it.

No, I think that's awesome, isn't it? And I think, like you say, it's just using technology to help us and actually make us much more efficient and actually get the, get the get the get the data that we need. I think, I think it's a really cool study actually, and just encourage our listeners to, to have a look at it.

So guys, just to wrap up, there's another one is places. Sorry, sorry. It's another one of these places we can then do trials. It's all about where we can put the trials. Exactly. Exactly. Exactly. So guys, just to sort of wrap up, I'll, I'll come to each and tell you then Liz. And then Dan, what, what do you feel in your own experience has been the, the, the, [00:17:00] the key really to the really impressive growth and success and all the amazing work that has occurred in the subspecialty over the past few years and maybe what you think the future holds?

Yael, maybe I'll start with yourself. I think that might be three of those. So you, you ask for one, you get three in return. So one is the perhaps establishing standards and agreeing on outcomes. So these are the, perhaps the foundation of every high quality research. The second is collaboration. We somehow manage to find a way to collaborate in the UK in particular, like nationally.

Join forces to do this. This funny looking man with glasses from Liverpool. That is pretty awesome. That's the third. I think that's a brilliant three, Yael. I love it. I love it. Liz, how about yourself? I would say it is all down to collaboration. I think as surgeons we often, you know, we're used to working alone.

Surgeons like to work alone. We don't like to share the glory. And [00:18:00] I think I agree with Yael, it is down to Dan. He has managed to unite basically the whole of the UK body of children's orthopaedic surgeons. And Dan, that is an awesome thing you have done because no one has managed it before. And I think in order to unite surgeons and.

To make them in a nice way, participate in studies where, you know, their name may not be top of the list. You know, they, they may go unnoticed a little bit, I think is an amazing feat. But I think that great things will come from these trials that are just starting now. I think that's brilliantly said, Liz and I totally agree, and I think the ability of, I'm not gonna, there's not the Dan appreciation podcast, but I would like to say that I think it is, impressive to get everybody to buy into that is really, it is really an achievement. Dan, I will come to you last and say, what do you feel it's about? Well, I'm very flattered. But ultimately it's a massive team game. Like it is just a massive team game children's orthopaedic surgeons are so. You, you know?

No, ev ev no one's got an agenda. Everyone just is doing it for, [00:19:00] for, you know, it sounds a bit corny, but doing it for the kids, doing it for the right reasons. Yeah. And everyone just wants to find the answers and just find out the best treatment and, and we all actually quite like each other. Which isn't the same in all spec subspecialties.

I think it's great and I think you can really see it when you guys are all together and actually with. With the amount of work that you've managed to develop and produce over a short period of time. I think you're right. It doesn't, it doesn't come without a good, good directional leadership, but also getting on in collaboration.

'cause ultimately you do have fun doing it, don't you as well. And I think that's really important. Well guys, I afraid that's all we have time for, but thank you so much to all three of you, that that was really fun and a really, really good overview of where things are in, in the subspecialty and just a really, a big, a big.

Gratitude, I think from orthopaedics in general actually for the amazing developments and achievements that have been made in the paediatric orthopaedic area. And certainly a lot to look forward to moving forward as well. And to our listeners, we do hope you've enjoyed joining us and we do encourage you all to share your thoughts and comments on the various platforms and the like.

Feel free to post about anything we've discussed here [00:20:00] today. And thanks again for joining us. Take care everyone.