What is a Registry?

In this episode, you will learn what a registry is, the roles that registries play in research, and how patients and families can help support them. 

In the second episode of The CARRA Podcast, Claudette Johnson talks with Dr. Mary Beth Son, who is the Co-PI of the CARRA Registry. Dr. Son is Clinical Chief of the Division of Immunology and Section Chief of the Rheumatology Program at Boston Children's Hospital, as well as an Associate Professor of Pediatrics at Harvard Medical School. 

The Childhood Arthritis and Rheumatology Research Alliance (CARRA) has the largest ongoing, observational registry in North America that collects clinical and patient-reported information about children and young adults with pediatric-onset rheumatic diseases. The CARRA Registry has more than 14,000 patients enrolled at 70 sites. It also serves as a biosample repository for select translational studies that leverage registry data. 

The CARRA registry is a large registry, and it is used to study pediatric rheumatic disease. It has a lot of people participating and supporting it. At the moment, there are three main diseases that we collect data on, and that includes juvenile arthritis, lupus, and juvenile dermatomyositis. These are three of the more common diseases that we see in pediatric rheumatology clinics.

The real advantage of studying pediatric rheumatic disease in a registry is that these are rare diseases. We really want to be able to answer the kinds of questions that patients and their families have, but any one center has a very difficult time robustly answering questions about these diseases because they're rare. If you pool everybody in, then you're really able to study things and hopefully get to some meaningful answers for patients and their families.

Dr. Son also explains exactly how the registry works, what kind of information is collected and what the roles of patients and families are. She also explains the importance of biosamples in pediatric rheumatic disease research.

Claudette and Dr. Son tackle a wide variety of topics related to the registry, including AI and data privacy.

Learn more about the CARRA Registry and ongoing research using the registry and biorepositories.

Transcript

What is a Registry?

Episode Two of The CARRA Podcast

Claudette Johnson: If you're a patient like me, you've probably heard the term “registry.” Maybe you’ve been asked to join the CARRA Registry.  

And if you're like me, you probably only have a sense of what that means. In this episode, we're going to learn what a registry is, the roles registries play in research, and how patient families can help support them.

Welcome to the CARRA Podcast. CARRA is the Childhood Arthritis and Rheumatology Research Alliance [00:01:00]. We are doctors, researchers, and patients all working together to prevent, treat, and cure pediatric rheumatic disease. My name is Claudette Johnson, and I'm your host. Today, I'm speaking with Dr. Mary Beth Son. She's the Section Chief of Rheumatology at Boston Children's Hospital and Associate Professor of Pediatrics at Harvard Medical School.

Last year, Dr. Son was among the principal investigators who received a $4. 4 million grant from the CDC to advance childhood lupus research through the CARRA Registry. Simply put, Dr. Son knows a lot about registries and how important they are to research. Dr. Son, thank you so much for joining us.

Dr. Mary Beth Son: Happy to be here today, Claudette.

Claudette Johnson: So, we first met when I went to Boston Children's Hospital to come get a second opinion on my diagnosis of linear scleroderma. I don't know if you remember me?

Dr. Mary Beth Son: I remember, I remember you very well. It was a long time ago. What [00:02:00] year was it?  

Claudette Johnson: Oh my gosh, I got diagnosed when I was like nine years old. So whatever year that was. 

Dr. Mary Beth Son: Many years ago.

Claudette Johnson: Yeah. What I remember most from there was you had these piano stairs, where if you stepped on the steps, it would play the keyboard sounds. That was just like the highlight of my entire trip. 

Dr. Mary Beth Son: We still have them. So that's good to hear. 

Claudette Johnson: Yeah. All right. So, this podcast episode, we're going to be talking about registries and what often happens is when patients are first diagnosed. It’s a question that's been asked by their doctors and their families about joining a registry. Now I thought we, it would be good to just start off with the question: What is a registry?  

Dr. Mary Beth Son: Sure. So, a registry generally refers to sort of like a repository or a collection of data. And, you can have, [00:03:00] typically research registries have a lot of clinical data regarding the patients that participate in them, and the data can really vary in terms of how much data you collect, what kind of data you collect and if you collect other things like biosamples. But a registry itself is a very broad term for a way to capture important information that you think will inform research questions and research projects.

Claudette Johnson: So, you said it collects data. What sort of data is that, if you could go more into detail?

Dr. Mary Beth Son: Sure, sure. Would you like me to talk about the CARRA registry, or just in general? So, the CARRA registry is a large registry and it is used to study pediatric rheumatic disease.

It has a lot of people participating and supporting it. At the moment there are three main diseases that we collect data on, and that includes juvenile arthritis, lupus, and juvenile [00:04:00] dermatomyositis. So, these are three of the more common things that we see in pediatric rheumatology clinics.

The real advantage, I think, of studying pediatric rheumatic disease in a registry is that these are rare diseases. So, we really want to be able to answer the kinds of questions that patients like you and families like yours come to us with. Any one center has a very difficult time, like, robustly answering questions about these diseases because they're rare.

But if you pool everybody in, then you're really able to study things and hopefully get to some meaningful answers for patients and their families. 

Claudette Johnson: So, why do researchers specifically need and use the registry? 

Dr. Mary Beth Son: What does it help? It helps with clinical questions like, what medications are best for either me to take or for my child to take?

What kinds of side effects do we see with different kinds of medications? What does it look like for a child [00:05:00] or an adolescent with arthritis? If they're five years into their diagnosis, how do things look like in regards to what kind of medications they're taking? What is their disease activity level?

Because there's different disease activity measures that we can look at, you know, in different diseases. It just gives you the ability to sort of build snapshots of what it looks like along an entire spectrum of disease for children and for adolescents with these specific diseases that I mentioned.

Claudette Johnson: So, on the part of a patient, what can patients do to help the registry?

Dr. Mary Beth Son: Yeah, thank you for asking that question. You know, I would love to hear what you think as well, Claudette, because I think sometimes, research means different things to different people, and participation in research can sometimes, I think, seem either unfamiliar, maybe it sounds like a little scary or it's unclear what people have to do. For a registry, especially one like the CARRA registry, which is what we call observational, [00:06:00] this means that we're collecting data at routine visits. We're not asking patients and families to do things outside of clinic visits.

I think if patients and families are interested, first of all, in any kind of research, I think they should always approach their doctors and ask because that is how questions get answered and we move things forward for clinical, um, clinical outcomes that I think patients and families care about.

So, but for a registry, all you have to do really is show up to clinic and report which medications you're on so that we can log that. And then we do ask either patients or their parents to fill out certain kinds of surveys. And those surveys are something called patient-reported outcomes because we want the patient voice involved in our clinical research.

So the patient reported outcomes have to do with how do you do during the day? Do you have pain? How is your function? How is going to school? Are there things that are hard for you? So, we do need patients engaged on that aspect of things. We ask them to fill out those surveys because that's [00:07:00] totally specific to them.

We can't answer those questions; they have to be able to do that themselves. But it all happens within a clinic visit. So, hopefully it doesn't feel like a lot of work. It's contributing to a really important goal. 

Claudette Johnson: And on the question of biosamples, is that also used? 

Dr. Mary Beth Son: Yes, so I'm glad you asked about biosamples.

Because if you think about it, we can collect all, all these different, we call it data points, or different data items on patients who are in a registry. And that has to do with how old they are, what disease do they have, what medications do they take. Is their disease active or not? But those are all questions that are answered um, without knowing what's going on inside that person's body itself.

So bio samples add in that whole dimension. That way you can study someone's blood, usually, for different kinds of either markers of disease or predictors of disease, [00:08:00] and that you can match that to the patient's clinical data and then we get a real picture of what's going on in that disease.

So biosamples, and again I'd love to talk about this because I want to dispel like any myths about what people might think about biosamples. Biosamples are just another way of doing research and helping us figure out what causes this disease? What makes them worse? What makes them better? And you know, what the outcomes are.

Claudette Johnson: Do you have patients who are concerned about their privacy? And if so, what do you say to them to help reassure them? 

Dr. Mary Beth Son: Yeah, thank you for asking that. So for sure, that is one of the main concerns that's either brought up either by the patient or the family. And I think that's a very legitimate concern, especially in this day and age of People always feeling like they're being hacked and their information's being stolen.

I mean, this is a really important question to ask for anything that you participate in. So what I would say for the CARRA Registry specifically, which is what I know the most about in this instance, is [00:09:00] that the data, the data that belong only to you, so like your name, and your birthday, and all that kind of information, is stored securely, on a server, and it's only given out to other people as de identified data, which means that no one will be able to figure out that it was you.

We take security risks extremely seriously and, we are happy to talk about it in clinic. But the risk is actually really quite low for these kind of data. 

Claudette Johnson: Who is invited to join the CARRA registry specifically? Is it every patient? 

Dr. Mary Beth Son: So ideally speaking, we would like every patient who comes to a pediatric rheumatology clinic in either the U.S. or in Canada who have either juvenile arthritis, lupus or juvenile dermatomyositis to be approached because we feel that research should be inclusive. And what I mean by that is that we learn the best about these diseases if we get everybody who has it into research studies that we [00:10:00] really know what happens across the spectrum of disease.

So, ideally speaking, we would like all patients at least to be approached. It doesn't mean that patients have to participate, just to be clear, right? Like, each patient and family needs to make their own decisions that they're comfortable with for participation. But we feel like that decision should be up to them.

They only get to make that decision if we ask them, if we approach them and say, “We have this study in mind, would you consider participating?” 

Claudette Johnson: You said you want to approach all patients about this. Does CARRA have a big registry encourage more research? Is this why patients should join? 

Dr. Mary Beth Son: Yes, the registry is one thing that we do in terms of CARRA's research, but there are other research studies that we also do.

And again, that's up to patients, they can do just the registry, and that's a hugely important contribution that we are very grateful for, or there's other studies that they can do as well, that also help further our field. So, it isn't just a one or nothing, it's, there are different [00:11:00] variations depending on what families are interested in.

Claudette Johnson: What do you think is the biggest change you've seen in a person's life from CARRA's registry? 

Dr. Mary Beth Son: So that's a good question because, you know, with registry data. It doesn't benefit sort of like any one person, right? So if you put your data in, you won't necessarily know, because we're protecting your privacy.

We don't publish anybody's name or anything, right? So we put all the data into groups and then we study it that way. But what we can say is that participation in research might help other people, which I think some kids and families, when they're feeling either frustrated by lack of answers or feeling worried that there's not enough information out there for them, Participating in research is a concrete way of saying I am helping to move this forward and it may not help me today But it might help me a few years down the road and it might also help lots of people behind me who are gonna have these diseases in a few years. So, I think the patients that I talk to who are most sort of inspired by [00:12:00] their journey, as a research participant, really focus on that.

They feel like with all the things that they may not have control over, they do have control over this. They have control over their participation and knowing that they're contributing to hopefully making it better for kids coming up behind them with similar or the same disease. 

Claudette Johnson: Perfect. And I also wanted to ask about AI, because in this day and age, it's in the news a lot.

And I feel like, do you see this new technology as helping these registries provide a new perspective on this data? 

Dr. Mary Beth Son: Yes. I mean, I think AI could play an important and really helpful role in terms of maybe like new kinds of analyses that we could do, new ways of looking at these data. 

I still think that the human touch of having a one-on-one conversation about research between either, you know, a physician or a nurse or you know, a person in clinic or a research coordinator is hugely important. That is [00:13:00] still really important and AI can never do that. But on the other side of like looking at the data, once it's collected, I think that the, it seems that we have more and more hope reasons to be hopeful that will get more and more figured out.

Claudette Johnson: For you personally, how do you think the registry has sort of changed your perspective on your practice of medicine? 

Dr. Mary Beth Son: I think the reason why I wanted to get involved and why I stayed involved in the CARRA registry, well there are probably a few reasons. One is that, you know, I mean, Claudette, I met you in clinic, right? And then you and your mom had really, had all the questions that I would have had, right? And we don't have, we don't have enough answers for everybody yet. And so being a part of this, again, it's almost, it makes me feel like I'm doing something concretely to try and help move the field forward.

As we collect more and more data and we look at the data in certain ways and try to answer questions that are important to people who are involved with this – patients, physicians, you know, everybody – it makes me feel like I'm doing some small part in in moving [00:14:00] that forward. 

I also just to say, I find it really meaningful and fun to collaborate with my colleagues and CARRA families. As you know, families are hugely important in CARRA. This is another way to connect with families, which is like the best part of my job. So, I do it for a few different reasons, but I think it's mostly it gives me hope that, you know, we'll study different facets of different diseases, and it'll help inform what we tell patients 10 years from now.

Claudette Johnson: If there's a hesitant patient listening to this, unsure about joining the registry, what would you say to them? 

Dr. Mary Beth Son: I think that I would ask them like, you know, what their concerns are because different people have different worries, right? So, I do think privacy is a big thing and I totally get that. I mean, I think sometimes other people aren't sure if it's going to make a difference, you know?

And like, why would they do it? And why would they take 10 more minutes in clinic to fill out those patient reported outcomes that I mentioned? So, I think I would try and figure out what their [00:15:00] concern is. And I would try to emphasize that the reason why CARRA was founded was to make things better for patients because we were all you know, all at different centers trying to do small studies and we really wanted to make it a more robust enterprise with lots of people working together. And so, I would say that, you know, research is the way to make things better, um, for patients and that we can't do it without them.

We can't do it without you, right? Like it's all, it's all about, you know, involving patients and, and getting answers for them. So, I would try to say that without ever pressuring them. I think it's also fine to say, “I'm not ready now.” Could you talk to me later about it? Like, again, that's fine.

But I would mostly try to spend time figuring out what the concern was and making sure that we're really hearing them. 

Claudette Johnson: Thank you, Dr. Mary Beth Son. Before we end this, I just wanted to ask one more bonus question. What is one thing that your patients might not know about you? 

Dr. Mary Beth Son: Oh, [00:16:00] Let's see. I have three daughters, and I love being a mom. And I really love Mike and Ikes. How about that? 

Claudette Johnson: Dr. Mary Beth Son, thank you so much for joining us. 

Dr. Mary Beth Son: Such a pleasure to be here, Claudette. Thank you.

Claudette Johnson: The CARRA Podcast wants to hear from you. If you have a topic for an upcoming episode, head over to our podcast page on the CARRA website and leave us a message through a SpeakPipe link. We might play your voice on a future show, and we'll do our best to answer your questions. Finally, we encourage you to share the CARRA podcast with friends, patients, and anyone else interested in learning more about the incredible work of the CARRA community, dedicated to creating a world free of limitations for children with rheumatic disease.

This podcast is produced and edited by Emily Nguyen. Our music is by Jonathan M. Horner. [00:17:00] Thanks for listening.