The Pediatric Epilepsy Learning Healthcare System Provides Big Data to Answer Big Questions

Zachary Grinspan, MD, MS

Pediatric epilepsy has been known to be associated with poor health-related quality of life (HRQOL) outcomes, and with massive amounts of data being collected in clinical trials and patient databases, the need to properly utilize these data is critical. Zachary Grinspan, MD, MS, a pediatric epilepsy specialist at Weill Cornell Medicine, in New York City, and colleagues are seeking to address this challenge, and in doing so, created the Pediatric Epilepsy Learning Healthcare System (PELHS).

At the 2022 American Epilepsy Society Annual Meeting, held December 2 to 6, in Nashville, Tennessee, Grinspan and colleagues presented data from a recent multicenter, cross-sectional study, the results of which showed that the Pediatric Epilepsy Learning Healthcare System Quality of Life (PELHS-QOL-2) prompt is a valid HRQOL prompt for children with epilepsy, and can be used in clinical care as a way to consistently start conversations about quality of life with caregivers on the children with epilepsy.¹

While on-site at the meeting, Grinspan spoke with NeurologyLive^® about what is occurring presently within the field of pediatric epilepsy based on the large amount of data given to researchers to analyze.² Additionally, he offered his insight on the hopes for the future in investigating how to improve clinical care for patients.

NeurologyLive^®: Can you provide an overview of what is currently happening in the clinical field of pediatric epilepsy?

Zachary Grinspan, MD, MS: Several of my colleagues and I realized that every time we see a patient with epilepsy, a child with epilepsy, there's an ocean of data that's created. We see the patient in the clinic, we write our notes, we draw labs. Sometimes they come to the hospital, we get EEGs on them, we do MRIs, we get genetic testing. They go for surgery, etc, etc. And all that data is sitting in all of our centers isolated just for clinical care.

Clinical care is important, but we want to learn, we want to do better. We want to give the best possible care to our children, for every child that comes through the door. We realized that we should use this ocean to learn. What we did is we founded the Pediatric Epilepsy Learning Healthcare System, with generous donations grants from the Pediatric Epilepsy Research Foundation, from our centers and from some other sources. We now have a data coordinating center at Weill Cornell Medicine, in New York City. We have data from 18 centers around the country. They ship us these huge datasets with raw electronic health record data that we then ingest into our data coordinating center, process it, standardize it, and then use it to learn.

For example, we are interested in a disease called infantile spasms. This is a devastating type of epilepsy that starts in the first year of life, and if you don't treat it well, if you don't do the right thing early in the course, then many of those children develop lifelong epilepsy. They're at higher risk for developmental delay. We know what the first line therapy is [adrenocorticotropic hormone], oral prednisolone, or a medicine called vigabatrin. But we weren't sure how often people were using that first-line therapy. We looked, and we found out that about 90% of the time we were doing the right thing. But in doing that, we also developed some methods to track how well we're doing as a community. We thought about other outcomes to measure, such as, do they have remission? Do they have relapse? What does their EEG look like? What is their development like? It spurred a whole series of new questions to ask about infantile spasms.

So based on that work, we're going to launch a clinical trial, not in the learning healthcare system, but based on what we found, to understand if oral steroids can prevent relapse of infantile spasms. We are thinking about whether some of our medication choices in early life can put children are at risk for infantile spasms. The main message, I think, is that we want to figure out a way for the work that we do every day in clinical care, as we're taking care of children in our clinics to take that information, aggregated across our centers across the United States, and then learn from it and do research, quality improvement, surveillance and epidemiology, health services, research, etc. So that we can optimize care for this vulnerable population of kids.

Do you have any hopes for what will be done in the future and what do you think should be done?

Yeah, so I'm going to nerd out a little bit with my answer to that question there. There are a lot of really exciting clinical things happening with the learning healthcare system. One thing that's very as granular is in order for us to learn from all of these data, we need to be doing the same thing in clinic. Now, that is a big challenge because part of what makes people want to be physicians is they really love that independence. They want to be able to practice in practice medicine in a way that they like. So we've thought very hard about what can we do to help physicians when they're in clinic to make their lives easier, while also standardizing the way data is collected at the time of care, so that we can learn from it.

So what we did, it was a four year process, was we got a bunch of experts together, and we thought about what questions are we always asking in the clinic to our families. And we realized that most of the questions were the same. What are the answers that were listening for? Again, across all of our centers, most of the answers were the same. So we took all those questions and answers, compiled them into a list, and developed a questionnaire. We then took that questionnaire to the electronic health record vendors. And we said, "Dear vendor, can you take our questionnaire and build it into your system, so we can all use it?" And they did. So now, in 3 or 4 different vendors' systems, our form is there, you can ask the IT people at your hospital to build out our form, the PELHS clinical form, and, and then use it in clinic.

The way we design the form is you kind of click through these big, beautiful buttons that allow you to ask all the questions you're going to ask anyway. And then there's some logic that allows you to pull that information into your clinical note, so it makes your life easier. You've got standardized data, faster note writing, and we get to learn from what's happening in clinic. We were a little bit afraid to do this because, again, doctors like to do their own thing. But we've gotten a lot of inspiration from other pediatric disease organizations, particularly this group called improve care. Now they do inflammatory bowel disease and they've been doing it for a decade. So we thought, Look, if the pediatric GI doctors can do it, we can do it, too, in pediatric epilepsy.

What do you think has been the greatest and most recent advance in this aspect of clinical care?

One of the things that we did, one of the things that makes a learning healthcare system work is that it's not just the physicians thinking together, it's that you bring in advocates, parents, researchers, and representatives from professional organizations as you ask and answer questions. You have lots of different stakeholders, helping guide which questions to ask what's important? Then, how to go about answering the questions? One of the things that we heard early on was about quality of life. The family said, "Yeah, my kids seizures are terrible, but quality of life is important." And so we pushed on that, and asked a lot of questions and looked around to see, well, how are we going to measure this? There are these standardized validated instruments to measure quality of life and they're 50 questions long.

Many of us said, how am I going to ask 50 questions, in addition to how are your seizures and how are your medicines, when coming to the epilepsy monitoring unit? It's just too much. We thought, can we reduce these 50 questions to 1 or 2 questions, so that we could ask them in the context of clinical care and learn about quality of life, inspired by the fact that the parents and the advocates are telling us this is so important. We came up with 2 questions. The first one says, "Think about your child's usual routines. How often do seizures disrupt those routines?" And the paired question, "Think about seizure medication side effects, how often do those side effects negatively affect your child's routines?" In both questions, thinking about the past 2 weeks, we nailed the questions. We figured out exactly what they wanted to be.

I have to tell you, it wasn't our idea. It was a parent's idea. It was a parent who told us that these were the right questions to ask and we're like, "OK, you got it." We took these questions and formulated them, we figured out exactly the right language, and we piloted them at 4 centers and compared them to several of these standard instruments to measure quality of life. We showed that this was a valid and reliable way to measure the quality of life for our children with epilepsy that we're taking care of every day. Those 2 questions are in our form.

Click here for more coverage of AES 2022.

REFERENCES
1. Shah PD, Yun M, Wu A, et al. Pediatric Epilepsy Learning Healthcare System Quality of Life (PELHS-QOL-2): A novel health-related quality of life prompt for children with epilepsy. Epilepsia. 2022;63(3):672-685. doi:10.1111/epi.17156
2. Grinspan Z. The Pediatric Epilepsy Learning Healthcare System (PELHS): Answering Big Questions with Bigger Data. Presented at: AES Annual Meeting; December 2-6, 2022; Nashville, TN, and virtual.