Helping Families Navigate Drug-Resistant Pediatric Epilepsy with Dr. Sandi Lam

Anti-seizure medications are the mainstay of epilepsy treatment, but these medications are not effective for all forms of epilepsy, such as Lennox-Gastaut syndrome or LGS. It's a rare form of epilepsy that has no cure. Dr. Sandi Lam, Division Head of Neurosurgery at Lurie Children’s, is working to help pediatric patients with LGS and their families manage this condition by investigating the outcomes that matter most to patients and their families. In this episode, Dr. Lam talks about this important work and how it may help build a roadmap that will empower the future of research for LGS and other pediatric epilepsies and rare diseases.

“Getting treatment for epilepsy can be a very bumpy journey … and it's very complex, but it's also something that we should all get behind and understand that we should not rest until we get the appropriate treatments and timely treatments to try to get the seizures under control. To help children and their medical health and their quality of life.”

Dr. Sandi Lam
Division Head, Neurosurgery

Show Notes

• Dr. Lam explains how epilepsy can have a far-reaching impact on the health, well-being and quality of life of the child affected and their and the family and the community.  Complex cases of epilepsy, such as Lennox-Gastaut Syndrome (LGS) can have an even greater impact on children and their families because of the limitations of standard anti-seizure medications.
• She discusses the issue of drug-resistant epilepsy and the underutilization of surgery in treating these cases. She believes such timely interventions could improve children’s quality of life.
• To better understand the needs of children with LGS and their families she is partnering with the Lennox-Gastaut Syndrome Foundation to do Patient-Centered Outcomes Research that explores outcomes that matter most to families, such as quality of life, behavior, and communication, which are often overlooked in traditional research.
• One of the studies she is leading compares the effectiveness of continued medication versus palliative epilepsy surgery, using multi-center data to help families make informed treatment decisions. Findings so far indicate that epilepsy surgery improves long-term survival rates for drug-resistant epilepsy patients, underscoring the need to raise awareness of surgical options.

Transcript 

[00:00:00] Erin Spain, MS: This is Precision, Perspectives on Children's Surgery from Ann and Robert H. Lurie Children's Hospital of Chicago. I'm your host, Erin Spain. On this podcast, we introduce you to surgeons at one of the country's most renowned children's hospitals to find out how they're transforming pediatric medicine. Anti-seizure medications are the mainstay of epilepsy treatment, but these medications are not effective for all forms of epilepsy, such as Lennox-Gastaut syndrome or LGS. It's a rare form of epilepsy that has no cure. Dr. Sandi Lam is working to help pediatric patients with LGS and their families manage this condition. And part of her approach entails investigating the outcomes that matter most to patients and their families. Dr. Lam, division head of neurosurgery at Lurie Children's joins me today to talk about this important work and how it may help build a roadmap that will empower the future of research for Lennox Gastaut Syndrome and other pediatric epilepsies and rare diseases. Welcome to the show.

[00:01:10] Dr. Sandi Lam: Thank you so much.

[00:01:11] Erin Spain, MS: Talk to me about pediatric epilepsy. What is the current treatment landscape right now?

[00:01:16] Dr. Sandi Lam: Pediatric epilepsy is a very challenging issue. Epilepsy is one of the most common neurologic conditions in the world. And in children, having recurrent seizures, which is epilepsy, can have a far reaching impact on health, on well being, on quality of life, and the quality of life of the child and the family and the community. There are many developmental aspects as well while the child is growing and has a developing brain, being held back with recurrent seizures has an additional impact on a child as opposed to an adult. So there really is an urgency to thinking about how we can control or stop the seizures, even though it is a chronic neurologic condition. 

[00:02:10] Erin Spain, MS: And unfortunately there are some types of pediatric epilepsy for which there doesn't seem to be an effective treatment. Can you tell me about that? 

[00:02:18] Dr. Sandi Lam: At least a third of pediatric epilepsy is classified as drug resistant, meaning that even on one or two medications that are anti seizure medications, children continue to have seizures. Drug resistant epilepsy actually should be worked up or have diagnostic tests to see if there are other alternatives beyond medications that can stop the seizures. Epilepsy surgery, and there's a whole spectrum of surgeries that can help, is actually underutilized in this country. Only about one in three kids who can benefit from epilepsy surgery actually ever receive the surgical treatment. So, it's a very complex issue in that there are many different forms of epilepsy and there are many different ways to understand the diagnosis and there are many different medications on the market, but the road to getting treatment for epilepsy can be a very bumpy journey, and can be what families sometimes describe as a journey around the world. And it's very complex, but it's also something that we should all get behind and understand that we should not rest until we get the appropriate treatment and timely treatments to try to get the seizures under control, to help children and their medical health and their quality of life and help their futures and help the children and the families.

[00:03:56] Erin Spain, MS: That's something that you are passionate about helping these families, helping these children. And you're working through patient centered outcomes research to do this, explain that concept to me and why it's such a good fit for pediatric epilepsy.

[00:04:11] Dr. Sandi Lam: Because epilepsy is a chronic illness, there are many ramifications to continuing to live with seizures. So right now I work with a patient and family advocacy group, the Lennox Gastaut Syndrome Foundation, and our PCORI sponsored project, from the Patient Centered Outcomes Research Institute. And we work together to understand Lennox Gastaut Syndrome. And Lennox Gastaut Syndrome is a rare form of pediatric epilepsy that is characterized by multiple seizure types and lifelong seizures that are drug resistant, and developmental delay of the children, cognitive impairments, and severe disabilities that could be physical disabilities, and also a shortened lifespan. So this is a severe form of epilepsy, but I think the, framework of how physicians and researchers work together with patient families is a really important one because what we're working on together is to actually understand outcomes that matter to families and outcomes that matter to the caregivers that are taking care of the children affected by LGS. In traditional research, we're able to measure certain things in our clinical care and in the healthcare system. So, in epilepsy, very common things would be seizure control or number of medications used. Other things like coming to the emergency room for seizures or getting admitted to the intensive care unit for seizures. When we work with patient families, we realize there's so many other things that are critically important to daily life and the things that, caregivers really, really care about in terms of looking at, is this going to help our child and the things that really impact daily life are behavior of the child, the communication ability of the child, and the quality of life. So, there really needs to be more partnership to understand is the healthcare system and in the way that we deliver healthcare really serving the needs of the families and are we addressing the major questions that will impact a family's quality of life? So I think in working together, we can all get better in terms of understanding our clinical treatments and understand how we can have better outcomes that matter to patients and families.

[00:06:54] Erin Spain, MS: Your current study looks at the comparative effectiveness of continued anti-seizure medications versus palliative epilepsy surgery for Lennox Gastaut Syndrome. Tell me about this study and some of the questions you were asking families. 

[00:07:10] Dr. Sandi Lam: We are looking at a question that many families have, which is, do we choose to start another medication or continue with our medical treatments? Or should we think about considering a surgery for epilepsy surgery to try to control the seizures? And this is a major decision point for many families, and this is something that many families want to know. So we are looking across a multi center study, including seven centers for intensive review of records and working with families for surveys to understand outcomes that matter to them. And then looking across 18 centers to see how care is actually being done in the United States and see how we can do better.

[00:08:00] Erin Spain, MS: This type of research is really a collaborative effort, as you've mentioned, with patient advocacy groups. Tell me how important these relationships are with these groups to help move this type of research forward.

[00:08:12] Dr. Sandi Lam: It's essential. Patient families are the ones with the lived experience, and we will never get better in healthcare to actually help improve quality of life or help improve health if we don't work with the people who are living with the disease. People who are closest to the problem, are also the closest to the solution. We need to work together with patients and their families to understand how health care treatments and health care interventions can help patients and families get to the quality of life and the quality of health that they can achieve. Without asking patients and their families what matters to them, we are missing a huge piece of the puzzle.

[00:09:02] Erin Spain, MS: And in fact, in a recent study you just published in Epilepsy and Behavior as an outcome of this project, you were able to gain a snapshot of patient and family perspectives and experiences, and you were able to uncover something very important that points to a potential gap in communication and understanding of patients needs. Tell me about this.

[00:09:24] Dr. Sandi Lam: So this was a survey study working with the Lennox-Gastaut Syndrome Foundation and other engaged pediatric epilepsy patient advocacy groups. The survey was actually trying to get to understanding how patient families see these identified areas of behavior, communication, and quality of life, and understand how important it is to them that their health care provider speak to them about it and, and try to work on this, and then also understand their experience of Is this actually happening in the course of their health care? To set the framework, in a previous survey with the LGS Foundation, many patient families reported significant difficulties with activities of daily living in the children with LGS. So there is a spectrum of physical abilities and other functional abilities in people living with LGS, but to give you an idea, about 40 percent of these patients, uh, are able to walk and about 20 percent are able to use the bathroom by themselves. Other responses that came up were that about 50 percent of these children are not able to speak with words, and about 58 percent need a mobility device, such as a wheelchair. And about half of patients are able to communicate even with people that they know, such as their families who know them very well. So when we're looking at a patient population that have impairments in daily function, it's really important for us to listen to patient families to understand what they're going through as caregivers and what the patients are going through as well as the parents can understand it. In working with the LGS Foundation for this particular study, many families identified that behavior, communication, and quality of life were the most important things to try to understand. That would be what impact on behavior, communication, and quality of life would these surgical or medical treatments have. So in doing that, we designed a survey study together to understand how important it is to families what they experience as happening in the healthcare that they're receiving now. So over 90 percent of patient families reported that behavior, communication, and quality of life are very important to them and very important for them to understand in terms of how the medical or surgical treatments would impact their child with LGS. But only about 50 percent of families reported that they perceived that this was actually being discussed or measured in the health care that they're receiving for their child now. So, this is really just a first step in understanding what is going on in patients lives and, and how their treatments are, and this has so many avenues that we can pursue and we will because we need to do better. This is an important study in that we are learning how to work with patients and families, understanding outcomes that matter to patients and families, and then understanding how we can work through our healthcare encounters and really be a way that we are a resource to families, not only in sickness, but in sickness and in health, and work in a partnership together.

[00:13:27] Erin Spain, MS: Tell me personally, why this is so important to you to really understand and pinpoint the patient's needs and the family's needs?

[00:13:35] Dr. Sandi Lam: That's a great question because sometimes people ask me, you're a neurosurgeon, why do you do this type of research? It actually doesn't matter if we can do great surgeries if we aren't actually getting to the patients and the families that we can help. I believe that we need to try to do more and try to do better to be able to reach the patients and families who are still severely affected by ongoing seizures and by epilepsy and be able to have patients and families understand that there are many treatment options and many ways that we can continue to try to get to better health and better quality of life for patients experiencing epilepsy. And the healthcare system in the U. S. is so difficult to navigate. We have to do better. We can't sit here and watch, and I think that patients and families go through so much when they're sick, especially with epilepsy. Seizures are not something that you can plan for. They're not something that you can control. They're unpredictable and they have far reaching impacts on a child's development, on a child's quality of life, on the parent's quality of life, and really on their communities. Parents have to go through so much to try to keep their children safe and try to keep them healthy and in preliminary findings of a study that we're working on, we see that one in four parents actually report that they have to reduce their work hours or quit their jobs altogether to take care of a child with epilepsy. So epilepsy is far reaching in that a child with seizures actually has a lot of needs, a lot of medical needs, a lot of social needs. And actually, the whole family is impacted in that they have a lot of social and financial challenges. In a survey study done with the LGS Foundation, looking at the parents' experience, over 50 percent of them report financial challenges. In the way that I see it, our surgeries really can help, right? Or our medicines really can help. And if we aren't doing a great job getting everybody the care that they can get and the care that they need, we're really holding patients and their families back from really fully being able to live life and fully being able to participate in the workforce or contribute to their communities. And there's a lot of social isolation and stigma that goes along with epilepsy as well. So I'm passionate about this because I know that we can help. I know that we can help with a variety of surgeries that are safe and effective. And I know that we can help achieve a better quality of life for patients and families, and we aren't getting to everybody who can benefit. Recently, I published a study with my team looking at long term survival and looking at the impact of epilepsy surgery, vagus nerve stimulation, or medical therapy on patients with drug resistant epilepsy in the pediatric population. We found that patients who underwent brain surgery for epilepsy actually were significantly more likely to be alive at 10 years versus those who were on continued medical therapy. The difference was significant, meaning that when we look at over 18,000 pediatric patients with drug resistant epilepsy those who had epilepsy surgery had a 98 percent chance of being alive at 10 years, while those who had vagus nerve stimulation had a 92 percent of being alive at 10 years, and those who were on continued medical therapy had an 89 percent chance of being alive at 10 years. So when you compare that, it means that we really need to try to reach more patients and families to have them understand all of the treatment options that they can look at, and they can choose, and then actually look at the outcomes that matter to them in terms of the lived experience, and if we don't ask, we won't know. 

[00:17:54] Erin Spain, MS: From your research and your interactions with these families, do you think that some of them feel that maybe surgery isn't a good match for their child because they do have the type of epilepsy of which there is no cure? Are there thoughts and feelings like that out there when you're talking about wanting to dispel some myths?

[00:18:12] Dr. Sandi Lam: Absolutely. The field of epilepsy surgery has evolved a lot over the past couple of decades. The technology has evolved, the way that we can diagnose and image the brain has evolved. And the tools with which we can do surgery have evolved, and also the medical devices that we can use to modulate or tell the brain to calm down has evolved. Some decades ago, the thinking for epilepsy surgery was to try to find the area of the brain that was causing the seizures and try to see if it is a safe area of the brain to remove. That was, in the most traditional sense, epilepsy surgery from a few decades ago. Now we have minimally invasive techniques such as endoscopes or lasers that can help achieve good seizure control. 

[00:19:11] Erin Spain, MS: For parents who are listening to this episode, maybe they have a new diagnosis of pediatric epilepsy or they have a child who is suffering from many, many seizures and has not explored all the options out there. What message would you like to send to those parents?

[00:19:26] Dr. Sandi Lam: We're here for you. We won't give up until we can all work together to try to find a cure or a better way to control the seizures and a way to help get a better quality of life and really to help your child reach their full potential. That's what every parent wants. Right. And that's what we want. That's why we're here to treat kids and try to get to all of the kids that we can help. And there are so many ways that we can address pediatric epilepsy now. And the field has gotten better and better. So there are so many tools in the toolbox. There are medications, there are Epilepsy surgery options and we don't think it is okay to have another year lost to continuing to have seizures. So there is urgency to treating children with epilepsy. So getting to the right team who will listen and explore all options, that's important.

[00:20:35] Erin Spain, MS: Well, Dr. Sandy Lam, thank you again for your expertise and sharing this message so thank you so much for your time today.

[00:20:42] Dr. Sandi Lam: Thank you very much.

[00:20:43] Erin Spain, MS: For more information, including how to make a referral or an appointment, visit LurieChildrens.org.