Focus on individualized patient care for cardiovascular disease and the future of homozygous familial hypercholesterolemia treatment

Carissa Baker-Smith, MD, MPH, associate professor of pediatrics and cardiology at Nemours Children’s Hospital in Wilmington, Delaware, discusses tailoring lifestyle recommendations based on individual patient factors and how screening can save a life.

Question:

You are recognized for your preventive care and work with pediatric patients who are at risk for cardiovascular disease. You also talk about the importance of individualized care. What approach do you take with patient management when you care for pediatric patients facing these cardiovascular disease risks?

Carissa M. Baker-Smith, MD, MPH:

I think every family is unique. I tend to start with the American Heart Life’s Essential Eight, which is the health factors and health behaviors. There’s 4 health factors, that’s your body mass index and blood pressure, cholesterol, blood sugar. We focus on those things. Then there’s the health behaviors. There’s your exercise and your diet, your sleep, making sure you don’t smoke or vape. We target those things and everybody’s a little different. You may live in an environment where you can’t exercise, so we come up with solutions for how you can exercise, and maybe it’s in-home dancing, but it’s something to keep you active. Then we focus on diet. Not everybody lives in an area where they have access to fresh fruits and vegetables, so you’re trying to figure out ways to improve access. We do that both clinically and then we’re trying to help families in other ways to achieve those goals.

When it comes to something like cholesterol management, blood pressure management, I do want to hear from patients and kids and from the parents. Make the effort to describe to them why it’s important to treat what their treatment options are. Then it’s a team. Then we decide sometimes there are not a ton of options. I say that like, “Look, you have high blood pressure. We have to treat your high blood pressure. We can try starting with this, lifestyle, but if that doesn’t work, there are these classes of medication that we may need to use.” You do try to individualize and try to give families and patients the opportunity to participate in the decision-making, but make them aware of what the risks are if untreated, and that’s important as well. Then recognizing that in terms of health equity, we don’t all have access to the same things or we may not all choose the same things.

How do we cater an intervention strategy in a way that’s suitable for the person? If you tell me you want to wear shoes, but you don’t want to wear high heels, well, I’m not going to give you a pair like Jimmy Choo’s, right? I’m going to maybe give you some tennis shoes or something that’s more comfortable for you. It’s in a way of making the solution something that’s feasible for families, something that’s acceptable, something that they can adopt and reinforcing the message of why it’s important to carry out, whether it’s a treatment or a lifestyle change.

Question:

What are some areas of unmet need and challenges as it relates to the treatment and management of the homozygous familial hypercholesterolemia patient population? Are there any areas of research that you are particularly interested in?

Carissa M. Baker-Smith, MD, MPH:

I really got involved with homozygous FH, and like I said, this trial really to give my patients a better quality of life. I saw what they were going through in terms of this weekly apheresis just being a tween or a teenager and having to deal with a child dealing with this. That’s how I got involved. I am excited that we do have these other medications on the market that can help to improve control of the cholesterol and in a way that doesn’t require our traditional therapies at least as often. Like I said, having something like evinacumab or other medications that can lower the LDL 50% or more and give individuals a longer period of time with control so that they don’t have to get that lipid apheresis so often.

I think the biggest challenge though that we have is also just recognizing we can’t hide from these diagnoses, and we do need to make sure that we’re advocating for screening and are working very closely with our primary care providers to say, “Look, we understand that lipid screening is something that maybe people feel uncomfortable ordering because they don’t know what to do with the results. Well, let’s help guide that a little bit.” We are doing some things within an Nemours health system to help improve recognition of lipid disorders and what to do and how to manage within even the primary care setting. I think that’s also going to be happening more at the national level, getting that word out.

But if I had to make one pitch, it’s don’t forget the importance of screening. Don’t forget the importance of testing for these conditions because you really can save a life. Usually, it’s not just 1 life. Usually what happens is that once we’ve diagnosed a child, we can screen the parents, and I’ll tell you, I’ve screened… We’ve recognized disease, identified disease in entire families and have helped entire families. I do think we’re going to see additional treatment options that are already coming into development that are going to be game changers in terms of the frequency with which people have to receive a medication to control their cholesterol and maybe some alternatives to some of the traditional therapeutic approaches that we’ve relied on because we didn’t have better ones.