The Dose - One Doctors Approach to Treating People with Sickle Cell Disease

Episode Date: September 20, 2019

Sickle cell disease is a life-threatening blood disorder that affects around 100,000 Americans, mostly African Americans. In addition to coping with the disease itself, many face a host of other challenges, like getting to the hospital for treatment, paying bills, and figuring out where their next meal will come from. On the latest episode of The Dose, Dr. Cecelia Calhoun talks about her work helping children and young adults with sickle cell cope with these issues day to day. Calhoun is one of the first experts to receive the Pozen-Commonwealth Fund Fellowship at Yale University to advance her work on health disparities.

The Dose is a production of the Commonwealth Fund, a foundation dedicated to affordable, high-quality health care for everyone. When I think about the patients that I see now, one is a young lady who is finishing up her sophomore year at UMSL, which is a university here in Missouri, and she's incredibly hardworking. She's pre-med and wants to be a doctor. She also works full-time, and she has sickle cell disease. And what that means for her as a student is that in addition to her coursework and to working her job,

that she comes to clinic every six to eight weeks to get an automated red cell exchange. What does that do to her life? And also, how does that make her feel physically, emotionally? It must be draining. Exactly. And so this particular treatment involves a lot of prep work. So she has to come and get labs one day to make sure that we have everything properly arranged and calculated for the red cell exchange. And then she comes for the treatment, which is patients kind of get hooked up to, it looks like a dialysis machine where you basically filter out their entire blood volume. So that takes a while too. And then we replace kind of some of the volume that we took away. Hi, everyone. Welcome to The Dose.

On the next three episodes, we're going to be talking about disparities in healthcare. You just heard from Cecilia Calhoun. Cecilia is a pediatric and young adult hematologist at Washington University in St. Louis. She treats patients who have sickle cell disease, which is a life-threatening blood disorder. Sickle cell disease affects around 100,000 Americans, mostly African Americans. Cecilia's work, helping children and young adults with sickle cell disease, shows how complex the connections are between health and socioeconomic factors. In 2018, she became one of the first experts to receive the Posen Commonwealth Fund Fellowship in Minority Health at Yale University. Maybe let's take a minute to cover some of the basics. Very simply,

what is sickle cell disease? Sickle cell disease is an inherited disorder where your red blood cells are misshapen and deformed, or they're shaped like a banana or a sickle. And in addition to being shaped differently, they're also quite sticky and quite rigid. And so what this means is that if our red blood cells are pipes, we can imagine a normal red blood cell flowing through the pipes, passing each other, swimming freely around in our plasma, making it to their final destination and delivering oxygen to our tissues. But with sickle cell disease, as those rigid, hard cells pass through those pipes, they're scratching up the side of those pipes, they're scratching up the side of those pipes. They're sticking to the sides of them, and they're sticking to one another, and they cause blockages.

And so if blood isn't able to get to where it needs to, that tissue doesn't get oxygen, and it kind of dies a little bit. So all the clinical manifestations that we see, and so most commonly that's pain, but also it can affect pretty much every organ in our body. So our lungs, our eyes, specifically our brain. And when I say our brain, I mean really, really bad strokes. And how does this young woman remind you of yourself as someone who was also young and also wanted to be a doctor? Yeah, so much like her, I didn't necessarily come from the rosiest background. You know, I grew up on the east side of Detroit and there was some socioeconomic disparity, but I had really dedicated parents and I had a support team. So I hope to be part of this young woman's support team. You know, I want to be one of the people that helps

her achieve her dreams because for me, that was absolutely essential. Where does your patient live? Where do you care for her? She lives in North County, which is a St. Louis suburb, which is predominantly African American. Our hospital is in downtown, I would say, sort of downtown. Is it difficult for her to get there for her treatment? So she actually takes public transportation. She takes the bus. And the reason that that's difficult is because of the time commitment, the irregularities, excuse me, irregularities of the bus schedule, and the fact that it just takes so long. And it's, that's

something that's not unique to my patients. You know, I think when I think about the two biggest logistical challenges that the adolescents and young adults with sickle cell disease that I see face, number one is insurance coverage, and number two is transportation. So she takes the public transportation to get here. I'm not exactly sure how many buses, but I know that it's over two. I know that it involves a transfer. And, you know, if it's a day like today when it's 90 degrees and she's still trying to get homework done and make it to work, you know, if it's a day like today when it's 90 degrees and she's still trying to get homework done and make it to work, you know, it can definitely be a challenge as I'm sure you can imagine. I can only imagine. And then after treatment, again, I'm sure it's,

she probably feels physically depleted and then she has to go back on a bus, transfer to another bus. That just sounds exhausting. And so making the time and also the emotional energy to go through something like this is really, really incredible. And as you say, it's really important that she's diligent about coming in to get this treatment so that she can manage her disease. I'm so, so incredibly proud of her because she's able to master this. And it's a challenge that I don't think that many people could, and certainly not that many of my young adults can do. Well, let's talk about one of those patients then. Yeah, so I would say most of the patients I see, they want to do the best for themselves. They want to live and thrive, but they really are just struggling to survive because of the circumstances in which they grew up.

So there's one patient in particular I think about who he was cared for at our hospital as a child, but subsequently got caught up in the criminal justice system and came to me when he was 19 to reestablish care. But he faces several social challenges in that he has almost no family support. He often is homeless. He has limited access to his medications. What happened? How did he lose his family support? How did he get caught up in the criminal justice system? He comes from a single parent home, and his mother had to work to support his family.

And so he was left alone a lot. And at a very young age, in an attempt to fit in, and we've talked about this very candidly, you know, he got arrested for car theft and then burglary. And he was about 16, I think, when that happened. And many of his, like, youth years have been kind of sucked up. So it's been a little bit of a cycle, you know. Even if you're young, immature, and you make a mistake, once you enter the criminal justice system, there's a certain stigma around that. And he hasn't really been able to escape that. How does somebody like that have the time and the ability to manage a disease that requires several hours every six weeks? So the short answer is I don't think they do. And that's one of the big problems when we think about how to support our young people with sickle cell disease. This is a population that's truly

affected by social determinants of health. So the things outside of the hospital, things external to the health care system that challenge the way that they're able to manage themselves. So if you are just trying to figure out how to pay your bills or how you're going to get from point A to point B or what your next meal is going to come from, it's going to be a challenge for you until you have bad pain to think about, hey, maybe I should go see my hematologist. Or, hey, I know that I have this treatment scheduled, but I don't really have a way to get there, and I have $5 as my whole entire food budget for the rest of the week. I think it's one of the areas that we really, really need to work on,

commit to, and find solutions to if we really want to help children and young adults and adolescents with sickle cell disease not just survive but thrive. And obviously this is something that you want to do but tell me a little bit about the beginning and how you got interested in studying sickle cell disease and also in treating patients with it? Yeah, so when I went to med school, I actually thought that I wanted to be a surgeon. And during your latter years in school, you do clinical rotations. And my favorite rotation was on the pediatric floor, which also happened to be the hematology and oncology floor.

But also, that was the first time that I actually got to care in some way for a patient with sickle cell disease. And it was the first time that I saw the deleterious consequences of sickle cell disease when I saw an eight-year-old girl who had had an overt stroke, and she couldn't move her entire left side. Through caring for patients with sickle cell, I realized the huge gap in transition as we help them move to the next phase of their life and receive adult care. And why isn't that a seamless process to begin with? So in a general sense, the transition from pediatric to adult care is a major issue for many disease processes. But when we think about special populations like children with type 1 diabetes or children with congenital heart disease or the children that I care for, sickle cell disease, it becomes even more complicated.

It's when the more insidious disease processes are becoming more apparent. You're going through puberty and your body is changing and the effects in your disease are greater. There may be a change in your home environment, certainly a change in resources like your insurance coverage, and changes in your academic environment. And then we also have to make sure that that process from going to the pediatric setting to the adult setting is clear, that there's good communication between providers, and that there are adult providers that are available to care for these patients. So it's pretty complex. And patients with sickle cell disease who, like I said before, are really affected by social determinants of health and stigma, that process becomes a lot more complicated.

So as I'm thinking through this idea of transitions, now children can stay on their parents' health insurance until they're 26 years old. But what happens to someone whose parents are uninsured? How are they getting care for sickle cell disease? So in the pediatric population, certainly here at our hospital, the majority of the children that we treat rely on government insurance. And so as these patients get older, either they don't meet criteria for continued government insurance or they don't know how to access it once it's fallen off. And that's a huge, huge barrier for the patients that I treat. It's almost like a, it's like a cliff. You go one day from being able to get care and then suddenly you're not able to have

that anymore. That's exactly it. And it's when they fall off, if they don't have a transition, that that's when the morbidity and mortality really starts to rise. And that's what the data shows us. What I really want to do is figure out how we as a healthcare system make that a bridge from pediatric care to adult care. And so my current work focuses on the design of structured transition programs so that hospitals can bridge that gap between pediatric and adult care. And do you have any stories about patients whom you have seen and have had a successful or smooth transition from pediatric to adult care?

So the first question I always ask a patient when I see them is, when you tell your friends you have sickle cell disease, how do you explain it to them? Because that tells me, one, if they're talking to people about sickle cell, because some of them aren't. And it also helps me understand how I can support them. When I think of kind of the extreme of this situation, I had one patient who came to me a little over a year ago. When I first saw him in clinic, we went through his 17 medications and I asked him which one he was taking and he was like, my pain medications. And we talked about how this is the only medication

that's proven to help his life last longer. We talked about how he can manage his own pain at home. We talked about how to call us and use us as a resource. I started seeing him weekly to optimize his care. And now he hasn't been hospitalized in months. And he wants his own apartment and he wants to drive and he wants to date. And now he's really able to kind of move towards those things because he's not living moment to moment wondering if he's going to be in the hospital. And that's amazing that you're able to get him up to this point.

Maybe what we should also talk about is treatment and cure. So you said that there's only one cure, and that's bone marrow transplants. But most patients don't have access to this. Yeah. So bone marrow transplant effectively is when you take stem cells from one person and you put them into another person and allow them to replicate. But that is a gross oversimplification. So for sickle cell disease and transplant, it's incredibly complicated because, number one, it's very challenging to find a match. Because African-Americans are such a heterogeneous population and a mix of different genes and DNA, it's hard to find someone that would be an exact match. And also because there are really not enough people of color on the bone marrow transplant registry, that places a huge challenge. And then also it's a very expensive procedure. The other treatment that

I've read a little bit about is gene therapy, but it seems like this isn't really being widely used. Can you talk about why? Yeah, so actually the promise of gene therapy is pretty exciting. One of the main challenges of bone marrow transplant is that it's really challenging for people to find a match. Gene therapy removes the need for you to find a match because with gene therapy, you're a match to yourself. Basically, what happens is your own stem cells are harvested. And outside of your body, your DNA is through a special process called CRISPR is modified so that you don't have sickle cell disease. And then you give it back to the person

and let those new cells kind of grow within their home space. And it's a very, very exciting therapy, but it's very new. And so it will take time and experience before it becomes widely available to patients. And I imagine it's also pretty expensive, right? Just like bone marrow transplant and even more so, this is using cutting edge technology to do these kinds of genetic manipulations. And so it is quite expensive and whether or not insurance companies are going to cover it, because certainly like we talked about, this is not a population that can afford very expensive medical procedures. This is pessimistic, but I can't help but think, given that sickle cell disease predominantly affects African-Americans, that it's not really a national priority to, one, do more research into how to treat it, but then to make sure that the treatment is widely accessible to more people. I think you're absolutely right. I think that really

what sickle cell disease represents is opportunity, opportunity for us to do better as a scientific community, opportunity for us to do better as healthcare providers and as a healthcare system, but also as a society. And I think that as we look at the challenges that patients with sickle cell disease face, if we are able to kick it in gear and make moves in this population, I think it is an example of what we can do overall. Well, I think that's the right note to end on then. So thank you so much for agreeing to be on the show. Thank you.

The Dose is hosted by me, Shanwar Sirvai. Our sound engineer is Joshua Tallman. We produced this show for the Commonwealth Fund with editorial support from Barry Scholl and design support from Jen Wilson. Special thanks to our team at the Commonwealth Fund. Our theme music is Arizona Moon by Blue Dot Sessions. Additional music by Pottington Bear.

Our website is thedose.show. There you'll find show notes and other resources. That's it for The Dose. Thanks for listening.