Mobilizing a System to Treat People with Sickle Cell Disease

Tell me a little bit about sickle cell disease and the experience of people with the disease.

Sickle cell disease is an inherited disorder—something that you’re born with. In the U.S., we estimate there are about a hundred thousand Americans with the disease, or probably more because we don’t have any registries that keep track, mostly of African descent.

What you inherit is just a tiny, tiny change in your genes—one little spot. So instead of the red blood cell, whose job is to carry oxygen, being shaped like a jelly donut, it’s shaped like a crescent or a banana or a sickle, which is why it’s called sickle cell disease. What that means is it doesn’t deliver oxygen the way it’s supposed to.

Oftentimes, those cells are quite brittle and break open quite easily, and they can be quite sticky. If you think of your blood vessels as pipes, those sickle cells are getting clogged up in there and not letting blood flow like it should. Maybe they are scratching up the sides of the pipe, causing inflammation. Everywhere blood goes, sickle cells go, so sickle cell disease affects every organ in our body.

The hallmark of the disease is unpredictable, severe pain and that’s the thing that mostly affects people’s quality of life. It prevents kids from going to school and adults from going to work. It really just affects their ability to thrive. And that’s against the backdrop of socioeconomic disparity that’s associated with the population that I treat.

Historically, we don’t have a lot of innovative treatments for sickle cell. We’ve been using the same medication to treat it since 1998. When you think about your blood, the fundamental fluid of life, being different, not delivering oxygen, affecting all of your organs, and then the most salient manifestation being invisible, you can imagine how challenging that would be for somebody living with sickle cell.

How did you come to lead the Sickle Cell Disease Program at Yale and what was the challenge that you wanted to address?

I didn’t anticipate having the opportunity to lead the program. I actually got connected with our sickle cell program here at Yale while I was studying in the Yale SOM EMBA program through the Pozen-Commonwealth Fund Fellowship. I was able to meet the leadership here and had an opportunity to take the work I was doing at my previous institution and some of the questions I was asking in the research setting and think about asking those questions here. Then about seven months after coming to Yale as a faculty member, an opportunity arose for me to lead the program.

At that time, there had been a lot of disruption in the program. So my first goal was to stabilize my team and the people around me. But when I think about the program itself, we have four pillars that we think about.

The first is providing evidence-based care to all of our patients. If there are new interventions, we want to be implementing them in clinic quickly.

Second, we want our patients to have access to the most cutting-edge therapies through clinical trials, which finally are starting to ramp up a bit for sickle cell. That’s really exciting.

A third pillar is around community engagement, partnering with the people that we live with in the New Haven community. Because of our location in the hospital, we’re a connection point between the university and the community. A really great hematologist who used to work here who has since passed away, Thomas Duffy, once told me, “Cece, the hospital is the intersection between the university and the community. How we care for patients here says a lot about how we care for the community around us.”

And then the final piece is around workforce and workforce development. For me as a leader, part of the privilege of leadership is being able to develop the people around you. And when I assumed leadership of the program, that was so critical. All the work that we do as a program, all the success that we have, the way that we’re able to deliver on our goals is really based on how we work together as a team. And I believe I have the best team in the hospital. They all know it, and I’ll say it to anybody who listens.

We’re a group that is committed to the patients we serve, on which everybody’s talents are leveraged. Our fellows, who are trainees who will be future sickle cell doctors; our social worker; our psychiatrist; our practice nurse, who really is the first interface with the patients—they’re just great. We have a really comprehensive team, which is necessary for good care of people with sickle cell disease.

If you ask somebody on my team, “What is your job?” we always say, “To keep our patients healthy and safe.” That’s the distillation of it.

What is it about the disease that requires that integrated approach and people from different fields?

One is that the population that we treat has limited options to actually treat the sickle cell disease. Oftentimes that means that we are working hard to address complications related to sickle cell— most notably pain. Sometimes it’s acute, but many of our patients live with chronic pain, especially adults with sickle cell. Over 70% of patients with sickle cell report some daily pain, and that takes a toll.

Maybe even more importantly, sickle cell disease primarily affects people of African descent. So it’s the complexity of being from a population that is marginalized in healthcare. Then we add onto that this layer of socioeconomic disparity that affects how people are able to receive and implement the recommendations that we give them. For example, there’s a lot of stigma around one of the main ways that we treat pain, which is opioids.

When you add up all of those things together, how our patients experience the health system is really complex. What they bring to us as their providers is incredibly complex. So we have to meet that complexity with a variety of experiences.

As a hematologist, I strive to treat the sickle cell disease and the complications related to the sickle cell disease, but I am not a psychiatrist and there’s an upper limit to the work that I can do around mental health support.

Our patients experience challenges with insurance and challenges with finding a home. For example, if you have sickle cell-related pain, one of the things that makes it worse is the cold weather because your blood vessels kind of clamp down; there’s not as much room for those sickle cells to move around. You get more vaso-occlusion; you get pain. If it’s winter and you can’t pay your heating bill, then your home is going to be cold and you’re going to have more pain. So we collaborate as a team to say, what can we do to support patients through this?

It’s really a privilege, because a patient has to be vulnerable enough to share that with us. I have a lot of pride in the work we do because we have created a space where patients feel vulnerable enough and comfortable and safe enough to share a lot of these challenges so that we can partner with them to address it.

Can you reflect on what the particular leadership challenges of this work are? What are the leadership lessons from the Yale SOM MBA program that you’re thinking about over the course of your day?

For me, one of the biggest leadership challenges has been connecting health outcomes with financial outcomes. I always say it’s my job to keep my patients healthy and safe, but how does that show up in a way that’s sustainable within a health system, especially for a population where many patients rely on government insurance and there aren’t drugs with high reimbursement? And how do we understand health equity as a quality outcome, rather than just something that is simply the right thing to do?

Many of my peers across the country face that challenge constantly: how, in conversations with their institutions, to show that providing safe and efficient care for people with sickle cell disease can inform how we care for many of our marginalized populations, and that the solutions we develop for this population can be scaled to others. And then there’s the challenge of translating that into language that resonates with healthcare administrators. I think that’s always a challenge when you care for people with sickle cell.

A lot of that is about cost avoidance—how doing the right thing for marginalized patients can help them move through the healthcare system more efficiently and save money.

One of the other challenges that I think is particularly salient to the work we did in EMBA was around building coalitions. Healthcare can be really siloed, but you really need everybody. It is a team sport. It’s never just a physician or a nurse or a nurse administrator. In order for a program to be successful, a department to be successful, a health system to be successful, you really need all hands on deck. We talked about that a lot in The Workforce course and the Managing Groups and Teams course. Those are two classes that really address building coalitions and aligning stakeholders. It’s not just about coming together, but also about getting on the same page and moving towards a common goal.

Another issue that we covered at SOM is burnout. The healthcare workforce, particularly nursing shortages post COVID, is an ongoing challenge for health system leaders—thinking about how to retain talent and make sure that individuals feel empowered in larger systems. For me in the sickle cell program, nurturing a workforce of providers who treat a patient population that’s stigmatized, that’s poor, that faces bias, comes with its own separate set of challenges.

You’ve done research on burnout among sickle cell disease providers. What were you hoping to learn and what have you found out?

Since the COVID-19 pandemic, it has become apparent to a lot of people outside of the healthcare industry how challenging healthcare delivery can be to an individual. Burnout is a triad: a decreased sense of accomplishment, depersonalization, and emotional exhaustion. It affects 50% of US physicians, and we know that within that group of the doctors who specialize in hematology and oncology, it affects about 45%.

What my colleagues and I wanted to know is how burnout shows up in physicians who care for people with sickle cell disease, particularly because the patient population is poor and faces bias and because there are often fewer providers dedicated to caring for persons with sickle cell, which means these challenges are distributed over a smaller workforce.

So we surveyed HemOnc [hematology-oncology] providers in the United States—those who cared for people with sickle cell and those who didn’t, to see if there was any difference in burnout. And I’m sure it comes as no surprise that physicians who care for people living with sickle cell disease experience more burnout.

But the interesting thing was that when we looked at our data using validated measures, there was no difference in resilience or grit. So it’s not that physicians who don’t care for persons with sickle cell have more grit or they’re more resilient or vice versa; there’s no statistical difference. What that implies to me is that this is not an individual-level challenge, but a systems-level challenge.

Which is exciting to me as a leader. When I think about myself as an individual, as a physician, my job is to provide care to the patient who is in front of me. But as medical director of the sickle cell program, it’s my job to support all of the people who are helping to provide care for the patients. And the privilege of leadership in any organization is that you have the authority and the ability to change, design, redesign, transform organizations—in this case, healthcare systems—to support that workforce and create processes that are equitable. You have the influence and ability to make systems-level change, which is a really cool thing.

We’re doing a second survey that’ll come out soon looking at modifiable factors in burnout for providers who work with sickle cell disease. We hope to pinpoint those systemic-level levers that we can pull to support our providers who work on sickle cell disease. Some of it is salary; usually providers of people living with sickle cell disease are paid less. Some of it is other institutional support. We have a Yale social worker and a practice nurse; not every program has that. I think there’s a lot more to discover there.

And I’m really grateful that American Society of Hematology is also really paying attention to the article now. I had the chance to talk to our Sickle Cell Disease Task Force yesterday about this article, and I think that they will be a great partner in trying to address some of these things as well. I’m excited for what happens next.

New gene therapies have emerged for sickle cell disease. How does that affect the work, and how does it interact with the existing issues of access to care?

Two therapies were approved in December 2023. I remember I was on the flight on the way to the largest annual meeting for hematologists in the world, and the FDA approval came through and everybody was posting about it. I got to the meeting and it was just this buzz. Everybody was just so excited. Our main treatment for sickle cell disease itself, and the one that we have the most data around, hydroxyurea, has been the same since 1998. Imagine the huge leap to jump from an oral medication around since the 1990s to a therapy with curative potential.

That’s not to say that we haven’t had other interventions; bone-marrow transplant is a cure for sickle cell, and it’s been around for quite some time, but it’s fraught with a lot of challenges. So this was really amazing. I also think any awareness that we bring to sickle cell disease is really important, because my patients often say it’s an invisible disease.

After the initial excitement got a chance to settle a little bit, we really had to confront the reality of cost and accessibility. We have a therapy that we know works, but how do we implement it? And it’s very expensive. I am thankful that sickle cell disease was one of the first diseases to be a part of the Cell and Gene Therapy Treatment Act, which allows us to negotiate on a state-by-state basis for coverage for our patients who have government insurance, and that increases access in a way.

But also what we’re learning is that even in the operational flows, there are some significant bottlenecks in being able to take a patient through the entire process. First is identifying the right patient for it. Then, currently gene therapy requires you to get full dose chemo, which is a really big barrier for people of reproductive age because sometimes fertility preservation isn’t covered. It can be quite frightening for patients and impact whether or not they choose to engage.

Gene therapy is relevant and applicable only to a small population. So we have to continue to have that buzz around innovation in sickle cell, but also recognize that we still need interventions for the majority of patients who are living with sickle cell. If any patient expresses interest, we do our due diligence; we talk through it; I send them to Transplant to have meaningful conversations. But ultimately who decides to go forward and who is able to, given financial constraints, operational constraints—it’s a really small group.

How small a percentage of patients is it really an option for?

Our center will probably do less than five a year when we get to maximum capacity. And we have, between our pediatric and adult populations, probably around 650 patients.

We’re on the front edge of this. It’s my hope that we get better over time. When you asked me about my vision for the program, one of the drivers was evidence-based care and another was cutting-edge therapy through clinical trials. It’s my hope that we continue to improve as a scientific community, but it’s important to recognize that we still have some really meaningful steps to accomplish.