Payal Desai, MD: How Patient Advocacy Can Shape Sickle Cell Treatment Decisions

Payal Desai, MD

Credit: Atrium Health

In an interview with The Educated Patient, Payal Desai, MD, director of Sickle Cell Enterprise at Levine Cancer Institute, discusses the complex challenges faced by patients with sickle cell disease, emphasizing the variability in symptoms and the need for personalized care. She also underscores the importance of patient advocacy, particularly in navigating treatment options, and recommends using tools like the American Society of Hematology (ASH)'s Sickle Cell Decision Aid to guide discussions with providers.

In your opinion, what are the biggest challenges that patients with sickle cell disease face in managing their condition?

Payal Desai, MD: Sickle cell disease is a highly complex condition—it's not one size fits all. Patients have different genotypes and, consequently, different manifestations of the disease. In medical terms, we say that genotype doesn’t equal phenotype, meaning what their genes encode doesn’t necessarily predict the complications they will experience. Every patient faces unique challenges based on how the disease affects their body, and these challenges often extend beyond the individual to impact their entire community.

For instance, if a patient is frequently hospitalized, it can affect their family, including their children or parents. This broader social impact is significant. Another key challenge that comes up repeatedly, especially in adults, is pain management. Finding optimal pain control is not straightforward, as pain can be acute, chronic or involve a nerve component. There are also factors beyond biology that influence pain, such as stress, depression and anxiety. Research shows that psychological and social stressors can have a direct impact on pain. For example, studies have demonstrated that showing patients stressful images can cause their blood vessels to constrict, leading to physical pain responses. This makes pain management for sickle cell patients a multi-faceted challenge.

Access to care is another major issue, especially for patients transitioning from pediatric to adult medical systems. Finding a consistent medical home where they can receive comprehensive care is often difficult.

How has the landscape of care for these patients changed during your time in the field?

PD: When I started treating sickle cell patients about 15 or 16 years ago, the only available treatments were transfusions, hydroxyurea and supportive care. Since 2017, we've seen significant advancements with five US Food and Drug Administration (FDA)-approved therapies—three new medications and two gene therapy products. Unfortunately, we’ve faced recent challenges with two of those approved therapies, so it's been five steps forward and maybe one or two steps back.

That said, I see a lot of progress and optimism. There’s growing interest in sickle cell disease, not just from a medication perspective but also in terms of improving access and advocating for better care. For example, ASH has a dedicated group focused on sickle cell disease, offering resources for healthcare providers, hosting educational sessions at their annual meetings and pushing for advocacy to improve patient access to care. While the medical advances are important, pain management is just one piece of the puzzle. It takes a dedicated group and a comprehensive approach to truly make a difference.

Speaking of advocacy, how can patients advocate for themselves during discussions about treatment options, especially when considering transplantation?

PD: It’s important for patients to understand the resources available to them, including what therapies and transplant options exist. Having informed discussions about both the benefits and potential complications of treatments, such as bone marrow transplantation, is essential. ASH recently released a Sickle Cell Decision Aid, which is a fantastic resource for patients to use alongside their medical providers. It’s designed to be used as a guide to help patients and doctors discuss the potential impacts of bone marrow transplantation—both the positives and the side effects. This tool provides a comprehensive view and is a great place to start when considering transplantation options with your healthcare team.

This transcript was edited for clarity using AI tools.