What support can we offer our patients with Sickle Cell Disease?
The biggest support that we can offer our patients with Sickle Cell Disease is Sickle Cell specific education to bring awareness of the disease process. Persons living with Sickle Cell have historically been misinformed about the disease process from healthcare professionals and myths in the community in general. With this knowledge in hand, patients can understand why taking disease modifying therapy, such as Hydroxyurea, reduces end-organ damage and mortality. The Sickle Cell Program at YNHH offers not only medical care but supportive care that is led by a team of experts in Sickle Cell Disease, including mental health support, chaplain services, and social work services.
What advances have made the biggest impact in the treatment of patients with Sickle Cell Disease in the last five years?
The biggest advancements in Sickle Cell have been in understanding the disease process. It was once thought that Sickle Cell Disease simply meant sickled cells "getting stuck" in the blood vessels and causing damage. We now know that Sickle Cell Disease is a multisystem disorder that is medically complex with excessive chronic inflammation. This involves inflammatory cytokines, the innate and adaptive immune systems, and endothelial dysfunction that leads to damage in any and every organ. With this background, investigators have now been able to study targeted disease modifying therapies. Allogeneic stem cell transplant is the only cure for Sickle Cell Disease. However, it is only available to a select few for many years, including limited by available donors. Gene therapy is currently in clinical trials but may soon be available. This is also limited to a select few due to the strict criteria of these trials. When it does become available it is unknown which insurers will cover this therapy, especially with ~70% of persons with Sickle Cell Disease receiving government medical insurance. Therefore, it is necessary to continue to develop disease modifying therapies that are available to the majority of persons living with sickle cell disease in the U.S.
What inspires you to work as a hematologist?
I have had a love of hematology since my first rotation on the hematology service as a resident. There is so much biochemistry involved. Specifically, with regards to Sickle Cell Disease, I am inspired by my patients. We learn from one another. As a hematologist, educating providers and patients is my passion. It is critical to decreasing mortality and improving overall healthcare outcomes. On the other hand, one of the key components to education, evidenced-based practice, is lacking when it comes to Sickle Cell Disease care. Education and research are inseparable. A theme that has emerged from my education research is that the paucity of data to guide management makes it less desirable to care for patients with Sickle Cell Disease.