The majority of the 100,000 Americans who suffer from Sickle Cell Disease, an inherited blood disorder, are diagnosed at birth. Patients with Sickle Cell Disease (SCD) grow up spending far too much time in hospitals and emergency rooms, debilitated by the severe pain that typifies the disease. Newly approved drugs, like crizanlizumab a monoclonal antibody medication that reduces pain crises from reduced blood flow caused by SCD, are helping. Patient pain is much easier to manage at home and patients find they can once again participate in their daily family and work activities. Patients who respond may not be in the hospital again for years, which is a stark contrast to 45 years ago when children with SCD usually died from infections before age five.
That statistic dramatically improved after two innovations in the 1980s and 1990s: daily doses of penicillin for young children with SCD, and vaccination against pneumococcus. However, people still die prematurely—between 45 and 55 in the United States, decades short of normal life expectancy. Several other drugs, like crizanlizumab, are available to help patients with SCD manage their pain. And although the only current cure for SCD is a bone marrow transplant from a sibling who is also a complete genetic match, other curative innovations are on the horizon.
Contributing to those advances in care is Cecelia Calhoun, MD, MPHS, MBA, Assistant Professor of Medicine and of Pediatrics, and Director of Smilow Cancer Hospital’s Sickle Cell Program. The program is designed to benefit pediatric, young adult, and adult patients in need of consistent services and a streamlined approach to the care of this often-misunderstood disease. There are about 200 adults and 200 children being seen through the program, and inconsistent episodic care in the emergency department (ED) or hospital has been replaced with long-term outpatient care. The programs are staffed with advanced practice professionals experienced in the care of patients with SCD and opioid use, and ED visits and hospitalizations have been reduced dramatically. To help manage the social and psychological issues that accompany an incurable disease and constant pain, both programs include access to social workers and a psychiatrist.
“Sickle Cell Disease is relatively rare, so providers encounter it infrequently and clinics dedicated to it are uncommon,” said Dr. Calhoun who joined Yale a year ago. “Part of the gift of being here is that people are really excited about ensuring our hospital is welcoming to patients with sickle cell disease.” The main symptom of SCD is pain, both chronic and acute, and the best treatment is opioids, typically administered in an emergency department. Inevitably, a small percentage of patients become addicted, which has led to unjust stigmatization of the disease.
The misunderstandings are exacerbated by two related issues—race and poverty. In the United States, most patients are Black; most are also poor. The consequences are discrimination and poor access to healthcare. At Smilow, 80 percent of SCD patients are on Medicaid, a reimbursement category avoided by many healthcare institutions. Dedicated care for younger patients diagnosed with SCD hopes to intervene early in care, before patients become overwhelmed or lost within the healthcare system.
“Many patients avoid the hospital, afraid they will be labeled as drug-seekers, even though their pain is very real and intense,” said Dr. Calhoun. “Taking responsibility for one’s healthcare is challenging for any young adult, without the added complication of a chronic disease. We need to get them excited about their future while they’re young. They can have a full life. We can’t control things outside of the hospital, but we can reach out and wrap our arms around them to help them through those things at a pivotal time. It’s a life’s work for me.”
A large part of Dr. Calhoun’s role is to help smoothly transition adolescent patients into the adult care setting. Her research efforts are focused on the relationship between health literacy, self-management, and cognition in adolescent and young adult patients. Dr. Calhoun is confident that the personal relationships built between Yale’s SCD providers and patients have formed the trust necessary to enroll participants in trials, which is critical to advancing care for these patients. She wants to expand that relationship into the community, to educate people about SCD.
“Awareness is such a critical part of making things better,” she said, “especially for young people trying to fit in, get jobs, or apply for college. As we get new therapies and we learn how to treat sickle cell better, it’s important for them to be able to say, ‘Yeah, I have sickle cell disease and sometimes I have bad pain but I’m still able to do what I want.’ How empowering is that? It’s not an overnight thing. It takes a consistent and continuous investment, but it has tremendous returns. When you can help people stay alive, what better return is there?”