September is National Sickle Cell Awareness Month, and we had the opportunity to speak with Dr. Michael DeBaun, MD, MPH, Professor of Pediatrics and Medicine at Vanderbilt University Medical School. He is the founding director of the Vanderbilt-Meharry Center for Excellence in Sickle Cell Disease. Dr. DeBaun was the primary physician author of the Sickle Cell Treatment Act, signed by President Bush into law on Oct. 22, 2004, as Title VII, creating regional networks for enhanced services for children and adults with sickle cell disease. Dr. DeBaun is known as one of the world’s leading physician-scientist in understanding the etiology, pathogenesis, and treatment of strokes in children and adults with sickle cell disease. He is an elected member of the National Academy of Medicine (2009) and recently received the Stanford University Medical School Lifetime Achievement Award (2019).
What progress has been made in the field?
The future is bright for children with sickle cell disease! Children born with the disease in 2020 will likely live to adulthood. Parent education about the disease, medical research, and improved care have combined to ensure children born in this era can look forward to prolonged life expectancy. It’s exciting that effective strategies for treatment have been identified during the past decade, and all of these new treatments are now available in the United States. Newborn screening is required in every state in the US. Thus, we know who has sickle cell disease, and we can start education and therapy to prevent life-threatening complications before 4 months of age. With intervention, the parents can be educated about the importance of evidence-based strategies to prevent death, strokes, and other complications
What is the biggest barrier in providing health care to children and adults with sickle cell disease?
The most significant barrier to providing health care to our families is access to health care for prevention and treatment. As a case in point, just 25 years ago, approximately 11% of all children born with sickle cell disease had strokes before their 18th birthday, but in 2020 with appropriate screening and preventive therapy, less than 1% of the children will have a stroke. However, only about 50% of the children are screened and receive treatment to prevent strokes.
Unfortunately, adults with the disease have not been the beneficiaries of medical advancements. Adults with sickle cell disease have a myriad of challenges, including the scarcity of specialists, the complexity of their medical care, and the high proportion of having public health insurance (Medicaid), which limits their access to comprehensive medical care. Many of the adults with sickle cell disease receive their medical care in emergency rooms because of the absence of primary care providers and specialists willing to take their insurance.
How has COVID-19 affected your work?
Preliminary data indicates, children and adults with sickle cell disease have a three to five times higher rate of dying from COVID-19. In early March of 2020, our Sickle Cell Disease Center of Excellence worked hard to become early adopters of telemedicine for our children and adults in families with sickle cell disease in our Center of Excellence. Thanks to the generous donation from a former St. Louisan, we made sure that our families had new iPads, and requisite technology for telemedicine visits. Notably, telemedicine has accelerated access to medical care for our families. Rather than parents and adults with the disease having to make difficult choices with competing demands of their time, they have much more flexibility. Telemedicine is decreasing the health care disparity for our families and decreasing difficult decisions, such as, “do I take my child to see their sickle cell doctor or take a vacation day from work?”
As part of our Center of Excellence’s philosophy that knowledge is a powerful tool in healthcare, I co-chaired with an adult advocate for sickle cell disease, weekly town hall meetings with our families for 10 weeks, from mid-March through May. The focus was to inform our families about the changing landscape of preventing SARS-CoV-2 infection. We continuously provided evidence-based information about the virus on the Tennessee Sickle Cell Disease Foundation’s Facebook page. The end result of this effort, that six months later, we have very few cases of SARS-CoV-2 infection and no related deaths.
Tell us about your most recent exciting initiatives.
Nine years ago, I traveled with my wife, Sandra, and or daughter Morgan to Nigeria, where 150,000 children with sickle cell anemia are born per year versus approximately 1400 per year in the US. My wife and daughter put on a mini “Camp Crescent,” for girls with sickle cell anemia. During the weekend, they orchestrated shared fun activities, while I presented an educational workshop for 40 parents of the girls. Unexpectantly, based on the interaction with the parents, I identified 6 of 42 girls with strokes and explained the implications to the family. We were all devastated because of the lack of awareness, coupled with the absence of any stroke prevention strategies specific to Africa, where 75% of the children with sickle cell disease are born. Unfortunately, the treatment to prevent strokes in children with SCA in the US did not apply in Africa.
To address the challenge, over the last 10 years, I have had the privilege of leading two National Institute of Health-funded and one foundation-funded stroke prevention trials in children with sickle cell anemia living in northern Nigeria. This year, we finished all three trials. The effort was a tremendous success. Not only did we demonstrate that we can prevent strokes, but we established a new paradigm of healthcare for the region that can be replicated in other African countries. Potentially our findings could be applied to enhance stroke prevention strategies in the US.
Importantly, after completion of the three-stroke prevention trials, the public health officials of three state governments in northern Nigeria, where 40,000 children with sickle cell anemia live, have agreed to provide free of charge the necessary treatment and infrastructure to prevent strokes. Rarely do you experience immediate government implementation of successful research results in the US or elsewhere in the world. The rapid implementation of our findings was not by accident. While a member of the Washington University School of Medicine faculty at St. Louis Children’s Hospital, from 1996-2010, I learned valuable lessons working with Missouri state representative Paula Carter from north St. Louis and US Senator Jim Talent of Missouri. Representative Carter and Senator Talent taught me how working with the local and federal government can facilitate sustainable government policies and improve clinical care of individuals with sickle cell anemia.
What is on the horizon for your next initiative?
A close friend, Dr. Adetola Kassim, the Director of the Adult Bone Marrow Transplant Program at Vanderbilt University Medical Center, and I co-chair the Vanderbilt Global Haploidentical Transplant Learning Collaborative to cure children and adults with sickle cell disease. We receive philanthropy funding from a Nigerian family, but 90% of the effort is the volunteer work of physicians, nurses, and coordinators from eight countries – Germany, Netherlands, Brazil, UK, Nigeria, India, and Saudi Arabia – to work as one team. Despite the absence of grant funding, and the different time-zones, we meet weekly to discuss the care of the children and adults being treated on our study protocols to cure sickle cell disease. Our preliminary results are promising, particularly for adults. We have cured over 30 children and adults with sickle cell disease, but we have had some setbacks. The knowledge that we can and will do better in the immediate future for children and adults with sickle cell disease is the team’s greatest motivation to work hard as one across many countries. I am honored to be part of this effort.
You have received two international awards for mentoring medical students, physician-scientists in the United States, Ghana, and Nigeria; one award was from the Society of Pediatric Research and the second from the American Society of Hematology. Why do you spend your time mentoring?
Growing up in University City, my family were life-long educators and coaches of youth. My grandfather, Henry Rutledge, was a teacher in the St. Louis Public School system, as well as my mother Everlouis (Maryville Class of 58’ and the first African American to graduate from Maryville), and my aunt and uncle (Doris and Jimmie Irons), were teachers and lived four houses away during my childhood. My father, Frank, was a coach for multiple little league teams in University City. My wife has a Master of Arts in Teaching and augmented our children’s education with home-lessons. My brother, Mark, is a teacher in Prince Georges County Public School System, and my sister, Michelle, was hired to be a teacher in the Normandy School system before her premature death in 1996. Our two adult children, Malcolm and Morgan, as part of their professional responsibilities, are both mentors. If I did not teach and mentor, I would not be honoring our family code to ‘lift as you climb.’