Description
Clinician-scientist sets sickle cell research agenda by engaging with the community, listening to families and patients
More than 30 years ago, the NHLBI funded the Stroke Prevention Trial in Sickle Cell Anemia (STOP) trial, which found that patients at high risk for stroke could be identified using trans-cranial Doppler (TCD) ultrasound, and that the incidence of stroke could be reduced by 90 percent in those children by periodic blood transfusion for at least 36 months. That important study began the NHLBI’s long legacy of transformative research into sickle cell disease (SCD), an area of research that remains a top priority for the institute today.
The importance of finding ways to mitigate the side effects and discovering potential cures for this rare disease – which affects 90,000-100,000 people in the U.S. – is one reason I recently spoke with clinician-scientist Dr. Michael DeBaun, professor of pediatrics and medicine at the Vanderbilt University School of Medicine and director of the Vanderbilt-Meharry Center for Excellence in Sickle Cell Disease. I also chose to speak with him because his work is so transformative, both in terms of what he is finding and how he goes about performing it, as you’ll see and hear below.
Dr. DeBaun’s research (literally) spans the globe and delves into topics ranging from the impact of silent infarcts on a child’s full-scale IQ and academic performance to connections among sickle cell, sleep, and asthma to the use of hydroxyurea in Nigerian children with SCD. His research priorities are often set by his interactions in the clinic, not the lab.
As the field of health disparities research grows, Dr. DeBaun’s intriguing work on the roles of poverty, parenting style, violence, and parental stress in relation to full-scale IQ in children with sickle cell disease will undoubtedly play a pivotal role. As Dr. DeBaun pointed out, it will become increasingly important that researchers become as sensitive to the social determinants of disease and outcomes as they are to the biological determinants of disease. The two factors are intertwined in a dynamic interplay that has important implications for defining more effective preventive interventions that enhance the well-being of patients with SCD.
His findings also could help other scientists move toward conducting research into rare diseases like SCD using the more “holistic” systems approach that typically has been applied to more “common” racial disparity disorders such as hypertension, asthma, and diabetes.
“Every R01 or foundation grant that I’ve received has been based on listening to [families] talk to me about the disease.”—Dr. DeBaun
Dr. DeBaun’s realization of the role of social determinants of disease undoubtedly is due, in part, to the fact that at two of the major locations where he has practiced and undertaken research – St. Louis and Nashville – 60-70 percent of the children with sickle cell disease that he’s worked with are on Medicaid.
His admirable dedication to these patients and his willingness to listen to their families has not only affected what he researches, but how he does that research. Or, more specifically, where he does that research. As a result, he is a wonderful example of a clinician-scientist who has integrated science and service to those with the greatest need by extending his reach beyond the laboratory and the clinic to touch the communities where his patients live, learn, play and pray.
Dr. DeBaun is a testament to the invaluable role that the clinician-scientist can play in moving medical and scientific research forward. His research into the use of hydroxyurea to help prevent stroke in children with sickle cell disease holds the promise of improving quality of life for thousands of children. His work into the social determinants of disease could change how doctors treat children with SCD who have suffered from stroke.
It is exciting to consider the possibilities of developing innovative family-based interventions that enhance the health literacy, resilience and child development skills of parents as an effective means of enhancing the efficacy of drug therapies and transfusions in changing the natural history of cognitive dysfunction in children with SCD. It is apparent from the Debaun research portfolio that by listening closely to the concerns of families and engaging patient’s within their social context, his work is making a difference – daily – to those in his care..
I look forward to Dr. DeBaun’s continued involvement with the NHLBI and to his voice being part of a national dialogue that the NHLBI plans to undertake with the sickle cell community to help advance important public health efforts for SCD patients everywhere.