Caitlyn Duffy, MD, MPH

In high-resource settings, the survival rate for childhood leukemia is upwards of 90%. Conversely, survival rates are drastically lower for those living in low- and middle-income countries. Much of this can be attributed to a failure to translate the science into usable treatment strategies that are tailored to the level of care of local healthcare environments. I use implementation science to address this issue. My approach takes the rigor used to develop scientific knowledge and applies it to the delivery of that knowledge so we can create meaningful impact for pediatric cancer patients globally.  

As we advance our understanding of the biology driving pediatric cancers and catastrophic diseases, we create more effective treatments and increase survival rates for the children of St. Jude. However, most children with cancer live in low- and middle-income countries, and they often do not have the same access to care compared to children living in high-income countries. Part of my work focuses on guideline implementation – taking the best of what we know and leveraging that information to create resource-adapted guidelines in a global capacity. I am also interested in how these guidelines are integrated into patient care as well as identifying reasons that may thwart that integration. 

Novel treatment strategies, such as immunotherapies like blinatumomab and chimeric antigen receptor CAR-T cells, have transformed how we treat leukemia and lymphoma in our pediatric patients. For example, blinatumomab is a bispecific T-cell engager (BiTE) cancer treatment that targets CD19 surface antigens on B cells. It couples a patient’s own T cells to kill B-cell leukemia. This therapy is FDA approved and has increased survival rates as well as lessened cytotoxic effects and adverse effects compared to chemotherapeutic treatment strategies. BiTE therapy has revolutionized the way we think about treating blood cancers and has the potential to make enormous impact in low-resource settings where the entire suite of treatment options—like transplantation—is not feasible. I am focused on creating resource-adapted guidance to make sure novel, effective treatments like BiTE therapy are readily available on a global scale. Futhermore, I aim to extend these lessons to translate new therapies like CAR-T cells as the standard of care evolves.

Just as we continue to lean on new discoveries and technologies to advance the standard of care, we must also lean on implementation science. I am interested in early integration of implementation science into the clinical research continuum to create a better understanding of how to integrate evidence-based practices and policies into drug design and clinical trials.