Overview
As a physician scientist and clinical innovator in pediatric hematopoietic cellular therapy (HCT), my primary focus of investigation is early diseases of endothelial dysfunction—primarily transplant associated thrombotic microangiopathy (TA-TMA), sinusoidal obstructive syndrome (SOS), and diffuse alveolar hemorrhage (DAH). All diseases are life threatening post HCT, with little known about the mechanisms, drivers of disease, and best treatments. Utilizing multiple research methods, my overarching goal of my investigation is to improve mortality in children with endothelial disorders by facilitating prompt diagnosis and investigating new therapies.
With the aid of multiple collaborators, we are working on identifying key pathways of these diseases, early prognostic biomarkers, and novel therapeutic targets. In addition to this translational work, I am a principal investigator of multiple interventional clinical trials testing the safety and efficacy of new agents in these diseases.
Current Projects
- Identifying novel blood and imaging biomarkers of TA-TMA that aid in diagnosis, prognosis and detection of subclinical disease
- A prospective cohort study of TA-TMA after implementation of screening guidelines identifying risk factors for developing TA-TMA and increased transplant related mortality
- Determining the cost associated with TA-TMA and the cost effectiveness of Eculizumab
- Novel interventional therapeutic agents for TA-TMA