I am an internationally recognized expert in every aspect of transplant-associated thrombotic microangiopathy (TMA), working on both clinical and research applications. My work was instrumental in linking TMA pathophysiology to the complement pathway, which demonstrated the relationship of the disease to specific clinical and genetic factors. Then, I established a new paradigm for therapy with inhibition of the complement pathway.
My ongoing research aims to develop new and better therapies for TMA. Currently, I lead multi-disciplinary international collaborative groups and research projects with clinicians and scientists from Germany, Switzerland, Italy and Japan. I also serve as a clinical consultant for physicians throughout the world regarding care of complex patients with TMA.
In previous work, I led the first prospective TMA biomarker and genetic predisposition studies. My efforts resulted in practical applications for clinical care, as well as comprehensive diagnostic laboratory panels now offered for patients worldwide. I also proposed new diagnostic algorithms and risk stratification data for TMA to help guide timely therapy initiation.
Additionally, I piloted ground-breaking complement blocking therapeutic agents for hematopoietic stem cell transplantation (HSCT) recipients living with severe TMA while also describing unique drug pharmacokinetics in this patient population. As a result of this work, the first prospective early intervention clinical study opened at four major United States pediatric healthcare centers. The study evaluates eculizumab for stem cell transplant recipients with high risk TMA.
My continuing research is supported by the National Institutes of Health (NIH) as well as institutional and industry-sponsored grants.
Bone marrow transplantation and cellular therapies
Thrombotic microangiopathies; organ toxicity prevention in transplant patients
Professor, UC Department of Pediatrics
Bone Marrow Transplantation BMT, Cancer and Blood Diseases
Bone Marrow Transplantation