My clinical efforts focus on pediatric hematopoietic stem cell transplantation (HSCT), with a specific emphasis on HSCT for patients with inherited and acquired bone marrow failure. Through my combined clinical activities in the Bone Marrow Failure Center within the Division of Hematology and the Blood and Marrow Transplant Section within the Division of Oncology, my goal is to facilitate outstanding care for patients with inherited bone marrow failure and acquired aplastic anemia during their transition from diagnostic evaluation to treatment through HSCT.
In coordination with these clinical efforts, my clinical research program seeks to retrospectively and prospectively assess outcomes for patients undergoing HSCT due to inherited and acquired bone marrow failure syndromes, defined and stratified by distinct cytogenetic abnormalities. Our ultimate goal is to develop targeted clinical trials which will serve to define optimal treatment strategies for patients with distinct genetic and immunologic etiologies of bone marrow failure.
The goal of my laboratory research program is to improve HSCT outcomes by identifying methods to decrease the incidence of graft failure following HSCT, which is a particularly critical challenge in the setting of transplant for non-malignant conditions such as bone marrow failure. We are specifically examining methods to enhance donor hematopoietic stem cell (HSC) engraftment efficiency following HSCT through enhancement of the functions of specialized areas of the bone marrow microenvironment, known hematopoietic stem cell niches. We are currently testing therapeutic strategies that can be applied to enhance niche activity and receptivity for donor stem cells, which could be utilized to improve donor engraftment efficiency following clinical HSCT.