My research interests are in hematopoietic stem cell transplantation and inherited immune deficiency disorders. In particular, my primary clinical and research focus is hematopoietic stem cell transplantation (HSCT) for inherited immune deficiency disorders, such as severe combined immune deficiency, Cartilage Hair Hypoplasia and X-Linked Hyper IgM syndrome.
I was first guided toward my research when working with young children. Very young children, including babies, undergo hematopoietic stem cell transplantation as a therapeutic and curative treatment for their main immune deficiency disorders. These children are given conditioning chemotherapy to assist with HSCT. However, several of the medications used in conditioning chemotherapy have very severe and toxic side effects.
The dosages of these medications are based on adult studies due to minimal data from pharmacokinetic trials in children. These dosages may not be accurate for all patients and may place certain children at a higher risk for toxic side effects. Therefore, pediatric patients have a greater need for individualized dosing of chemotherapeutic drugs used during conditioning chemotherapy.
As such, my research is involved in pharmacokinetic (PK) and pharmacodynamic (PD) assessments of chemotherapeutic agents used during HSCT and clinical trial development.
Among the goals my colleagues and I have is designing precision dosing strategies for individualized drug treatment to enable patients to obtain the best possible clinical outcome with the fewest side effects. I’ve developed and executed a clinical trial for pharmacokinetic-guided precision dosing of melphalan in pediatric patients undergoing HSCT. I have also developed a reduced toxicity conditioning regimen with pharmacokinetic-guided dosing of busulfan in pediatric patients undergoing HSCT for a variety of primary immune deficiency disorders.
In terms of my patient care, I focus on bone marrow transplantation and immune deficiency. I work with pediatric patients with a wide variety of disorders that require hematopoietic stem cell transplantation for long-term survival. This type of treatment is associated with a multitude of difficulties, including life-threatening issues and major challenges for the patients and their families.
I am passionate about making a difference in a child's life and their family’s life during a very challenging time. I have always been impressed with how resilient children are, and I find it very fulfilling when children undergo a successful transplant.
During my career, I have been recognized with the American Society of Bone Marrow Transplantation (ASBMT) New Investigator Award from 2015 to 2017. The ASBMT New Investigator Award inspires young researchers to pursue clinical or laboratory investigations in the field of blood and marrow transplantation.
My research has been published in respected journals such as Biology of Blood and Marrow Transplantation, Journal of Clinical Immunology, Blood, Blood Advances and Pediatric Blood and Cancer.