The UCSF Pediatric Rheumatology research program aims to advance our understanding of complex rheumatic diseases and to improve outcomes for affected children. Our researchers are involved in:
- Understanding the underlying basic mechanisms of autoimmunity
- Determining the best treatments for children with lupus nephritis and juvenile arthritis
- Studying the causes of osteoporosis
- Helping teens and young adults transition to adult providers
- Studies on the use of musculoskeletal ultrasound for diagnosing juvenile arthritis and guiding intra-articular injections
In addition, we actively participate in Childhood Arthritis and Rheumatology Research Alliance (CARRA), our national research network, which provides our patients opportunities to participate in national multicenter registries and therapeutic trials.
Dr. Lawson's research seeks to predict long-term outcomes of childhood-onset rheumatic diseases and to improve the transition to adulthood for young adults with childhood-onset rheumatic disease. Her work focuses on health outcomes, education and employment outcomes, health care access and patient self-management.
Dr. von Scheven's research focuses on improving the long-term outcomes of patients with childhood-onset rheumatic diseases through understanding and preventing the collateral damage and secondary morbidities that these children face as they become adults. Most of Dr. von Scheven's early work focused on children with systemic lupus erythematosus (SLE). The increased survival of children with SLE over the past 25 years has been associated with the emergence of morbidities, such as osteoporosis, premature atherosclerosis, cancer, and psychosocial problems not previously seen in this patient population. Some of these complications result directly from long-term consequences of uncontrolled inflammation and other disease-related phenomenon, while others result from the treatments provided for the SLE itself. As children with SLE survive longer they face many challenges related to disease management which become particularly important as they transition from pediatric to adult providers. Dr. von Scheven's thesis is that prevention of the complications that appear in adulthood begins in childhood, and that better understanding and risk prediction is the first step in developing effective preventative strategies.
The evaluation of drugs for childhood SLE has always been challenged by the high cost of conducting randomized controlled trials and small sample sizes. Thus, as part of a national cross-disease pediatric rheumatology initiative through CARRA (Childhood Arthritis and Rheumatology Research Alliance) Dr. von Scheven has been leading a national effort to utilize comparative effectiveness methods to determine best treatments for lupus nephritis in children. Our strategy is to utilize the CARRA Registry (a national dataset) to capture patient and physician reported outcomes, and in parallel to reduce practice variability which thwarts the comparison of treatments in real-life data by developing Consensus Treatment Plans (CTPS) for use in clinical practice.
Dr. von Scheven's research emphasizes patient engagement and she currently serves as an investigator for PARTNERS, a pediatric rheumatology PPRN (Patient Powered Research Network) that is supported by PCORI and aims to transform the conduct of research by incorporating the patient’s voice. As Chair of the PARTNERS Outreach and Communication committee, Dr. von Scheven oversees patient engagement, patient and physician training, and the development of governance documents.
Dr. Waterfield is a basic science immunologist who is currently studying the role of AIRE, a protein involved with the development of autoimmunity. He oversees the basic science component of the fellowship training program. Dr. Waterfield sees patients in the in- and out-patients units at UCSF.
Autoimmune disease affects up to 5% of the population and can cause significant morbidity and mortality. Our labs main focus is to understand the basic mechanisms by which immune tolerance is broken in order to identify novel therapeutic targets for the treatment of autoimmune diseases. We utilize a variety of mouse models to study both central tolerance and peripheral tolerance. Central tolerance is the process by which autoreactive T cells are deleted in the thymus through negative selection. We are currently utilizing a variety of novel conditional knockout mice to study the roles of specific proteins in thymic tolerance.
A second area of active research in the lab is the role of effector T cells in autoimmune disease. One subtype of CD4+ effector T cell, termed T helper 17 (Th17) cells have been found to be important in the pathogenesis of multiple autoimmune diseases and Th17 cells have been targeted therapeutically for treatment. We have identified the activating transcription factor 7 interacting protein (ATF7ip) as a novel regulator of Th17 cell differentiation and are currently studying its mechanism of action in Th17 cells and other immune cells.
Dr. Lionetti’s research has focused on identifying risk factors for disease and treatment-related sequelae, such as avascular necrosis in Lupus and medication adverse events. She has an interest in autoinflammatory diseases and is an active member of the Childhood Arthritis & Rheumatology Research Alliance (CARRA) Periodic Fever, Aphthous Stomatitis, Pharyngitis, Cervical Adenitis (PFAPA) group which has an emphasis on further characterizing treatment plans and outcomes of this fairly common periodic fever syndrome. In collaboration with other researchers internationally, she has worked on further characterizing and treating patients with hyperzincemia/hypercalprotectinema, which is now considered an autoinflammatory-related disease. She also has participated in the CARRA Juvenile DermatoDr. von Scheven'sositis and Kawasaki Disease working groups.
Dr. Bernal’s research focuses on treatment and outcomes of pain amplification syndromes in children.
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