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Pediatric rheumatologists,researchers and families, working hand in hand to support groundbreaking,
life-changing research and treatment for children living with arthritis and lupus.

Research Project Summaries

Southern California's pediatric rheumatologists are on the cutting edge of research and are currently developing several innovative projects, each a revolutionary study that would advance understanding of lupus and/or improve care of patients with lupus.

Genes and Biomarkers

Information learned from our studies is being utilized to develop a predictive model for kidney and other organ damage in pediatric lupus, leading to individualized prediction of disease severity, risk of disease exacerbation and risk of kidney damage in children and adolescents with lupus. Such a platform of biomarkers to identify risk for serious disease and organ damage could become an important tool for determination of appropriate medical therapy and optimization of outcome, leading to more personalized care of pediatric patients with lupus. Ultimately this will help to determine potential new therapeutic targets in lupus and improve care now and in the future.

Quality of life and the impact of illness on youths with lupus nephritis and their healthy siblings

Pediatric-onset systemic lupus erythematosus (pSLE; age of onset < 18 years) is a complex chronic autoimmune disease that can involve multiple organ systems. Utilizing an electronic data capture system, the goals of this study are to: 1) assess the quality of life (QOL) of 50 pSLE patients with LN and 60 of their healthy siblings; 2) identify the relationship between QOL of siblings of pSLE patients and health-related quality of life (HRQOL) and disease activity in pSLE; and 3) explore potential correlations between QOL of siblings discordant for disease and their perception of the impact of pSLE on their life in relation to their gender, age and birth-rank.

Results from this study will be used to address the need for family-oriented care for families coping with LN and appropriately target psychological and social interventions to benefit the function and adaptation of the entire family unit.

Leptin Modulates Apoptosis and Clearance of Autoimmune T Cells

This project aims to study the influence of leptin on apoptosis (billions of cells dying in programmed fashion on a daily basis) and the clearance of apoptotic T cells and apoptosis in Systemic Lupus Erythematosus (SLE). Thus far, the program has concluded that leptin facilitates autoreactive T-cell survival in SLE through a leptin-dependent apoptosis impairment. Moreover, leptin promotes availability of self-antigen to autoreactive T cells in inflammatory macrophages.  This newly described dual role of leptin in facilitating autoreactive T cell activity in SLE may have implications for new targeted therapeutic approaches in the disease. We hope in the future to try to get recovery of this process by exploring human systems and treat human cells with anti-leptin agent that is already available for lab research.

Leptin in Neonatal Lupus

In neonatal lupus, congenital heart block can be a fatal and/or severely disabling complication that can lead to congestive heart failure and/or the need of placement of a pacemaker (in half of the patients). This proposal will study the possibility for the use of leptin as a new biomarker in neonatal lupus.Since deaths may also occur later in life, early monitoring and/or intervention could lead to reduced morbidity and better prognosis and management for this disease.

Role of Th17 cells in SLE 

T helper 17 cells (Th17) are newly discovered lymphocyte cells (part of the immune system) that found to play major and essential role in inflammation and immune response against infection. Study findings suggest a direct role of IL-17 in the SLE pathogenesis and may point towards a new drug therapy pathway for this disease. Researchers now want to explore the mechanism by which these cells induce lupus and to add another layer to the complex not fully solved pathogenetic puzzle of the disease.

Influence of Sex Chromosome Complement and Sex Hormone on Autoimmunity

One clinical group plans to study the link between genes on the X chromosome  and the greater incidence of autoimmune diseases in females. The study will look at individuals with abnormal numbers of sex chromosome, measure the concentration of circulating sex hormone E2 and testosterone in the same cohort prior starting hormone replacement therapy, and analyze the relationship of autoimmunity between individuals with abnormal number of the sex chromosomes and healthy controls with normal number of sex chromosomes.

This proposed study will not only provide new insights into the role of varying sex chromosome complement and sex hormone in autoimmunity but also lay a theoretical groundwork for future basic science studies on the pathogenesis of autoimmune diseases.

DNA Repair in SLE
Deborah McCurdy, Hailiang Hu, Nathan Martin, Ornella Rullo, Jennifer Woo

Clinical Manifestations and Disease Characteristics of Pediatric Onset
Systemic Lupus Erythematosus
Ornella Rullo, Jennifer Woo, Deborah McCurdy

Deficiency of the DNA repair enzyme, ATM kinase in juvenile arthritis
Gil Amarilyo, Deborah McCurdy

Research Biomarkers for Prediction of Risk in Pediatric SLE
Ornella Rullo, Deborah McCurdy, Jennifer Woo, Betty Tsao

CARRA Registry for Childhood Arthritis and Rheumatology
Ornella Rullo, Jennifer Woo, Deborah McCurdy

Ultrasound Use in Juvenile Idiopathic Arthritis
Miriam Parsa, Deborah McCurdy

T cell Homeostasis and Cytokine Production in Females with Turner's Syndrome 
Eric Yen, Deborah McCurdy, Ram Singh

Genetic Risk Factors in Juvenile Idiopathic Arthritis (JIA) and Uveitis
Miriam Parsa, Deborah McCurdy

Trends in the use of folate with methotrexate in children with rheumatic disease

Barriers to optimal pediatric rheumatology healthcare at UCLA

Implementation of an electronic note in UCLA pediatric rheumatology outpatient clinics

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