Grants & Funding

2021 CARRA-Arthritis Foundation Grant Awardees

Congratulations to the 2021 Fall Grant Cycle Awardees!

The following projects have been funding from March 1, 2021 through February 28, 2023. 


Large Grant Recipients

 

AnneMarie Brescia, MD

Nemours

Project Title: "Predicting and Preventing Extension in Juvenile Idiopathic Arthritis"

Lay Summary: Development of synovial and serum biomarkers allows families to have more complete information regarding prognosis, allowing for informed decisions regarding treatment. Addressing juvenile idiopathic arthritis (JIA) as early as possible to prevent joint damage will significantly improve the long-term outcome and life enjoyment of children with arthritis. This project focuses on prediction of extension in JIA. Since disease progression increases the risk for disability, early prediction of this course is essential; unfortunately, no reliable clinical or laboratory predictors have been established. Within this proposal, Dr. Brescia will validate the candidate synovial biomarkers that she has developed to predict disease progression from persistent to extended oligoarticular JIA on a larger, independent cohort of well-characterized existing samples from collaborating institutions within the CARRA network. Additionally, she will study how cells which line the joint contribute to progression of disease.

Linda Hiraki, MD, FRCPC, ScD 

The Hospital for Sick Children

Project Title: "Understanding the Genetics of Childhood-onset Systemic Lupus Erythematosus"

Lay Summary: Lupus is a life threatening, chronic disease. One in 5 patients are children or adolescents when diagnosed, and are at increased risk of suffering permanent organ damage from disease and its toxic medications. Prior studies have shown that genetic differences are important in determining who will get lupus and what body systems are affected. Yet no prior studies have had adequate numbers of patients with detailed clinical information collected over time. On behalf of the CARRA Lupus Genetics Work Group, we propose to initiate genetic studies of lupus patients enrolled in the CARRA Lupus Registry. The Registry includes more than 800 children and adolescents with lupus from sites across the CARRA network. Biologic samples are not collected in all patients. This is a missed opportunity to understand the biology of lupus. We propose studying both rare and common changes in genes of lupus patients to better understand lupus genetics.

**This grant is being sponsored by the Alpha Omicron Pi (AOII) Foundation. We thank them for their support!

Nadine Saad, MD

University of Michigan

Project Title: "Validation of the Auto-Inflammatory Diseases Activity Index (AIDAI) in Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Cervical Adenitis (PFAPA) Syndrome"

Lay Summary: Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome is the most common recurrent fever syndrome in children. Children with PFAPA have fevers that last 3-7 days and recur every 3-6 weeks, in the presence of the symptoms listed in the disease name. Although many different therapies are used to treat PFAPA, it is difficult to compare them because there are no reliable tests to measure disease activity. A disease activity index is a scoring system that accounts for different signs and symptoms of a disease to determine disease severity. The goal of this study is to validate the already established Auto-Inflammatory Diseases Activity Index (AIDAI) as an activity measure for PFAPA. This would be helpful to conduct further studies to compare treatments for PFAPA with the goal of treating children on a more individual level.

Erin Treemarcki, DO

University of Utah

Project Title: "Emotional Distress and Acceptance of Mental Health Interventions in Pediatric Rheumatology"

Lay Summary: For this CARRA Large Grant, we aim to survey children who take part in the CARRA registry to find out if they experience increased emotional distress. Emotional distress results from exposure to stress and can lead to more significant mental health problems, which is particularly concerning in light of current traumatic events (e.g., COVID-19 pandemic, natural disasters). The goal is to determine if emotional distress is related to disease outcomes in children with rheumatologic conditions (juvenile idiopathic arthritis, juvenile dermatomyositis, or systemic lupus erythematosus). We will design an educational tool to empower children and parents to recognize emotional distress and other mental health problems and to self-advocate for mental health interventions. The tool will be developed and tested with a co-investigative team of key stakeholders including pediatric rheumatologists, mental health providers, patients, and their caregivers. We will then have the tool reviewed by patients and parents for acceptability.

**This grant is being sponsored by the Alpha Omicron Pi (AOII) Foundation. We thank them for their support!

Christy Zigler, PhD, MSEd 

Duke University

Project Title: "Facilitating Collaborations in Pediatric Localized Scleroderma Research: International Validation of Outcome Measures"

Lay Summary: Juvenile localized scleroderma (jLS) is a rare condition that can impair health-related quality of life, especially as it affects other body parts besides the skin. Current outcome measures fail to capture this complexity, limiting our understanding of the full impact of jLS. Recently, measures have been developed specifically for jLS, and there is a critical need for coordinated, international partnerships to ensure their validity. This study builds upon existing collaborations between CARRA investigators and members of the Paediatric Rheumatology European Society (PReS) & Paediatric Rheumatology INternational Trials Organisation (PRINTO) to support three aims; the international validation of the Localized Scleroderma Quality of Life Instrument (patient-reported; specific to jLS), further refine the Total Morbidity Score (clinician-reported), and validate Patient-Reported Outcomes Measurement Information System (patient-reported; general). This study will generate the first international jLS dataset to support the use of multiple outcome measures, both of which can be used for future studies.

 


Small Grant Recipients

 

Colleen Correll, MD

University of Minnesota

Project Title: “Mortality and Cancer Incidence in Pediatric Rheumatic Disease: Linking the CARRA Registry with the National Death Index and Virtual Pooled Registry-Cancer Linkage System"

Lay Summary: Previous studies have shown higher mortality in children with rheumatic diseases, particularly in children with dermatomyositis and systemic lupus erythematosus, but most of these studies were small and/or dated. Studies have also demonstrated an increase in cancer incidence in children with juvenile idiopathic arthritis. Newer studies are needed to determine mortality and cancer rates in children with rheumatic diseases. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) registry is an ideal database to study mortality and cancer rates because it is the largest observational pediatric rheumatology research registry in the United States. We propose to link the CARRA Registry to a national death and national cancer database to calculate death and cancer rates in pediatric rheumatic diseases. We will investigate whether patient characteristics, disease type or status, and medications increase risk of death. If we identify risk factors associated with death we may be able to prevent it.

Yuki Kimura, MD

Joseph M. Sanzari Children's Hospital/Hackensack U l

Project Title: “Biologic Switching and Treatment Effectiveness in JIA"

Lay Summary: Treatment choices for children with JIA have grown to include several biologics that work on the immune system in different ways. Some children with JIA continue to have active disease even after being on the most commonly used medicines (methotrexate and etanercept). Right now, if a child doesn’t respond to these medicines, we don’t know which medication should be tried next. This study will look at the medications doctors most often tried next for these children and which medications seem to work the best in the “real world”. This will help to answer this important question of what to try next. This study will use information about thousands of children with JIA from 2 large insurance companies in the US. Analyzing this insurance information will let us see how children are actually treated so that we can understand how these treatments are used and how children respond to them.

**This grant is being sponsored by the Alpha Omicron Pi (AOII) Foundation. We thank them for their support!

Natalia Vazquez-Canizares

Children's Hospital at Montefiore

Project Title: “Identifying Current Assessment and Treatment Strategies in Juvenile Systemic Sclerosis: An International Effort to Improve Patient Outcomes"

Lay Summary: Juvenile Systemic Sclerosis (jSSc) is an inflammatory and scaring disease that affects the skin and multiple organs. More than 1/3 of children develop disability and have a substantial mortality risk. Studies are needed to identify the most effective therapies, but the rarity of jSSc makes conducting such studies in a single country difficult. Standard tools and definitions are needed to evaluate treatment response and define if patients have improved on or failed a given therapy. This study aims to develop tools and standards needed for trials by surveying pediatric rheumatologists in multiple countries to identify currently used assessments, treatments, and definitions. A group of jSSc experts will review and discuss this information in comparison to best available evidence from the literature to come to agreement on what standards are feasible and effective to use in future international treatment trials, to be able to work towards identifying the most effective treatments.

 


Implementation Science Design Grant Recipients

 

Project Title: "Optimizing Care Delivery in Pediatric Lupus"

 

 

Project Title: "Designing an Equitable and Efficient Process for Depression Screening for Children with Rheumatic Disease in the Southeastern United States"