Juvenile idiopathic arthritis (JIA) affects as many as 300,000 children in the United States; more than half of these children have a limited form of JIA at presentation (i.e., less than four joints are affected during the first six months of disease). After the first six months, up to half of these children will develop arthritis in more joints and/or develop inflammation in their eyes at some point during their disease course. This makes the disease more difficult to treat. In addition, some children with persistent limited JIA may be severely affected, requiring prolonged courses of systemic medication. Arthritis and eye inflammation can cause pain, suffering, school and work absence, and less ability to engage in normal activities. In children, arthritis can cause joint damage, deformities, and growth problems. Eye inflammation can cause permanent damage to the eyes, including blindness.
Preventing the development of these outcomes is important to parents, patients, and healthcare providers. Children with limited JIA are treated with nonsteroidal anti-inflammatory drugs (e.g., ibuprofen and naproxen) in combination with steroid injections into the inflamed joints. This study will determine whether a six-month course of abatacept given soon after diagnosis of JIA will prevent the development of increased joint inflammation, eye inflammation, or need for stronger treatment. Abatacept is a biologic medication that acts specifically on a part of the immune system known to be involved in the development of arthritis. Abatacept is FDA-approved for children with polyarticular JIA (JIA with more than four joints involved), is commonly used, and is known to be safe and effective.
Another aim of the study is to better understand the experience of children and families participating in a clinical trial. It is often overwhelming to consider enrolling in a clinical trial at the same time as receiving a new diagnosis. However, studies suggest that how JIA is treated at the beginning makes a difference in long-term outcome, making it critical to test treatment effectiveness from early in the course of the disease. Using home visits, telephone interviews, and electronic surveys, we will learn what it is like for children and parents on the day of diagnosis and how they feel about being approached about participating in a clinical trial at the same time. We will continue to collect information from families about trial participation throughout the duration of the study and use this information to improve the experience of enrolling and participating in a research study.
Only patients and their families truly know how challenging limited JIA can be. Only children and families understand the worry about developing more severe disease, further disease complications, and what it is like to have to make choices about treatment. Only children and families know how much their daily life can be affected by the disease and its treatments—and being in a clinical trial. Patient stakeholders will tell us what aspects of their lives are most affected and how we can best measure these, tell us whether we are conducting the study correctly, and help us to involve other patients and families in the research. Perhaps most important, they will help us get the study results out to patients and healthcare providers, which could change future care.
*All proposed projects, including requested budgets and project periods, are approved subject to a programmatic and budget review by PCORI staff and the negotiation of a formal award contract.