May 14, 2018 – The study is one of four recently announced by PCORI.
The DCRI’s Laura Schanberg, MD, and the University of Washington’s Sarah Ringold, MD, working with a team of investigators from the Childhood Arthritis and Rheumatology Research Alliance (CARRA), have been approved for a $7 million funding award by the Patient-Centered Outcomes Research Institute (PCORI) to study treatments for juvenile idiopathic arthritis (JIA). This project is also supported by CARRA, the Arthritis Foundation, and Bristol-Myers Squibb.
JIA affects as many as 300,000 children in the United States. The condition can cause pain, suffering, school and work absence, and diminished ability to engage in normal activities. In younger patients, arthritis can cause joint damage, deformities, and growth problems. Eye inflammation can cause permanent damage to the eyes, including blindness. Studies suggest that how JIA is treated at the beginning makes a difference in long-term outcomes, making it critical to test treatment effectiveness from early in the course of the disease.
The study will determine whether a six-month course of abatacept given soon after diagnosis of limited JIA (affecting four or fewer joints) will prevent the involvement of more joints, 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.
Another aim of the study is to better understand the experience of children and families participating in a clinical trial, starting at the time of JIA diagnosis. Using home visits, telephone interviews, and electronic surveys, the researchers will collect information from families about trial participation in real time throughout the duration of the study, starting at the time of enrollment, and use this information to improve the experience of enrolling and participating in a research study.
“This will be the first time that a preventive approach to JIA has been tried,” Schanberg said. “If we are able to decrease the more severe forms of JIA and eye disease, it will substantially reduce the morbidity associated with disease and change standard of care. Another unique part of the study is embedding ethnographic assessment of subjects in the trial, making it possible to learn about study participation as it is being experienced by study subjects.”
The study will utilize the infrastructure of the CARRA Registry, for which the DCRI serves as data coordinating center. An outcomes team led by Kristin Siebenaler and Anne Dennos will work with network clinical operations to model a pragmatic trial executed in an ongoing registry. The proposal was developed under the auspices of PARTNERS, a PCORI-funded patient powered research network, led by DCRI’s Renee Leverty.
The study will also leverage PCORnet, the National Patient-Centered Clinical Research Network. PCORnet is PCORI’s initiative to improve the nation’s capacity to conduct patient-centered comparative clinical effectiveness research more efficiently by harnessing the power of large amounts of health data and patient partnerships.
“This project was selected for PCORI funding not only for its scientific merit and commitment to engaging patients and other stakeholders, but also for its potential to improve our nation’s capacity to conduct clinical research more efficiently and to answer important questions that patients and clinicians face,” said PCORI Executive Director Joe Selby, MD, MPH. “We look forward to following the study’s progress and working with the DCRI to share the results.”
Schanberg’s study was selected for PCORI funding through a highly competitive review process in which patients, clinicians, and other stakeholders joined clinical scientists to evaluate the proposals. Applications were assessed for scientific merit, how well they will engage patients and other stakeholders, and their methodological rigor among other criteria. The award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.
PCORI is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed healthcare decisions. For more information about PCORI’s funding, visit www.pcori.org.
CARRA is a nonprofit research network of more than 500 pediatric rheumatology investigators spanning more than 120 sites across the U.S. and Canada, dedicated to improving the treatment and health related outcomes of children with rheumatic diseases through research. CARRA’s mission is to conduct collaborative research to prevent, treat, and cure pediatric rheumatic diseases. For more information on CARRA, visit www.carragroup.org.