Researchers from the Betty Irene Moore School of Nursing at UC Davis and the University of California, San Diego School of Medicine and Rady Children’s Hospital-San Diego received a $2.8 million award from the Patient-Centered Outcomes Research Institute (PCORI) for a three-year study on the effectiveness of two different therapies for children with Kawasaki disease, as well as the burdens of treatments on those children and their families.
Jane C. Burns, professor of pediatrics at UC San Diego and director of the Kawasaki Disease Research Center, and Katherine Kim, assistant professor at UC Davis, serve as co-primary investigators on the project, “A Stakeholder-driven Comparative Effectiveness Study of Treatments to Prevent Coronary Artery Damage in Patients with Resistant Kawasaki Disease.”
Kawasaki disease is an uncommon childhood illness that affects the blood vessels and is the leading cause of acquired heart disease in children. Experts do not know the cause of the disease, which is most common in children ages 1 to 2 years. However, two medication regimens are widely used in treatment. More than 100 new patients were treated at Rady Children’s Hospital-San Diego in 2015. That year, UC Davis Children’s Hospital reported more than 100 visits, including new cases and those patients diagnosed earlier with the condition.
“After bringing together 120 parents of children with the disease, clinicians and researchers, we learned their top priority was to test the effectiveness of treatments to prevent heart damage,” Burns said. “We know that 10 to 20 percent of patients are resistant to intravenous immunoglobulin (IVIG), the standard treatment for Kawasaki disease. These patients are at higher risk of heart complications. We want to find out which of two widely used treatments will stop the fevers and inflammation the fastest.”
For those with persistent fever, some receive a second dose of IVIG, while others receive the biologic, infliximab. This comparative effectiveness study is a superiority trial comparing these two approaches.
“In addition to comparing the effectiveness of these drugs, we will also evaluate patient reported outcomes to determine the burden that treatment has on patients and their families,” Kim said. “We will then factor in those reported experiences when evaluating the efficacy of the treatment.”
Burns and Kim will use a parent observation tool to record discomfort, psychosocial concerns and other experiences during both the in-hospital stay and once they go home using mobile technology.
“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 fill an important gap in our health knowledge and give people information to help them weigh the effectiveness of their care options,” said PCORI executive director Joe Selby. “We look forward to following the study’s progress and working with Drs. Burns and Kim to share the results.”
Burns’ and Kim’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. 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.