What Works to Alleviate Juvenile Arthritis?

When people think about arthritis, most envision adults rather than children with this serious chronic illness. Yet about 300,000 children in the United States have long-term joint inflammation. During July, designated Juvenile Arthritis Awareness Month, we call attention to this common illness, which is often misdiagnosed when a child first experiences joint pain, stiffness, and swelling. Although appropriate treatment can reduce damage to a child’s joints, there is currently no cure.

At PCORI, we have a variety of projects addressing this widespread, high-burden condition. Some PCORI staff, as well as awardees, have personal experience with the juvenile arthritis and related diseases that led us to our interest in patient-centered clinical research.

Suzanne Schrandt

This summer marks my 25th year of living with juvenile arthritis. While I’m not celebrating this anniversary, I am thrilled about the remarkable advances in research and medicine that have occurred during this period. As a child, I was fortunate to be diagnosed promptly and referred to a wonderful medical team, but the path forward for my treatment was very unclear.

My disease was very aggressive. At first, I was unable to dress or eat without help. Since those early days, it has been a whirlwind of drugs, surgeries, and hospitalizations. Now, after the discovery of biologics and increased knowledge about the disease, I am living a full and relatively healthy life.

I have long been a patient advocate heavily involved in patient education, patient-centered clinical education, and health policy. Until I learned about PCORI, however, I never realized the profound power of the patient voice in research or the opportunities for patients to be involved in this aspect of health care. PCORI provides a unique chance for patients to become part of the solution to their medical challenges, and I was thrilled to join PCORI and help to carry our mission forward.

Seth D. Ginsberg

I was diagnosed with spondyloarthropathy, an illness related to arthritis, when I was in eighth grade, just weeks before my Bar Mitzvah. At the time, the diagnosis was hard to take in—I had thought I just had growing pains, but arthritis ran in the family, so my mother suspected otherwise. The diagnosis brought relief, but that came at a price: taking medicine throughout the day, every day and physical therapy four days a week. And when I missed a dose or skipped a therapy session, I felt it the next day, so there was little room for cheating. 

When I went away to college, I was 18, just a pup who had never been away from home before, and I realized very quickly how much I relied on my support network of family and friends. Being on a college campus and sleeping in a dorm room was basically the opposite from being surrounded by people who understood what I was going through. That isolation prompted me to seek strength and support from others out there on the internet. 

And voila! Our online community of arthritis patients and caregivers, CreakyJoints, was born.

Today, CreakyJoints has a world-wide network of more than 80,000 people who will never need to feel alone again. One of our four areas of focus is patient-centered research, and we now have a PCORI-funded Patient-Powered Research Network called Arthritis Power (AR-PoWER), where people with arthritis can help guide promising, meaningful research. Within AR-Power, CreakyJoints collaborates with the University Alabama Birmingham Center for Education and Research and Therapeutics of Musculoskeletal Diseases, the American College of Rheumatology, and Corrona, a doctor-led arthritis research network.

PCORI-funded Research Projects

PCORI has funded various research projects focused on juvenile arthritis. Currently, patients, families, and doctors must often make decisions on which medications to use without information about how they compare with one other, and the right time to start each one. There is little evidence regarding the effectiveness and safety of even the most commonly used medications.

A recently approved New Jersey study will compare the effectiveness of three different first treatment strategies in 400 children and their families. The research team will measure arthritis activity after 12 months of treatment and collect data on pain, mobility, medication side effects, quality of life, and other important factors affecting patients and their families.  

Another study addresses what treatments clinicians should offer when earlier treatments fail. It was motivated by a question commonly asked by parents of patients—“Given my child’s responses to the previous treatments, what is the best next treatment option for my child?”—and a question asked by clinicians—“What treatment should we recommend to patients who fail to respond to the first (or second) line of treatment?” The project, based at the Cincinnati Children’s Hospital Medical Center, is developing analytic methods that take into account an individual child’s disease course and responses to earlier treatment. The team plans to evaluate adaptive treatment strategies by applying their methods to patient registries and electronic health records. The team’s long-term goal is to improve health outcomes by helping to create easy-to-use tools that assist patients, families, and clinicians in shared decision making.

PCORI is also supporting the Patients, Advocates and Rheumatology Teams Network for Research and Service (PARTNERS), a PPRN run by patients, their families, and advocacy organizations, along with clinical research partners. Led by an investigator at Duke University, it provides a common venue for patients and researchers focused on moving research forward. It has a registry of more than 9,000 children with rheumatic diseases, of which juvenile arthritis and lupus are the most common. While encouraging registered patients to contribute via data collection at clinic visits and outside the clinic using electronic devices, the PPRN is also contacting new patients to expand the network. In particular, PARTNERS is focusing on increasing data collection from diverse patient groups, especially underserved populations.

This PPRN includes three patient-advocacy groups (Arthritis Foundation, Friends of CARRA, and Lupus Foundation of America), healthcare providers, a clinician scientist-initiated research network (Childhood Arthritis and Rheumatology Research Alliance [CARRA]), and a quality-improvement learning network (Pediatric Rheumatology Care and Outcomes Improvement Network ). The goal is a patient-centered health system that benefits from complementary perspectives, infrastructures, and expertise to empower patients, clinicians, and researchers to learn from every patient interaction, conduct patient-centered outcomes research, and implement the findings.

Welcoming Collaboration

Identifying the best treatments for juvenile arthritis and improving outcomes will require the efforts of many groups. We know that we can’t do it alone. So, we are pleased to be collaborating with a variety of effective organizations, including the Arthritis Foundation, within PARTNERS and in other initiatives.

Says Ann Palmer, president and CEO of the Arthritis Foundation, “We’re proud to be working closely with PCORI, PARTNERS, CARRA, and several other organizations to address the unique and urgent needs of juvenile arthritis patients and their families across the country,” She adds, “Together, we’re having a huge impact on children, teens, young adults, and their families who live daily with arthritis and rheumatic conditions. We host family days in communities across the country; provide local juvenile arthritis camps, as well as programs for teens and young adults; and hold an annual juvenile arthritis conference, which brings together thousands of families to learn, connect, and share their experiences.  Many families tell us the conference is life changing.” 

With its emphasis on scientific discovery and improving access to care through advocacy efforts, the Arthritis Foundation shares PCORI’s commitment to research, engagement, and outreach. "There’s tremendous work to be done in the years to come," Palmer says, "and we look forward to ongoing collaboration on behalf of the arthritis community."