Spondyloarthritis is one of the most common forms of juvenile arthritis, accounting for as much as one-third of all cases. The characteristics of spondyloarthritis include inflammatory arthritis, tender tendon attachments to bone, swollen fingers, back pain, inflammatory bowel disease, eye inflammation, and psoriasis.
Patients with spondyloarthritis are often treated with a type of medication called tumor necrosis factor inhibitors (TNFi), which can help patients get to a point where they no longer have any symptoms—known as quiet disease. Once an inactive disease state is reached, there is almost no information to guide the next steps. Researchers want to know if children who have maintained inactive disease for at least six months can maintain quiet disease without taking their medication as frequently or stop the TNFi therapy. They also want to know the safest method to bring patients off medication. For example, if a flare (disease worsening) does occur during therapy reduction, can doctors predict when it is most likely to happen and can inactive disease be quickly recaptured?
This trial will compare how patients with inactive spondyloarthritis on TNFi treatment respond to three current treatment options: (1) continue standard TNFi medication, (2) space out the time between usual TNFi medication doses, or (3) stop the TNFi medication. If it is possible to safely prolong the time between doses or completely take away TNFi therapy in this population, this could remove unnecessary medication exposure, improve patient quality of life, and lower medication costs.
Patients, caregivers, and physicians can use the findings from this study to decide the best treatment option for children with spondyloarthritis who have inactive disease.
The research team will enroll 198 children with spondyloarthritis from 20 participating healthcare centers across the country. These patients must be receiving TNFi therapy and have maintained quiet disease for at least six months. Children will be randomly assigned to one of the three treatment strategies to compare the risk of flare and each patient’s lived experiences between the strategies.
Participants in each treatment group will follow their routine care schedule and see their usual doctor approximately every three months for up to 12 months. Each visit will include a routine physical exam and a health survey to measure outcomes most important to patients and families. For participants for whom a flare occurs, their active study participation will end and they will work with their doctor to get back to an inactive disease state. We will use electronic health record data to track long-term participant disease flares after active trial participation ends.
In preparation for this study, we met with families and patients within our population of interest to gain insight into their priorities and preferences, and to hear their suggestions and concerns about the study design and recruitment plan. The study team has formed a Research Partners Group comprising patients, caregivers, rheumatologists, representatives from national arthritis professional/advocacy organizations, and payer representatives. This group will help design the study around the questions most important to the spondyloarthritis population and advise the research team as the study progresses.
*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.