A treatment’s long-term safety and the impact of its side effects on quality of life are the top concerns of parents with children who have juvenile idiopathic arthritis (JIA), a new study shows.
The study, “Adding patient-reported outcomes to a multisite registry to quantify quality of life and experiences of disease and treatment for youth with juvenile idiopathic arthritis,” was published in the Journal of Patient-Reported Outcomes.
Despite advances in treatment, children with JIA frequently have poor health-related quality of life (HRQOL) due to disease-associated relapses and the unpredictable course of JIA and its symptoms. Furthermore, adverse effects of medications and other treatment complications, such as frequent injections and intravenous infusions, may also affect well-being.
Aiming to improve the care of children with JIA, patient-centered outcomes may be particularly useful by evaluating both disease and treatment-related factors, with an emphasis on patient-reported concerns, which may be different from the assessment of clinicians.
As data on underlying conditions in JIA are rare, disease registries may help elucidate disease features and clinical factors toward improved patient well-being.
In this study, the research team used the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry and enabled parents of patients with JIA to provide patient-centered outcomes regarding the child’s HRQOL. The team also analyzed experiences of disease and treatment burden, including pain interference, morning stiffness, a medication’s adverse effects, and intolerance to the immunosuppressant therapy methotrexate.
Researchers hypothesized that parents’ major concerns would be treatment safety and side effects, which would negatively impact HRQOL.
A total of 180 patients (81 percent white non-Hispanic and 76.7 percent women) were included in The Learning Cohort study. Patients’ mean age was 11.8 years and mean disease duration was 7.7 years.
Mean Total Pediatric Quality of Life, a parent-reported assessment of the child’s physical, emotional, social, and school functioning in the previous month, was 76.7, which is below the threshold for suboptimal HRQOL (set at 78.6).
In addition, 17.8 percent of JIA patients experienced over 15 minutes of morning stiffness on a typical day, and 26.7 percent reported one or more serious adverse effects from treatment. Children aged 13 or older showed higher incidence of both morning stiffness and adverse effects.
The top concerns reported by parents were serious long-term side effects of medications (63.3 percent) and the safety of medications (46.1 percent).
Furthermore, 38 out of 90 methotrexate users (42.2 percent) revealed methotrexate intolerance, meaning they experienced adverse effects caused by the treatment.
Measures of disease and treatment burden were negatively correlated with HRQOL, meaning that the worse the disease or treatment burden, the worse the patient’s quality of life.
“Understanding threats to HRQOL – including those related to treatment – is vital to improving care of youth with JIA,” the researchers wrote. “Adverse treatment experiences undermine HRQOL even after accounting for disease symptoms and disease activity and should be assessed routinely to improve wellbeing.”
Among the study’s limitations, the authors cited a lack of reports from children and the impossibility of generalizing findings for all children with JIA.