Early aggressive treatment with biologic and synthetic disease-modifying anti-rheumatic drugs (DMARDs) improved the quality of life of children with polyarticular juvenile idiopathic arthritis (pJIA) in Finland, according to one-year data from a Phase 3 trial.
The study, “Health-related quality of life during early aggressive treatment in patients with polyarticular juvenile idiopathic arthritis: results from randomized controlled trial,” was published in the journal Pediatric Rheumatology.
Children with JIA often experience a lower health-related quality of life (HrQoL), which includes impacts on their well-being, physical health, and psychological state.
With evidence suggesting that quality of life can predict disease activity and functional disability, improving quality of life is an important clinical goal for JIA treatments.
To date, a few studies have evaluated the effects of treatment on the quality of life of JIA patients. A recent German study showed that treatment helped JIA children achieve comparable well-being to that of healthy children.
In the new study, researchers in Finland assessed the one-year impact of several DMARD-based treatments on the quality of life of pJIA children, who are more likely to fail to respond to the treatments than those with other JIA types.
However, biologic DMARDs, such as infliximab — sold under the brand name Remicade, among others — have shown considerable effectiveness in easing symptoms and managing disease activity in pJIA.
The study was part of the Phase 3 ACUTE-JIA clinical trial (NCT01015547), which evaluated the safety and effectiveness of different DMARD-based regimens in 60 Finnish children newly diagnosed with pJIA.
Participants, 4 to 14 years old, were randomly assigned to receive one of three regimens: methotrexate alone, methotrexate with Remicade, or methotrexate with both Oxiklorin (hydroxychloroquine) and Salazopyrin (sulfasalazine), which is a triple combo of conventional or synthetic DMARDs.
Disease activity and HrQoL were assessed before treatment and at several study visits over a year.
Changes in disease activity were measured by the American College of Rheumatology pediatric score and the juvenile arthritis disease activity score. HrQoL was evaluated through parents’ reports on the Childhood Health Assessment Questionnaire (CHAQ), which includes 50 questions, and physical and psychosocial summary scores, with higher scores reflecting better quality of life.
A total of 47 children (78%) remained on treatment: 20 on methotrexate and Remicade, 16 on the triple combo, and 11 on methotrexate alone. Treatment was discontinued due to lack of efficacy in 11 children (eight on methotrexate and three on the triple combo) and to adverse events in two children (one on methotrexate and one on the triple combo).
Results showed that, after one year, treatment with methotrexate and Remicade was the most effective in halting disease activity in these children. Infections and temporary elevations of liver enzymes were most common in the methotrexate group, while nausea was more frequent in the triple combo group.
Before treatment, pJIA children had lower scores than the normal population in several domains of health-related quality of life: global health perception, bodily pain, role or social limitations due to physical reasons, physical functioning, and parental emotional impact. After one year of treatment, all scores except global health perception reached normal values, regardless of treatment regimen.
“Despite their different efficacy and safety profiles, infliximab [Remicade] and synthetic DMARDs, when used early and aggressively, were effective in improving health-related quality of life,” the researchers wrote.
Notably, the physical dimension of HrQoL showed improvements earlier than the psychosocial domain, and at the same time as the decrease in disease activity.
Further analyses showed that physical aspects of quality of life in these children were influenced by CHAQ total scores, level of pain, and time spent in inactive disease. In addition, gender and CHAQ total scores contributed to psychosocial aspects.
Overall, these findings illustrated the importance of managing disease activity to ensure good quality of life, and suggested that measuring pain could be used to assess well-being in JIA children, the scientists said.
Of note, the team added that the lack of differences in quality of life improvements between the different regimens may be due to the low number of participants and the study’s short follow-up period.
“In the future, a long-term follow-up study on HrQoL, assessing the effects of early aggressive treatment and factors associated with HrQoL might direct treatment decisions towards better patient-reported outcomes,” the researchers wrote.