Health-Related Quality of Life in Juvenile Idiopathic Arthritis: A Systematic Review of Phase III Clinical Trials.

Background/objectives: Juvenile idiopathic arthritis (JIA) is the most common rheumatic disease in childhood, leading to severe disability and negatively affecting patients' health-related quality of life (HRQoL). The aim of this systematic review was to evaluate the adoption, reporting and assessment methodology of HRQoL in phase III clinical trials involving children with JIA. Methods: An electronic and manual search was conducted to identify primary and secondary publications of pharmacological trials conducted between 2012 and 2023. Data were extracted and recorded in duplicate. Results: A total of 222 studies were screened and 24 articles (22 primary and 2 secondary publications) were included in the review. HRQoL was not listed among the endpoints in 10 trials (45.5%), while it was a secondary endpoint in 12 trials (54.5%). The proportion of trials that did not consider HRQoL was equally relevant in both for-profit and no-profit settings (44.4% versus 50.0%), but it was higher in studies on systemic JIA compared to other JIA subtypes (62.5%), and on IL inhibitor treatment (72.7%) with respect to other disease-modifying antirheumatic drugs. Information on HRQoL was usually collected from parents/caregivers, and only three studies were categorized as "probably robust" with regard to HRQoL assessment. Conclusions: Systematic incorporation of HRQoL measures represents an urgent need in pediatric rheumatology, aiding clinicians in their decision-making in relation to treatment effectiveness and considering the children's perspective.