Utility of patient-reported outcomes for pulmonary symptoms and sleep disturbance and impairment in children with systemic juvenile idiopathic arthritis.

Background: Patient-reported outcomes (PROs) are critical assessment tools for clinical practice, observational studies, and interventional trials. While families of children with systemic juvenile idiopathic arthritis (SJIA) and SJIA-associated lung disease (SJIA-LD) report significant limitations in their quality of life, existing PROs for juvenile idiopathic arthritis may not properly measure the full impact of these disorders. Our objective was to utilize a newly developed lung symptom survey as well as existing, validated Patient-Reported Outcomes Measurement Information System (PROMIS) measures in children with SJIA with and without LD.

Methods: Participants were parents/guardians of SJIA patients ≤ 18 years and were invited to participate using the Cincinnati Children's Hospital Medical Center (CCHMC) JIA Registry, and memberships in the Systemic JIA Foundation, and SJIA Facebook Group. Participants provided proxy-reports for their child using several PRO questionnaires [CCHMC Lung Symptom Survey; PROMIS Asthma Impact, Sleep Disturbance, Sleep Impairment Forms] and selected demographic and SJIA specific information.

Results: There were 139 responses, of which 40.3% (n = 57) reported some lung disease including 12.9% (n = 20) with interstitial lung disease (ILD), pulmonary alveolar proteinosis (PAP) and/or pulmonary artery hypertension (PAH). All SJIA patients with any lung disease and those with ILD/PAP/PAH had significantly higher total questionnaire scores than patients without lung disease on the CCHMC Lung Symptoms Survey. Both the full survey and individual questions showed good ability to distinguish patients with ILD/PAP/PAH from those without (area under the curve (AUC) > 0.7). The majority of patients reported some level of sleep disturbance (n = 71/139 = 51.1%) and sleep impairment 53.2% (n = 74) regardless of presence or absence of lung disease, including moderate to severe sleep impairment and/or disturbance in 48% of SJIA patients.

Conclusions: Children with SJIA and lung problems had higher scores on the CCHMC Lung Symptom Survey; however, these measures did not discriminate between SJIA-LD and other pulmonary conditions such as asthma. Based on PROMIS measures, a majority of children with SJIA had sleep disturbance and impairment, regardless of steroid use or presence of lung disease.