Validation of Patient‐Reported Outcomes Measurement Information System ( PROMIS ) Pediatric Measures for Children With Chronic Nonbacterial Osteomyelitis

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OBJECTIVE: To assess the validity of the Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric measures in patients with chronic nonbacterial osteomyelitis (CNO). METHODS: Within the longitudinal patient registry of CNO, English-speaking patients aged 8 years and older self-reported PROMIS Pediatric measures of fatigue, pain interference (PI), pain behavior (PB), mobility, upper extremity (UE), physical activity (PA), and strength impact (SI), and external validation measures. Log-transformed linear mixed-effects models with random patient intercepts were used to assess PROMIS T-score changes. Wilcoxon signed-rank test was performed to determine the PROMIS T-score changes among the improved, worsened, and unchanged groups. Spearman rank correlation test determined the relationship of PROMIS T-scores with disease status reported by patients/families. RESULTS: More than 1,000 clinical visits from 184 patients included PROMIS Pediatric measures entries. All PROMIS T-scores correlated significantly (P < 0.01) with patient-reported variables and physician global assessment (PHGA). The correlation between function and mobility, PB, and PI was good (r = 0.4-0.6). The correlation of patient-reported disease status was strong with PHGA (r = 0.75); moderate with mobility, PB, and PI; and weak with fatigue, SI, UE, and PA. The changes of PROMIS T-scores over time for mobility, PB, PA, and PI compared with the self-reported status change were significant (P < 0.05). After effective treatment, when clinical disease activity score improved by at least three points (n = 18), the change of PROMIS T-scores for mobility, PB, PI, and UE were significant (P < 0.05). CONCLUSION: This study provided evidence supporting the use of PROMIS Pediatric mobility, PB, and PI measures for CNO clinical disease monitoring.