Establishing clinical meaning and defining important differences for Patient-Reported Outcomes Measurement Information System (PROMIS®) measures in juvenile idiopathic arthritis using standard setting with patients, parents, and providers

Abstract

Background Patient Reported Outcomes Measurement Information System (PROMIS) measures are used increasingly in clinical care. However, for juvenile idiopathic arthritis (JIA), scores lack a framework for interpretation of clinical severity, and minimally important differences (MID) have not been established, which are necessary to evaluate the importance of change. Methods We identified clinical severity thresholds for pediatric PROMIS measures of mobility, upper extremity function (UE), fatigue, and pain interference working with adolescents with JIA, parents of JIA patients, and clinicians, using a standard setting methodology modified from educational testing. Item parameters were used to develop clinical vignettes across a range of symptom severity. Vignettes were ordered by severity, and panelists identified adjacent vignettes considered to represent upper and lower boundaries separating category cut points (i.e., from none/mild problems to moderate/severe). To define MIDs, panelists reviewed a full score report for the vignettes and indicated which items would need to change and by how much to represent “just enough improvement to make a difference”. Results For fatigue and UE, cut points among panels were within 0.5 SD of each other. For mobility and pain interference, cut-scores among panels were more divergent, with parents setting the lowest cut-scores for increasing severity. The size of MIDs varied by stakeholders (parents estimated largest, followed by patients, then clinicians). MIDs also varied by severity classification of the symptom. Conclusions We estimated clinically relevant severity cut points and MIDs for PROMIS measures for JIA from the perspectives of multiple stakeholders and found notable differences in perspectives.

Can Patient-Reported Outcomes Measurement Information System® (PROMIS) measures accurately enhance understanding of acceptable symptoms and functioning in primary care?

“…Affirming the current categories, the PASS thresholds for PF, PI, and Fatigue essentially align with mild or moderate severity, depending on the measure. Multiple studies have used patient-informed benchmarking techniques for PROMIS [9,45,46], and discrepancies between patient and provider definitions of severity have been reported [9]. This study reinforces the need to establish PROMIS T-score thresholds in alignment with patient health experiences, toward improved interpretability for providers.…”

Pediatric pain screening identifies youth at risk of chronic pain in sickle cell disease

“…Each standardized measure was dichotomized based on established cutoffs to determine “cases” and “noncases.” Case or noncase was determined for each measure independently. Established reference standard cases were defined as pain frequency ≥ 15 days/month or SCPBI item 1 ≥ 3 (i.e., “many” or “every” days/month); pain duration ≥ 6 months; FDI ≥ 13; Pain Interference T‐Score ≥ 65; CDI‐2 T‐score ≥ 65; PCS‐C ≥ 26; FOPQ‐C ≥ 51; admissions ≥ 3; or ED visits ≥3 . Responses that did not meet the established cutoff for a measure were categorized as noncases for that measure.…”

Parent-reported outcomes in young children with disorders/differences of sex development

“…As such, scores on these latter measures can be used to compare adaptation and the influence of care strategies across specific DSD populations and against patients with other chronic paediatric conditions. Aggregating such data longitudinally and across multiple centres will create the opportunity for developing robust reference data which can be employed as clinical benchmarks [38] useful in guiding a process of continuous quality improvement in the care of these patients and their families.…”