Identifying Minimal and Meaningful Change in a Patient‐Reported Outcomes Measurement Information System for Rheumatoid Arthritis: Use of Multiple Methods and Perspectives

Abstract

Objective Rheumatoid arthritis (RA) is chronic, painful, disabling condition resulting in significant impairments in physical, emotional, and social health. Our objective was to use different methods and perspectives to evaluate the responsiveness of Patient‐Reported Outcomes Measurement Information System (PROMIS) short forms (SFs) and to identify minimal and meaningful score changes. Methods Adults with RA who were enrolled in a multisite prospective observational cohort completed PROMIS physical function, pain interference, fatigue, and participation in social roles/activities SFs, the PROMIS 29‐item form (PROMIS‐29), and pain and patient global assessment, and rated change in specific symptoms and RA (a little versus lot better or worse) at the second visit. Physicians recorded joint counts, physician global assessment, and change in RA at visit 2. We compared mean score differences for minimal and meaningful improvement/worsening using patient and physician change ratings and distribution‐based methods, and we visually inspected empirical cumulative distribution function curves by change categories. Results The 348 adults were mostly female (81%) with longstanding RA. Using patient ratings, generally 1–3‐point differences were observed for minimal change and 3–7 points for meaningful change. Larger differences were observed with patient versus physician ratings and for symptom‐specific versus RA change. Mean differences were similar among SF versions. Prespecified hypotheses about change in PROMIS physical function, pain interference, fatigue, and participation and legacy scales were supported. Conclusion PROMIS SFs and the PROMIS‐29 profiles are responsive to change and generally distinguish between minimal and meaningful improvement and worsening in key RA domains. These data add to a growing body of evidence demonstrating the robust psychometric properties of PROMIS and supporting its use in RA care, research, and decision‐making.

Meaningful Change Thresholds for Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue and Pain Interference Scores in Patients With Rheumatoid Arthritis

“…21 Moreover, using adults with RA enrolled in a multisite observation cohort, a recent study also found a 1-3 points change in PROMIS scores for several PRO measures when patients felt a little better or a little worse. 22 In addition, the mean and IQRs of the PROMIS t-score for fatigue and pain interference scores in our study cohort do not indicate an issue with floor or ceiling effects.…”

Meaningful Improvement in General Health Outcomes with Guselkumab Treatment for Psoriatic Arthritis: Patient-Reported Outcomes Measurement Information System-29 Results from a Phase 3 Study

“…However, it is noteworthy that reported minimally important differences vary across the different PROMIS domains and across different patient populations. For example, in patients with rheumatoid arthritis, changes as small as 1-2 points are associated with noticeable improvements in domains such as fatigue and pain interference [15,24,32], and meaningful change thresholds for these domains have been defined as 2-3 points [34]. Minimal important differences have been reported to be approximately 2 points for PROMIS domains of physical function, fatigue, pain interference, and social participation in patients with systemic lupus erythematosus [33] and to be approximately 2 points for physical function and pain interference in patients with knee osteoarthritis or other chronic musculoskeletal pain [19,25].…”

“…Modified cumulative distribution curves [23] were generated to show the proportions of patients achieving various levels of improvement in each PROMIS-29 domain T-score from ≥ 1 to ≥ 21 points at Week 24 by treatment group. Based on published studies estimating clinically meaningful changes and minimally important differences in PROMIS domain scores in patients with rheumatic and musculoskeletal disorders [15,[24][25][26][27], two prespecified thresholds (≥ 5and ≥ 3-point improvement) were used to define clinically meaningful changes in each domain T-score. At Week 24, achievement of point improvement thresholds was calculated for all patients who had not met treatment failure criteria; patients with missing data were considered non-responders.…”

“…The PROMIS-29 Profile includes 29 items assessing the following seven domains of health: physical function, pain interference, fatigue, sleep disturbance, satisfaction with participation in social roles and activities, depression, and anxiety; plus a single pain-intensity numeric rating scale (NRS) [11]. Validated PROMIS Profile measures are increasingly being used in clinical research and in real-world practice to evaluate general health outcomes that affect quality of life, especially in patients with rheumatic and musculoskeletal diseases [11][12][13][14][15][16][17][18][19].…”

Response to Treatment with Intravenous Golimumab or Infliximab in Rheumatoid Arthritis Patients: PROMIS Results from the Real-World Observational Phase 4 AWARE Study

“…The Patient-Reported Outcomes Measurement Information System (PROMIS Ò ), developed by the National Institutes of Health [11,12], is a disease-agnostic set of health assessment instruments designed to efficiently evaluate and monitor HRQoL across numerous aspects of physical, emotional, and social health [11][12][13]. PROMIS can be used to assess the health status of individuals in the general population as well as in those with chronic conditions and has been validated in patients with RA [14,15]. PROMIS measures have greater precision than the 36-item Short-Form Health Survey [SF-36] and improved responsiveness to change when assessing physical function [16].…”