Ability of PROMIS Pediatric Measures to Detect Change in Children With Cerebral Palsy Undergoing Musculoskeletal Surgery

Abstract

Background The Patient Reported Outcomes Measurement Information System (PROMIS®) was developed to provide patient-reported outcome (PROs) measures that are designed as being universally relevant across health conditions, low burden and precise. A major problem for research and clinical practice in cerebral palsy (CP) is the void of outcomes instruments that are capable of evaluating the wide range of abilities and broad age spectrum inherent in this clinical population. Given the tremendous potential of PROMIS® , the research questions for this study were “How do PROMIS® pediatric computer adaptive tests (CAT) and short forms (SF) detect change in children with CP following elective musculoskeletal surgery?” and “How do PROMIS® instruments compare to the Pediatric Quality of Life Inventory™ Cerebral Palsy Module Version 3.0 (PedsQL CP), Pediatric Outcomes Data Collection Instrument (PODCI), the Timed Up and Go (TUG) and the Gross Motor Functional Measure (GMFM). Methods PROMIS® Pediatric CATs and SFs and the PedsQL, PODCI, TUG and GMFM were administered before and after surgery. Effect Size (ES) and Standardized Response Mean (SRM) were calculated. Floor and ceiling effects were evaluated and, exposure rates for the PROMIS® item banks were examined. Results ES and SRM for all PROMIS® Pediatric Measures were non-significant. PedsQL CP detected significant, positive change in mobility at 6 (ES =0.26; SRM = 0.31) and 12 (ES = 0.36; SRM=0.36) months; pain at 12 months (ES=0.29; SRM=0.34); and fatigue at 6 (ES=0.24; SRM=0.22) and 12 (ES=0.36; SRM=0.41) months. Significant negative changes were detected by the PODCI (ES= - 0.20; SRM= - 0.26), GMFM (ES= - 0.13; SRM = -0.24) and TUG (ES= - 0.29; SRM= - 0.25). Ceiling effects were high. Exposure to an appropriate range of the PROMIS® Mobility item bank was limited. Conclusion PROMIS® measures were less able to detect change than other measures. PROMIS® measures may be improved by tailoring start\stop rules or by adding items to include content appropriate for children with mobility impairments. Level of Evidence Diagnostic Study, Level III

Differential item functioning in the Patient Reported Outcomes Measurement Information System Pediatric Short Forms in a sample of children and adolescents with cerebral palsy

“…The Wald χ 2 test was used to examine the equality of item parameters (including discrimination and threshold) across groups and identify DIF items. Because this method for DIF detection requires multiple tests of significance, we applied the Benjamini–Hochberg adjustment 6, 12 to control for Type 1 error in multiple comparisons. Items without DIF identified in this step were used as anchor items in the next step.…”

Patient-reported outcomes of pain and physical functioning in neurofibromatosis clinical trials

“…Good agreement between child self-report and parent proxy (upper extremity r 5 0.69; mobility r 5 0.63) 44 Sensitive to change after spine augmentation in adults 28 ; no change after surgery in children with cerebral palsy 29 CAT version less accurate then the SF in children with cerebral palsy 38 ; CAT was more precise than SF but CAT administered more items to use the longest PF SF that is feasible for a particular study (e.g., 20-item SF was the most responsive in populations with physical impairments) 37 or the CAT in adult studies. The CAT alone is not recommended for pediatric studies at this time since it was less accurate than the SF in children with functional limitations.…”

“…Good preliminary psychometric data in adult patients 23 Responsive to change in adults 28 ; no changed detected in one pediatric longitudinal study to date 29 SF and CAT highly correlated; CAT often more precise than SF in adults 36 ; SF generally more precise than CAT in one pediatric study appropriate for consideration; 5 met basic criteria to be rated. Of these, the PROMIS Physical Functioning scale (PROMIS-PF), 25 Health Assessment Questionnaire, 31 and Functional Disability Inventory 32 were the toprated measures.…”

“…20,26,27 The PROMIS-PI also has been shown to be responsive to changes in pain in a limited number of clinical trials with adults 28 but not yet with children. 29 Published studies using both the pediatric selfreport and parent-proxy pain interference scales currently are scarce. The pediatric and parent forms are parallel and general parent-child agreement is moderate.…”

“…38 In clinical trials, the PROMIS-PF showed sensitivity to change after spine augmentation in adults 28 but not after surgery in children with cerebral palsy. 29 Physical functioning recommendation. Although published studies have not used the PROMIS-PF in NF to date, the evaluation of both upper and lower extremity function as well as the range, content, and wording of the items are well-suited to assess the spectrum of complications affecting physical function in NF.…”