Assessing responsiveness over time of the PROMIS (R) pediatric symptom and function measures in cancer, nephrotic syndrome, and sickle cell disease

• Type of Material: Article
• Authors: Bryce B. Reeve, University of North Carolina; Lloyd J. Edwards, University of North Carolina; Byron C. Jaeger, University of North Carolina; Pamela S. Hinds, Children's National Health System; Carlton Dampier, Emory University; Debbie S. Gipson, University of Michigan; David T. Selewski, University of Michigan; Jonathan P. Troost, University of Michigan; David Thissen, University of North Carolina; Vaughn Barry, Emory University; Heather E. Gross, University of North Carolina; Darren A. DeWalt, University of North Carolina
• Language: English
• Date: 2018-01-01
• Publisher: Springer Verlag (Germany)
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2017, Springer International Publishing AG.
• Final Published Version (URL): http://dx.doi.org/10.1007/s11136-017-1697-z
• Title of Journal or Parent Work: Quality of Life Research
• ISSN: 0962-9343
• Volume: 27
• Issue: 1
• Start Page: 249
• End Page: 257
• Grant/Funding Information: The contents of this article uses data developed under PROMIS.; PROMIS® was funded with cooperative agreements from the National Institutes of Health (NIH) Common Fund Initiative (Northwestern University, PI: David Cella, PhD, U54AR057951, U01AR052177; Northwestern University, PI: Richard C. Gershon, PhD, U54AR057943; American Institutes for Research, PI: Susan (San) D. Keller, PhD, U54AR057926; State University of New York, Stony Brook, PIs: Joan E. Broderick, PhD and Arthur A. Stone, PhD, U01AR057948, U01AR052170; University of Washington, Seattle, PIs: Heidi M. Crane, MD, MPH, Paul K. Crane, MD, MPH, and Donald L. Patrick, PhD, U01AR057954; University of Washington, Seattle, PI: Dagmar Amtmann, PhD, U01AR052171; University of North Carolina, Chapel Hill, PI: Harry A. Guess, MD, PhD (deceased), Darren A. DeWalt, MD, MPH, Bryce B. Reeve, PhD U01AR052181; Children’s Hospital of Philadelphia, PI: Christopher B. Forrest, MD, PhD, U01AR057956; Stanford University, PI: James F. Fries, MD, U01AR052158; Boston University, PIs: Alan Jette, PT, PhD, Stephen M. Haley, PhD (deceased), and David Scott Tulsky, PhD (University of Michigan, Ann Arbor), U01AR057929; University of California, Los Angeles, PIs: Dinesh Khanna, MD (University of Michigan, Ann Arbor) and Brennan Spiegel, MD, MSHS, U01AR057936; University of Pittsburgh, PI: Paul A. Pilkonis, PhD, U01AR052155; Georgetown University, PIs: Carol. M. Moinpour, PhD (Fred Hutchinson Cancer Research Center, Seattle) and Arnold L. Potosky, PhD, U01AR057971; Children’s Hospital Medical Center, Cincinnati, PI: Esi M. Morgan DeWitt, MD, MSCE, U01AR057940; University of Maryland, Baltimore, PI: Lisa M. Shulman, MD, U01AR057967; and Duke University, PI: Kevin P. Weinfurt, PhD, U01AR052186).; These contents do not necessarily represent an endorsement by the US Federal Government or PROMIS.; This study was funded by the National Institutes of Health (NIH) Common Fund Initiative (grant #: U01AR052181; U2CCA186878).
• Keywords: Life Sciences & Biomedicine; ADOLESCENTS; MEASUREMENT INFORMATION-SYSTEM; PATIENT-REPORTED OUTCOMES; Health Care Sciences & Services; Sickle cell disease; Cancer; MODEL; Responsiveness; Public, Environmental & Occupational Health; QUALITY-OF-LIFE; Science & Technology; Pediatrics; RESPONSE THEORY ANALYSIS; NEPHROLOGY CONSORTIUM; Nephrotic syndrome; ITEM BANK; CHILDREN; Patient-reported outcomes; SCALE; Health Policy & Services
• Research Categories: Health Sciences, Health Care Management; Health Sciences, Oncology; Biology, Biostatistics

Abstract

Purpose: Previous studies provided evidence for the validity of the PROMIS Pediatric measures in cross-sectional studies. This study evaluated the ability of the PROMIS Pediatric measures to detect change over time in children and adolescents with cancer, nephrotic syndrome (NS), or sickle cell disease (SCD). Methods: Participants (8–17 years) completed measures of fatigue, pain interference, anger, anxiety, depressive symptoms, mobility, upper extremity, and peer relationships at three or four time points (T1–T4). Between T1 and T2, children with cancer received chemotherapy and children with SCD experienced a pain exacerbation. Children with NS were first assessed during active disease (T2), with T3 and T4 conducted at disease remission. For the primary analysis of responsiveness, we expected better scores at T3 (recovery) compared to T2 (event) for all diseases. T1 and T4 are also expected to have better scores than T2. Linear mixed models were used and adjusted for time, gender, age, race/ethnicity, education, comorbid conditions, and disease. Results: Enrolled were 96 children with cancer, 121 children with SCD, and 127 children with NS. Fatigue, pain interference, mobility, and upper extremity scores worsened from T1 (baseline) to T2 (event) (p < 0.01), and significantly improved from T2 to T3 and T4 (p < 0.01). Similarly, anxiety and depressive symptoms significantly improved from T2 to T3 and T4 (p < 0.01). Conclusions: This study provides evidence for the responsiveness of seven PROMIS Pediatric measures to clinical disease state in three chronic illnesses. The findings support use of PROMIS Pediatric measures in clinical research.