Responsiveness of the PROMIS (R) measures to changes in disease status among pediatric nephrotic syndrome patients: a Midwest pediatric nephrology consortium study

• Type of Material: Article
• Authors: David T. Selewski, University of Michigan; Jonathan P. Troost, University of Michigan; Danyelle Cummings, University of Michigan; Susan F. Massengill, Levine Children's Hospital; Rasheed A. Gbadegesin, Duke University; Larry Greenbaum, Emory University; Ibrahim F. Shatat, Sidra Medical and Research Center; Yi Cai, DeVos Children's Hospital; Gaurav Kapur, Children’s Hospital of Michigan; Diane Hebert, The Hospital for Sick Children; Michael J. Somers, Boston Children's Hospital; Howard Trachtman, New York University Langone Medical Center; Priya Pais, Medical College of Wisconsin; Michael E. Seifert, University of Alabama Birmingham; Jens Goebel, Cincinnati Children's Hospital; Christine B. Sethna, Cohen Children’s Medical Center of New York; John D. Mahan, Ohio State University; Heather E. Gross, Uni North Carolina; Emily Herreshoff, University of Michigan; Yang Liu, University of North Carolina; Noelle E. Carlozzi, University of Michigan; Bryce B. Reeve, University of North Carolina; Darren A. DeWalt, University of North Carolina; Debbie S. Gipson, University of Michigan
• Language: English
• Date: 2017-08-23
• Publisher: BioMed Central
• Publication Version: Final Published Version
• Copyright Statement: © 2017 The Author(s).
• License: Creative Commons Attribution 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1186/s12955-017-0737-2
• Title of Journal or Parent Work: Health and Quality of Life Outcomes
• ISSN: 1477-7525
• Volume: 15
• Issue: 1
• Start Page: 166
• End Page: 166
• Grant/Funding Information: The Patient-Reported Outcomes Measurement Information System® (PROMIS®) is an NIH Roadmap initiative to develop valid and reliable measures of patient-reported outcomes in individuals with a wide range of chronic diseases and demographic characteristics.; PROMIS II was funded by cooperative agreements with a Statistical Center (Northwestern University, PI: David Cella, PhD, 1U54AR057951), a Technology Center (Northwestern University, PI: Richard C. Gershon, PhD, 1U54AR057943), a Network Center (American Institutes for Research, PI: Susan (San) D. Keller, PhD, 1U54AR057926) and thirteen Primary Research Sites which may include more than one institution (State University of New York, Stony Brook, PIs: Joan E. Broderick, PhD and Arthur A. Stone, PhD, 1U01AR057948; University of Washington, Seattle, PIs: Heidi M. Crane, MD, MPH, Paul K. Crane, MD, MPH, and Donald L. Patrick, PhD, 1U01AR057954; University of Washington, Seattle, PIs: Dagmar Amtmann, PhD and Karon Cook, PhD, 1U01AR052171; University of North Carolina, Chapel Hill, PI: Darren A. DeWalt, MD, MPH, 2U01AR052181; Children’s Hospital of Philadelphia, PI: Christopher B. Forrest, MD, PhD, 1U01AR057956; Stanford University, PI: James F. Fries, MD, 2U01AR052158; Boston University, PIs: Stephen M. Haley, PhD and David Scott Tulsky, PhD (University of Michigan, Ann Arbor), 1U01AR057929; University of California, Los Angeles, PIs: Dinesh Khanna, MD and Brennan Spiegel, MD, MSHS, 1U01AR057936; University of Pittsburgh, PI: Paul A. Pilkonis, PhD, 2U01AR052155; 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, 1U01AR057940; University of Maryland, Baltimore, PI: Lisa M. Shulman, MD, 1U01AR057967; and Duke University, PI: Kevin P. Weinfurt, PhD, 2U01AR052186).
• Keywords: SCALE; Nephrotic syndrome; REPORTED OUTCOMES; Quality of life; Science & Technology; Patient-reported outcomes; CHILDREN; RESPONSE THEORY ANALYSIS; QUALITY-OF-LIFE; Pediatrics; Life Sciences & Biomedicine; Health Policy & Services; Children; IMPORTANT DIFFERENCE; HEALTH-STATUS; Health Care Sciences & Services; IMPACT; PERSPECTIVE; INFORMATION-SYSTEM PROMIS
• Research Categories: Health Sciences, Medicine and Surgery

Abstract

Background: Nephrotic syndrome represents a condition in pediatric nephrology typified by a relapsing and remitting course, proteinuria and the presence of edema. The PROMIS measures have previously been studied and validated in cross-sectional studies of children with nephrotic syndrome. This study was designed to longitudinally validate the PROMIS measures in pediatric nephrotic syndrome. Methods: One hundred twenty seven children with nephrotic syndrome between the ages of 8 and 17years participated in this prospective cohort study. Patients completed a baseline assessment while their nephrotic syndrome was active, a follow-up assessment at the time of their first complete proteinuria remission or study month 3 if no remission occurred, and a final assessment at study month 12. Participants completed six PROMIS measures (Mobility, Fatigue, Pain Interference, Depressive Symptoms, Anxiety, and Peer Relationships), the PedsQL version 4.0, and two global assessment of change items. Results: Disease status was classified at each assessment: nephrotic syndrome active in 100% at baseline, 33% at month 3, and 46% at month 12. The PROMIS domains of Mobility, Fatigue, Pain Interference, Depressive Symptoms, and Anxiety each showed a significant overall improvement over time (p < 0.001). When the PROMIS measures were compared to the patients' global assessment of change, the domains of Mobility, Fatigue, Pain Interference, and Anxiety consistently changed in an expected fashion. With the exception of Pain Interference, change in PROMIS domain scores did not correlate with changes in disease activity. PROMIS domain scores were moderately correlated with analogous PedsQL domain scores. Conclusion: This study demonstrates that the PROMIS Mobility, Fatigue, Pain Interference, and Anxiety domains are sensitive to self-reported changes in disease and overall health status over time in children with nephrotic syndrome. The lack of significant anchoring to clinically defined nephrotic syndrome disease active and remission status may highlight an opportunity to improve the measurement of HRQOL in children with nephrotic syndrome through the development of a nephrotic syndrome disease-specific HRQOL measure.