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

Health and Quality of Life Outcomes

Research

Yi Cai¹  Rasheed A. Gbadegesin²  Howard Trachtman³  Larry A. Greenbaum⁴  Noelle E. Carlozzi⁵  Yang Liu⁶  Jens Goebel⁷  Michael J. Somers⁸  Jonathan P. Troost⁹  Emily Herreshoff⁹  David T. Selewski⁹  Debbie S. Gipson⁹  Danyelle Cummings⁹  Diane Hebert^1,10  Christine B. Sethna^1,11  Michael E. Seifert^1,12  Susan F. Massengill^1,13  John D. Mahan^1,14  Gaurav Kapur^1,15  Priya Pais^1,16  Ibrahim F. Shatat^1,17  Heather E. Gross^1,18  Darren A. DeWalt^1,18  Bryce B. Reeve^1,18 

[1] DeVos Children’s Hospital, Grand Rapids, MI, USA;Department of Pediatrics, Division of Nephrology, Duke University Medical Center, Durham, NC, USA;Department of Pediatrics, Division of Nephrology, New York University Langone Medical Center, New York, NY, USA;Department of Pediatrics, Emory University and Children’s Healthcare of Atlanta, Atlanta, GA, USA;Department of Physical Medicine and Rehabilitation, University of Michigan, Ann Arbor, USA;Department of Psychology, University of North Carolina, Chapel Hill, NC, USA;Division of Nephrology and Hypertension, Cincinnati Children’s Hospital, Cincinnati, OH, USA;Division of Nephrology, Boston Children’s Hospital, Harvard Medical School, Boston, MA, USA;Division of Nephrology, Department of Pediatrics & Communicable Diseases, C.S. Mott Children’s Hospital, University of Michigan, 1500 E Medical Center Drive, SPC5297, 48109-5297, Ann Arbor, MI, USA;Division of Nephrology, The Hospital for Sick Children, Toronto, ON, Canada;Division of Pediatric Nephrology, Cohen Children’s Medical Center of New York, New Hyde Park, NY, USA;Division of Pediatric Nephrology, Department of Pediatrics, University of Alabama Birmingham, Birmingham, AL, USA;Division of Pediatric Nephrology, Levine Children’s Hospital, Charlotte, NC, USA;Nationwide Children’s Hospital, The Ohio State University, College of Medicine, Columbus, OH, USA;Pediatric Nephrology and Hypertension Division, Children’s Hospital of Michigan, Detroit, MI, USA;Pediatric Nephrology and Hypertension, Medical College of Wisconsin, Milwaukee, WI, USA;Pediatric Nephrology and Hypertension, Sidra Medical and Research Center, Doha, Qatar;University of North Carolina, Chapel Hill, NC, USA;

关键词: Patient-reported outcomes;    Quality of life;    Nephrotic syndrome;    Pediatrics;    Children;   

DOI  :  10.1186/s12955-017-0737-2

 received in 2017-04-06, accepted in 2017-08-07,  发布年份 2017

来源: Springer

【 摘 要 】

BackgroundNephrotic 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.MethodsOne hundred twenty seven children with nephrotic syndrome between the ages of 8 and 17 years 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.ResultsDisease 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.ConclusionThis 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.

【 授权许可】

CC BY   
© The Author(s). 2017