The longitudinal relationship between patient-reported outcomes and clinical characteristics among patients with focal segmental glomerulosclerosis in the Nephrotic Syndrome Study Network
Author:
Troost Jonathan P1, Waldo Anne1, Carlozzi Noelle E2, Murphy Shannon3, Modersitzki Frank4, Trachtman Howard5, Nachman Patrick H6, Reidy Kimberly J7, Selewski David T8, Herreshoff Emily G1, Srivastava Tarak9, Gibson Keisha L3, Derebail Vimal K3, Lin Jen Jar10, Hingorani Sangeeta11, Fornoni Alessia12, Fervenza Fernando C13, Sambandam Kamalanathan14, Athavale Ambarish M15, Kopp Jeffrey B16, Reich Heather N17, Adler Sharon G18, Greenbaum Larry A19, Dell Katherine M20, Appel Gerald21, Wang Chia-shi19ORCID, Sedor John22, Kaskel Frederick J7, Lafayette Richard A23, Atkinson Meredith A24, Lieske John C13, Sethna Christine B25, Kretzler Matthias26, Hladunewich Michelle A17, Lemley Kevin V27, Brown Elizabeth28, Meyers Kevin E29, Gadegbeku Crystal A30, Holzman Lawrence B31, Jefferson Jonathan Ashley32, Tuttle Katherine R3334, Singer Pamela25, Hogan Marie C13, Cattran Daniel C35, Barisoni Laura3637, Gipson Debbie S1, Sedor J, Dell K, Schachere M, Lemley K, Whitted L, Srivastava T, Haney C, Sethna C, Gurusinghe S, Appel G, Toledo M, Greenbaum L, Wang C, Lee B, Adler S, Nast C, La Page J, Stroger John H, Athavale A, Itteera M, Neu A, Boynton S, Fervenza F, Hogan M, Lieske J, Chernitskiy V, Kaskel F, Reidy K, Kopp J, Castro-Rubio E, Brede E, Blake J, Trachtman H, Zhdanova O, Modersitzki F, Vento S, Lafayette R, Mehta K, Gadegbeku C, Johnstone D, Pfeffer Z, Cattran D, Hladunewich M, Reich H, Ling P, Romano M, Fornoni A, Barisoni L, Bidot C, Kretzler M, Gipson D, Williams A, Pitter R, Derebail V, Gibson K, Grubbs S, Froment A, Holzman L, Meyers K, Kallem K, Cerecino F J, Sambandam K, Brown E, Johnson N, Jefferson A, Hingorani S, Tuttle K, Klepach K, Kelton M, Cooper A, Freedman B, Lin J J, Spainhour M, Gray S, Kretzler M, Barisoni L, Gadegbeku C, Gillespie B, Gipson D, Holzman L, Mariani L, Sampson M, Song P, Troost J, Zee J, Herreshoff E, Kincaid C, Lienczewski C, Mainieri T, Williams A, Avila-Casado C, Bagnasco S, Gaut J, Hewitt S, Hodgin J, Lemley K, Mariani L, Palmer M, Rosenberg A, Royal V, Thomas D, Zee J, Barisoni L, Nast C, Abbott K, Roy C, Urv T, Brooks P J, Sedor J, Dell K, Schachere M, Lemley K, Whitted L, Srivastava T, Haney C, Sethna C, Gurusinghe S, Appel G, Toledo M, Greenbaum L, Wang C, Lee B, Adler S, Nast C, La Page J, Stroger John H, Athavale A, Itteera M, Neu A, Boynton S, Fervenza F, Hogan M, Lieske J, Chernitskiy V, Kaskel F, Reidy K, Kopp J, Castro-Rubio E, Brede E, Blake J, Trachtman H, Zhdanova O, Modersitzki F, Vento S, Lafayette R, Mehta K, Gadegbeku C, Johnstone D, Pfeffer Z, Cattran D, Hladunewich M, Reich H, Ling P, Romano M, Fornoni A, Barisoni L, Bidot C, Kretzler M, Gipson D, Williams A, Pitter R, Derebail V, Gibson K, Grubbs S, Froment A, Holzman L, Meyers K, Kallem K, Cerecino F J, Sambandam K, Brown E, Johnson N, Jefferson A, Hingorani S, Tuttle K, Klepach K, Kelton M, Cooper A, Freedman B, Lin J J, Spainhour M, Gray S, Kretzler M, Barisoni L, Gadegbeku C, Gillespie B, Gipson D, Holzman L, Mariani L, Sampson M, Song P, Troost J, Zee J, Herreshoff E, Kincaid C, Lienczewski C, Mainieri T, Williams A, Avila-Casado C, Bagnasco S, Gaut J, Hewitt S, Hodgin J, Lemley K, Mariani L, Palmer M, Rosenberg A, Royal V, Thomas D, Zee J, Barisoni L, Nast C, Abbott K, Roy C, Urv T, Brooks P J,
Affiliation:
1. Department of Pediatrics, Division of Nephrology, University of Michigan, Ann Arbor, MI, USA 2. Department of Physical Medicine and Rehabilitation, University of Michigan, Ann Arbor, MI, USA 3. University of North Carolina Kidney Center at Chapel Hill, Chapel Hill, NC, USA 4. Department of Medicine, Division of Nephrology, New York University Langone Health, New York, NY, USA 5. Department of Pediatrics, Division of Nephrology, New York University Langone Health, New York, NY, USA 6. Division of Renal Diseases and Hypertension, University of Minnesota, Minneapolis, MN, USA 7. Department of Pediatrics, Division of Nephrology, Albert Einstein College of Medicine, Montefiore Medical Center, New York, NY, USA 8. Department of Pediatrics, Division of Nephrology, Medical University of South Carolina, Charleston, SC, USA 9. Section of Nephrology, Children's Mercy Hospital and University of Missouri at Kansas City, Kansas City, MO, USA 10. Division of Pediatric Nephrology, Brenner Children's Hospital, Wake Forest University, Winston-Salem, NC, USA 11. Department of Pediatrics, Division of Nephrology, Seattle Children's Hospital and University of Washington, Seattle, WA, USA 12. Department of Medicine, Katz Family Division of Nephrology and Hypertension, University of Miami Miller School of Medicine, Miami, FL, USA 13. Division of Nephrology and Hypertension, Mayo Clinic, Rochester, MN, USA 14. Department of Internal Medicine, Division of Nephrology, UT Southwestern Medical Center, Dallas, TX, USA 15. Division of Nephrology, Core Faculty, Internal Medicine Residency Program, John H. Stroger Jr. Hospital of Cook County, Chicago, IL, USA 16. Kidney Disease Section, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA 17. Department of Medicine, Division of Nephrology, Sunnybrook Health Sciences Centre, University of Toronto, Toronto, ON, Canada 18. Division of Nephrology and Hypertension, Los Angeles Biomedical Research Institute at Harbor-University of California Los Angeles Medical Center, Torrance, CA, USA 19. Department of Pediatrics, Division of Pediatric Nephrology, Emory University School of Medicine and Children's Healthcare of Atlanta, Atlanta, GA, USA 20. Center for Pediatric Nephrology, Cleveland Clinic Children's and Case Western Reserve University, Cleveland, OH, USA 21. Division of Nephrology, Columbia University Medical Center, New York, NY, USA 22. Department of Nephrology, Cleveland Clinic, Cleveland, OH, USA 23. Department of Medicine, Division of Nephrology, Stanford University, Stanford, CA, USA 24. Division of Pediatric Nephrology, Johns Hopkins University School of Medicine, Baltimore, MD, USA 25. Pediatric Nephrology, Cohen Children's Medical Center of New York, Zucker School of Medicine at Hofstra/Northwell, Hempstead, NY, USA 26. Department of Internal Medicine, Division of Nephrology, University of Michigan, Ann Arbor, MI, USA 27. Division of Nephrology, Children's Hospital Los Angeles, Los Angeles, CA, USA 28. Department of Pediatrics, Division of Nephrology, University of Texas Southwestern Medical Center, Dallas, TX, USA 29. Department of Pediatrics, Division of Nephrology, Children's Hospital of Philadelphia, Philadelphia, PA, USA 30. Division of Nephrology, Temple University School of Medicine, Philadelphia, PA, USA 31. Renal-Electrolyte and Hypertension Division, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA 32. Department of Medicine, Division of Nephrology, University of Washington, Seattle, WA, USA 33. Providence Health Care, Providence Medical Research Center, Spokane, WA, USA 34. Nephrology Division, Kidney Research Institute and Institute for Translational Health Sciences, University of Washington, Seattle, WA, USA 35. Department of Medicine, Division of Nephrology, University Health Network, University of Toronto, Toronto, ON, Canada 36. Department of Pathology, Division of Nephrology, Duke University, Durham, NC, USA 37. Department of Medicine, Division of Nephrology, Duke University, Durham, NC, USA
Abstract
Abstract
Background
Understanding the relationship between clinical and patient-reported outcomes (PROs) will help support clinical care and future clinical trial design of novel therapies for focal segmental glomerulosclerosis (FSGS).
Methods
FSGS patients ≥8 years of age enrolled in the Nephrotic Syndrome Study Network completed Patient-Reported Outcomes Measurement Information System PRO measures of health-related quality of life (HRQoL) (children: global health, mobility, fatigue, pain interference, depression, anxiety, stress and peer relationships; adults: physical functioning, fatigue, pain interference, sleep impairment, mental health, depression, anxiety and social satisfaction) at baseline and during longitudinal follow-up for a maximum of 5 years. Linear mixed-effects models were used to determine which demographic, clinical and laboratory features were associated with PROs for each of the eight children and eight adults studied.
Results
There were 45 children and 114 adult FSGS patients enrolled that had at least one PRO assessment and 519 patient visits. Multivariable analyses among children found that edema was associated with global health (−7.6 points, P = 0.02) and mobility (−4.2, P = 0.02), the number of reported symptoms was associated with worse depression (−2.7 per symptom, P = 0.009) and anxiety (−2.3, P = 0.02) and the number of emergency room (ER) visits in the prior 6 months was associated with worse mobility (−2.8 per visit, P < 0.001) and fatigue (−2.4, P = 0.03). Multivariable analyses among adults found the number of reported symptoms was associated with worse function in all eight PROMIS measures and the number of ER visits was associated with worse fatigue, pain interference, sleep impairment, depression, anxiety and social satisfaction. Laboratory markers of disease severity (i.e. proteinuria, estimated glomerular filtration rate and serum albumin) did not predict PRO in multivariable analyses, with the single exception of complete remission and better pain interference scores among children (+9.3, P = 0.03).
Conclusions
PROs provide important information about HRQoL for persons with FSGS that is not captured solely by the examination of laboratory-based markers of disease. However, it is critical that instruments capture the patient experience and FSGS clinical trials may benefit from a disease-specific instrument more sensitive to within-patient changes.
Funder
Nephrotic Syndrome Study Network Consortium National Institutes of Health Rare Disease Clinical Research Network Office of Rare Diseases Research National Center for Advancing Translational Sciences NIDDK University of Michigan NephCure Kidney International Halpin Foundation Charles Woodson Pilot Research Award University of Michigan Department of Pediatrics
Publisher
Oxford University Press (OUP)
Subject
Transplantation,Nephrology
Cited by
12 articles.
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