The longitudinal relationship between patient-reported outcomes and clinical characteristics among patients with focal segmental glomerulosclerosis in the Nephrotic Syndrome Study Network-Reference-Cited by-同舟云学术

The longitudinal relationship between patient-reported outcomes and clinical characteristics among patients with focal segmental glomerulosclerosis in the Nephrotic Syndrome Study Network

Published:2019-08-05 Issue:4 Volume:13 Page:597-606
ISSN:2048-8513
Container-title:Clinical Kidney Journal
language:en
Short-container-title:

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-shi¹⁹^ORCID,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