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

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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.

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Most cited references 18

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Design of the Nephrotic Syndrome Study Network (NEPTUNE) to evaluate primary glomerular nephropathy by a multi-disciplinary approach

Crystal Gadegbeku, Debbie S. Gipson, Larry Holzman … (2013)

The Nephrotic Syndrome Study Network (NEPTUNE) is a North American multi-center collaborative consortium established to develop a translational research infrastructure for Nephrotic Syndrome. This includes a longitudinal observational cohort study, a pilot and ancillary studies program, a training program, and a patient contact registry. NEPTUNE will enroll 450 adults and children with minimal change disease, focal segmental glomerulosclerosis and membranous nephropathy for detailed clinical, histopathologic, and molecular phenotyping at the time of clinically-indicated renal biopsy. Initial visits will include an extensive clinical history, physical examination, collection of urine, blood and renal tissue samples, and assessments of quality of life and patient-reported outcomes. Follow-up history, physical measures, urine and blood samples, and questionnaires will be obtained every 4 months in the first year and bi-annually, thereafter. Molecular profiles and gene expression data will be linked to phenotypic, genetic, and digitalized histologic data for comprehensive analyses using systems biology approaches. Analytical strategies were designed to transform descriptive information to mechanistic disease classification for Nephrotic Syndrome and to identify clinical, histological, and genomic disease predictors. Thus, understanding the complexity of the disease pathogenesis will guide further investigation for targeted therapeutic strategies.

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Focal and segmental glomerulosclerosis: definition and relevance of a partial remission.

Stéphan Troyanov, Catherine Wall, Judith Miller … (2005)

Focal and segmental glomerulosclerosis (FSGS) is one of the most common primary glomerular diseases to terminate in ESRD. A complete remission (CR) confers an excellent long-term prognosis, but the quantitative benefits of partial remissions (PR) have not been defined. This study evaluated the rate of renal function decline (slope of creatinine clearance) and renal survival in nephrotic FSGS patients with CR, PR, or no remission. It also examined relapse rate from remission and its impact on outcome. Multivariate analysis included clinical and laboratory data at presentation and over follow-up, BP control, the agents used, and immunosuppressive therapy. The study cohort was 281 nephrotic FSGS patients who had a minimum of 12 mo of observation and were identified from the Toronto Glomerulonephritis Registry. Over a median follow-up of 65 mo, 55 experienced a CR, 117 had a PR, and 109 had no remission. A PR was independently predictive of slope and survival from renal failure by multivariate analysis (adjusted time-dependent hazard ratio, 0.48; 95% confidence interval, 0.24 to 0.96; P = 0.04). Immunosuppression with high-dose prednisone was associated with a higher rate of PR and CR. Relapse from PR was frequent (56%) and associated with a more rapid rate of renal function decline and worse renal survival compared with relapse-free partial remitters. Only female gender and the nadir of proteinuria during remission were associated with a sustained remission. A PR in proteinuria and its maintenance are important therapeutic targets in FSGS, with implications for both slowing progression rate and improving renal survival.

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Rituximab treatment of adult patients with steroid-resistant focal segmental glomerulosclerosis.

S Alexandru, Ester González, Gema Fernández-Fresnedo … (2009)

Isolated case reports have shown a beneficial effect of rituximab on pediatric patients with primary FSGS, but there is no information about rituximab treatment of FSGS in adults. All patients who had biopsy-proven FSGS and were treated with rituximab in Spain were identified, independent of their positive or negative response, among the nephrology departments that belong to the Spanish Group for the Study of Glomerular Diseases (GLOSEN). Their characteristics and outcome after rituximab treatment were studied. Eight patients were identified. Rituximab failed to improve nephrotic syndrome in five of eight patients, who continued to show massive proteinuria and exhibited a rapidly deteriorating renal function in two cases. Among the remaining three patients, two of them showed an improvement of renal function and a remarkable proteinuria reduction and one experienced a beneficial but transitory effect after rituximab. There were no differences in clinical or laboratory characteristics or in the CD20 B lymphocyte count after rituximab between these three patients and the five who had a negative response. The only difference was in the regimen of rituximab administration: Whereas the five patients with a negative response received only four weekly consecutive infusions of 375 mg/m(2), the three remaining patients received additional doses of rituximab. Only a minority (three of eight) of patients in our series of adult patients with FSGS showed a positive influence of rituximab. More studies are necessary to characterize further the optimal dosages and the mechanisms of action of rituximab in FSGS.

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Author and article information

Journal

Journal ID (nlm-ta): Clin Kidney J

Journal ID (iso-abbrev): Clin Kidney J

Journal ID (publisher-id): ckj

Title: Clinical Kidney Journal

Publisher: Oxford University Press

ISSN (Print): 2048-8505

ISSN (Electronic): 2048-8513

Publication date Collection: August 2020

Publication date (Electronic): 05 August 2019

Publication date PMC-release: 05 August 2019

Volume: 13

Issue: 4

Pages: 597-606

Affiliations

[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

Author notes

Correspondence and offprint requests to: Jonathan P. Troost; troostj@ 123456med.umich.edu

Author information

Chia-shi Wang http://orcid.org/0000-0001-6941-0057

Article

Publisher ID: sfz092

DOI: 10.1093/ckj/sfz092

PMC ID: 7467600

PubMed ID: 32905199

SO-VID: 4134113d-0a9c-4cf1-aa2e-4693a29f429e

License:

This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License ( http://creativecommons.org/licenses/by-nc/4.0/), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited. For commercial re-use, please contact journals.permissions@oup.com

History

Date received : 24 April 2019

Date accepted : 18 June 2019

Page count

Pages: 10

Funding

Funded by: Nephrotic Syndrome Study Network Consortium;

Award ID: U54‐DK‐083912

Funded by: National Institutes of Health Rare Disease Clinical Research Network;

Funded by: Office of Rare Diseases Research;

Funded by: National Center for Advancing Translational Sciences, DOI 10.13039/100006108;

Funded by: NIDDK, DOI 10.13039/100000062;

Funded by: University of Michigan, DOI 10.13039/100007270;

Funded by: NephCure Kidney International;

Funded by: Halpin Foundation;

Funded by: Charles Woodson Pilot Research Award;

Funded by: University of Michigan Department of Pediatrics;

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

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Abstract

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Conclusions

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Karger: Nephrology

Most cited references 18

Design of the Nephrotic Syndrome Study Network (NEPTUNE) to evaluate primary glomerular nephropathy by a multi-disciplinary approach

Focal and segmental glomerulosclerosis: definition and relevance of a partial remission.

Rituximab treatment of adult patients with steroid-resistant focal segmental glomerulosclerosis.

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