Dataset Information

Health-Related Quality of Life in Focal Segmental Glomerular Sclerosis and Minimal Change Disease: A Qualitative Study of Children and Adults to Inform Patient-Reported Outcomes.

ABSTRACT:

Rationale & objective

Assessment of how patients feel and function is needed for clinical care and research for focal segmental glomerulosclerosis (FSGS) and minimal change disease (MCD). The objective of this study was to develop a patient-reported outcome assessment appropriate for use in children and adults with FSGS and MCD.

Study design

Qualitative study using semi-structured interviews.

Setting & participants

48 semi-structured interviews with children aged 8 to 17 years (n = 11) and adults (n = 10) with FSGS and children aged 8 to 17 (n = 11) and adults (n = 16) with MCD recruited from 3 academic medical centers.

Analytical approach

Latent content analysis.

Results

FSGS and MCD have a pervasive and comparable impact on physical, social, and mental health-related quality of life regardless of age or diagnosis. Physical symptoms of swelling, fatigue, and pain were articulated by most participants. Disease management was also a frequent topic of discussion; participants described their experiences with medication and associated side effects, as well as lifestyle changes made to manage their disease (ie, dietary changes and frequent medical appointments). These discussions often identified a profound impact on physical abilities and life participation. In many instances, participants described the negative impact these symptoms had on their mood and sense of self, with most participants reporting feelings of anxiety.

Limitations

Participants were primarily non-Hispanic White and English speaking, which may limit generalizability.

Conclusions

Our results suggest that there are commonalities to the FSGS-MCD patient experience of health-related quality of life that will enable the generation of a disease-specific FSGS-MCD patient-reported outcomes instrument for use in children and adults. The development of this tool is intended to facilitate better care and support clinical research for these individuals.

SUBMITTER: Carlozzi NE

PROVIDER: S-EPMC8350833 | biostudies-literature | 2021 Jul-Aug

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Health-Related Quality of Life in Focal Segmental Glomerular Sclerosis and Minimal Change Disease: A Qualitative Study of Children and Adults to Inform Patient-Reported Outcomes.

Carlozzi Noelle E NE Massengill Susan F SF Trachtman Howard H Walsh Liron L Singhal Neena N LaVigne Joseph M JM Miner Jennifer A JA Desmond Hailey E HE Lynam Christian C Gipson Debbie S DS

Kidney medicine 20210420 4

<h4>Rationale & objective</h4>Assessment of how patients feel and function is needed for clinical care and research for focal segmental glomerulosclerosis (FSGS) and minimal change disease (MCD). The objective of this study was to develop a patient-reported outcome assessment appropriate for use in children and adults with FSGS and MCD.<h4>Study design</h4>Qualitative study using semi-structured interviews.<h4>Setting & participants</h4>48 semi-structured interviews with children aged 8 to 17 ye ...[more]

PMID: 34401716

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Project description:Primary focal segmental glomerulosclerosis (FSGS), along with minimal change disease (MCD), are diseases with primary podocyte damage that are clinically manifested by the nephrotic syndrome. The pathogenesis of these podocytopathies is still unknown, and therefore, the search for biomarkers of these diseases is ongoing. Our aim was to determine of the proteomic profile of urine from patients with FSGS and MCD. Patients with a confirmed diagnosis of FSGS (n = 30) and MCD (n = 9) were recruited for the study. For a comprehensive assessment of the severity of FSGS a special index was introduced, which was calculated as follows: the first score was assigned depending on the level of eGFR, the second score-depending on the proteinuria level, the third score-resistance to steroid therapy. Patients with the sum of these scores of less than 3 were included in group 1, with 3 or more-in group 2. The urinary proteome was analyzed using liquid chromatography/mass spectrometry. The proteome profiles of patients with severe progressive FSGS from group 2, mild FSGS from group 1 and MCD were compared. Results of the label free analysis were validated using targeted LC-MS based on multiple reaction monitoring (MRM) with stable isotope labelled peptide standards (SIS) available for 47 of the 76 proteins identified as differentiating between at least one pair of groups. Quantitative MRM SIS validation measurements for these 47 proteins revealed 22 proteins with significant differences between at least one of the two group pairs and 14 proteins were validated for both comparisons. In addition, all of the 22 proteins validated by MRM SIS analysis showed the same direction of change as at the discovery stage with label-free LC-MS analysis, i.e., up or down regulation in MCD and FSGS1 against FSGS2. Patients from the FSGS group 2 showed a significantly different profile from both FSGS group 1 and MCD. Among the 47 significantly differentiating proteins, the most significant were apolipoprotein A-IV, hemopexin, vitronectin, gelsolin, components of the complement system (C4b, factors B and I), retinol- and vitamin D-binding proteins. Patients with mild form of FSGS and MCD showed lower levels of Cystatin C, gelsolin and complement factor I.

Dataset Information

Health-Related Quality of Life in Focal Segmental Glomerular Sclerosis and Minimal Change Disease: A Qualitative Study of Children and Adults to Inform Patient-Reported Outcomes.

Rationale & objective

Study design

Setting & participants

Analytical approach

Results

Limitations

Conclusions

Publications

Health-Related Quality of Life in Focal Segmental Glomerular Sclerosis and Minimal Change Disease: A Qualitative Study of Children and Adults to Inform Patient-Reported Outcomes.

Similar Datasets

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