Patient-Reported Outcomes Over 24 Months in Pediatric CKD: Findings From the MyKidneyHealth Cohort Study.

2023

03/2023

1523-6838

RATIONALE AND OBJECTIVE: The lived experience of children with chronic kidney disease (CKD) is poorly characterized. We examined the associations between patient-reported outcomes (PROs) measuring children's fatigue, sleep health, psychological distress, family relationships, and global health with clinical outcomes over time in children with CKD and investigated how PROs of children with CKD compare with those of other children.

STUDY DESIGN: Prospective cohort study.

SETTING AND PARTICIPANTS: 212 children 8-21 years-old with CKD and their parents recruited from 16 nephrology programs across North America.

PREDICTORS: CKD stage, disease etiology, sociodemographic and clinical variables.

OUTCOMES: PROs over 2 years.

ANALYTICAL APPROACH: We compared PROs in the CKD sample with a nationally representative general pediatric population. Change of PROs over time and association of sociodemographic and clinical variables with PROs were assessed using multivariable regression models.

RESULTS: 84% parents and 77% children completed PROs at all time points. Baseline PRO scores for children with CKD revealed higher burden of fatigue, sleep-related impairment, psychological distress, impaired global health, and poorer family relationships compared with the general pediatric population, with median score differences ≥ one standard deviation for fatigue and global health. Baseline PRO scores did not differ by CKD stage or glomerular vs. non-glomerular etiology. Over two years, PROs were stable with < 1-point annual change on average on each measure and intraclass correlation coefficients ranging 0.53 to 0.79, indicating high stability. Hospitalization and parent-reported sleep problems were associated with worse fatigue, psychological health and global health scores (all p<0.04).

LIMITATIONS: Unable to assess responsiveness to change with dialysis or transplant.

CONCLUSIONS: Children with CKD experience high, yet stable burden of impairment across numerous PRO measures, especially fatigue and global health, independent of disease severity. These findings underscore the importance of assessing PRO, including fatigue and sleep measures, in this vulnerable population.

10.1053/j.ajkd.2022.12.014

Am J Kidney Dis

36889426

Using Electronic Health Record Data to Rapidly Identify Children with Glomerular Disease for Clinical Research.

2019

2427-2435

2019 Dec

1533-3450

<p><strong>BACKGROUND: </strong>The rarity of pediatric glomerular disease makes it difficult to identify sufficient numbers of participants for clinical trials. This leaves limited data to guide improvements in care for these patients.</p>

<p><strong>METHODS: </strong>The authors developed and tested an electronic health record (EHR) algorithm to identify children with glomerular disease. We used EHR data from 231 patients with glomerular disorders at a single center to develop a computerized algorithm comprising diagnosis, kidney biopsy, and transplant procedure codes. The algorithm was tested using PEDSnet, a national network of eight children's hospitals with data on &gt;6.5 million children. Patients with three or more nephrologist encounters (=55,560) not meeting the computable phenotype definition of glomerular disease were defined as nonglomerular cases. A reviewer blinded to case status used a standardized form to review random samples of cases (=800) and nonglomerular cases (=798).</p>

<p><strong>RESULTS: </strong>The final algorithm consisted of two or more diagnosis codes from a qualifying list or one diagnosis code and a pretransplant biopsy. Performance characteristics among the population with three or more nephrology encounters were sensitivity, 96% (95% CI, 94% to 97%); specificity, 93% (95% CI, 91% to 94%); positive predictive value (PPV), 89% (95% CI, 86% to 91%); negative predictive value, 97% (95% CI, 96% to 98%); and area under the receiver operating characteristics curve, 94% (95% CI, 93% to 95%). Requiring that the sum of nephrotic syndrome diagnosis codes exceed that of glomerulonephritis codes identified children with nephrotic syndrome or biopsy-based minimal change nephropathy, FSGS, or membranous nephropathy, with 94% sensitivity and 92% PPV. The algorithm identified 6657 children with glomerular disease across PEDSnet, ≥50% of whom were seen within 18 months.</p>

<p><strong>CONCLUSIONS: </strong>The authors developed an EHR-based algorithm and demonstrated that it had excellent classification accuracy across PEDSnet. This tool may enable faster identification of cohorts of pediatric patients with glomerular disease for observational or prospective studies.</p>

10.1681/ASN.2019040365

J. Am. Soc. Nephrol.

31732612