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

Validating a Computable Phenotype for Nephrotic Syndrome in Children and Adults Using PCORnet Data.

2021

1979-1986

2021 Dec 30

2641-7650

<p><strong>Background: </strong>Primary nephrotic syndromes are rare diseases which can impede adequate sample size for observational patient-oriented research and clinical trial enrollment. A computable phenotype may be powerful in identifying patients with these diseases for research across multiple institutions.</p>

<p><strong>Methods: </strong>A comprehensive algorithm of inclusion and exclusion ICD-9 and ICD-10 codes to identify patients with primary nephrotic syndrome was developed. The algorithm was executed against the PCORnet CDM at three institutions from January 1, 2009 to January 1, 2018, where a random selection of 50 cases and 50 noncases (individuals not meeting case criteria seen within the same calendar year and within 5 years of age of a case) were reviewed by a nephrologist, for a total of 150 cases and 150 noncases reviewed. The classification accuracy (sensitivity, specificity, positive and negative predictive value, F1 score) of the computable phenotype was determined.</p>

<p><strong>Results: </strong>The algorithm identified a total of 2708 patients with nephrotic syndrome from 4,305,092 distinct patients in the CDM at all sites from 2009 to 2018. For all sites, the sensitivity, specificity, and area under the curve of the algorithm were 99% (95% CI, 97% to 99%), 79% (95% CI, 74% to 85%), and 0.9 (0.84 to 0.97), respectively. The most common causes of false positive classification were secondary FSGS (nine out of 39) and lupus nephritis (nine out of 39).</p>

<p><strong>Conclusion: </strong>This computable phenotype had good classification in identifying both children and adults with primary nephrotic syndrome utilizing only ICD-9 and ICD-10 codes, which are available across institutions in the United States. This may facilitate future screening and enrollment for research studies and enable comparative effectiveness research. Further refinements to the algorithm including use of laboratory data or addition of natural language processing may help better distinguish primary and secondary causes of nephrotic syndrome.</p>

10.34067/KID.0002892021

Kidney360

35419531