Pediatric Chronic Kidney Disease (CKD) results from health conditions that reduce kidney function for >3 months. It can progress to end-stage kidney disease (ESKD), which requires dialysis or kidney transplant. In adults, CKD is common and caused mainly by hypertension and diabetes. CKD in childhood is rare and caused primarily by congenital anomalies of the genitourinary system and immune-mediated disorders. The best estimate of pediatric CKD prevalence is <1/15,000 pediatric population. Hypertension occurs in 50% of affected children, and is a major risk factor for decline in kidney function. Several clinical practice guidelines have offered recommendations for blood pressure (BP) management in pediatric CKD; however, clinical trial and large-scale observational data are limited, leading to a weak evidence base and substantial practice variation.
Preserving Kidney Function in Children with Chronic Kidney Disease (PRESERVE) is a three-year, multi-institutional clinical trial evaluating blood pressure management and urine protein monitoring strategies to preserve kidney function in pediatric chronic kidney disease (CKD) patients. The purpose of PRESERVE is to provide new knowledge to inform shared decision-making regarding BP management for pediatric CKD. We will leverage the PCORnet® infrastructure to conduct large-scale observational studies that will address BP management knowledge gaps for pediatric CKD and sub-groups for whom antihypertensive treatment and outcome associations may be different (e.g., cause of kidney disease and proteinuria).
The >11,000 children included in this project represents the largest study of pediatric CKD to date (by an order of magnitude). Leveraging data for >11,000 children with CKD, PRESERVE aims to:
- Expand and improve the PCORnet CDM for research in children with kidney disease by adding new pediatric- and kidney-specific variables and linking patients’ EHR data to other kidney disease databases including the Chronic Kidney Disease in Children (CKiD) cohort and United States Renal Data System (USRDS)
- Compare the effectiveness of alternative strategies for monitoring and treating hypertension on preserving kidney function
- Assess the lived experiences of patients and caregivers related to BP management
PRESERVE is supported by the Glomerular Learning Network (GLEAN), a leading rare disease partner advocacy organization. Motivated by the challenges of enrolling patients with rare conditions into traditional clinical trials, Co-PI Michelle Denburg, MD, initiated and co-leads the multi-institutional Glomerular Learning Network in partnership with PEDSnet, which now includes nine large pediatric health systems (seven of which are in PEDSnet, and eight of which are participating in PRESERVE).
The vision of GLEAN is to improve the health and well-being of children with glomerular disease by conducting and integrating multi-institutional research and quality improvement that helps children, families, and health professionals make better health and healthcare decisions. The development of this network involved engaging multiple partners and collaborators in the nephrology community, as well as obtaining several sources of independent funding (industry, PCORI, NIH) to support its activities (computable phenotype development and validation, infrastructure, research, quality improvement, patient education and engagement). The goal of GLEAN is to provide a robust, sustainable and efficient resource for high priority comparative effectiveness, interventional research and outcomes improvement in children with glomerular disease.
Since its inception in 2018, the GLEAN Patient and Family Partners group has provided feedback on research and co-produced and continue to co-produce educational and engagement activities. Following this model, PRESERVE strives to develop authentic engagement of patients, caregivers and clinicians in all elements of research in order to build bridges between the patient-family experience and the development of clinical practice guidelines. PRESERVE will leverage data science work on key kidney-specific variables and outcomes performed for several observational studies conducted by GLEAN and PEDSnet to set the standard for patient-centered outcomes research design, both within the PRESERVE centers and in the broader pediatric nephrology community.