Pediatric chronic kidney disease (CKD) is the target group of rare diseases for the proposed project PRESERVE. Fewer than one in 15,000 children are affected by CKD, a condition characterized by reduced kidney function for three months or longer. Over time, progressive loss of kidney function results in end-stage kidney disease, which requires long-term dialysis or kidney transplantation. Patients, parents/caregivers, and clinicians agree that the most important outcome for pediatric CKD is preservation of kidney function. The limited research that has been conducted suggests that hypertension, which affects 50 percent of children with CKD, is a major and potentially modifiable contributor to loss of kidney function. Thus, regardless of why the CKD arose, the treatment is the same: control blood pressure (BP) using medications. The purpose of PRESERVE is to provide new knowledge to inform shared decision making regarding BP management for pediatric CKD. To conduct this project, we have formed a consortium of 16 healthcare institutions that are part of the National Patient-Centered Clinical Research Network (PCORnet). We plan to study over 11,000 children with CKD. Specifically, this project will do the following:
- Expand and improve the PCORnet common data model for research in children with kidney disease by adding new pediatric- and kidney-specific variables and linking patients’ electronic health record (EHR) data to other kidney disease databases.
- 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.
The first two parts of this project will involve analyses of EHR data, and the third part will involve a survey of patients and their caregivers. EHR data will be obtained from 2009 to 2022, providing a rich resource for studying patients from the time they are diagnosed with CKD to years later. This will help determine if alternative BP clinical management strategies are associated with preservation of kidney function. The results of this study will provide answers to key questions that patients/parents and clinicians have regarding BP management: (1) Can BP measurements performed in physicians’ offices replace 24-hour ambulatory blood pressure monitoring, a procedure that is performed at home and involves BP readings taken every 20–30 minutes? (2) How high should BP be before starting medications? (3) When starting medications, what is the best medication choice? (4) How low should BP be to protect kidney function over time, while minimizing potentially adverse clinical events? (5) How do different strategies for BP management affect patients’ pain, fatigue, sleep, emotions, relationships with friends, and satisfaction with their lives?
The study population will include children with mild to moderate CKD (stages 2–3), when treatment to preserve kidney function is most likely to be beneficial. The primary outcome will be kidney function decline assessed as any of the following events happening: 50 percent reduction in kidney function, starting dialysis, receiving a kidney transplant, or experiencing a drop in kidney function to levels deemed to be end-stage kidney disease. For the patient survey, we will involve 800 children with CKD and their parents, who will respond to an electronic questionnaire.
A major goal of the study is to ensure that the engagement of patients/parents and clinicians in all parts of the research process is authentic. Three parent partners are investigators who will participate in all project decisions. We will establish a Patient/Parent Partners Work Group and a Clinician Partners Work Group from across the participating centers. These groups will meet regularly to provide input on all phases of the study. The patient/parent partners will design the patient survey. We will hold semiannual town halls, open to the public, to discuss study progress. Results will also be disseminated to over 180 pediatric nephrology centers, facilitating their widespread uptake, and directly returned to families who participate in the patient survey.
PRESERVE will be the largest study for pediatric CKD among children who have not yet reached end-stage kidney disease. Not only will our project address meaningful questions related to BP management for patients with pediatric CKD and their clinicians, but it will also create new PCORnet infrastructure that can be used in future studies, such as clinical trials on BP control strategies in pediatric CKD.