PCORnet® Studies Improve the Health and Healthcare of Children

PCORnet® is intended to improve the nation’s capacity to efficiently conduct patient-centered health research, particularly comparative clinical effectiveness research (CER), by providing a large, highly representative network of health data, research expertise, and patient insights. PCORnet has been developed with funding from the Patient-Centered Outcomes Research Institute® (PCORI®).

December 2025

Pediatric Research Summary

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SOLVING KEY CHALLENGES IN PEDIATRIC RESEARCH

Challenge: Small study populations

Challenge: Burdensome study designs inhibit recruitment and retention rates

Challenge: Low-impact study outcomes

Solution: Real-world data from everyday healthcare encounters with nearly 13 million pediatric patients ages birth to 20 years old

Solution: Proven, low-burden patient-centered models for pragmatic research

Solution: Patient and caregiver engagement throughout the study lifecycle drives meaningful research and results

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CASE STUDIES: PEDIATRIC RESEARCH POWERED BY PCORnet®

Greenlight Plus Study: A Randomized Study of Approaches to Early Childhood Obesity Prevention

Study Design: Intervention Trial

Funder: Patient-Centered Outcomes Research Institute® (PCORI®)

Principal Investigator: William Heerman, Vanderbilt University Medical Center

Study Question

Does text-messaging in addition to clinic-based health behavior counseling improve healthy weight trajectories among children ages 0–2 compared to health behavior counseling alone?

Using PCORnet, the study team:

  • Partnered with six healthcare institutions to enroll 900 English- and Spanish-speaking parent-infant pairs
  • Leveraged the PCORnet® Common Data Model to standardize EHR data across sites
  • Compared the study population to unenrolled parent-infant pairs across sites

Dissemination

Study results were published in JAMA

Preserving Kidney Function in Children with Chronic Kidney Disease (PRESERVE)

Study Design: Retrospective Observational

Funder: Patient-Centered Outcomes Research Institute® (PCORI®)

Principal Investigator: Christopher Forrest, The Children’s Hospital of Philadelphia

Study Questions

Which monitoring strategies and blood pressure medications best preserve kidney function in pediatric patients with chronic kidney disease (CKD)?

What are the lived experiences of patients and families managing pediatric CKD?

Using PCORnet, the study team:

  • Partnered with 15 healthcare institutions participating in PCORnet
  • Included 20,100 children (ages 1–17) with CKD
  • Expanded the PCORnet® Common Data Model for pediatric and rare kidney disease research

Dissemination

Published a framework to assess the quality of real-world data in PLOS Digital Health

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Pediatric KIDney Stone (PKIDS) Care Improvement Network

Study Design: Prospective Observational

Funder: Patient-Centered Outcomes Research Institute® (PCORI®)

Principal Investigator: Gregory Tasian, The Children's Hospital of Philadelphia

Study Question

What are the differences in kidney stone clearance and the lived experiences of youth following ureteroscopy, shock wave lithotripsy, and percutaneous nephro-lithotomy for the removal of kidney and ureteral stones?

Using PCORnet, the study team:

  • Partnered with 20 healthcare institutions to enroll 1,290 youth (ages 8–21) receiving kidney stone surgery
  • Assessed the generalizability of their findings

Dissemination

Utilizing PCORnet to Support Transition from Pediatric to Adult Centered Care and Reduce Gaps in Recommended Care in Patients with Congenital Heart Disease (CHI-RON)

Study Design: Retrospective Observational

Funder: Patient-Centered Outcomes Research Institute® (PCORI®)

Principal Investigators: Thomas Carton and Anitha John, Louisiana Public Health Institute

Study Questions

  • How does receiving current recommended care affect long-term outcomes and healthcare needs among the numerous rare disease subtypes of congenital heart defects?
  • What factors are associated with gaps in recommended care?
  • Do patients report feeling better when they remain in specialty care?

Using PCORnet, the study team:

  • Established a system to study different ways to reduce gaps in care for people with Adult Congenital Heart Disease
  • Leveraged the PCORnet® Common Data Model to assess outcomes

Dissemination

“PKIDS at its core has turned around the way we conduct research in pediatric surgery. We turn to our patient and family partners to learn what are the questions we should be asking and what outcomes we should be measuring.”

 

Gregory Tasian, Principal Investigator for PKIDS

POWER YOUR PEDIATRIC RESEARCH WITH PCORnet®

PCORnet resources are available to academic and industry researchers, patient advocacy groups, and other organizations from all funding sources and affiliations. PCORI also offers funding for broad pragmatic studies using PCORnet.

 

Are you ready to find out how PCORnet can support your next research effort? Contact the PCORnet® Front Door to start the conversation.

PCORnet® is a national resource, funded by PCORI, that enables insights from high-quality health data, patient partnership, and research expertise to deliver fast, trustworthy answers that advance health outcomes. The network supports both observational and interventional research.