Findings from the Pediatric KIDney Stone (PKIDS) trial, a PCORnet® Study conducted by the PKIDS Care Improvement Network, are helping to shape how clinicians approach kidney stone surgery in children and adolescents, demonstrating the power of patient-centered research at scale. The prospective observational study compared three surgical treatments for the removal of kidney and ureteral stones – ureteroscopy, shock wave lithotripsy, and percutaneous nephrolithotomy – to determine differences in stone clearance and time to return to school or work.
The PKIDS study found that while ureteroscopy and shockwave lithotripsy did not differ in stone clearance, patients reported better experiences with shockwave lithotripsy than ureteroscopy one week after surgery. For patients with large kidney stones, percutaneous nephrolithotomy was more effective than ureteroscopy, and patients reported a better experience.
Engagement Expertise and Patient Partners Drive Meaningful Research
A hallmark of PKIDS, the largest observational study to date comparing ureteroscopy and shock wave lithotripsy in pediatric patients, was its deeply engaged, multidisciplinary network of partners. The PKIDS advisory council brought together physicians specializing in urology, nephrology, and radiology, alongside patients, caregivers, and representatives from health insurers and industry.
PKIDS Patient and Family Partners were integral members of the research team and informed the study design, recruitment strategies, and dissemination efforts. “We have the real-life experiences of living with kidney stones and can help provide crucial information that you would only know by living with the condition,” said Hunter Beck, a PKIDS Patient Partner.
Drawing on lived experiences, the PKIDS Patient and Family Partners selected patient-reported outcomes and collaborated with investigators to develop the Questionnaire for Urinary Issues—Kidney Stone Surgery (QUIKSS), a novel tool that captures patient-reported symptoms and recovery experiences not measured in existing validated instruments.
The study’s successful integration of Patient and Family Partners was facilitated by engagement experts from PEDSnet, a PCORnet® Clinical Research Network (CRN), and the Patient-Centered Outcomes Research Institute® (PCORI®), who funded the study.
“The engagement leads provided excellent insight into how we could deepen our engagement with patients, caregivers, and patient advocates,” said Gregory Tasian, MD, MSc, MSCE, PKIDS Principal Investigator. “That guidance helped us move beyond asking for feedback at isolated points and instead embed patient and family partners throughout the study. Our Patient and Family Partners helped us select outcomes that reflect what families experience after surgery, refine recruitment and retention strategies, and think carefully about how to return results in a way that is useful to patients and clinicians. The meaningful engagement of our Patient and Family Partners made PKIDS a stronger study because it ensured that the evidence we generated was clinically rigorous, patient-centered, and directly relevant to shared decision making.”
Research-Ready Community Surfaces Real-World Data Insights
The PKIDS study enrolled 1,290 participants ages 8 to 21 years across 20 sites in five PCORnet® Clinical Research Networks (CRNs) and 11 sites outside of the network, illustrating how the PCORnet infrastructure can efficiently support large, multi-site studies in pediatric populations. The PKIDS study team used the PCORnet® Common Data Model (CDM) to assess the generalizability of their findings, but the benefits of partnering with PCORnet expanded beyond standardized health data.
By comparing stone clearance and patient experiences across ureteroscopy, shock wave lithotripsy, and percutaneous nephrolithotomy, the PKIDS study strengthened the evidence base for shared patient-clinician decision making and contributed findings that support a 15 mm size threshold for pediatric patients with kidney stones. The prospective observational design of the study required the team to collect and analyze a large amount of clinical, patient, surgeon, and site-level data to account for important contextual factors that could make one treatment appear more or less effective than it really is. Gathering these detailed data required a strong data coordinating center and reliable research coordinators at each site, underscoring a comparative advantage of PCORnet – support from research experts across the healthcare community who help investigators interpret real-world data and translate their findings into practice-relevant evidence.
Patient-Centered Health Research Returns Actionable Results
The PKIDS study was designed in response to questions that directly impact patients and their caregivers: Which surgical approaches most effectively remove kidney stones? How do those procedures affect daily life? What does recovery look like?
By comparing stone clearance and patient experiences across ureteroscopy, shock wave lithotripsy, and percutaneous nephrolithotomy, the PKIDS study strengthened the evidence base for shared patient-clinician decision making and contributed findings that support a 15 mm size threshold for pediatric patients with kidney stones.
PKIDS results were presented at the American Urological Association (AUA) annual meeting as a “paradigm-shifting, practice-changing plenary session,” underscoring the study’s significance to the field. The study also contributed to the evidence base informing the AUA’s Surgical Management of Kidney and Ureteral Stones Guideline, where PKIDS findings were cited as supporting evidence.
The PKIDS study highlights how the PCORnet infrastructure enables researchers to generate timely, meaningful evidence that reflects patient experiences, clinical outcomes, and the local contexts in which care is delivered. By combining real-world data with engagement expertise and close connections to patients, caregivers, clinicians, and other community partners, research supported by PCORnet offers a distinctive model for advancing patient-centered health research in pediatric populations.
Contact the PCORnet® Front Door to learn how the PCORnet infrastructure can support your patient-centered health research.