Researchers at Children’s Hospital of Philadelphia (CHOP), along with several academic partners, announced the primary results of the Pediatric KIDney Stone (PKIDS) trial, the largest comparative effectiveness study of surgical interventions for children and adolescents with kidney stones. The CHOP-led PKIDS trial, with two published studies, marks a significant breakthrough by offering stronger evidence for treating stones of varying sizes, including new information on patient experiences after surgery, thus reducing uncertainty and empowering informed decision-making for patients, caregivers, and physicians.
Kidney stones were once largely a disease that affected adults. However, kidney stones in children have been on the rise in recent decades, doubling the likelihood that a child will develop a kidney stone. As a result, CHOP founded the PKIDS Care Improvement Network in 2019, which now includes 31 sites in the United States and Canada.
Ureteroscopy (an endoscopic outpatient procedure), shockwave lithotripsy (a noninvasive outpatient procedure) and percutaneous nephrolithotomy (a minimally invasive surgery with a short hospital stays) are the procedures used to treat children and adults with kidney stones. Most children and adolescents with kidney and ureteral stones are treated with ureteroscopy despite uncertainty of which procedure is more effective and their impact on patients’ lives. As pioneers in urology, CHOP leaders aimed to enhance pediatric patient and caregiver decision-making for kidney stone surgeries while enabling urologists to adopt techniques for optimal outcomes, including patient-selected experiences.
In the first study in JAMA Network Open, Tasian and his team enrolled 1142 patients aged 8 to 21 with kidney and/or ureteral stones between 2020 and 2023 at all 31 sites in the United States and Canada. Researchers evaluated ureteroscopy against shockwave lithotripsy and found that shockwave lithotripsy was associated with less pain and fewer urinary symptoms compared with those who had ureteroscopy. No meaningful differences were detected in stone-free rates for the procedures.
In the other CHOP-led study in JAMA Network Open, Jonathan S. Ellison, MD, an Associate Professor of Urology at the Medical College of Wisconsin and Pediatric Urologist at Children’s Wisconsin, and the PKIDS team compared percutaneous nephrolithotomy (PCNL) and ureteroscopy. That study found that for children with larger stones, PCNL not only cleared more stones effectively but also led to a better overall recovery experience than ureteroscopy.
Overall, the authors emphasised the post-surgery experiences of children, noting that quality of life factors, such as the loss of school time for children and work time for caregivers, are crucial in determining effective treatment options. While the authors are planning further research, they also hope these findings will lead to immediate improvements for families.
“The PKIDS trial demonstrated that ureteroscopy and shockwave lithotripsy remove stones equally well and that patients having shockwave lithotripsy recover more quickly after surgery with less pain and fewer urinary symptoms. Our findings provide new information that allow for tailored approaches to kidney stone treatment for children and their families,” said Gregory E. Tasian, MD, MSc, MSCE, Director of the PKIDS network and an attending pediatric urologist in the Division of Urology at Children’s Hospital of Philadelphia. “Although future clinical trials are important, we hope that clinical practice guidelines will consider outcomes that matter to patients.”
