Landmark PKIDS Trial Challenges Standard Pediatric Kidney Stone Treatment Approaches
核心洞察
The PKIDS trial, the largest comparative effectiveness study of surgical interventions for pediatric kidney stones (搜索), enrolled 1,142 patients across 31 medical centers in the US and Canada.
Shockwave lithotripsy demonstrated equivalent stone clearance rates to ureteroscopy but with significantly better patient-reported outcomes including less pain and fewer urinary symptoms.
For kidney stones (搜索) larger than 15 mm, percutaneous nephrolithotomy achieved superior stone clearance rates (94% vs 55%) compared to ureteroscopy with better recovery experiences.
The largest comparative effectiveness study of surgical interventions for pediatric kidney stones (搜索) has revealed significant differences in patient outcomes that challenge current treatment preferences, according to results from the Pediatric KIDney Stone (PKIDS) trial published in JAMA Network Open.
The PKIDS trial, led by researchers at Children's Hospital of Philadelphia (CHOP (搜索)), enrolled 1,142 patients aged 8 to 21 with kidney and/or ureteral stones (搜索) between 2020 and 2023 across 31 medical centers in the United States and Canada. The study addresses a critical knowledge gap in pediatric urology, as most children and adolescents with kidney stones (搜索) are currently treated with ureteroscopy despite uncertainty about which procedure is most effective and their impact on patients' lives.
Shockwave Lithotripsy Shows Superior Patient Experience
In the comparison between ureteroscopy and shockwave lithotripsy involving 953 and 189 patients respectively, researchers found no significant difference in stone clearance rates. The stone clearance rate was 71.2% in the ureteroscopy arm versus 67.5% in the shockwave lithotripsy arm, a difference that did not achieve statistical significance (risk difference, 3.6%; 95% CI, −6.2 to 13.5).
However, patients who underwent shockwave lithotripsy reported markedly better recovery experiences. Compared with shockwave lithotripsy, ureteroscopy was associated with greater pain interference (T-score difference, 5.0 [95% CI, 2.3-7.8]) and urinary symptoms (symptom score difference, 3.9; 95% CI, 1.2 to 6.7) at one week after surgery.
The impact extended beyond clinical measures to practical considerations for families. In the ureteroscopy arm, patients missed more school (risk difference, 21.3%; 95% CI, 9.7 to 32.8) and caregivers missed more work (risk difference, 23.0%; 95% CI, 11.0 to 35.0) in the week following the procedure.
PCNL Demonstrates Clear Advantage for Larger Stones
The second study compared percutaneous nephrolithotomy (PCNL) with ureteroscopy in 1,039 patients, with 93 undergoing PCNL and 946 undergoing ureteroscopy. While overall stone clearance rates were similar between the procedures (67.2% for PCNL vs 73.4% for ureteroscopy), a striking difference emerged for patients with stones larger than 15 mm.
For these larger stones, PCNL achieved a stone clearance rate of 94% (95% CI, 83.3 to 100) compared with 55% (95% CI, 32.9 to 77.1) for ureteroscopy, representing a statistically significant risk difference of 39.0% (95% CI, 14.4 to 63.5).
Patients who underwent PCNL also reported significantly better experiences across multiple domains at one week post-surgery, including lower scores for pain intensity (T score difference, −5.42; 95% CI, −10.38 to −0.46), pain interference (T score difference, −5.88; 95% CI, −11.02 to −0.75), anxiety (T score difference, −5.74; 95% CI, −9.26 to −2.22), psychological stress experiences (T score difference, −7.90; 95% CI, −13.13 to −2.67), sleep disturbance (T score difference, −5.57; 95% CI, −8.56 to −2.58), and urinary symptoms (symptom score difference, −6.37; 95% CI, −11.71 to −1.03).
Clinical Practice Implications
"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," said Gregory E. Tasian, MD, MSc, MSCE, Director of the PKIDS network and an attending pediatric urologist in the Division of Urology at CHOP (搜索). "Our findings provide new information that allow for tailored approaches to kidney stone treatment for children and their families."
The researchers suggest that the better experiences with PCNL may be explained by the lower use of ureteral stents and reduced ureteral manipulation compared to ureteroscopy. However, they note that further investigation is needed to understand whether the patient-reported outcome differences are primarily related to ureteral stenting or to ureteroscopy itself.
Growing Pediatric Health Challenge
The study addresses an increasingly important clinical problem, as kidney stones (搜索) in children have been rising in recent decades, doubling the likelihood that a child will develop a kidney stone. This trend prompted CHOP (搜索) to establish the PKIDS Care Improvement Network in 2019, which now includes 31 sites across North America.
The authors emphasize that quality of life factors, including loss of school time for children and work time for caregivers, are crucial considerations in determining effective treatment options. While planning further research, they hope these findings will lead to immediate improvements for families and influence clinical practice guidelines to consider outcomes that matter most to patients.
The findings support PCNL as a first-line surgical intervention for pediatric patients with kidney stones (搜索) larger than 15 mm, while suggesting that shockwave lithotripsy may be preferable to ureteroscopy for smaller stones when equivalent efficacy can be achieved with better patient experiences.