Comparative Effectiveness of Surgery for Pediatric Kidney Stones Using a LHS

Specific Aims: 

The rapid increase in the prevalence of kidney stones among youth has resulted in a large population of patients for whom little evidence exists to guide clinical care. This proposal arises from patients’ expressed need to better understand the effectiveness of different surgical approaches to remove kidney stones, the impact of surgery on their lives, and how long it takes to recover. To address this critical knowledge gap, this proposal leverages the Pediatric KIDney Stone (PKIDS) Care Improvement Network, which is a community of patients, caregivers, and clinicians who perform collaborative studies of kidney stones at 15 pediatric healthcare systems in the United States. PKIDS arose out of the 8 institutions that comprise PEDSnet, a member of the PCORI-funded PCORnet. PKIDS leverages the PEDSnet infrastructure to efficiently perform studies and translate the findings into clinical practice. The long-term goal of PKIDS is to generate and apply knowledge that improves outcomes for pediatric patients with kidney stones. The immediate objective of the proposed study is to compare stone clearance and patients’ experiences for three surgical approaches to remove stones-ureteroscopy (URS), shock wave lithotripsy (SWL), and percutaneous nephrolithotomy (PCNL). The resultant knowledge of these outcomes that matter the most to patients will improve shared clinical decision-making about the surgical management of stones. In the proposed 6-week prospective cohort study, we apply a conceptual framework for the examination of procedural interventions that accounts for the patient, surgeon, technical, hospital, and access factors that influence the choice of surgery and outcomes for patients 8 to 21 years of age. Accordingly, we will prospectively measure characteristics that impact the real-world delivery of surgical care and its outcomes in15 healthcare systems at which almost 1000 pediatric patients underwent kidney stone surgery in 2018.

Aim 1: To compare stone clearance for alternative approaches for kidney stone removal.We will use a standardized ultrasound protocol to assess stone clearance, a primary determinant of surgical retreatment. SA1a: To examine heterogeneity of treatment effect by clinical status (stone size and stone location). SA1b: To identify modifiable surgical techniques associated with higher stone clearance for each surgery. 

Aim 2: To compare patients’ experiences after URS, SWL, and PCNL. We will assess the immediate (1-week) and delayed (3-week)impact of surgery on patient-reported outcomes selected by the patients in PKIDS: pain interference, anxiety, psychological stress, peer relationships, and family relationships (PROMIS) and urinary tract symptoms. SA2a: To examine heterogeneity of treatment effect by age and sex. SA2b: To identify modifiable surgical techniques that impact these experiences for each surgery.


The proposed study will improve the ability of pediatric patients and their caregivers to select surgical treatment options for kidney stones and will improve the ability of urologists to use techniques that result in the best outcomes for these surgeries. As a cooperative consortium of 15 large healthcare systems, PKIDS is uniquely positioned to rapidly implement the study results into clinical care. This proposal will also generate highly useable and generalizable knowledge that can be quickly adopted and scaled across other healthcare systems. In addition, PKIDS will provide a model for examining the effectiveness of procedural interventions for other surgical diseases in children and adults.