In 2005, investigators in the United Kingdom embarked upon perhaps one of the most ambitious randomized controlled trials (RCTs) ever proposed for urological disease, the Percutaneous vesicoamniotic shunting versus conservative management for fetal Lower Urinary Tract Obstruction (PLUTO) trial.¹ PLUTO randomized pregnant women with singleton fetuses exhibiting signs of lower urinary tract obstruction to intervention versus observational management. In 2010, 5 years after study design inception, the study was terminated early owing to poor accrual with approximately 20% of anticipated participants enrolled. At the time of study publication in 2013, nearly a decade had passed since the initial trial design was proposed and the trial’s namesake was no longer considered a planet.²

The PLUTO trial highlights the limitations of RCTs of surgical intervention. Clinical equipoise, implying uncertainty regarding the optimal treatment choice, serves as the ethical basis for RCTs, thereby creating a framework for procedural randomization. However, treatment effectiveness in surgical intervention is dependent on more factors than disease and patient characteristics, including surgeon experience and the technical aspects of the intervention.³ Lack of equipoise, combined with a reluctance of a patient or caregiver to submit their surgical care choice to the fates of randomization, can lead to accrual failure.⁴ Lastly, technological advances and procedural learning curves may render initial outcomes obsolete by the time the study reaches publication.⁵

Opportunities of a Surgical Learning Health System (LHS)

A LHS is neither a data repository nor a pure clinical collaborative, but rather an amalgamation of these concepts whose whole is greater than the sum of its parts. The LHS is built upon quality improvement concepts of “Plan-Do-Study-Act” cycles to engender continuous process improvement. As opposed to a RCT, clinical trials within a LHS allow for pragmatic recruitment embedded within natural variations in clinical care and provide a natural infrastructure to rapidly disseminate knowledge generated back to key users, namely surgeons and other health care providers (see figure). Krapohl et al identifies 3 key steps in a surgical LHS.⁶ First, Performance to Data, where current practices and key outcomes are identified for improvement efforts. Second, Data to Knowledge, where data collected are analyzed and disseminated back to the end-users. Third, Knowledge to Performance, where the knowledge generated then spurs further efforts at practice change on an individual or system level. Unique to surgery, a LHS may provide insight into procedural learning curves and adoption of new techniques or technologies in surgical practice. Thus, a surgical LHS may serve as an ideal platform for simultaneously studying and improving surgical or procedural based techniques and outcomes.

Figure. Representative construct of patient-centered learning health system, with central collaboration of researcher, clinician, and patient both contributing to and benefitting from process and output of surgical learning cycle.

The Pediatric KIDney Stone (PKIDS) Care Improvement Network: A National Surgical Learning Health System for Pediatric Kidney Disease

PKIDS was developed with the following mission: “To improve outcomes for children with kidney stone disease through collaborative patient-centered research.” Currently, with funding support from the Patient-Centered Outcomes Research Institute (PCORI), PKIDS is performing a prospective observational trial on the comparative effectiveness of surgical interventions (namely shock wave lithotripsy, ureteroscopy, and percutaneous nephrolithotomy) for pediatric kidney stone disease. Spanning 26 hospitals and embedded within standard clinical care, this study is assessing stone clearance and the lived experiences of children following surgical intervention and harnesses a structured and comprehensive data collection process to capture specific information on surgical practices. Actionable knowledge generated from this trial will then be taken directly back to the network for future iterative improvement efforts.⁷

Patient Engagement in Research Design

Patient-reported outcomes have long been recognized as key, if yet underutilized, outcome measures in urological disease.⁸ More recently, the value of engaging patients in research that will directly impact them has gained momentum in the urological community. This effort is particularly evident in the work supported by Smith et al, where patient-directed research efforts generated an entire research agenda for bladder cancer.⁹ Within PKIDS, we have been fortunate to partner with several thoughtful and engaged patients and their parents who have direct experience with kidney stones and kidney stone surgery. These collaborators have provided invaluable insight into our study design. First, they provided input into the selection of patient-reported outcome measures which they felt were the most important experiences after surgery. Second, they identified gaps in validated questionnaires which prompted an internally generated questionnaire so as not to miss other key aspects of recovery. Third, they recommended engagement strategies (ie texting) and interval followup timing so as to enhance participant engagement and collection of key outcomes. Additionally, participating in the study design provided a great deal of satisfaction for our patient partners, as expressed by Hunter Beck, a teenage member of PKIDS’ Patient and Family Partners: “Having kidney stones is very stressful and by participating in the studies I feel that I am helping the future outcome for me and other kids. Participating in studies is a way for those that suffer from kidney stones to feel like they are involved and helping find a way to treat them. I feel it is important to do something positive to combat a negative situation. PKIDS is a great way to connect with the doctors and stay engaged. I feel like we are making a difference by collaborating on ways to include more patients’ outcomes in the forms for studies, and it is great to connect with others that are going through the same experiences you are.”

Future Opportunities

As modern surgical innovation continues to rapidly outpace the ability to assess comparative effectiveness through the lumbering and rigid processes of the RCT, more malleable strategies for studying and simultaneous improving outcomes are needed, such as the LHS. Engaging patients in these efforts will serve to enhance these pursuits by defining outcomes that matter most to patients, ensuring feasible study designs from the perspective of a potential participant, and engaging in dissemination efforts to reach the most important of end-users: our patients. Accordingly, concerted efforts must be taken to develop and amplify these partnerships, creating a network of engaged patients and caregivers eager and primed to partner in generating knowledge that has a direct impact on patient care.