Ellison JS. et al., 2022: Comparative effectiveness of paediatric kidney stone surgery (the PKIDS trial): study protocol for a patient-centred pragmatic clinical trial
Ellison JS, Lorenzo M, Beck H, Beck R, Chu DI, Forrest C, Huang J, Kratchman A, Kurth A, Kurth L, Kurtz M, Lendvay T, Sturm R, Tasian G.
Urology, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Perelman School of Medicine, Philadelphia, Pennsylvania, USA.
Division of Urology, Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.
Department of Urology, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.
Department of Biostatistics, Epidemiology, & Informatics, University of Pennsylvania, Philadelphia, Philadelphia, Pennsylvania, USA.
Boston Children's Hospital, Boston, Massachusetts, USA.
Seattle Children's Hospital, Seattle, Washington, USA.
David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California, USA.
Abstract
Introduction: The strength of the evidence base for the comparative effectiveness of three common surgical modalities for paediatric nephrolithiasis (ureteroscopy, shockwave lithotripsy and percutaneous nephrolithotomy) and its relevance to patients and caregivers are insufficient. We describe the methods and rationale for the Pediatric KIDney Stone (PKIDS) Care Improvement Network Trial with the aim to compare effectiveness of surgical modalities in paediatric nephrolithiasis based on stone clearance and lived patient experiences. This protocol serves as a patient-centred alternative to randomised controlled trials for interventions where clinical equipoise is lacking.
Methods and analysis: The PKIDS is a collaborative learning organisation composed of 26 hospitals that is conducting a prospective pragmatic clinical trial comparing the effectiveness of ureteroscopy, shockwave lithotripsy and percutaneous nephrolithotomy for youth aged 8-21 years with kidney and/or ureteral stones. Embedded within clinical care, the PKIDS trial will collect granular patient-level, surgeon-level and institution-level data, with a goal enrolment of 1290 participants over a 21-month period. The primary study outcome is stone clearance, defined as absence of a residual calculus of >4 mm on postoperative ultrasound. Secondary outcomes include patient-reported physical, emotional and social health outcomes (primarily using the Patient-Reported Outcome Measurement Information System), analgesic use and healthcare resource use. Timing and content of secondary outcomes assessments were set based on feedback from patient partners. Heterogeneity of treatment effect for stone clearance and patient-reported outcomes by participant and stone characteristics will be assessed.
Ethics and dissemination: This study is approved by the central institutional review board with reliance across participating sites. Participating stakeholders will review results and contribute to development dissemination at regional, national and international meetings.
Trial registration number: NCT04285658; Pre-results.
BMJ Open. 2022 Apr 5;12(4):e056789. doi: 10.1136/bmjopen-2021-056789. PMID: 35383073. FREE ARTICLE
Comments 1
The authors claim that RCTs, so commonly used today and considered as optimal scientific procedures, are associated with obvious shortcomings. In their opinion it is thus difficult to correctly analyse the clinical value of the three surgical procedures used for stone removal in the paediatric population: URS, SWL and PCNL. I fully agree on their doubts about the uncritical value of RCTs.
Accordingly, the authors have developed a trial protocol in which both stone clearance and patient experience are included.
The second part of the analysis is based on patient-reported outcomes, whereas the first part describes stone clearance expressed as no residuals > 4mm. The stone situation will be determined with standardized ultrasound protocol (developed for urinary stone disease). The outcomes will be recorded after 6 weeks and 3 months. Children’s experience will be summarized by reports from patients and family partners.
The points raised by the authors are both logical and important. It is necessary to consider, however, that the article presents what the authors hope to achieve, not what they already have achieved. The protocol is ambitious but given the well-recognized difficulties with studies of this kind I wish the authors good luck in their efforts and hope that the possibility to draw definite conclusions corresponds to their expectations. It is with excitement I look forward to the outcome in the 1290 children planned to be included at the end of June 2022 (starting August 2020).
Hans-Göran Tiselius