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. 2022 Apr 5;12(4):e056789.
doi: 10.1136/bmjopen-2021-056789.

Comparative effectiveness of paediatric kidney stone surgery (the PKIDS trial): study protocol for a patient-centred pragmatic clinical trial

Jonathan S Ellison  1 , Matthew Lorenzo  2   3 , Hunter Beck  2   3 , Ruth Beck  2   3 , David I Chu  4   5 , Christopher Forrest  2   3 , Jing Huang  2   3   6 , Amy Kratchman  2   3 , Anna Kurth  2   3 , Laura Kurth  2   3 , Michael Kurtz  7 , Thomas Lendvay  8 , Renae Sturm  9 , Gregory Tasian  2   3   6 ; Pediatric KIDney Stone Care Improvement Network
Collaborators, Affiliations

Comparative effectiveness of paediatric kidney stone surgery (the PKIDS trial): study protocol for a patient-centred pragmatic clinical trial

Jonathan S Ellison et al. BMJ Open. .

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.

Keywords: Clinical trials; Protocols & guidelines; UROLOGY.

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Conflict of interest statement

Competing interests: JSE is a consultant for Dicerna Pharmaceuticals and Alnylam Pharmaceuticals. TL is cofounder of MicrobiomX and Singletto. GT is on the Scientific Advisory Board for Allena Pharmaceuticals, Dicerna Pharmaceuticals and Novome Biotechnology. The other authors have no relevant conflicts of interest to disclose.

Figures

Figure 1
Figure 1
PKIDS Care Improvement Network organisational chart. CHOP, Children’s Hospital of Philadelphia; PKIDS, Pediatric KIDney Stone.
Figure 2
Figure 2
Comparative effectiveness of Pediatric KIDney Stone surgery study flowchart. ED, emergency department; EHR, electronic health record.
Figure 3
Figure 3
QUIKKS. QUIKKS, Questionnaire for Urinary Issues–Kidney Stone Surgery.
Figure 4
Figure 4
Conceptual framework for evaluating the effectiveness of URS, SWL and PCNL with factors that influence the impact on choice of surgical intervention and outcomes. PCNL, percutaneous nephrolithotomy; SWL, shockwave lithotripsy; URS, ureteroscopy.
See this image and copyright information in PMC

References

    1. Tasian GE, Ross ME, Song L, et al. Annual incidence of nephrolithiasis among children and adults in South Carolina from 1997 to 2012. Clin J Am Soc Nephrol 2016;11:488-96. 10.2215/CJN.07610715 - DOI - PMC - PubMed
    1. Assimos D, Krambeck A, Miller NL, et al. Surgical management of stones: American urological Association/Endourological Society guideline, part I. J Urol 2016;196:1153–60. 10.1016/j.juro.2016.05.090 - DOI - PubMed
    1. Silay MS, Ellison JS, Tailly T, et al. Update on urinary stones in children: current and future concepts in surgical treatment and shockwave lithotripsy. Eur Urol Focus 2017;3:164–71. 10.1016/j.euf.2017.07.005 - DOI - PubMed
    1. He Q, Xiao K, Chen Y, et al. Which is the best treatment of pediatric upper urinary tract stones among extracorporeal shockwave lithotripsy, percutaneous nephrolithotomy and retrograde intrarenal surgery: a systematic review. BMC Urol 2019;19:98. 10.1186/s12894-019-0520-2 - DOI - PMC - PubMed
    1. Schaeffer AJ, Freeman M, Giambarresi L. Introduction to the National urology research agenda: a roadmap for priorities in urological disease research. Philadelphia, PA: Wolters Kluwer, 2010. - PubMed

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