The PUrE randomised controlled trial 1: Clinical and cost effectiveness of flexible ureterorenoscopy and extracorporeal shockwave lithotripsy for lower pole stones of ≤10 mm

Abstract

Background and objective: The European Association of Urology guidelines recommend that extracorporeal shockwave lithotripsy (ESWL) and flexible ureterorenoscopy (FURS) are equally appropriate first-line treatment options for lower pole renal stones (LPSs). We report a pragmatic multicentre, open-label, superiority randomised controlled trial (RCT) comparing FURS versus ESWL for quality of life, and clinical and cost effectiveness for LPSs of ≤10 mm-one of two RCTs that were part of the Percutaneous nephrolithotomy, flexible Ureteroscopy and Extracorporeal shockwave lithotripsy for lower pole kidney stone study (PUrE), with the other PUrE RCT 2 investigating larger stones.

Methods: In total, 231 and 230 patients were randomised to FURS and ESWL, respectively. The primary outcome was health status area under the curve (AUC) 12 wk after intervention using the EQ-5D-5L questionnaire. The primary economic outcome was the incremental cost per quality-adjusted life-year (QALY) gained at 12 mo after randomisation.

Key findings and limitations: The mean health status AUCs over 12 wk were 0.807 (standard deviation [SD] 0.205) for FURS (n = 164) and 0.826 (SD 0.207) for ESWL (n = 188); the between-group difference was 0.024 (95% confidence interval -0.004, 0.053), a small nonsignificant difference in favour of FURS adjusted for an initial baseline imbalance. Complete stone clearance was higher with FURS (72%) than with ESWL (36%). The incremental cost-effectiveness ratio between FURS and ESWL was £65 163, meaning that at a threshold value of £20 000 per QALY, ESWL has a >99% chance of being cost effective.

Conclusions and clinical implications: PUrE RCT 1 found that there was no evidence of a difference in health status between FURS and ESWL for LPSs of ≤10 mm, with FURS leading to a higher stone-free rate. ESWL was more cost effective than FURS.

Keywords: Extracorporeal shockwave lithotripsy; Flexible ureterorenoscopy; Lower pole stone; Randomised controlled trial; Renal stone.