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

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.