PUrE Randomised Controlled Trial 2: Clinical and Cost Effectiveness of Flexible Ureterorenoscopy and Percutaneous Nephrolithotomy for Lower-pole Stones of 10-25 mm.

Background and objective: The European Association of Urology guidelines for lower-pole stones (LPS) recommend percutaneous nephrolithotomy (PCNL) for stones >20 mm, flexible ureterorenoscopy (FURS) or PCNL for stones of 10-20 mm if there are unfavourable factors for extracorporeal shockwave lithotripsy (ESWL), or any of these options for stones of 10-20 mm if there are no unfavourable factors for ESWL. PUrE RCT 2 was a pragmatic multicentre, open-label, superiority, randomised controlled trial comparing FURS versus PCNL in terms of quality of life, clinical effectiveness, and cost effectiveness for patients with LPS of 10-25 mm.

Methods: A total of 159 adults with LPS of 10-25 mm were randomised, 73 to FURS and 86 to PCNL. The primary outcome was health status area under the curve (AUC) measured weekly using the EQ-5D-5L questionnaire up to 12 wk after intervention. The primary economic outcome was the incremental cost per quality-adjusted life year (QALY) gained at 12 mo from randomisation.

Key finding and limitations: Mean health status AUC slightly favoured PCNL (difference -0.07, 95% confidence interval [CI] -0.11 to -0.02) when adjusted for baseline status. The complete stone clearance rate was higher with PCNL (71%) than with FURS (48%). PCNL was less costly (adjusted mean difference £733, 95% CI -£508 to £1973) and resulted in greater QALYs (adjusted mean difference -0.001, 95% CI -0.044 to 0.042). At a threshold value of £20 000 per QALY, PCNL has an 87% chance of being cost-effective.

Conclusions and clinical implications: Results from PUrE RCT 2 show that PCNL resulted in marginally better health status and a higher stone-free rate and was more cost-effective than FURS among patients with LPS of 10-25 mm. These outcomes are important for patient counselling and joint decision-making on LPS treatment.