Marguet, Charles G.

Purpose: The ASPiration to Improve Renal Calculi Removal Effectiveness study showed steerable ureteroscopic renal evacuation (SURE) with CVAC that significantly reduced residual stone volume at 30 days vs standard ureteroscopy (URS). This report presents 2-year health care consumption and stone events (SE) and their link to residual stone volume and other variables ( n = 101 at primary end point; n = 93 at 2 years). Materials and Methods: Logistic regression identified predictors of health care consumption events (HCEs), that is, emergency department visits, hospitalization, surgical retreatment, and SE across the study population. Incidence was compared between the treatment groups with Kaplan–Meier survival analysis. Results: At 2 years, residual stone volume was significantly associated with both HCEs and SE. For every 100 mm 3 increase in residual stone volume, the HCE risk increased by 50% to 54%, and the SE risk increased by 70%. Stone-free rate, the number of residual fragments (RFs), and RF total stone burden (based on diameter) were not predictors. Significantly fewer HCEs occurred in the SURE group (3 vs 20 events, p = 0.0004). Survival analysis confirmed this (4.3% vs 20%, log-rank p = 0.02), with a 73% risk reduction (hazard ratio 0.27, 95% Cl 0.09–0.80, p = 0.02). Fewer SE occurred in the SURE group, although not statistically significant. These subjects also had longer event-free survival. Conclusion: High-quality volumetric analysis supports evaluation of stone removal therapies, as residual stone volume predicts HCEs and SE. SURE significantly reduces the downstream incidence and risk of HCEs compared with standard URS.

Purpose: We report the first multicenter, prospective, randomized noninferiority controlled trial of steerable ureteroscopic renal evacuation (SURE) for nephrolithiasis treatment. Materials and Methods: Candidates for laser lithotripsy ≥18 years with ≥1 renal stone ≥7 mm and 7–20 mm stone burden were randomized 1:1 SURE vs ureteroscopy (URS). SURE was performed using the CVAC Aspiration System, a novel steerable irrigation–aspiration catheter. The primary efficacy end point was noninferiority in stone-free rate (SFR) based on zero residual fragments (RFs) 30 days postprocedure on noncontrast computed tomography. Secondary end points were superiority tests, stone clearance (percent stone volume reduction), residual stone volume (RSV), SFR (no RF >2 mm), and SFR (no RF >4 mm) 30 days postprocedure. Results: Of the subjects, 123 were randomized and 101 qualified for efficacy analysis (SURE 46 vs URS 55). Despite randomization, baseline URS stone volume was higher (SURE 485.0 ± 432.5 mm 3 vs URS 713.3 ± 558.5 mm 3 ). The primary noninferiority end point was achieved (SURE SFR 48% vs URS SFR 49%, p = 0.027, −1.3% [90% confidence interval; −18%, 15%]). Stone clearance was significantly higher for SURE vs URS subjects (SURE 96.9 ± 5.6% vs URS 92.9 ± 11.6%, p = 0.036); RSV was significantly lower (SURE 14.3 ± 3 0.9 mm 3 vs URS 70.2 ± 144.9 mm 3 , p = 0.012). SURE stone clearance and RSV were independent of baseline stone volume, but for URS degraded with increasing baseline stone volume. Safety was comparable between groups. Conclusions: SFR for SURE subjects was noninferior compared with URS subjects; stone clearance and RSV were significantly better with SURE, independent of baseline stone volume.

ABSTRACTAimsThis study aimed to determine the efficacy and safety of iltamiocel investigational autologous muscle cell therapy in females with stress urinary incontinence (SUI).MethodsAdult females were randomized 2:1 to iltamiocel (150 × 106 cells) or placebo and stratified by severity and prior SUI surgery. The primary objective was efficacy based on the frequency of stress incontinence episodes (SIE) recorded in a 3‐day diary at 12 months posttreatment. After 12 months, placebo participants could elect to receive open‐label iltamiocel. Efficacy and safety analyses were performed using all patients as treated populations.ResultsThe study enrolled 311 patients, 297 were randomized to either iltamiocel (n = 199) or placebo (n = 98). Of the 295 participants that completed 12 months blinded follow‐up, the proportion achieving the primary endpoint of ≥ 50% SIE reduction was not statistically different between treatment groups (52% vs. 53.6%; p = 0.798). A significantly greater proportion of iltamiocel participants in the prior SUI surgery stratum group achieved ≥ 75% SIE reduction compared with placebo, (40% vs. 16%; p = 0.037). Treatment response was maintained at 24 months in 78.4% and 64.9% of iltamiocel participants who achieved ≥ 50% and ≥ 75% SIE reduction, respectively, at Month 12. Adverse events related to the treatment were reported in 19 (9.5%) iltamiocel participants and 6 (6.1%) placebo participants.ConclusionThe study did not meet its primary endpoint however, iltamiocel cell therapy is safe and may be ideally suited to female patients who have undergone prior surgery for SUI. Additional study in this group of patients with high unmet medical needs is warranted.Trial RegistrationClinicalTrials.gov identifier: NCT01893138; EudraCT number: 2014‐002919‐41.