Saunders, Rhodri

Introduction: Guideline recommended surveillance of nonmuscle invasive bladder cancer (NMIBC) involves frequent cystoscopies. Cxbladder Monitor (CxbM) is a noninvasive urinary genomic test with high negative predictive value for NMIBC recurrence. Incorporation of CxbM into surveillance could reduce the frequency, burden, and cost of surveillance. We studied this impact using a theoretical health economic model. Materials and Methods: A Markov model for disease dynamics and decision tree model for surveillance pathways were created for 100 patients with theoretical NMIBC risk stratified by AUA criteria over 5 years from diagnosis. In the standard of care (SOC) arm, cystoscopy was performed per AUA guidelines. In the CxbM surveillance arm, CxbM was used starting 9 months after diagnosis. If positive, cystoscopy was performed per guidelines, but if negative, the cystoscopy was not performed and on the next scheduled encounter. Primary outcome was total budgetary impact. Additional outcomes included number of diagnostic tests, test-related complications, and patients with delayed diagnosis. Results: Compared with SOC, CxbM reduced mean total costs by $68,621 for 100 patients over 5 years or $137.24 per patient per year. Cystoscopy was reduced by 129 (0.31 per patient per year), and there was no difference in delayed cancer diagnosis. Using a probabilistic model, the median cost reduction per patient per year was $160 (95% credible interval −520 to 109) with 86.2% probability of cost savings. Conclusions: In this Markov model, incorporation of CxbM into surveillance for NMIBC reduced cystoscopy without negative impact on cancer detection with cost savings for the majority of simulations run.

Summary The importance of serum immunoglobulin (Ig)G concentration in IgG replacement therapy for primary immunodeficiency diseases is established in certain settings. Generally, IgG is infused via the intravenous (IVIG) or subcutaneous (SCIG) route. For IVIG infusion, published data demonstrate that higher IgG doses and trough levels provide patients with improved protection from infection. The same conclusions are not yet accepted for SCIG; data from two recent Phase III studies and a recent post-hoc analysis, however, suggest the same correlation between higher SCIG dose and serum IgG concentration and decreased incidence of infection seen with IVIG. Other measures of clinical efficacy have not been considered similarly. Thus, combined analyses of these and other published SCIG studies were performed; a full comparison of the 13 studies was, however, limited by non-standardized definitions and reporting. Despite these limitations, our analyses indicate that certain clinical outcomes improve at higher SCIG doses and associated higher serum IgG concentrations, and suggest that there might be opportunity to improve patient outcomes via SCIG dose adjustment.