Randomized, controlled trials can provide high-quality, unbiased evidence for therapeutic interventions but are not always a practical or viable study design for certain healthcare decisions, such as those involving prognostic or predictive testing. Studies using large, real-world databases may be more appropriate and more generalizable to the intended target population of physicians and patients to answer these questions but carry potential for hidden bias. We illustrate several emerging methods of analyzing observational studies using propensity score matching (PSM) and coarsened exact matching (CEM). These advanced statistical methods are intended to reveal a "hidden experiment" within an observational database, and so refute or confirm a potential causal effect of assignment to an intervention and study outcome. We applied these methods to the Optum™ Research Database (ORD; Eden Prairie, MN) of electronic health records and administrative claims data to assess the effect of the 17-gene Genomic Prostate Score® (GPS™; Genomic Health, Redwood City, CA) assay on use of active surveillance (AS). In a traditional multivariable logistic regression, the GPS assay increased the use of AS by 29% (95% CI, 24%-33%). Upon applying the matching methods, the effect of the GPS assay on AS use varied between 27% and 80% and the matched data were significant among all algorithms. All matching algorithms performed well in identifying matched data that improved the imbalance in baseline covariates. By using different matching methods to assess causal inference in an observational database, we provide further confidence that the effect of the GPS assay on AS use is statistically significant and unlikely to be a result of confounding due to differences in baseline characteristics of the patients or the settings in which they were seen.
There is a paucity of studies addressing the accuracy of 24-hour urine collection for assessing stone risk parameters. Collection accuracy is thought to be essential for assigning optimal therapy for stone prevention. The objective of this study was to determine factors associated with accurate and inaccurate collections. During a 2-year period (2015-2016), 241 stone formers completed 24-hour urine collections. They were divided into accurate collectors (AC), defined as at least one accurate urine collection, and inaccurate collectors (IC). Accuracy was assessed by 24-hour urine creatinine (Cr) excretion indexed to body weight (normal: males, 20-25 mg Cr/kg; females, 15-20 mg Cr/kg). Demographic data analyzed included age, gender, race, insurance status, partner status, income, and education. Statistical analysis methods included the chi-square test, Fisher's exact test, and the two-group t-test. Average age was 50.7 years at the time of collection; 50.2% were men, 86% were white, and 14% were black. Overall, 51.0% of collections were inaccurate. There was no statistical significance between AC and IC for gender (P = 0.85), race (P = 0.90), insurance status (P = 0.85), recurrence (P = 0.87), stone type (P = 0.57), education (P = 0.35), income (P 5 0.42), or poverty (P = 0.35). Older age (P = 0.017) and having a partner (P = 0.022) were significantly associated with AC. The high rate of inaccurate 24-hour urine collections is a concern. The only factors we identified as influencing collection accuracy were age and partner status. These results underscore the importance of developing methods to improve the accuracy of collecting 24-hour urine samples.
The challenge to the urology community is to reduce the risks of screening and treatment by reducing the number of men undergoing unnecessary biopsy and whole-gland curative treatment of low-risk disease. There is compelling evidence that focal ablation of prostate cancer is truly minimally invasive and offers major functional advantages over whole-gland treatment.
Nocturia is a condition that has a tremendous impact on a patient's health and wellbeing. Getting up 2 or more times a night to urinate fragments sleep, preventing deep, restorative stages of the sleep cycle. With safer and more effective therapies, nocturia is a treatable condition that no longer should be overlooked. The simplicity of directly targeting the cause of nocturia, the overproduction of urine (ie, nocturnal polyuria) should be considered. Noctiva™ (desmopressin acetate) nasal spray (Avadel Pharmaceuticals plc, Chesterfield, MO), a novel FDA-approved microdose desmopressin nasal spray, can reduce nighttime urine production and potentially mitigate the potential harm of nocturia.