Phenotypic severity scoring system and categorisation for prune belly syndrome: application to a pilot cohort of 50 living patients.

OBJECTIVE: To design a novel system of scoring prune belly syndrome (PBS) phenotypic severity at any presenting age and apply it to a large pilot cohort. PATIENTS AND METHODS: From 2000 to 2017, patients with PBS were recruited to our prospective PBS study and medical records were cross-sectionally analysed, generating individualised RUBACE scores. We designed the pragmatic RUBACE-scoring system based on six sub-scores (R: renal, U: ureter, B: bladder/outlet, A: abdominal wall, C: cryptorchidism, E: extra-genitourinary, generating the acronym RUBACE), yielding a potential summed score of 0-31. The 'E' score was used to segregate syndromic PBS and PBS-plus variants. The cohort was scored per classic Woodard criteria and RUBACE scores compared to Woodard category. RESULTS: In all, 48 males and two females had a mean (range) RUBACE score of 13.8 (8-25) at a mean age of 7.3 years. Segregated by phenotypic categories, there were 39 isolated PBS (76%), six syndromic PBS (12%) and five PBS-plus (10%) cases. The mean RUBACE scores for Woodard categories 1, 2, and 3 were 20.5 (eight patients), 13.8 (25), and 10.6 (17), respectively (P < 0.001). CONCLUSIONS: RUBACE is a practical, organ/system level, phenotyping tool designed to grade PBS severity and categorise patients into isolated PBS, syndromic PBS, and PBS-plus groups. This standardised system will facilitate genotype-phenotype correlations and future prospective multicentre studies assessing medical and surgical treatment outcomes.

Managing Ureteropelvic Junction Obstruction in the Young Infant.

In the last decade, management of congenital UPJ obstruction has become progressively observational despite the lack of precise predictors of outcome. While it is clear that many children will have resolution of their hydronephrosis and healthy kidneys, it is equally clear that there are those in whom renal functional development is at risk. Surgical intervention for the young infant, under 6 months, has become relatively infrequent, yet can be necessary and poses unique challenges. This review will address the clinical evaluation of UPJO in the very young infant and approaches to determining in whom surgical intervention may be preferable, as well as surgical considerations for the small infant. There are some clinical scenarios where the need for intervention is readily apparent, such as the solitary kidney or in child with infection. In others, a careful evaluation and discussion with the family must be undertaken to identify the most appropriate course of care. Further, while minimally invasive pyeloplasty has become commonly performed, it is often withheld from those under 6 months. This review will discuss the key elements of that practice and offer a perspective of where minimally invasive pyeloplasty is of value in the small infant. The modern pediatric urologist must be aware of the various possible clinical situations that may be present with UPJO and feel comfortable in their decision-making and surgical care. Simply delaying an intervention until a child is bigger may not always be the best approach.

Comparison of Outcomes Between Preoperatively Potent Men Treated with Focal Versus Whole Gland Cryotherapy in a Matched Population.

BACKGROUND AND PURPOSE: The refinement in the localization of prostate cancer tumor foci through transperineal template-mapping biopsies and MRI has led to an increased interest in lesion-directed focal prostatic cryoablation. Data are lacking, however, that compare the outcomes of whole-gland (WG) to focal ablation therapy (FT). The aim of our study was to assess both oncologic and functional outcomes between WG and FT cryoablation of the prostate after having matched patients for preoperative characteristics. PATIENTS AND METHODS: We matched with a 1:1 ratio 317 men who underwent FT with 317 who underwent WG treatment in the Cryo Online Data (COLD) registry between 2007 and 2013. All patients were low-risk according to the D'Amico risk groups and were matched according to age at surgery. We only included preoperatively potent men. Oncologic outcomes were biochemical recurrence (BCR) free-survival defined according to the American Society for Radiation Oncology (ASTRO) and Phoenix criteria and assessed by Kaplan-Meier curves. Only patients with prostate-specific antigen (PSA) nadir data were included in oncologic outcome analysis. Functional outcomes were assessed at 6, 12, and 24 months after the procedure for erectile function (defined as ability to have intercourse with or without erectile aids), urinary continence, urinary retention, and rates of fistula formation. RESULTS: Median age at the time of the procedure was 66.5 years (standard deviation [SD] 6.6 y), and median follow-up time was 58.3 months. After surgery, 30% (n=95) and 17% (n=55) of the men who received WG and FT, respectively, underwent biopsy, with positive biopsy rates of 11.6% and 14.5%, respectively. BCR-free survival rates at 60 months according to the Phoenix definition were 80.1% and 71.3% in the WG and FT cohorts, respectively, with a hazard ratio of 0.827; according to the ASTRO definition, they were 82.1% and 73%, respectively (all P ≥ 0.1). Erectile function data at 24 months was available for 172 WG and 160 FT treated men. Recovery of erection was achieved in 46.8% and 68.8% of patients in the WG and FT cohorts, respectively (P=0.001). Urinary function data at 24 months was available for 307 WG and 313 FT patients. Continence rates were 98.7% and 100% for WG and FT groups, respectively (P=0.02). Urinary retention at 6, 12, and 24 months was reported in 7.3%, 1.9%, and 0.6%, respectively, in the WG arm, and in 5%, 1.3%, and 0.9%, respectively, in the FT arm. Finally, only one fistula was reported in each group. CONCLUSIONS: Men with low-risk prostate cancer who underwent FT cryoablation had comparable BCR-survival rates at 60 months to patients treated with WG. However, FT patients had higher erectile function preservation rates at 24 months post-procedure. Urinary continence, retention and fistula rates were similar between the two treatment groups.

Impact of the Site of Metastases on Survival in Patients with Metastatic Prostate Cancer.

BACKGROUND: Limited data exist on the impact of the site of metastases on survival in patients with stage IV prostate cancer (PCa). OBJECTIVE: To investigate the role of metastatic phenotype at presentation on mortality in stage IV PCa. DESIGN, SETTING, AND PARTICIPANTS: Overall, 3857 patients presenting with metastatic PCa between 1991 and 2009, included in the Surveillance Epidemiology and End Results-Medicare database were evaluated. OUTCOME MEASUREMENTS AND STATISTIC ANALYSES: Overall and cancer-specific survival rates were estimated in the overall population and after stratifying patients according to the metastatic site (lymph node [LN] alone, bone, visceral, or bone plus visceral). Multivariable Cox regression analyses tested the relationship between the site of metastases and survival. All analyses were repeated in a subcohort of patients with a single metastatic site involved. RESULTS AND LIMITATIONS: Respectively, 2.8%, 80.2%, 6.1%, and 10.9% of patients presented with LN, bone, visceral, and bone plus visceral metastases at diagnosis. Respective median overall survival and cancer-specific survival were 43 mo and 61 mo for LN metastases, 24 mo and 32 mo for bone metastases, 16 mo and 26 mo for visceral metastases, and 14 mo and 19 mo for bone plus visceral metastases (p<0.001). In multivariable analyses, patients with visceral metastases had a significantly higher risk of overall and cancer-specific mortality versus those with exclusively LN metastases (p<0.001). The unfavorable impact of visceral metastases persisted in the oligometastatic subgroup. Our study is limited by its retrospective design. CONCLUSIONS: Visceral involvement represents a negative prognostic factor and should be considered as a proxy of more aggressive disease in patients presenting with metastatic PCa. This parameter might indicate the need for additional systemic therapies in these individuals. PATIENT SUMMARY: Patients with visceral metastases should be considered as affected by more aggressive disease and might benefit from the inclusion in clinical trials evaluating novel molecules.

Head-to-head comparison of lymph node density and number of positive lymph nodes in stratifying the outcome of patients with lymph node-positive prostate cancer submitted to radical prostatectomy and extended lymph node dissection.

OBJECTIVE: The aim of this study was to compare the predictive ability of lymph node density (LND) and number of positive lymph nodes in patients with prostate cancer and lymph node invasion. MATERIALS AND METHODS: We included 568 patients with lymph node invasion treated with radical prostatectomy and extended pelvic lymph node dissection between January 1990 and July 2011 at a single center. The Kaplan-Meier method and multivariable Cox regression models tested the association between the number of positive lymph nodes or LND and cancer-specific survival (CSS). The predictive accuracy of a baseline model was assessed using Harrell's concordance index and then compared with that of a model including either the number of positive nodes or LND. RESULTS: The median number of positive lymph nodes was 2, whereas the median LND was 11.1%. At 5, 8, and 10 years, CSS rates were 92.5%, 83.9%, and 82.8%, respectively. At multivariable analyses, number of positive lymph nodes and LND, considered as continuous variables, were independent predictors of CSS (all P≤0.01). A 30% LND cutoff was found to be highly predictive of CSS (P = 0.004), and a cutoff of 2 positive nodes was confirmed to be a strong predictor of CSS (P = 0.02). The number of positive nodes and LND similarly, continuous or dichotomized, increased the accuracy for CSS predictions (0.68-0.69 vs. 0.61 of baseline model). LND cutoff of 30% increased the discrimination the most (0.69; +0.083). CONCLUSIONS: The number of positive lymph nodes and LND showed comparable discriminative power for long-term CSS predictions. A cutoff of 30% LND might be suggested for the selection of patients candidate for adjuvant systemic therapy, because it increased the model's discrimination the most.

Incidence, survival and mortality rates of stage-specific bladder cancer in United States: a trend analysis.

PURPOSE: To examine the overall and stage-specific age-adjusted incidence, 5-year survival and mortality rates of bladder cancer (BCa) in the United States, between 1973 and 2009. MATERIALS AND METHODS: A total of 148,315 BCa patients were identified in the Surveillance, Epidemiology and End Results database, between years 1973 and 2009. Incidence, mortality, and 5-year cancer-specific survival rates were calculated. Temporal trends were quantified using the estimated annual percentage change (EAPC) and linear regression models. All analyses were stratified according to disease stage, and further examined according to sex, race, and age groups. RESULTS: Incidence rate of BCa increased from 21.0 to 25.5/100,000 person-years between 1973 and 2009. Stage-specific analyses revealed an increase incidence for localized stage: 15.4-20.2 (EAPC: +0.5%, p < 0.001) and distant stage: 0.5-0.8 (EAPC: +0.7%, p = 0.001). Stage-specific 5-year survival rates increased for all stages, except for distant disease. No significant changes in mortality were recorded among localized (EAPC: -0.2%, p = 0.1) and regional stage (EAPC: -0.1%, p = 0.5). An increase in mortality rates was observed among distant stage (EAPC: +1.0%, p = 0.005). Significant variations in incidence and mortality were recorded when estimates were stratified according to sex, race, and age groups. DISCUSSION: Albeit statistically significant, virtually all changes in incidence and mortality were minor, and hardly of any clinical importance. Little or no change in BCa cancer control outcomes has been achieved during the study period.

Assessing the most accurate formula to predict the risk of lymph node metastases from prostate cancer in contemporary patients treated with radical prostatectomy and extended pelvic lymph node dissection.

BACKGROUND AND PURPOSE: The aim of this study was to perform a head-to-head comparison of the Roach formula vs. two other newly developed prediction tools for lymph node invasion (LNI) in prostate cancer, namely the Nguyen and the Yu formulas. MATERIAL AND METHODS: We included 3115 patients treated with radical prostatectomy and extended pelvic lymph node dissection (ePLND), between 2000 and 2010 at a single center. The predictive accuracy of the three formulas was assessed and compared using the area-under-curve (AUC) and calibration methods. Moreover, decision curve analysis compared the net-benefit of the three formulas in a head-to-head fashion. RESULTS: Overall, 10.8% of patients had LNI. The LNI-predicted risk was >15% in 25.5%, 3.4%, and 10.2% of patients according to the Roach, Nguyen and Yu formula, respectively. The AUC was 80.5%, 80.5% and 79%, respectively (all p>0.05). However, the Roach formula demonstrated more favorable calibration and generated the highest net-benefit relative to the other examined formulas in decision curve analysis. CONCLUSIONS: All formulas demonstrated high and comparable discrimination accuracy in predicting LNI, when externally validated on ePLND treated patients. However, the Roach formula showed the most favorable characteristics. Therefore, its use should be preferred over the two other tools.

Selecting the optimal candidate for adjuvant radiotherapy after radical prostatectomy for prostate cancer: a long-term survival analysis.

BACKGROUND: The role of adjuvant radiotherapy (ART) after radical prostatectomy (RP) on survival of patients with prostate cancer (PCa) is still controversial. OBJECTIVE: We tested the impact of ART on cancer-specific mortality (CSM) and overall mortality (OM) in PCa patients according to pathologic PCa features. DESIGN, SETTING, AND PARTICIPANTS: We evaluated 1049 PCa patients treated with RP and extended pelvic lymph node dissection alone or in combination with adjuvant treatments between 1998 and 2008. All patients had positive surgical margins and/or pT3/pT4 disease with or without positive lymph nodes. OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS: Cox regression analyses tested the relationship between pathologic characteristics and CSM rates. Independent predictors of survival were used to develop a novel risk score based on the number of risk factors. Finally, Cox regression models tested the relationship between ART and survival according to the number of risk factors. RESULTS AND LIMITATIONS: On multivariable analyses, only pathologic Gleason score ≥ 8, pT3b/T4 stage, and presence of positive lymph nodes represented independent predictors of CSM (all p ≤ 0.02). The cumulative number of these pathologic findings was used to develop a risk score, which was 0, 1, 2, and 3 in 43.6%, 22.1%, 20.7%, and 13.6% of patients, respectively. In patients sharing more than two mentioned predictors of CSM (primarily having a risk score of 0 or 1), ART did not significantly improve survival (all p ≥ 0.4). Conversely, in patients with a risk score ≥ 2, ART was associated with lower CSM and OM rates (all p=0.006). The observational nature of the cohort represents a limitation of the study. CONCLUSIONS: ART significantly improved survival only in patients with at least two of the following pathologic features at RP: Gleason score ≥ 8, pT3/pT4 disease, and positive lymph nodes. These patients represent the ideal candidates for ART after RP.

Performance characteristics of computed tomography in detecting lymph node metastases in contemporary patients with prostate cancer treated with extended pelvic lymph node dissection.

BACKGROUND: Computed tomography (CT) is a commonly used noninvasive procedure for prostate cancer (PCa) staging. All previous studies addressing the ability of CT scan to predict lymph node invasion (LNI) were based on historical patients treated with limited pelvic lymph node dissection (PLND). OBJECTIVE: Assess the value of CT in predicting LNI in contemporary PCa patients treated with extended PLND (ePLND). DESIGN, SETTING, AND PARTICIPANTS: We evaluated 1541 patients undergoing radical prostatectomy and ePLND between 2003 and 2010 at a single center. All patients were preoperatively staged using abdominopelvic CT scan. All lymph nodes with a short axis diameter ≥ 10 mm were considered suspicious for metastatic involvement. INTERVENTION: All patients underwent preoperative CT scan, radical retropubic prostatectomy, and ePLND, regardless of PCa features at diagnosis. MEASUREMENTS: The performance characteristics of CT scan were tested in the overall patient population, as well as according to the National Comprehensive Cancer Network (NCCN) classification and according to the risk of LNI derived from a nomogram developed on an ePLND series. Logistic regression models tested the relationship between CT scan findings and LNI. Discrimination accuracy was quantified with the area under the curve. RESULTS AND LIMITATIONS: Overall, a CT scan that suggested LNI was found in 73 patients (4.7%). Of them, only 24 patients (32.8%) had histologically proven LNI at ePLND. Overall, sensitivity, specificity, and accuracy of CT scan were 13%, 96.0%, and 54.6%, respectively. In patients with low-, intermediate-, or high-risk PCa according to NCCN classification, sensitivity was 8.3%, 96.3%, and 52.3%, respectively; specificity was 3.6%, 97.3%, and 50.5%, respectively; and accuracy was 17.9%, 94.3%, and 56.1%, respectively. Similarly, in patients with a nomogram-derived LNI risk ≥ 50%, sensitivity, specificity, and accuracy were only 23.9%, 94.7%, and 59.3%, respectively. At multivariable analyses, inclusion of CT scan findings did not improve the accuracy of LNI prediction (81.4% compared with 81.3%; p=0.8). Lack of a central scan review represents the main limitation of our study. CONCLUSIONS: In contemporary patients with PCa, the accuracy of CT scan as a preoperative nodal-staging procedure is poor, even in patients with high LNI risk. Therefore, the need for and the extent of PLND should not be based on the results obtained by CT scan.