Zinner syndrome with contralateral hydronephrosis: A rare congenital condition.

Zinner Syndrome (ZS) is a rare congenital genitourinary abnormality defined by seminal vesicle cysts, ejaculatory duct obstruction, and unilateral renal dysplasia or agenesis. Patients can be asymptomatic, while others experience pain, urinary or ejaculatory symptoms and infertility. A patient that presented with painless gross hematuria was found to have a large pelvic cystic structure, an absent left kidney, multiple fluid collections in the region of the left seminal vesicle and right hydronephrosis. Hydronephrosis is atypical in ZS. This patient eventually developed right flank and pelvic pain treated with robotic-assisted laparoscopic excision of the pelvic cystic structure and extravesical ureteral reimplantation.

Robot-Assisted Repair of Bladder Rupture following Penile Ring Entrapment.

Penile rings have been used to help sustain erection and enhance sexual pleasure for centuries. Constriction of the penis reduces the outflow of blood from the cavernosal tissue. However, if left for an extended time period, a condition called penile ring entrapment can occur. This may result in severe edema, gangrene, necrosis, and even penile amputation. Penile ring entrapment is a very rare condition; complete urinary obstruction with concomitant bladder rupture as a result renders this case even more extraordinary. We discuss our experience in the management of a 64-year-old man, who presented with altered mental status and inability to urinate, found to have penile ring entrapment and intraperitoneal bladder rupture. Removal of the constricting ring was performed in the ED, and bladder injury and penile necrosis were subsequently repaired with robot-assisted laparoscopic cystorrhaphy, penectomy, and perineal urethrostomy.

Femoral-Femoral Bypass for Limb Salvage to Correct Common Iliac Occlusion after Lower Extremity Trauma.

BACKGROUND: Roadway injuries are a leading cause of lower extremity vascular trauma. Treating these injuries involves controlling life-threatening hemorrhage and restoring distal perfusion. MATERIALS AND METHODS: We describe a unique presentation of chronic iliac artery occlusion in the setting of blunt trauma requiring extra-anatomic bypass for maximal limb salvage. RESULTS: A 50-year-old male presented after a pedestrian versus auto accident. He had mangled bilateral lower extremities and was taken emergently for lower extremity amputations. He was found to have chronic left common iliac occlusion and a femoral-femoral bypass was performed to assist with healing his left below-the-knee amputation. DISCUSSION: Lack of adequate perfusion can cause poor outcomes in limb salvage. This case demonstrated that lower extremity trauma can be complicated by chronic vascular disease. Reperfusion and adequate wound healing can be accomplished by using bypass grafting after more traditional reperfusion techniques fail.

Role of the androgen, estrogen, and progesterone receptors in adherent perinephric fat in robotic partial nephrectomy.

To determine whether androgen, estrogen, and/or progesterone signaling play a role in the pathophysiology of adherent perinephric fat (APF). We prospectively recruited patients undergoing robotic assisted partial nephrectomy during 2015-2017. The operating surgeon documented the presence or absence of APF. For those with clear cell renal cell carcinoma (ccRCC), representative sections of tumor and perinephric fat were immunohistochemically stained with monoclonal antibody to estrogen α, progesterone, and androgen receptors. Patient characteristics, operative data, and hormone receptor presence were compared between those with and without APF. Of 51 patients total, 18 (35.3%) and 33 (64.7%) patients did and did not have APF, respectively. APF was associated with history of diabetes mellitus (61.1% vs 24.2%, p = 0.009) and larger tumors (4.0 cm vs 3.0 cm, p = 0.017) but not with age, gender, BMI, Charleston comorbidity index, smoking, or preoperative estimated glomerular filtration rate. APF was not significantly associated with length of operation, positive margins, or 30-day postoperative complications but incurred higher estimated blood loss (236.5 mL vs 209.2 mL, p = 0.049). Thirty-two had ccRCC and completed hormone receptor staining. The majority of tumors and perinephric fat were negative for estrogen and progesterone while positive for androgen receptor expression. There was no difference in hormone receptor expression in either tumor or perinephric fat when classified by presence or absence of APF (p > 0.05). APF is more commonly present in patients with diabetes or larger tumors but was not associated with differential sex hormone receptor expression in ccRCC.

Comparing Off-clamp and On-clamp Robot-assisted Partial Nephrectomy: A Prospective Randomized Trial.

OBJECTIVE: To determine whether performing robot-assisted partial nephrectomy without warm ischemia "off-clamp" results in favorable postoperative renal functional outcomes compared with the on-clamp method. METHODS: We conducted a prospective trial of 80 patients who underwent robot-assisted partial nephrectomy. They were randomized in a 1:1 ratio to undergo the procedure with renal artery clamping or without clamping. The groups were compared across demographics, operative information, perioperative outcomes, and postoperative renal function. We assessed renal function by estimated glomerular filtration rate and renal scintigraphy both preoperatively and at 3 months postoperatively. RESULTS: Patients in the on-clamp and off-clamp groups were similar in age, gender, body mass index, comorbidities, clinical tumor size, nephrometry score, and laterality. Off-clamp procedures were lengthier at an average 178.0 minutes vs 156.0 minutes for on-clamp (P = .011). Estimated blood loss, rates of pelvicalyceal repair, postoperative complications, and positive margins were not different. At a median 3-month follow-up, no significant differences were seen in change in postoperative estimated glomerular filtration rate or percent split renal function between both groups. CONCLUSION: In this prospective study, off-clamp robot-assisted partial nephrectomy resulted in similar perioperative outcomes compared with the on-clamp technique. No benefit was demonstrated in the preservation of renal function. Urologists may safely employ either an on-clamp or off-clamp strategy depending on surgeon preference and patient-specific factors including baseline renal insufficiency, multiple masses, or solitary kidney.

Development of Reliable, Valid and Responsive Scoring Systems for Endoscopy and Histology in Animal Models for Inflammatory Bowel Disease.

BACKGROUND AND AIMS: Although several endoscopic and histopathologic indices are available for evaluating the severity of inflammation in mouse models of colitis, the reliability of these scoring instruments is unknown. Our aim was to evaluate the reliability of the individual items in the existing indices and develop new scoring systems by selection of the most reliable index items. METHODS: Two observers scored the histological slides [n = 224] and endoscopy videos [n = 201] from treated and untreated Interleukin[IL]-10 knock-out and T-cell transferred SCID mice. Intra-rater and inter-rater reliability for endoscopy and histology scores, and each individual item, were measured using intraclass correlation coefficients [ICCs]. The Mouse Colitis Histology Index [MCHI] and Mouse Colitis Endoscopy Index [MCEI] were developed using the most reliable items. Both were correlated to the colon density and to each other and were evaluated for their ability to detect changes in pathobiology. RESULTS: The intraclass correlation coefficients (ICCs) for inter-rater agreement (95% CIs) for the total histology and endoscopy scores were 0.90 [0.87-0.92] and 0.80 [0.76-0.84], respectively. The MCHI and MCEI were highly correlated with colon density, with a Spearman Rho = 0.81[0.75-0.85] and 0.73 [0.66-0.79], respectively, and with each other, Spearman Rho = 0.71 [0.63-0.77]. The MCHI and MCEI were able to distinguish between the experimental groups within the models, with pairwise differences between the treated and untreated groups being statistically significant [p < 0.001]. CONCLUSIONS: These histological and endoscopic indices are valid and reliable measures of intestinal inflammation in mice, and they are responsive to treatment effects in pre-clinical studies.

Performance of Crohn's disease Clinical Trial Endpoints based upon Different Cutoffs for Patient Reported Outcomes or Endoscopic Activity: Analysis of EXTEND Data.

Background: Clinical trial endpoints for Crohn's disease (CD) activity correlate poorly with mucosal inflammation; to assess treatment efficacy, patient-reported outcomes and endoscopic assessments are preferred. This study assessed the impact on treatment efficacy estimations of using different definitions of clinical and endoscopic remission and endoscopic response, and of using site- or central-based endoscopy evaluation. Methods: This post hoc analysis of data fromEXTEND (extend the safety and efficacy of adalimumab through endoscopic healing), a placebo (PBO)-controlled, randomized trial of adalimumab (ADA) for mucosal healing, included adults with moderate-to-severe CD. Subsets of patients meeting specified Simplified Endoscopic Score for CD (SES-CD) inclusion criteria, according to site or central reading, and baseline stool frequency (SF) and/or abdominal pain score (AP) thresholds were evaluated. Various endpoint definitions based on the Crohn's Disease Activity Index (CDAI), its SF and AP components, SES-CD, and composite endpoints were compared between treatment groups. Results: Increased stringency of Week 12 clinical endpoints compared to CDAI<150 to SF≤3.0/1.5&AP≤1.0 reduced PBO response rates by ≥12% and increased treatment effects by ≤10%. Amending the SES-CD endpoint from ≤4 to ≤2 reduced the treatment effect from 24% to 8%. Composite endpoints further diminished response rates and effect sizes. Site-based evaluation was associated with lower remission rates versus central reading in the PBO group and, thus, greater ADA-related treatment effects. Conclusions: This analysis is the first to demonstrate that increasing the stringency of clinical and endoscopic endpoint definitions in CD trials, especially lowering SF or SES-CD definitions, reduces the ability to detect treatment-related change in CD activity; focus on endpoints that reflect clinical change is warranted.

Development and Validity of a Silicone Renal Tumor Model for Robotic Partial Nephrectomy Training.

OBJECTIVE: To provide a training tool to address the technical challenges of robot-assisted laparoscopic partial nephrectomy, we created silicone renal tumor models using 3-dimensional printed molds of a patient's kidney with a mass. In this study, we assessed the face, content, and construct validity of these models. MATERIALS AND METHODS: Surgeons of different training levels completed 4 simulations on silicone renal tumor models. Participants were surveyed on the usefulness and realism of the model as a training tool. Performance was measured using operation-specific metrics, self-reported operative demands (NASA Task Load Index [NASA TLX]), and blinded expert assessment (Global Evaluative Assessment of Robotic Surgeons [GEARS]). RESULTS: Twenty-four participants included attending urologists, endourology fellows, urology residents, and medical students. Post-training surveys of expert participants yielded mean results of 79.2 on the realism of the model's overall feel and 90.2 on the model's overall usefulness for training. Renal artery clamp times and GEARS scores were significantly better in surgeons further in training (P ≤.005 and P ≤.025). Renal artery clamp times, preserved renal parenchyma, positive margins, NASA TLX, and GEARS scores were all found to improve across trials (P <.001, P = .025, P = .024, P ≤.020, and P ≤.006, respectively). CONCLUSION: Face, content, and construct validity were demonstrated in the use of a silicone renal tumor model in a cohort of surgeons of different training levels. Expert participants deemed the model useful and realistic. Surgeons of higher training levels performed better than less experienced surgeons in various study metrics, and improvements within individuals were observed over sequential trials. Future studies should aim to assess model predictive validity, namely, the association between model performance improvements and improvements in live surgery.

Reliability of Measuring Ileo-Colonic Disease Activity in Crohn's Disease by Magnetic Resonance Enterography.

Background: Magnetic resonance enterography is increasingly utilized for assessment of luminal Crohn's disease activity. The Magnetic Resonance Index of Activity and the London Index are the most commonly used outcome measures in clinical trials. We assessed the reliability of these indices and several additional items. Methods: A consensus process clarified scoring conventions and identified additional items based on face validity. Four experienced radiologists evaluated 50 images in triplicate, in random order, at least 1 month apart, using a central image management system. Intra- and interrater reliability were assessed by calculating and comparing intraclass correlation coefficients. Results: Intrarater intraclass correlation coefficients (95% confidence intervals) for the Magnetic Resonance Index of Activity, London, and London "extended" indices and a visual analogue scale were 0.89 (0.84 to 0.91), 0.87 (0.83 to 0.90), 0.89 (0.85 to 0.92), and 0.86 (0.81 to 0.90). Corresponding interrater intraclass correlation coefficients were 0.71 (0.61 to 0.77), 0.67 (0.55 to 0.75), 0.70 (0.61 to 0.76), and 0.71 (0.62 to 0.77). Reliability for each index was greatest in the terminal ileum and poorest in the rectum. All 3 indices were highly correlated with the visual analogue scale; 0.79 (0.71 to 0.85), 0.78 (0.71 to 0.84), and 0.79 (0.72 to 0.85) for the Magnetic Resonance Index of Activity, London, and the London "extended" indices, respectively. Conclusions: "Substantial" interrater reliability was observed for all 3 indices. Future studies should assess responsiveness to treatment in order to confirm their utility as evaluative indices in clinical trials and clinical practice.

Retroperitoneal access for robotic renal surgery.

INTRODUCTION AND OBJECTIVE: Retroperitoneal access for robotic renal surgery is an effective alternative to the commonly used transperitoneal approach. We describe our contemporary experience and technique for attaining retroperitoneal access. MATERIALS AND METHODS: We outline our institutional approach to retroperitoneal access for the instruction of urologists at the beginning of the learning curve. The patient is placed in the lateral decubitus position. The first incision is made just inferior to the tip of the twelfth rib as described by Hsu, et al. After the lumbodorsal fascia is traversed, the retroperitoneal space is dilated with a round 10 millimeter AutoSutureTM (Covidien, Mansfield, MA) balloon access device. The following trocars are used: A 130 millimeter KiiR balloon trocar (Applied Medical, Rancho Santa Margarita, CA), three robotic, and one assistant. Key landmarks for the access and dissection are detailed. RESULTS: 177 patients underwent a retroperitoneal robotic procedure from 2007 to 2015. Procedures performed include 158 partial nephrectomies, 16 pyeloplasties, and three radical nephrectomies. The robotic fourth arm was utilized in all cases. When compared with the transperitoneal approach, the retroperitoneal approach was associated with shorter operative times and decreased length of stay (1). Selection bias and surgeon preference accounted for the higher proportion of patients who underwent partial nephrectomy off-camp via the retroperitoneal approach. CONCLUSIONS: Retroperitoneal robotic surgery may confer several advantages. In patients with previous abdominal surgery or intra-abdominal conditions, the retroperitoneum can be safely accessed while avoiding intraperitoneal injuries. The retroperitoneum also provides a confined space that may minimize the sequelae of potential complications including urine leak. Moreover, at our institution, retroperitoneal robotic surgery is associated with shorter operative times and a decreased length of stay when compared with the transperitoneal approach (2). In selected patients, the retroperitoneal approach is a viable alternative to the transperitoneal approach for a variety of renal procedures.

Disease activity indices in coeliac disease: systematic review and recommendations for clinical trials.

OBJECTIVE: Although several pharmacological agents have emerged as potential adjunctive therapies to a gluten-free diet for coeliac disease, there is currently no widely accepted measure of disease activity used in clinical trials. We conducted a systematic review of coeliac disease activity indices to evaluate their operating properties and potential as outcome measures in registration trials. DESIGN: MEDLINE, EMBASE and the Cochrane central library were searched from 1966 to 2015 for eligible studies in adult and/or paediatric patients with coeliac disease that included coeliac disease activity markers in their outcome measures. The operating characteristics of histological indices, patient-reported outcomes (PROs) and endoscopic indices were evaluated for content and construct validity, reliability, responsiveness and feasibility using guidelines proposed by the US Food and Drug Administration (FDA). RESULTS: Of 19 123 citations, 286 studies were eligible, including 24 randomised-controlled trials. Three of five PROs identified met most key evaluative criteria but only the Celiac Disease Symptom Diary (CDSD) and the Celiac Disease Patient-Reported Outcome (CeD PRO) have been approved by the FDA. All histological and endoscopic scores identified lacked content validity. Quantitative morphometric histological analysis had better reliability and responsiveness compared with qualitative scales. Endoscopic indices were infrequently used, and only one index demonstrated responsiveness to effective therapy. CONCLUSIONS: Current best evidence suggests that the CDSD and the CeD PRO are appropriate for use in the definition of primary end points in coeliac disease registration trials. Morphometric histology should be included as a key secondary or co-primary end point. Further work is needed to optimise end point configuration to inform efficient drug development.

Off-clamp robot-assisted partial nephrectomy does not benefit short-term renal function: a matched cohort analysis.

In the interest of renal functional preservation, partial nephrectomy has supplanted radical nephrectomy as the preferred treatment for T1 renal masses. This procedure usually involves the induction of renal warm ischemia by clamping the hilar vessels prior to tumor excision. Performing robot-assisted partial nephrectomy (RAPN) "off-clamp" can theoretically prevent renal functional loss associated with warm ischemia. We describe our institutional experience and compare perioperative and renal functional outcomes using a propensity score matched cohort. We conducted a retrospective comparison from a prospectively maintained database of all patients who underwent RAPN from 2009 to 2015. Of those patients, 143 underwent off-clamp RAPN. Fifty off-clamp RAPN patients were propensity score matched with fifty clamped RAPN patients based on renal function, tumor size, and R.E.N.A.L. nephrometry score. The cohorts were compared across demographics, operative information, perioperative outcomes, and renal functional outcomes. For all off-clamp RAPN patients, mean nephrometry score was 7.1, mean estimated blood loss (EBL) was 236.9 mL, perioperative complication rate was 7.7%, and mean decrease in estimated glomerular filtration rate (eGFR) was 7.1% at a median follow-up of 9.2 months. In the propensity score matched cohorts, off-clamp RAPN resulted in a shorter mean operative time (172.0 versus 196.0 min, p = 0.025) and a lower mean EBL (179.7 versus 283.2 mL, p = 0.046). A lower complication rate of 6.0% in the off-clamp group compared with 20.0% in the clamped group approached significance (p = 0.071). Mean preoperative eGFR was similar in both cohorts. Importantly, there was no significant difference in decrease in eGFR between the clamped cohort (9.8%) and off-clamp cohort (11.9%) at a median follow-up of 9.0 months (p = 0.620). Off-clamp RAPN did not result in improved renal functional preservation in our experience. Surprisingly, the off-clamp cohort experienced lower intraoperative blood loss, shorter operative times, and fewer complications.

Systematic Review: Disease Activity Indices in Eosinophilic Esophagitis.

OBJECTIVES: There is no clear consensus regarding the most appropriate measure(s) of eosinophilic esophagitis (EoE) disease activity. We aimed to identify all scoring indices used for the measurement of disease activity in EoE, appraise their operating properties, and discuss their value as outcome measures. METHODS: MEDLINE, EMBASE, and CENTRAL (The Cochrane library) were searched from inception to 11 May 2016. Randomized controlled trials (RCTs), cohort, case-control, and cross-sectional studies that reported outcomes to measure EoE disease activity or response to treatment were eligible. Operating properties of histologic, endoscopic, and patient reported/symptomatic and health-related quality of life measures were critically appraised according to guidelines proposed by the United States Food and Drug Administration. RESULTS: Of 4,373 citations, 130 studies were eligible, of which 20 were RCTs. Although no index met all evaluative criteria, we found that: (1) the EoE histologic scoring system (EoEHSS) is the most valid histologic measure; (2) the Endoscopic Reference Score (EREFS) is the most reliable and responsive endoscopy measure; and (3) the Eosinophilic Esophagitis Activity Index (EEsAI) or the Dysphagia Symptoms Questionnaire (DSQ) had superior construct validity and responsiveness in adults. The Pediatric Quality of Life Inventory EoE was the most valid pediatric symptomatic measure. CONCLUSIONS: Current evidence supports the use of the EoEHSS and EREFS as measures of histologic and endoscopic EoE disease activity, respectively, and the EEsAI, DSQ, or Pediatric Quality of Life Inventory EoE as measures of adult and pediatric symptoms. Additional research is needed to optimize endpoint configuration to facilitate development of new therapies.

Trends in the Management of Small Renal Masses: A Survey of Members of the Endourological Society.

Treatment modalities for small renal masses (SRMs) include open or minimally invasive radical or partial nephrectomy, and laparoscopic or percutaneous ablations. Members of the Endourological Society were surveyed to evaluate how practitioner and clinical practice characteristics may be associated with the management of SRMs over time. The survey assessed characteristics of urologists (recency of residency and fellowship training, clinical practice type and location, and treatment modalities available) and their management of SRMs over the past year and over the course of the year 5 years prior. Of the 1495 surveys e-mailed, there were 129 respondents (8.6%). Comparing the past year to 5 years prior, there was increasing utilization of robotic partial nephrectomy (p < 0.001) and robotic radial nephrectomy (p = 0.031). In contrast, there was decreasing utilization of open partial nephrectomy (p < 0.001), open radical nephrectomy (p = 0.039), laparoscopic partial nephrectomy (p = 0.002), and laparoscopic radical nephrectomy (p = 0.041). Employment of laparoscopic ablation decreased (p = 0.001), but that of percutaneous ablation did not change significantly. For masses treated with image-guided therapy, there was increasing utilization of microwave ablation (p = 0.008) and decreasing usage of radiofrequency ablation (p = 0.002). Future studies should focus on the most effective treatment modalities based on provider, patient, and tumor characteristics.

Histologic scoring indices for evaluation of disease activity in ulcerative colitis.

BACKGROUND: Disease activity can be determined using clinical, endoscopic or histologic criteria in patients with ulcerative colitis (UC). Persistent disease activity is associated with poor outcomes. Histologic disease activity has been shown to be associated with relapse, colectomy and colorectal cancer. The ability to objectively evaluate microscopic disease activity using histology is important for both clinical practice and clinical trials. However, the operating properties of the currently available histologic indices remain unclear. OBJECTIVES: A systematic review was undertaken to identify and evaluate the development and operating characteristics of histologic disease activity indices used to assess disease activity in people with ulcerative colitis. SEARCH METHODS: We searched MEDLINE, EMBASE, PubMed, CENTRAL and the Cochrane IBD Review Group Specialized Trials Register from inception to 2 December 2016 for applicable studies. There were no language or document type restrictions. SELECTION CRITERIA: Any study design (e.g. randomized controlled trials, cohort studies, case series) that evaluated a histologic index in patients with UC were considered for inclusion. Eligible patients were adults (> 18 years), diagnosed with UC using conventional clinical, radiographic, endoscopic and histologic criteria. DATA COLLECTION AND ANALYSIS: Two authors (MHM and CEP) independently reviewed the titles and abstracts of the studies identified from the literature search. A standardized form was used to assess eligibility of trials for inclusion and for data extraction.Two authors (MHM and CEP) independently extracted and recorded data, which included the number of patients enrolled, number of patients per treatment arm, patient characteristics including age and gender distribution, and the name of the histologic index. Outcomes (i.e. intra-rater reliability, inter-rater reliability, internal consistency, content validity, criterion validity, construct validity, responsiveness, and feasibility) were recorded for each trial. MAIN RESULTS: In total, 126 reports describing 30 scoring indices were identified through the screening process. Eleven of the 30 scoring indices have undergone some form of index validation. Intra-rater reliability was assessed for eight scoring indices. Inter-rater reliability was evaluated for all 11 of the scoring indices. Three of the indices underwent content validation. Two of the included scoring indices assessed criterion validity. Six of the included scoring indices explored content validity. Two of the included scoring indices were tested for responsiveness. AUTHORS' CONCLUSIONS: The Nancy Index and the Robarts Histopathology Index have undergone the most validation in that four operating properties including reliability, content validity, construct validity (hypothesis testing) and criterion validity have been tested. However, none of the currently available histologic scoring indices have been fully validated. In order to determine the optimal endpoint for histologic healing in UC, more research is required. The optimal index would need to be fully validated.

Systematic review of the current status of programs and general knowledge of diagnosis and management of retinoblastoma / Revisión sistemática del estado actual de los programas y conocimiento general de la diagnosis y manejo de retinoblastoma

Abstract: Background: This systematic review aims to report the current knowledge of retinoblastoma (Rb) and its implications in Mexico. We analyzed clinical and demographic data of patients with Rb at select hospitals with Rb programs or that treat and refer patients with Rb, and identified the gaps in practice. We propose solutions to improve diagnosis, provide adequate treatment, and improve patient uptake. Methods: A general review was conducted on PubMed of peer-reviewed literature on Rb in Mexico. Ophthalmology Department Heads or Directors of Rb programs at seven hospitals in Mexico were contacted for data available on their patients with Rb. Results: Five hospitals provided clinical data on 777 patients with Rb in a period spanning 2000-2015. Of the 122 patients with treatment, 83.4% underwent enucleation. From 33 to 45.3% of Rb tumors in Mexico reach an advanced intraocular stage of development. Knowledge of the disease is limited, despite the fact that the Mexican Retinoblastoma Group has elaborated Rb treatment guidelines and is developing a national Rb registry. Especially in the Southern states, prevalence and outcomes are comparable to African and Asian countries, and only few patients are referred to national treatment centers. Only three institutions have comprehensive Rb programs. Conclusions: There is an immediate need in Mexico to expand primary care providers' knowledge of Rb and to expand and upgrade current Rb programs to meet the needs of the population adequately. Diagnosis and care of Rb patients in Mexico can also be improved by the establishment of a national Rb registry and a national early detection program, and by increased use of the national treatment protocol.

Resumen: Introducción: Esta es una revisión sistemática de los conocimientos actuales del retinoblastoma (Rb) y sus implicaciones en los centros de referencia más importantes del país. Se presenta un análisis situacional de los programas de Rb en México, se identificaron las brechas en la práctica, y se proponen soluciones para mejorar el diagnóstico, tratamiento y referencia oportuna de pacientes. Métodos: Se realizó una revisión general de la literatura publicada sobre Rb en México a través de PubMed. Los datos sociodemográficos de pacientes con Rb fueron obtenidos a través de los directores de programas de retinoblastoma en siete hospitales. Resultados: Casi una tercera parte de los casos Rb se diagnostican en estadios avanzados. A pesar de la existencia del Grupo Mexicano de Retinoblastoma, el conocimiento de esta patología entre los médicos es limitado. Las diferencias en el tratamiento son notorias en el sur del país, donde la prevalencia y los resultados son comparables con África y Asia. Solamente tres instituciones a nivel nacional tienen un programa establecido de Rb. Conclusiones: Existe la necesidad inmediata de consolidar los programas de Rb para cubrir las necesidades reales de la población. Se requiere mejorar la educación del médico de primer contacto, establecer el registro nacional de casos y el programa de detección temprana, establecer los programas de salvamento ocular nacional, y reforzar las instituciones que brindan tratamiento.

Responsiveness of Endoscopic Indices of Disease Activity for Crohn's Disease.

OBJECTIVES: The Crohn's Disease Endoscopic Index of Severity (CDEIS) and the Simple Endoscopic Score for Crohn's Disease (SES-CD) are commonly used to assess Crohn's disease (CD) activity; however neither instrument is fully validated. We evaluated the responsiveness to change of the SES-CD and CDEIS using data from a trial of adalimumab, a drug therapy of known efficacy. METHODS: Paired video recordings (N=112) of colonoscopies (baseline and week 8-12) obtained from patients with CD who participated in a trial of adalimumab therapy were reviewed in random order, in duplicate, by four central readers (56 pairs of videos by 2 groups of readers). Responsiveness of the SES-CD and the CDEIS was evaluated by comparing correlations between the observed and pre-specified predictions of change scores for these endoscopic indices with a global endoscopic evaluation of severity (GELS), a patient reported outcome (PRO2), and the Crohn's disease activity index (CDAI), and by calculation of the standardized effect size, and Guyatt's Responsiveness statistic (GRS) using 2 definitions of change; (1) treatment assignment and (2) an absolute change in total PRO2 of 50. The potential application of effect size estimates was demonstrated by calculating hypothetical sample sizes for comparing two independent groups. The impact of removing stenosis as an index item and adjusting for the number of segments observed was also assessed. RESULTS: Changes in both endoscopic instruments and the GELS were highly correlated. The SES-CD displayed numerically higher effect sizes for both definitions of change. The standardized effect size and GRS estimates (95% confidence interval) for the SES-CD based on treatment assignment were 0.84 (0.53, 1.15) and 0.79 (0.48, 1.09). Corresponding values for the CDEIS were 0.72 (0.42, 1.02) and 0.75 (0.45, 1.06). The standardized effect size and GRS estimates for the SES-CD based on an absolute change in total PRO2 of 50 points or greater were 0.76 (0.49, 1.02) and 0.93 (0.64, 1.21). Corresponding values for CDEIS were 0.70 (0.44, 0.97), 0.83 (0.55, 1.10). Removal of stenosis as an index item and adjusting for observed segments did not improve responsiveness estimates. CONCLUSIONS: Although both the SES-CD and CDEIS are valid measures of endoscopic disease activity that are moderately responsive to changes in endoscopic disease activity, the SES-CD displayed numerically greater responsiveness in this data set.

Advances in Use of Endoscopy, Radiology, and Biomarkers to Monitor Inflammatory Bowel Diseases.

Crohn's disease and ulcerative colitis are heterogeneous inflammatory bowel diseases, and therapeutic requirements vary among patients. We have a limited capacity to predict disease progression for individual patients, therefore it is important that they are evaluated for the presence of active disease when symptoms are mild or even absent, when patients are more likely to respond to new treatment interventions. It then is important to monitor responses to treatment, to quickly identify those therapies that are ineffective, modify or change therapy, and avoid disease complications. Studies are underway to assess the effects of different monitoring strategies. Because of the heavy burden of severe inflammatory bowel disease on patients' health and quality of life, and the association between intestinal healing and disease progression in high-risk patients, a treat-to-target strategy (based on tissue healing) is likely to be optimal.