Predicting childhood chronic kidney disease severity in infants with posterior urethral valve: a critical analysis of creatinine values in the first year of life.

BACKGROUND: Posterior urethral valve (PUV) is a leading cause of chronic kidney failure in children. Studies have shown that a creatinine nadir above historical cutoff values of 0.8 or 1.0 mg/dL correlates with worse kidney outcomes. The ability to use nadir creatinine more discriminately as a test of kidney outcomes is otherwise limited. METHODS: We performed a retrospective review of 102 infants treated with primary valve ablation prior to 1 year of age. Patient factors including creatinine at presentation (Cr-P), nadir within 6 weeks after ablation (Cr-6 W), and nadir within 1 year after ablation (Cr-1Y) were assessed as predictors of final chronic kidney disease (CKD) severity. An optimal threshold for four CKD levels was defined in incremental fashion using binary outcome with receiver operating characteristic (ROC). Multivariable logistic regression models compared Cr-P, Cr-6 W, and Cr-1Y while adjusting for patient factors. RESULTS: Boys were ablated at mean age of 36.3 days and followed for 6.6 years (± 3.7). When compared to other demographics, only creatinine remained independently predictive of CKD outcomes on multivariable analysis. ROC analysis demonstrated excellent diagnostic accuracy for Cr-6 W and Cr-1Y (p < 0.001) and acceptable accuracy for Cr-P (p < 0.005). Using the Cr-6 W and Cr-1Y models, high sensitivity and specificity creatinine nadir cutoffs were determined to predict each CKD outcome. CONCLUSIONS: The severity of childhood CKD can be predicted with high accuracy using the creatinine nadir within 6 weeks of ablation. The cutoff values described can be incorporated into a clinical setting for patient counseling and individual risk stratification.

Role of a Preoperative Catheter Regimen in Achieving Early Primary Endoscopic Valve Ablation in Neonates with Posterior Urethral Valves.

PURPOSE: Primary valve ablation is preferred to vesicostomy in the initial management of posterior urethral valves. However, some neonates have a prohibitively small urethra. We describe our experience with a preoperative urethral catheter regimen to enhance the likelihood of neonatal valve ablation. MATERIALS AND METHODS: We performed a retrospective review of 126 neonates with posterior urethral valves treated between 2003 and 2019 with valve ablation prior to 10 weeks of age. The preoperative indwelling catheter either was gradually upsized to an 8Fr (progressive urethral dilation), was not upsized (nondilated) or was initially larger bore (8Fr only). The primary outcome was the ability to perform primary ablation by neonatal resectoscope. The secondary objective was to establish the parameters for considering progressive urethral dilation as well as its associated risks. RESULTS: Overall 97% could be ablated. The progressive urethral dilation group had the lowest mean weight (p <0.001). Only a larger catheter at the time of ablation was significantly associated with feasible ablation (p <0.001) and not urethral dilation, the infant's weight or his gestational age. Progressive urethral dilation was associated with a longer duration of catheterization as well as double the rate of febrile urinary tract infections (8.5%) over the nondilated group (3.6%). CONCLUSIONS: A much higher rate of primary ablation is feasible (97%) than previously reported (82%). More important than the infant's weight is whether a 6Fr to 8Fr catheter is in place at ablation. If an initial 6Fr to 8Fr catheter cannot be placed, urethral dilation to 8Fr should be performed before attempting ablation. This is both a technique and preoperative assessment that is useful for operative planning.

Early effect of American Academy of Pediatrics Urinary Tract Infection Guidelines on radiographic imaging and diagnosis of vesicoureteral reflux in the emergency room setting.

PURPOSE: In 2011 the AAP revised practice parameters on febrile urinary tract infection in infants and children 2 to 24 months old. New imaging recommendations invigorated the ongoing debate regarding the diagnosis and management of vesicoureteral reflux. We compared evaluations in these patients with febrile urinary tract infection before and after guideline publication. MATERIALS AND METHODS: During 2 separate 6-month periods 350 patients 2 to 24 months old were evaluated in the emergency room setting. Demographics, urine culture, renal-bladder ultrasound, voiding cystourethrogram and admission status were assessed. RESULTS: A total of 172 patients presented with initial febrile urinary tract infection in 2011, of whom 47 (27.3%) required hospitalization, while 42 of 178 (23.6%) were admitted in 2012. Admission by year did not significantly differ (p = 0.423). After guideline revision 41.2% fewer voiding cystourethrograms were done (72.1% of cases in 2011 vs 30.9% in 2012, p <0.001). A 17.2% decrease in renal-bladder ultrasound was noted (75.6% in 2011 vs 58.4% in 2012, p <0.001). Of 55 voiding cystourethrograms 21 (38.2%) were positive in 2012 compared to 36.3% in 2011 (p = 0.809). Mean ± SD maximum vesicoureteral reflux grade was unchanged in 2011 and 2012 (2.9 ± 1.2 and 2.5 ± 0.93, respectively, p = 0.109). There was no association between abnormal renal-bladder ultrasound and voiding cystourethrogram positivity (p = 0.116). CONCLUSIONS: AAP guidelines impacted the treatment of infants and young children with febrile urinary tract infection. We found no relationship between renal-bladder ultrasound and abnormal voiding cystourethrogram, consistent with previous findings that call ultrasound into question as the determinant for additional imaging. Whether forgoing routine voiding cystourethrogram results in increased morbidity is the subject of ongoing study.

Emergency Hospital Admissions for Initial Febrile Urinary Tract Infection: Do Patient Demographics Matter?

BACKGROUND: In 2011, the American Academy of Pediatrics revised practice parameters regarding febrile urinary tract infection (fUTI) in children aged 2-24 months. The Section on Urology opposed the omission of voiding cystourethrogram (VCUG), and expressed concern that potential untoward consequences of deferring VCUG may be most felt by children on Medicaid. OBJECTIVE: We ascertained imaging and characteristics of children presenting to the Emergency Department (ED) with initial fUTI to determine the impact of patient demographics on admissions for pyelonephritis. METHODS: Children aged 2-24 months presenting to the ED with initial fUTI were identified. Demographics, insurance status, laboratory studies, renal-bladder ultrasound (RBUS), VCUG, and hospital admission status were evaluated. RESULTS: Three-hundred fifty patients met inclusion criteria; 88 (25.1%) were admitted. Admitted patients were significantly (p < 0.001) younger (mean 0.31 ± 0.33 years) than those managed as outpatients (mean 0.91 ± 0.7 years). On univariate analysis, male gender (p < 0.001), Medicaid insurance (p < 0.05), and non-Hispanic race (p < 0.05) were associated with admission. Race retained significance on multivariate analysis; Caucasian children were 2.35 times (95% confidence interval [CI] 0.79-7.23) and African-American children 3.8 times more likely to be admitted than Hispanic patients (95% CI 1.88-7.63). Children with abnormal RBUS were 12.8 times more likely to require admission (95% CI 4.44-37.0). Medicaid was also independently predictive of admission; such patients were 2.6 times more likely to be admitted than those with private insurance (95% CI 1.15-5.88). CONCLUSIONS: Abnormal ultrasound, non-Hispanic race, and public insurance were strongly associated with hospital admission in children presenting to the ED with initial febrile urinary tract infection.

Infantile versus childhood posterior urethral valve diagnosis: management patterns and clinical outcomes at opposite ends of the spectrum.

BACKGROUND: Management patterns and outcomes are poorly defined in cases of late PUV diagnosis. OBJECTIVE: To compare post-ablation management and clinical outcomes of patients with infantile (<1 year) versus childhood (>5 year) PUV diagnosis to gain insight into the pathologies at opposite ends of the PUV spectrum. STUDY DESIGN: A multicenter retrospective cohort study was conducted using the TriNetX research network between 2006 and 2022. TriNetX synthesizes insurance claims and electronic medical record data for over 110 million patients from 92 healthcare organizations. We defined two cohorts: 1) The <1 year arm had an index diagnosis of PUV and cystoscopy with valve ablation within 1 year of life, 2) the >5 year arm had an index diagnosis of PUV and valve ablation after age 5. We report rates and time-to-first use of antispasmodics, alpha-blockers, CIC, bladder botox, enterocystoplasty or Mitrofanoff or secondary cutaneous vesicostomy, and CKD. RESULTS: We identified 569 patients (323 <1 year; 246 >5 year). Median age at diagnosis was 1 month (median follow-up 8 years) and 9 years (median follow-up 10 years) for the <1 year and >5 year cohorts, respectively. Following ablation, both arms were primarily managed with antispasmodics, with no difference between groups. The >5 year arm was significantly more likely to receive alpha-blockers or bladder botox. The <1 year arm was significantly more likely to be started on CIC, undergo enterocystoplasty, Mitrofanoff or secondary cutaneous vesicostomy, or renal transplantation. The <1 year arm had significantly higher rates and shorter time-to-progression to all stages of CKD. DISCUSSION: Despite higher utilization of conservative strategies among patients with a late PUV diagnosis, these patients had superior renal outcomes and low rates of progression to invasive treatments. Limitations include potential inaccuracies in medical coding as well as variations in thresholds to initiate CIC, perform surgical reconstruction, or proceed with renal transplantation at participating centers. CONCLUSIONS: These findings provide evidence that a late PUV diagnosis reflects an overall milder disease process.

Giant Congenital Melanocytic Nevus of the Male External Genitalia: A Pediatric Case Report of Diagnosis, Evaluation and Management.

Congenital melanocytic nevi are present at birth or develop within the first few months of life. Giant congenital melanocytic nevi are a rare variant and may involve the external genitalia with a confluent "bathing trunk" distribution. Rapid growth of proliferative nodules of melanocytic cells may cause disfigurement and anatomical distortion resulting in psychological distress and loss of functionality. We report the case of a neglected 17-year-old nonverbal male who received a resection of a Giant Congenital Melanocytic Nevi (GMN) engulfing the penis and scrotum with final resected dimensions of 36.0×20.0×8.0 cm.

Use of Perirectal Hyaluronic Acid Spacer Prior to Radiotherapy in a Pediatric Patient With Bladder Rhabdomyosarcoma: A Case Report.

Rhabdomyosarcoma (RMS) treatment involves surgery, chemotherapy, and radiotherapy. A radioprotective space between the bladder/prostate and rectum reduces postradiation complications, as reported in adult patients. Describe pediatric preradiotherapy perirectal hyaluronic acid (HA) spacer injection for bladder/prostate RMS. We present a case of a 17-month-old male with bladder/prostate RMS. Before radiotherapy, an HA spacer was injected peri-rectally. Under general anesthesia, a transrectal ultrasound was positioned and 1mL of HA spacer was injected into the perirectal space. No complications were reported at 6-month follow-up. This is the first report of pre-radiation therapy spacer injection for pediatric bladder/prostate RMS.

An African ancestry-specific allele of CTLA4 confers protection against rheumatoid arthritis in African Americans.

Cytotoxic T-lymphocyte associated protein 4 (CTLA4) is a negative regulator of T-cell proliferation. Polymorphisms in CTLA4 have been inconsistently associated with susceptibility to rheumatoid arthritis (RA) in populations of European ancestry but have not been examined in African Americans. The prevalence of RA in most populations of European and Asian ancestry is approximately 1.0%; RA is purportedly less common in black Africans, with little known about its prevalence in African Americans. We sought to determine if CTLA4 polymorphisms are associated with RA in African Americans. We performed a 2-stage analysis of 12 haplotype tagging single nucleotide polymorphisms (SNPs) across CTLA4 in a total of 505 African American RA patients and 712 African American controls using Illumina and TaqMan platforms. The minor allele (G) of the rs231778 SNP was 0.054 in RA patients, compared to 0.209 in controls (4.462 x 10(-26), Fisher's exact). The presence of the G allele was associated with a substantially reduced odds ratio (OR) of having RA (AG+GG genotypes vs. AA genotype, OR 0.19, 95% CI: 0.13-0.26, p = 2.4 x 10(-28), Fisher's exact), suggesting a protective effect. This SNP is polymorphic in the African population (minor allele frequency [MAF] 0.09 in the Yoruba population), but is very rare in other groups (MAF = 0.002 in 530 Caucasians genotyped for this study). Markers associated with RA in populations of European ancestry (rs3087243 [+60C/T] and rs231775 [+49A/G]) were not replicated in African Americans. We found no confounding of association for rs231778 after stratifying for the HLA-DRB1 shared epitope, presence of anti-cyclic citrullinated peptide antibody, or degree of admixture from the European population. An African ancestry-specific genetic variant of CTLA4 appears to be associated with protection from RA in African Americans. This finding may explain, in part, the relatively low prevalence of RA in black African populations.

Posterior urethral morphology on initial voiding cystourethrogram correlates to early renal outcomes in infants with posterior urethral valves.

INTRODUCTION: Posterior urethral valves (PUV) is a leading cause of chronic renal failure in childhood. Bladder and posterior urethral deformity in infants with PUV are highly variable on initial voiding cystourethrogram (VCUG). Some types of deformity may be more important than others in determining the severity of the condition. Identification of specific VCUG features may allow for a simple, early screening assessment for patients. OBJECTIVE: Determine whether morphologic features on the initial VCUG correlate to renal outcomes as measured by the creatinine nadir in the first year after ablation. METHODS: Children with PUV treated by primary valve ablation before 12 months old and followed >1 year were identified. Initial diagnostic VCUG was evaluated for the bladder height to width ratio (HW-B), posterior urethral height to width ratio (HW-PU), and posterior-anterior urethral ratio (PA-UR). A trabeculation grade was assigned by three pediatric urologists, and reflux was noted. Univariable analyses with chi-square and t-test were used to compare bladder and posterior urethral morphology factors among those who obtained a creatinine nadir <0.8 or ≥0.8 mg/dL in the first year after ablation. Linear regression was performed to correlate morphology values with true creatinine nadir. RESULTS: A total of 120 boys were ablated at mean age of 40.5 days (range 0-342) and followed for 5.9 years (± 3.85). Among these, 21 (17.5%) had a creatinine nadir ≥0.8 mg/dL. Mean overall HW-B and mean PA-UR were not significantly different between those with creatinine nadir <0.8 versus ≥0.8 mg/dL. Bladder trabeculation grade was not associated with creatinine nadir. For the entire cohort, only the difference in HW-PU was statistically significant between creatinine nadir groups with a much higher ratio among those with a nadir ≥0.8 (p < 0.001). Linear regression demonstrated a significant positive correlation between the HW-PU and creatinine nadir (R 2 = 0.097, p = 0.002). The presence of bilateral reflux is significantly associated with creatinine nadir ≥0.8 mg/dL (p = 0.001). DISCUSSION: We investigate for the first time the association of morphology features on the initial VCUG with renal outcome in PUV patients. Posterior urethral deformity as quantified by a higher HW-PU ratio is significantly correlated with a higher creatinine nadir, whereas measured bladder morphology metrics are not. HW-PU may be an indirect measure of the severity of obstruction in posterior urethral valves. CONCLUSION: The HW-PU appears to be a meaningful early morphologic metric for renal outcome.

Role of progressive urethral dilation and primary valve ablation in the long-term renal outcomes of small, preterm infants with posterior urethral valve.

INTRODUCTION: PUV patients managed with primary vesicostomy instead of primary valve ablation (PVA) historically are preterm, low-birth-weight (LBW) infants with inadequate urethral size. We previously described progressive urethral dilation (PUD) as an effective method of enhancing the likelihood of PVA in these infants, allowing equal access to PVA as an initial management method. OBJECTIVE: We aim to characterize renal outcomes in patients managed with PUD + PVA and compare this to outcomes with PVA alone. We also re-examine the effect of LBW and gestational age on renal outcomes in PUV with a cohort treated uniformly by PVA. METHODS: We performed retrospective review of 78 neonates with PUV treated with PVA prior to 10 weeks of age with >1 year of follow up. Before valve ablation, boys either underwent PUD (serial upsizing of a smaller bore urethral catheter to an 8Fr catheter; PUD + PVA) or non-dilation (smaller bore catheter was maintained; PVA-only). PUD + PVA versus PVA-only was compared using chi-square and t-test. Logistic regression was performed to assess the effect of PUD, preterm (<37 weeks), LBW (<2.5 kg), and other predictors on the final outcomes of CKD3+ and ESRD. RESULTS: 31 of 78 patients underwent PUD + PVA. Mean follow up was 5.2 years (SD 3.4), with no significant difference between PUD + PVA and PVA-only. The PUD + PVA group included significantly lower gestational age infants with lower birth weight and ablation weight. There was no significant effect of PUD on final CKD3+ or ESRD outcome on univariable or multivariable analysis. When adjusted for other variables, only Cr nadir >0.5 remained an independent predictor of CKD3+ (OR 41.2; p < 0.001) and ESRD (OR 18.9; p = 0.015). DISCUSSION: We previously demonstrated that PUD is an effective means to achieve PVA in small neonates who might otherwise require vesicostomy. The data herein demonstrates no significant effect of PUD on renal outcomes. In this unique cohort of newborns treated with PVA, only creatinine nadir and not gestational age or an independent predictor of outcomes. CONCLUSION: In small preterm infants who would have been excluded from PVA due to limited urethral size, PUD + PVA confers the same renal outcomes as PVA alone in larger infants. This novel data will assist in the risk-benefit analysis of using PUD before PVA in newborns diagnosed with PUV. When primary intervention is uniformly PVA, preterm birth and LBW are not independent predictors of renal outcomes.

Generalized bone loss as a predictor of three-year radiographic damage in African American patients with recent-onset rheumatoid arthritis.

OBJECTIVE: To examine the association between baseline bone mineral density (BMD) and radiographic damage at 3 years of disease duration in a longitudinal cohort of African Americans with recent-onset rheumatoid arthritis (RA). METHODS: African American RA patients with a disease duration of <2 years (n = 141) were included in the study. All patients underwent baseline BMD measurements (femoral neck and/or lumbar spine) using dual x-ray absorptiometry. T scores were calculated using normative data from the general population of African Americans. Patients were categorized as having osteopenia/osteoporosis (T score less than or equal to -1) or as being healthy. Hand and wrist radiographs, obtained at baseline and at 3 years of disease duration, were scored using the modified Sharp/van der Heijde method. The association between baseline BMD and total radiographic score at 3 years of disease was examined using multivariable negative binomial regression. RESULTS: At baseline, the mean age and the mean disease duration were 52.4 years and 14.8 months, respectively; 85.1% of the patients were women. The average total radiographic scores at baseline and at 3 years of disease were 2.4 and 5.7, respectively. In the final reduced multivariable model, adjusting for age, sex, anti-cyclic citrullinated peptide antibody positivity, and the presence of radiographic damage at baseline, the total radiographic score at 3 years disease in patients with osteopenia/osteoporosis of the femoral neck was twice that in patients with normal bone density, and the difference was statistically significant (P = 0.0084). No association between lumbar spine osteopenia/osteoporosis and radiographic score was found. CONCLUSION: Our findings suggest that reduced generalized BMD may be a predictor of future radiographic damage and support the hypothesis that radiographic damage and reduced generalized BMD in RA patients may share a common pathogenic mechanism.

Most common single-nucleotide polymorphisms associated with rheumatoid arthritis in persons of European ancestry confer risk of rheumatoid arthritis in African Americans.

OBJECTIVE: Large-scale genetic association studies have identified >20 rheumatoid arthritis (RA) risk alleles among individuals of European ancestry. The influence of these risk alleles has not been comprehensively studied in African Americans. We therefore sought to examine whether these validated RA risk alleles are associated with RA risk in an African American population. METHODS: Twenty-seven candidate single-nucleotide polymorphisms (SNPs) were genotyped in 556 autoantibody-positive African Americans with RA and 791 healthy African American control subjects. Odds ratios (ORs) and 95% confidence intervals (95% CIs) for each SNP were compared with previously published ORs for RA patients of European ancestry. We then calculated a composite genetic risk score (GRS) for each individual based on the sum of all risk alleles. RESULTS: Overlap of the ORs and 95% CIs between the European and African American populations was observed for 24 of the 27 candidate SNPs. Conversely, 3 of the 27 SNPs (CCR6 rs3093023, TAGAP rs394581, and TNFAIP3 rs6920220) demonstrated ORs in the opposite direction from those reported for RA patients of European ancestry. The GRS analysis indicated a small but highly significant probability that African American patients relative to control subjects were enriched for the risk alleles validated in European RA patients (P = 0.00005). CONCLUSION: The majority of RA risk alleles previously validated for RA patients of European ancestry showed similar ORs in our population of African Americans with RA. Furthermore, the aggregate GRS supports the hypothesis that these SNPs are risk alleles for RA in the African American population. Future large-scale genetic studies are needed to validate these risk alleles and identify novel RA risk alleles in African Americans.

A functional RANKL polymorphism associated with younger age at onset of rheumatoid arthritis.

OBJECTIVE: We previously observed the association of the co-occurrence of the HLA-DRB1 shared epitope (SE) and RANKL single-nucleotide polymorphisms (SNPs) with younger age at the onset of rheumatoid arthritis (RA) in 182 rheumatoid factor (RF)-positive European American patients with early-onset RA. The aim of this study was to fine-map the 48-kb RANKL region in the extended cohort of 210 European American RF-positive patients with early RA, to seek replication of RA-associated SNPs in additional RA cohorts of 501 European Americans and 298 African Americans, and to explore the functional consequences of RA-associated SNPs. METHODS: SNP genotyping was conducted using pyrosequencing or TaqMan polymerase chain reaction (PCR) assays. Associations of rs7984870 with RANKL expression in plasma, peripheral blood mononuclear cells, and isolated T cells were quantified using enzyme-linked immunosorbent assay and reverse transcription-PCR. Site-directed mutagenesis of rs7984870 within the 2-kb RANKL promoter was performed to drive the luciferase reporter gene in osteoblast and stromal cell lines. Interaction of DNA and protein was determined by electrophoretic mobility shift assay. RESULTS: A single promoter SNP, rs7984870, was consistently significantly associated with earlier age at the onset of RA in 3 independent seropositive (RF or anti-cyclic citrullinated peptide antibody) RA cohorts but not in seronegative RA patients. The C risk allele of rs7984870 conferred 2-fold higher plasma RANKL levels in RF-positive patients with RA, significantly elevated RANKL messenger RNA expression in activated normal T cells, and increased promoter activity after stimulation in vitro via differential binding to the transcription factor SOX5. CONCLUSION: The RANKL promoter allele that increased transcription levels upon stimulation might promote interaction between activated T cells and dendritic cells, predisposing to a younger age at the onset of RA in seropositive European American and African American patients.

Inflammatory Myofibroblastic Tumor Presenting as Gross Hematuria in a Pediatric Patient With VACTERL Syndrome Following Bladder Augmentation.

Inflammatory myofibroblastic tumors (IMT) are rare and poorly understood inflammatory neoplasms. Most commonly occurring in the liver and gastrointestinal tract, cases of bladder involvement have been rarely reported. Bladder IMT generally presents with gross hematuria and can be differentiated from other bladder tumors by expression of anaplastic lymphoma kinase. We report the occurrence of an Bladder IMT detected following lower urinary tract reconstruction with bladder augmentation.

A Review of Mullerian Anomalies and Their Urologic Associations.

Structural anomalies of the female reproductive tract, known as Mullerian anomalies, can occur in isolation or in association with anomalies of other organ systems. Due to shared embryology, the most common association in up to 40% of patients is with renal, ureteral, and bladder anomalies. Affected girls can have a wide range of genitourinary symptoms with urologists playing an integral role in their diagnosis and treatment. To facilitate the recognition and management of these conditions, we provide a review of Mullerian anomalies including the embryology, classifications, syndromes, evaluation, and treatments with attention to their urologic applicability.

Preliminary experience with epsilon aminocaproic acid for treatment of intractable upper tract hematuria in children with hematological disorders.

PURPOSE: Gross, intractable hematuria is rare in children. Although the role of epsilon aminocaproic acid in the management of refractory hematuria is well established in the adult population, few data exist about its use in children for this indication. We present our initial experience with epsilon aminocaproic acid for the treatment of intractable hematuria after more conservative measures failed, and propose an algorithm for administration of epsilon aminocaproic acid in children. MATERIALS AND METHODS: We reviewed the charts of all patients treated with epsilon aminocaproic acid for intractable gross hematuria at our institution during a period of 36 months. All patients underwent hematological evaluation and any underlying bleeding dyscrasias were addressed. All patients also underwent renal and bladder ultrasound, retrograde pyelogram and ureteroscopy. Demographic information, medical and surgical histories, and epsilon aminocaproic acid dosing and outcomes were recorded. RESULTS: Three boys and 1 girl 11 to 17 years old were treated with epsilon aminocaproic acid. Three patients had sickle trait (1 with nutcracker phenomenon) and 1 had hemophilia A. Three patients required packed red blood cell transfusions to maintain hematocrit. Three renal angiograms were performed, all of which were nondiagnostic. Duration of hematuria ranged from 1 to 52 weeks before administration of epsilon aminocaproic acid. Endoscopic evaluation demonstrated hematuria localized to 1 ureteral orifice in all 4 patients. All patients received 100 mg/kg epsilon aminocaproic acid orally every 6 hours, which uniformly led to cessation of hematuria. CONCLUSIONS: Epsilon aminocaproic acid is useful for the management of gross refractory hematuria when more conservative measures fail. Because of its potential side effects, it should be used cautiously.

Use of rectus abdominis muscle flap as adjunct to bladder neck closure in patients with neurogenic incontinence: preliminary experience.

PURPOSE: Vesicoureteral fistula is a well-known potential complication following bladder neck closure for neurogenic incontinence. Various maneuvers, including omental interposition, have been described to prevent this problem. Unfortunately omentum is not always available or feasible for use. We describe the surgical anatomy and use of a rectus abdominis muscle flap as an adjunctive maneuver during bladder neck closure to correct or prevent development of bladder neck fistula. MATERIALS AND METHODS: We performed a retrospective chart review of all patients at our institution undergoing rectus abdominis muscle flap by a single surgeon (EAS). Patient demographics, indications for surgery, intraoperative and postoperative complications, and long-term efficacy were assessed. Cadaveric dissection was also performed to gain a greater understanding of the surgical anatomy relevant to this procedure. RESULTS: In 6 patients with neurogenic bladder dysfunction a rectus abdominis muscle flap was interposed between the bladder neck and urethral stump at bladder neck closure. There were no intraoperative or postoperative complications associated with this procedure. At a mean followup of 45.5 months (range 18 to 120) all 6 patients were continent of urine. There have been no urinary fistulas related to use of the rectus abdominis muscle flap. Cadaveric dissections confirmed the inferior epigastric artery to be the dominant and readily mobile blood supply of the rectus abdominis muscle flap. CONCLUSIONS: The rectus abdominis muscle flap is easily harvested without significant risk of morbidity and offers a well vascularized tissue for coverage of a bladder neck closure when an omental flap is not available.

Fate of Pediatric Renal Angiomyolipoma During mTOR Inhibitor Treatment in Tuberous Sclerosis Complex.

OBJECTIVE: To evaluate the clinical and radiographic follow-up of renal angiomyolipoma (AML) in pediatric patients with tuberous sclerosis complex (TSC) on mTOR inhibitors. METHODS: We performed retrospective chart review of children who were diagnosed with TSC between 2000 and 2019 and prescribed everolimus at age ≤18 years. Treatment assessment was performed in patients who were medically-compliant by serum drug trough levels and who had at least a baseline and one subsequent renal imaging study. RESULTS: Nineteen patients were analyzed. Average age of everolimus initiation was 9 years, and indication was neurologic in 17 (90%). Fourteen patients (73.6%) had AML with average size of 1.9 (0.4-5) cm. Medication was discontinued due to side effects in 3 (16%) patients. Treatment assessment was analyzed for 15 patients with median medication exposure 5.1 (0.8-8.5) years. Among 13 with AML, the dominant lesion decreased in size in 9 (69%) and stayed stable in 4 (31%). Greatest absolute size decrease was seen for lesions ≥2 cm. No new AML lesions formed during treatment. CONCLUSION: Although not currently approved for this indication, everolimus appears to be well-tolerated with similar efficacy for pediatric AML as in adult AML. Use may be most warranted in children with AML ≥2 cm.