Citations

Summary: While the current approach to precursor hematologic conditions is to “watch and wait,” this may change with the development of therapies that are safe and extend survival or delay the onset of symptomatic disease. The goal of future therapies in precursor hematologic conditions is to improve survival and prevent or delay the development of symptomatic disease while maximizing safety. Clinical trial considerations in this field include identifying an appropriate at-risk population, safety assessments, dose selection, primary and secondary trial endpoints including surrogate endpoints, control arms, and quality-of-life metrics, all of which may enable more precise benefit–risk assessment.

BACKGROUND: Radiographic imaging of abdomen and pelvis plays an important role in the diagnosis and management of ileal pouch disorders with modalities including computed tomography, magnetic resonance imaging, contrasted pouchography, and defecography. OBJECTIVES: A systematic review of the literature was performed and applications of cross-sectional imaging, pouchography, defecography, and ultrasonography are described. DATA SOURCES: PubMed, Google Scholar, and Cochrane database. STUDY SELECTION: Relevant articles on endoscopy in ileal pouches published between Jan 2003 and June 2023 in English were included based on PRISMA guidelines. INTERVENTION: Main abdominal and pelvic imaging modalities and their applications in the diagnosis of ileal pouch disorders were included. MAIN OUTCOME MEASURES: Accuracy in characterization of the ileal pouch disorders. RESULTS: Computed tomography is the test of the choice for the evaluation of acute anastomotic leaks, perforation, and abscess(es). Magnetic resonance imaging of the pelvis is suitable for the evaluation of chronic anastomotic leaks and their associated fistulas and sinus tracts, as well as for the penetrating phenotype of Crohn’s disease of the pouch. Computed tomography enterography and magnetic resonance enterography are useful in the assessment of intraluminal, intramural, and extraluminal disease processes of the pouch and prepouch ileum. Water-soluble contrast pouchography is particularly useful for the evaluation of acute or chronic anastomotic leaks and outlines the shape and configuration of the pouch. Defecography is the key modality to evaluate structural and functional pouch inlet and outlet obstructions. Ultrasonography can be performed to assess the pouch in experienced IBD centers. LIMITATIONS: This is a qualitative, not quantitative review of mainly case series and case reports. CONCLUSIONS: Abdominopelvic imaging along with clinical and endoscopic evaluation is imperative for the accurate assessment of structural, inflammatory, functional, and neoplastic disorders. See video from symposium.

A woman in her 40s presented to the emergency department (ED) with a 3-week history of nausea, vomiting and diarrhoea. Blood cultures were positive for Serratia and Pantoea agglomerans spp. One month before her ED visit, she underwent targeted uterine artery embolisation with particles by an interventional radiologist. Uterine artery embolisation is considered a safe alternative to surgical removal of fibroids or hysterectomy. The patient was initially treated with targeted antibiotics for a large infected uterine fibroid but ultimately required a hysterectomy for source control. To our knowledge, this is the first documented case of P. agglomerans infecting a uterine fibroid.

Abstract Introduction High medication costs for vaginal estrogen may create a barrier for patients seeking treatment for numerous urinary and vaginal symptoms and lead to poor medication adherence. The Mark Cuban Cost Plus Drug Company (CPD) and GoodRx tools offer patients a method to purchase prescription medications at a discount, which may lead to improved medication adherence and decreased national expenditures on such medications. Objective To compare the pricing of vaginal estrogen as advertised by retail pharmacies, Medicare, and discount listings through CPD and GoodRx. Methods A review of publicly available data from CPD and GoodRx was performed to obtain listed retail and discounted prices for Estrace (topical), Vagifem (tablet), and Premarin (topical). GoodRx prices were obtained using prices from Costco, CVS, Walgreens, and Walmart pharmacies in the most from populous zip code from each of the 3 most populous U.S. states: California, New York, and Texas. Prices were adjusted to account for dosage and prescribed quantity to estimate an average cost per dosage unit ($/adu). The Medicare Part D Drug Spending and Utilization data from 2021 were used to report the average spending per dosage unit and total expenditure for each of the listed medications. These data were compared to create an estimate of average national savings after use of CPD and/or GoodRx. Results Average spending per dosage unit for Estrace, Vagifem, and Premarin was lesser when discount tools were used (0.50 $/adu, 3.75 $/adu, and 4.30 $/adu respectively), compared to Medicare pricing (6.14 $/adu, 20.95 $/adu, and 10.10 $/adu respectively) or retail pricing (3.14 $/adu, 10.19 $/adu, and 5.43 $/adu respectively). When adjusted for total dosage units, use of CPD over Medicare pricing for the three medications would result in a combined annual national saving of $64.2M. Use of either CPD or GoodRx compared to retail pricing would similarly result in a combined annual national saving of $39.2M. Conclusions The utilization of CPD and GoodRx in the prescription of vaginal estrogen led to a substantial national average cost savings when compared to retail and Medicare pricing. CPD and GoodRx may offer a way to decrease the cost burden for patients seeking treatment for urinary and vaginal symptoms and significantly reduce national expenditures on medications. Disclosure Any of the authors act as a consultant, employee or shareholder of an industry for: The utilization of CPD and GoodRx in the prescription of vaginal estrogen led to a substantial national average cost savings when compared to retail and Medicare pricing. CPD and GoodRx may offer a way to decrease the cost burden for patients seeking treatment for urinary and vaginal symptoms and significantly reduce national expenditures on medications.

Abstract Introduction Krabbe Disease (KD) or globoid cell leukodystrophy is a rare autosomal recessive lysosomal storage disorder affecting approximately 1 in 100,000 people worldwide. GALC gene mutations associated with KD involve a galactosylceramidase deficiency, which damages the oligodendrocytes and Schwann cells. Therefore, nervous system demyelination is a hallmark of KD. While only a few infants survive beyond two years in early-onset Krabbe disease, patients with late-onset Krabbe Disease exhibit a milder phenotype and longer life expectancy. Late-onset KD symptoms include progressive muscle weakness and stiffness, developmental regression, behavioral changes, dementia, hearing loss, vision problems, and seizures. Only a few cases of sexual dysfunction associated with KD have been reported in literature but they highlight the wide-ranging impact of this condition on young adults and adults. Objective This review aims to examine sexual dysfunction in KD, as reported in literature. We employ a broad definition of sexual dysfunction (erectile dysfunction, dyspareunia, orgasmic disorder, muscle weakness, etc.) and its incidence in both sexes with KD. A comprehensive review of this topic has not yet been conducted, and we offer here preliminary information for healthcare providers when addressing sexual concerns in patients with lysosomal storage disorders. Methods A literature search was conducted by two separate investigators who independently screened studies and abstracted data. Data sources included peer reviewed publications from the following databases: PubMed, Scopus, Web of Science, EMBASE, Cochrane, Google Scholar and Clinical trials.gov. There were no restrictions on language nor date of publication. The search algorithm utilized to generate the review list was: (Globoid cell leukodystrophy OR Galactosylcerebrosidase deficiency OR Galactosylceramidase deficiency OR Krabbe disease OR Late-onset Krabbe disease OR GALC mutation). Results were further filtered by article type, restricting articles to Case Reports, Clinical Trials, Reviews, and Systematic Reviews. Results were then screened for reports containing data on sexual dysfunction in patients with KD. Results A total of four case reports and one review paper contained reports of sexual dysfunction in KD patients. All cases involved late-onset Krabbe disease, characterized by a milder and more insidious presentation. Genitourinary symptoms reported included urinary incontinence (N=3) and erectile dysfunction (N=2), however, there were no reports (N=0) of symptoms related to female sexual dysfunction. Other symptoms commonly reported in late-onset KD that might be linked to sexual function were ataxia, spasticity and weakness of lower limbs (N=3), and sensory dysfunction due to peripheral neuropathy (N=4). Conclusions This pioneering review reveals that patients with KD experience symptoms of sexual dysfunction. While the rarity of KD makes it challenging to study it clinically, sexual dysfunction may be consistent with the known pathophysiology of late-onset KD. This review also highlights that sexual dysfunction in female patients with KD is an under-investigated component of the disease presentation. Future clinical research into this important comorbidity, especially in female patients where the literature is lacking, may improve the care of patients with KD. Disclosure No.

Introduction Burnout in medicine is an occupational hazard and has emerged as a pressing concern in recent years. Organizational changes can be impactful in countering burnout (1). The factors leading to burnout in medicine are multifaceted, encompassing organizational factors such as workload, inadequate support, and inefficient administrative systems. The purpose of this study is to investigate current organizational measures to support physician well‐being. This study was an initiative led by the SVIN wellness committee. Methods A 39‐question online survey investigating current organizational well‐being practices was distributed to physicians both nationally and internationally practicing stroke, neurocritical care, and interventional neurology. Data analysis was performed using Python, utilizing the libraries “pandas” and “sklearn”. Results This study analyzed burnout among 109 healthcare professionals, predominantly from the U.S. (93.6%) and Canada (6.4%). The majority were neurointerventional specialists (53.2%), aged 35‐44 years (52.3%), and male (62.4%). Burnout frequency was measured on a 0 (Never) to 4 (Every day) scale. Using a Random Forest model, the study identified key burnout predictors from questions, which covered organizational leadership, wellness resources, compensation, and workload. The most influential predictors were: adequacy of compensation relative to specialty, workload, and stress (17.7% importance); leadership accountability for workforce wellbeing (9.1%); and establishment of wellness or burnout as a critical metric (7.5%). Other factors included provision of wellness screenings (7.1%), and dedication of resources toward professional well‐being (4.9%). Conclusion The study identifies compensation, leadership accountability, and wellness resources as key predictors of burnout among healthcare professionals. These findings highlight the need for targeted organizational strategies to enhance physician well‐being and mitigate burnout. Further research is warranted to validate and expand upon these findings.