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The American Association of Pediatric Urologists (AAPU) 2025 annual meeting- Beaver Creek, Colorado
No full textA scan of pleiotropic immune mediated disease genes identifies novel determinants of baseline FVIII inhibitor status in hemophilia A
No full textHemophilia-A (HA) is the X-linked bleeding disorder caused by heterogeneous factor (F)VIII gene (F8)-mutations and deficiencies in plasma-FVIII-activity that prevent intrinsic-pathway mediated coagulation-amplification. Severe-HA patients (HAPs) require life-long infusions of therapeutic-FVIII-proteins (tFVIIIs) but ~30% develop neutralizing-tFVIII-antibodies called “FVIII-inhibitors (FEIs)”. We investigated the genetics underlying the variable risk of FEI-development in 450 North American HAPs (206 and 244 respectively self-reporting black-African- or white-European-ancestry) by analyzing the genotypes of single-nucleotide-variations (SNVs) in candidate immune-mediated-disease (IMD)-genes using a binary linear-mixed model of genetic association with baseline-FEI-status, the dependent variable, while simultaneously accounting for their genetic relationships and heterogeneous-F8-mutations to prevent the statistical problem of non-independence. We a priori selected gene-centric-association-scans of pleiotropic-IMD-genes implicated in the development of either ≥2 autoimmune-/autoinflammatory-disorders (AADs) or FEIs and ≥1 AAD. We found that baseline-FEI-status was significantly associated with NOS2A (rs117382854; p = 3.2 × 10−6) and B3GNT2 (rs10176009; p = 5.1 × 10−6)—pleiotropic-IMD-genes known previously to function in anti-microbial-/-tumoral-immunity but not in the development of FEIs—and confirmed associations with CTLA4 (rs231780; p = 2.2 × 10−5). We also found that baseline-FEI-status has a substantial heritability (~55%) that involves (i) a F8-mutation-specific component of ~8%, (ii) an additive-genetic contribution from SNVs in IMD-genes of ~47%, and (iii) race, which is a significant determinant independent of F8-mutation-types and non-F8-genetics
Emergency Medical Services Utilization of Medication for Opioid Use Disorder: A Narrative Review of the Literature and Analysis of Prehospital Buprenorphine Protocols
No full textObjectives: This narrative review examines the current literature and prehospital buprenorphine protocols, discusses the available outcomes data of existing protocols, identifies protocol variations, and highlights challenges and opportunities associated with prehospital medication for opioid use disorder (MOUD) initiation. Methods: PubMed was searched for articles from 1/1/2010 to 2/1/2025, using keywords “Prehospital Buprenorphine,” “Emergency Medical Services Buprenorphine,” “EMS Medication for Opioid Use Disorder,” and “Prehospital MOUD.” All EMS protocols from the website http://www.emsprotocols.org (Acid Remap, LLC) were reviewed, supplemented with internet searches. A data dictionary was developed to categorize protocol characteristics; categories of responses included minimum Clinical Opiate Withdrawal Scale (COWS) scores, initial and maximum buprenorphine dosages, adjunct medications for withdrawal symptoms, online medical control (OMC) consultation requirements, minimum age, contraindications and exclusion criteria, and post-induction follow-up. Results: Six statewide prehospital MOUD protocols were identified, along with regional protocols in 21 states and Washington, DC. Details were available for 42 state or regional protocols. Initial dosing across all protocols ranged between 4 and 24 mg. Maximum dosing ranged between 8 and 48 mg. The median initial and total dosage was 16 mg and 24 mg, respectively. Adjunctive medication for withdrawal was available in 61.9% of protocols. Treatment with MOUD was limited to mobile integrated health/community paramedicine programs in 19% of protocols. Otherwise, administration was permitted via standing order in 52.4% and required physician authorization in 26.2% of protocols. The minimum COWS scores ranged between 5 and 8, with 71.5% of protocols requiring a COWS score \u3e7. Most (59.5%) protocols specified 18 years as the minimum age, while 23.8% specified 16 years. Buprenorphine was contraindicated for pregnant patients in 38% of protocols, while 9.6% of protocols required OMC consultation. Ten peer-reviewed studies evaluating EMS MOUD protocols outcomes were identified, most of which reported successful initiation, linkage to care, and retention for significant proportions of patients evaluated for opioid overdose and/or opioid withdrawal symptoms. Conclusions: Prehospital MOUD protocol design varies significantly, and best practices remain unknown, particularly regarding protocol inclusion criteria, dosing regimens, adjunctive medications, and resources to ensure continuity. The available data suggest protocols with more permissive inclusion criteria and comprehensive service provision are associated with higher rates of linkage to care
Safety and preliminary efficacy of ivaltinostat, an HDAC inhibitor, plus capecitabine in patients with pretreated metastatic pancreatic adenocarcinoma (mPDAC): Results of a phase Ib study
No full text2025 ASCO Gastrointestinal Cancers Symposium, January 23 - 25, 2025, San Francisco, C
Markers of growth and nutrition in children with acquired chylothorax post CHD surgery
Get PDFBackground: Acquired chylothorax is an established complication of CHD surgery, affecting 2-9% of patients. CHD places a child at risk for failure to thrive, with subsequent chylothorax imposing additional risk. Objective: We conducted a retrospective chart review to ascertain quantitative markers of nutrition and growth in children affected by chylothorax following CHD surgery between 2018 and 2022 compared to controls. Methods: We utilised electronic medical record system, EPIC, at Children\u27s Hospital, New Orleans, targeting subjects \u3c 18 years old who underwent CHD surgery between 2018 and 2022 and developed a subsequent chylothorax. Study subjects were identified using the 10th revision of the International Classification of Diseases codes (ICD-10 codes: J94.0, I89.8, and J90.0). Each chylothorax case (n = 20) was matched by procedure type and age to a control with no chylothorax (n = 20). Data were recorded in REDCap and analysed using SPSS. Results: After removal of outliers, we analysed 19 total matched pairs. There was no statistical difference in growth velocity (p = 0.12), weight change (operation to discharge) (p = 0.95), weight change (admission to discharge) (p = 0.35), Z-score change (operation to discharge) (p = 0.90), Z-score change (admission to discharge) (p = 0.21), serum protein (p = 0.88), or serum albumin (p = 0.82). Among cases, linear regression demonstrated no significant association between maximum chylous output and growth velocity (p = 0.91), weight change (operation to discharge) (p = 0.15), or weight change (admission to discharge) (p = 0.98). Conclusions: We did not observe statistically significant markers of growth or nutrition in children with chylothorax post-CHD surgery compared to those without chylothorax. Multisite data collection and analysis is required to better ascertain clinical impact and guide clinical practice
Analyzing data with chatgpt\u27s data analyst tool: Perks, pitfalls, and a demonstration
No full textInternational Society of Psychoneuroendocrinology, September 3 - 5, 2025, New Orleans, L
