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The Spectrum of Influenza-Associated Acute Necrotizing Encephalopathy of Childhood: A Case Series.
No full textBACKGROUND: Acute necrotizing encephalopathy of childhood (ANEC) is a rare inflammatory neurological disease with characteristic neuroimaging findings commonly leading to disability or death. It is associated with a variety of infectious agents, most commonly influenza.
METHODS: This study is a retrospective case series describing three children presenting with ANEC during the 2023-2024 influenza season.
RESULTS: All patients were influenza A positive with a variable duration of prodromal infectious symptoms. Each presented with witnessed or suspected seizures and persistent encephalopathy. Initial computed tomography (CT) was unremarkable in two patients; however, on follow-up neuroimaging all evolved multifocal brain lesions, including symmetric bilateral thalamic involvement consistent with ANEC. All patients received intravenous pulse methylprednisolone, but the timing and selection of additional immunomodulatory interventions differed between patients. Outcomes spanned from slight disability to death.
CONCLUSIONS: These patients illustrate the spectrum of ANEC presentations and outcomes and underscore the importance of prompt investigations in children with persistent encephalopathy and prodromal infectious symptoms, even with unremarkable findings on initial CT. The variability in treatment and outcomes within a single center emphasizes the need for further research to establish a better pathophysiologic understanding of ANEC and optimal treatment protocols
Awake versus Asleep Craniotomy for Eloquent Glioma Resection: Is There Truly a Difference in Intraoperative Costs? A Time-Driven Activity-Based Cost Analysis
No full textVACTERL Screening in Newborns With Anorectal Malformations - An Opportunity to Optimize Screening Practices, add Gynecologic and Spinal Conditions, and Utilize a New Acronym: VACTE(G)RLS.
No full textINTRODUCTION: Spinal cord and gynecologic anomalies may be overlooked as part of the traditional VACTERL screening for patients with anorectal malformations (ARM). We investigated the rates of associated anomalies in patients with ARM using the Pediatric Health Information Systems (PHIS) database.
METHODS: A retrospective multi-institutional cohort study was performed between 1/2016-12/2022. ARM patients were identified using ICD10 diagnosis and procedure codes. We assessed the rate of associated anomaly diagnosis at a patient\u27s initial newborn admission and across all hospital encounters using ICD10 diagnosis codes.
RESULTS: 1467 patients (34 % female) were observed across 46 hospitals. The rates of VACTERL diagnoses on initial admission were 35.0 % vertebral/spinal, 72.9 % cardiac, 9.5 % TEF, 34.7 % renal, and 13.3 % limb. Diagnosis rates were all higher across all hospital encounters: 45.4 % vertebral/spinal, 77.4 % cardiac, 10.2 % TEF, 39.9 % renal, and 15.7 % limb. Among female patients, 16.1 % had a congenital gynecologic malformation diagnosed at birth and 25.8 % across all hospital encounters. Among sub-category diagnoses, the rates of lower Mullerian tract malformations (+11 %), spinal cord malformations (+12 %), and vesicoureteral reflux (+14 %) had the greatest discrepancy between newborn diagnosis and diagnosis across all hospital encounters on a one-tailed comparison of proportions (all p \u3c 0.001) CONCLUSION: The most commonly associated anomalies in ARM are cardiac, vertebral/spinal, renal, and gynecologic (in females). Many are diagnosed as newborns however some are diagnosed later, suggesting an opportunity to improve screening, especially with spinal cord and gynecologic malformations. We suggest changing the VACTERL acronym to VACTE(G)RLS to emphasize the importance and frequency of these malformations in patients with ARM.
LEVEL OF EVIDENCE: Observational study, III.
STUDY TYPE: Multi-institutional retrospective cohort IRB APPROVAL: STUDY00000902
Cold atmospheric plasma effectively kills chordoma cells through induction of intracellular reactive oxygen species.
No full textChordomas remain one of the most difficult-to-treat of skull base tumors. The best chance of survival and cure to date is with en bloc radical surgical resection, followed by adjuvant radiotherapy. Oftentimes, tumor infiltration into functionally critical, or difficult-to-access areas, precludes optimal resection. The median overall survival for these tumors is 116 months. Cold atmospheric plasma (CAP) is generated by applying a high voltage electric field to helium or argon feed gases, resulting in reactive atmospheric species. Over the past decade, CAP has been applied experimentally in a number of oncologic conditions and has demonstrated anti-tumor effects both in vitro and in vivo. Additionally, CAP has been shown to increase cancer cells\u27 sensitivity to radiation and could therefore be a useful tool in improving chordoma recurrence rates as an intraoperative adjuvant therapy to the current standard of care. To date, there are no studies in the literature examining the efficacy of CAP in inducing cytotoxicity in chordoma. We treated CH2, CH7 and UM-Chor1 chordoma cells with CAP, measuring resulting cell viability and intracellular ROS accumulation. Here, we show a dose-dependent increase in intracellular ROS and cell death with direct CAP exposure in vitro, finding an exquisite sensitivity of chordoma cells to CAP-mediated cytotoxicity
FIBROSIS REGRESSION WITH RESMETIROM: EARLY OBSERVATIONS IN REDUCING FIBROSIS IN PATIENTS WITH METABOLIC DYSFUNCTION-ASSOCIATED STEATOHEPATITIS
No full textEfficacy and safety of rigosertib in patients with recessive dystrophic epidermolysis bullosa-associated advanced/metastatic cutaneous squamous cell carcinoma.
No full textBACKGROUND: Recessive dystrophic epidermolysis bullosa (RDEB) is an epithelial fragility disease primarily affecting the skin and is caused by variants in the COL7A1 gene. Individuals with RDEB are predisposed to develop highly aggressive cutaneous squamous cell carcinomas (SCCs) which are the most common cause of premature death. There is a lack of effective prevention or treatment options for patients with RDEB-SCC.
OBJECTIVES: To evaluate the antitumour activity and safety of the polo-like kinase-1 (PLK1) inhibitor, rigosertib, two investigator-initiated open-label, single-arm phase II studies were opened in Europe and the USA and enrolled five patients with RDEB diagnosed with locally advanced and/or metastatic SCCs whose disease had not previously responded successfully to standard care.
METHODS: Using a common protocol, patients were offered either oral or intravenous (IV) administration of rigosertib with consultation from the treating physician. Patients were monitored with clinical photography, biopsy, positron emission tomography/computed tomography scans and quality of life (QoL) questionnaires over the 12-month duration of the trial. The pharmacokinetics of drug absorption was monitored in four patients.
RESULTS: Antitumour efficacy with acceptable toxicity was seen in patients on IV or oral therapy and two patients had a complete response within 6 months of treatment. Their QoL was not negatively impacted by treatment and drug absorption exceeded that seen in previous patient populations presumably due to the relatively high dosing in a cohort of underweight patients.
CONCLUSIONS: These data identify rigosertib as a promising drug therapy for patients with RDEB-SCC where there is a substantial unmet need, absence of approved therapies and where tumours arise on a background of a unique fibrotic and inflammatory environment -characterized by germline mutations in COL7A1 that promote the development of homogenous primary tumours with aberrant PLK1 -activity
