Citations

Abstract Introduction: Patient and stakeholder involvement enhances the conduct and applicability of comparative effectiveness research (CER). However, examples of engagement practices for CER leveraging real-world data (i.e., data from routine clinical practice) are scarce. Notably, these studies differ from traditional clinical trials in their technical complexity and minimal prospective data collection, posing unique challenges for stakeholder involvement. This paper describes patient and stakeholder engagement in a CER study of type 2 diabetes (T2D) medications using real-world data from a large administrative claims database. Methods: A Patient and Stakeholder Advisory Group (PSAG) was formed to guide study design, conduct, and dissemination. The PSAG (n = 12) included individuals with T2D, clinicians, health systems leaders, professional society representatives, and a payer representative. Members were surveyed post-study initiation to assess their participation goals and experiences to date. Results: PSAG members influenced key design and methodological decisions, including cohort selection and adding an aim focused on patient preference elicitation. Survey results indicated high satisfaction with engagement processes and a desire for ongoing involvement. Most PSAG members cited their main goals as impacting the lives of people with T2D and ensuring the research’s relevance to clinicians. Conclusions: Best practices for engaging stakeholders in CER using real-world data are underdeveloped. Our experience suggests that an inclusive, stakeholder-engaged approach enriches the research process and ensures diverse perspectives are integrated into study design and conduct. Ongoing efforts will focus on assessing long-term engagement outcomes and PSAG member satisfaction.

Importance Typical cysteine-altering NOTCH3 ( NOTCH3 cys ) variants are highly prevalent (approximately 1 in 300 individuals) and are associated with a broad spectrum of small vessel disease (SVD), ranging from early-onset stroke and dementia (cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy [CADASIL]) to nonpenetrance. A staging system that captures the full NOTCH3 -SVD severity spectrum is needed and currently lacking. Objective To design a simple disease severity staging system that captures the broad clinicoradiological NOTCH3 -SVD severity spectrum. Design, Setting, and Participants A cohort study was performed in which the NOTCH3 -SVD severity staging system was developed using a discovery cohort (2019-2020) and validated in independent international CADASIL cohorts (1999-2023) and the UK Biobank. Clinical and imaging data were collected from participants originating from 23 international CADASIL cohorts and from the UK Biobank. Eligibility criteria were presence of a NOTCH3 cys variant, availability of brain magnetic resonance imaging, and modified Rankin Scale score. The discovery cohort consisted of 195 NOTCH3 cys -positive cases from families with CADASIL; the validation set included 1713 NOTCH3 cys -positive cases from 15 countries. The UK Biobank cohort consisted of 101 NOTCH3 cys -positive individuals. Data from 2-year (2019-2023) and 18-year (1999-2017) follow-up studies were also analyzed. Data analysis was performed from July 2023 to August 2024. Main Outcomes and Measures Percentage of cases following the sequence of events of the NOTCH3 -SVD stages, and the association between the stages and ischemic stroke, intracerebral hemorrhage, global cognition, processing speed, brain volume, brain microstructural damage, and serum neurofilament light chain (NfL) level. Results The NOTCH3 -SVD staging system encompasses 9 disease stages or substages, ranging from stage 0 (premanifest stage) to stage 4B (end stage). Of all 1908 cases, which included 195 in the discovery cohort (mean [SD] age, 52.4 [12.2] years) and 1713 in the validation cohorts (mean [SD] age, 53.1 [13.0] years), 1789 (94%) followed the sequence of events defined by the NOTCH3 -SVD staging system. The NOTCH3 -SVD stages were associated with neuroimaging outcomes in the NOTCH3 cys -positive cases in the CADASIL cohorts and in the UK Biobank and with cognitive outcomes and serum NfL level in cases from the CADASIL cohorts. The NOTCH3 -SVD staging system captured disease progression and was associated with 18-year survival. Conclusions and Relevance The NOTCH3 -SVD staging system captures the full disease spectrum, from asymptomatic individuals with a NOTCH3 cys variant to patients with end-stage disease. The NOTCH3 -SVD staging system is a simple but effective tool for uniform disease staging in the clinic and in research.

An extended orbital exenteration defect in a male in his mid-80s was successfully reconstructed with a medial sural artery perforator flap. To our knowledge, this flap has not been described in the literature for such defects until now. While it may require meticulous intramuscular perforator dissection, it provides several advantages; it is thin, pliable, has a long pedicle of large calibre facilitating microanastomosis and provides adequate coverage where the amount of chimeric muscle can easily be tailored to the defect’s surface area and volume. In addition, its robust vascular supply can withstand volume changes post radiation therapy. Two years after surgery, the patient has healed well with an excellent functional outcome.

ABSTRACT Background Flat bone chondrosarcomas have worse outcomes than extremity tumors, but there is no data directly comparing the different flat bones. The aim of this study was to examine differences in recurrence and survival between pelvic and scapular chondrosarcoma. Methods One hundred and sixty‐nine (42 scapula, 127 pelvic) patients with chondrosarcoma who underwent surgical resection were reviewed. High‐grade tumors were defined as lesions that were Grade 3 or dedifferentiated on surgical pathology. Results Patients with low‐intermediate grade lesions of the scapula were more likely to have positive margins during definitive surgical management (14% vs. 3%, OR 5, 95% CI [1.15, 22.6], p = 0.02), however, this did not translate to differences in recurrence or survival ( p > 0.05). The presence of an associated soft tissue mass in low‐intermediate grade lesions was not associated with increased recurrence. Among high‐grade tumors, patients with scapular lesions had worse DSS (HR 2.99, 95% CI [1.05, 8.51], p = 0.04). One‐ and 2‐year DSS for high‐grade pelvic tumors was 75% and 57%, respectively, and 50% and 33% for scapular tumors. Conclusion Survival for high‐grade chondrosarcoma of the flat bones is poor, particularly for those of the scapula. Despite a higher rate of positive margins for low‐intermediate grade lesions of the scapula, there was no significant difference in survival compared to low‐intermediate grade lesions of the pelvis. Additional studies with larger sample sizes are needed to further elucidate differences between these locations.

Abstract Psychiatry has a long and controversial history, and its legacy is often questioned in the arts. But how is psychiatry used by filmmakers, what do they think of mental illness, and how do they use it? What is their frame of mind? Psychoanalytically? Biologically? Sociologically or anthropologically? Cinema has not only produced a full spectrum of elements of mental illness but also showed its treatment (or lack thereof). Frames of Minds traces the historical display of interpreted neuropsychiatry and explains why it is a story about medicine and why it should interest us. Patterns and paradigm shifts in neuropsychiatry over the last century have emerged over the decades, and mental disorders on screen became more specific. The influence of the anti-psychiatry movement in documentary filmmaking was evident. The takeaway is that filmmakers like to break the rules and take chances with the awkwardness of mental illness and psychotic breakdown.

Purpose of review To review role of artificial intelligence in medicine. Recent findings Artificial intelligence is continuing to revolutionize access, diagnosis, personalization of medicine, and treatment in healthcare. As a matter of fact, artificial intelligence contributed to the research that resulted in 2024 Nobel Prizes in physics, chemistry, and economics. We are only at the tip of the iceberg in utilizing the abilities of artificial intelligence in medicine to improve accuracy of diagnoses and to enhance patient outcomes. Artificial intelligence has allowed better image analysis, prediction of progression of disease, personalized treatment plans, incorporations of genomics, and improved efficiency in care and follow-up utilizing home monitoring. In ocular health diagnosis and treatment of diabetic retinopathy, macular degeneration, glaucoma, corneal infections, and ectasia are only a few examples of how the power of artificial intelligence has been harnessed. Even though there are still challenges that need more work in the areas of patient privacy, Health Insurance Portability and Accountability Act (HIPAA) compliance, reliability, and development of regulatory frameworks, artificial intelligence has revolutionized and will continue to revolutionize medicine. Summary Artificial intelligence is enhancing medical diagnosis and treatment, as well as access and prevention. Ocular imaging, visual outcome, optics, intraocular pressure, and data points will continue to see growth it the field of artificial intelligence.

Background: Culture-negative vertebral osteomyelitis presents a significant diagnostic challenge. Neisseria sicca (N. sicca) is a typically benign commensal organism of the upper respiratory tract that rarely causes invasive infections, warranting cautious interpretation if isolated in a single positive culture. This case study details a 62-year-old male diagnosed with vertebral osteomyelitis caused by N. sicca, examining diagnostic challenges, treatment, and outcomes. Methods: We conducted a comprehensive search on MEDLINE using the keywords “Neisseria sicca”, “osteomyelitis”, and “diskitis”. An additional search excluding N. gonorrhea and N. meningitidis was also performed, revealing a total of four cases of N. sicca osteomyelitis in the literature. Results: A 62-year-old male with a history of hypertension, type 2 diabetes, atrial fibrillation, and previous L5-S1 spinal fusion presented with fever and back pain in May 2023. Initial imaging revealed T8-T9 vertebral osteomyelitis. Despite a six-week course of IV daptomycin, his symptoms worsened, prompting further evaluation. A CT-guided biopsy with comprehensive testing, including histopathology and microbial cultures, initially identified N. sicca. Due to its rarity, additional biopsies were conducted, confirming the infection. IV ceftriaxone was initiated, leading to significant pain improvement, and a subsequent MRI showed near resolution. The patient transitioned to oral cefuroxime, with follow-up confirming complete infection resolution by December 2023. Conclusions: This case underscores the importance of a structured diagnostic approach in culture-negative vertebral osteomyelitis to differentiate between commensal contamination and true infection. Repeated positive cultures of N. sicca from a sterile site confirmed its role as the causative agent. Early identification and targeted antibiotic therapy are critical to improving outcomes in rare cases of N. sicca vertebral osteomyelitis.

Abstract Background The recognition of autoimmune causes of encephalitis has led to epidemiological shifts in the worldwide characteristics of encephalitis. N-methyl-D-aspartate receptor (NMDAR) antibody encephalitis leads to well-established complex neuropsychiatric manifestations. In low- and middle-income countries, including Vietnam, its relative incidence, especially in children, is unknown and most neurologists currently consider infectious encephalitis prior to autoimmune etiologies. Methods The study was prospectively conducted at Children's Hospital 1 in Ho Chi Minh City between March 2020 and December 2022. Any child admitted to the Department of Infectious Diseases and Neurology fulfilling the case definition of encephalitis was eligible to participate. Cerebrospinal fluid samples were collected alongside meta-clinical data for analysis. Results We recruited 164 children with a clinical diagnosis of encephalitis. Etiologies were determined as NMDAR antibody encephalitis in 23 of 164 cases (14.0%), Japanese encephalitis virus in 14 of 164 (8.5%), and herpes simplex virus in 4 of 164 (2.4%). Clinical categorizations suggested idiopathic viral encephalitis in another 71 (43.3%), and autoimmune encephalitis of unknown origin in the remaining 52. Factors including demographics, specific clinical features, cerebrospinal fluid and electroencephalogram findings, and length of hospital stay were significantly different between NMDAR antibody encephalitis and Japanese encephalitis. Conclusions At a tertiary children's hospital in Vietnam, the prevalence of NMDAR antibody encephalitis exceeds that of Japanese encephalitis, the most common infectious encephalitis cause in Southeast Asia. NMDAR antibody encephalitis is associated with long hospital stay and poor outcomes. These findings should change pediatric diagnostics, to earlier consider autoimmune treatments in this clinical setting.