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Background: Incidence rates of autism, attention-deficit/hyperactivity disorder (ADHD), and gender dysphoria (GD) are rising not only in the general population, but particularly among children, adolescents, and young adults with eating disorders (EDs). While ED rates have risen during the COVID pandemic, trends in co-occurring autism, ADHD, and GD have yet to be investigated in detail or at scale by way of large electronic medical record data. Objectives: To investigate trends in rates of co-occurring autism, ADHD, and GD among children, adolescents, and young adults with EDs in years prior to and during the COVID-19 pandemic. Methods: We utilized a de-identified multinational electronic health records database (TriNetX) with 48,558 individuals aged 5-26 diagnosed with eating disorders (EDs) at least twice between 2017 and 2022. The primary predictor variable differentiated between the years of each person's index (first) ED diagnosis (2017-2019 vs. 2020-2022). The primary outcome variable was the rate of new co-occurring psychiatric diagnoses of autism, ADHD, and GD in the year following each patient's first ED diagnosis. We applied propensity score-matched multivariable logistic regressions to compare primary outcomes between 2017-2019 and 2020-2022. Results: Our analysis included 17,445 individuals diagnosed with EDs in 2017-2019 (8% autism, 13.5% ADHD, 1.9% GD) and 31,113 diagnosed with EDs in 2020-2022 (8% autism, 14.6% ADHD, 3.2% GD). After 1:1 propensity score matching, 17,202 individuals from the 2017-2019 cohort were matched to peers mirroring the 2020-2022 cohort. Those diagnosed in 2020-2022 showed a 19% (aOR[95%CI]=1.19[1.07-1.33]), 25% (aOR=1.25[1.04-1.49]), and 36% (aOR=1.36[1.07-1.74]) increase in odds for autism, ADHD, and GD diagnoses, respectively, within the 365 days after the index EDs diagnosis, compared to the 2017-2019 cohort. Discussion: Rates of autism, ADHD, and GD are significantly higher in individuals with ED in the post-pandemic 2020-2022 cohort in comparison to the pre-pandemic 2017-2019 cohort, even after controlling for baseline levels of co-occurring psychiatric diagnoses. Such findings reveal a critical gap in our current understanding of the totality of ways in which COVID-19 may have impacted the onset and clinical course of EDs, autism, ADHD, and GD among children, adolescents, and young adults.

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BACKGROUND: Two-stage models of heterogenous treatment effects (HTE) may advance personalized medicine in multiple sclerosis (MS). Brain atrophy is a relatively objective outcome measure that has strong relationships to MS prognosis and treatment effects and is enabled by standardized MRI. OBJECTIVES: To predict brain atrophy outcomes for patients initiating disease-modifying therapies (DMT) with different efficacies, considering the patients' baseline brain atrophy risk measured via brain parenchymal fraction (BPF). METHODS: Analyses included patients enrolled in the Multiple Sclerosis Partners Advancing Technology and Health Solutions (MS PATHS) network who started DMT and had complete baseline data and ≥ 6-month brain MRI follow-up. All brain MRIs were acquired using standardized acquisition sequences on Siemens 3T scanners. BPF change risk was derived by linear mixed effects models using baseline covariates. Model performance was assessed by predicted versus actual BPF change R2. Propensity score (PS) weighting was used to balance covariates between groups defined by DMT efficacy (high: natalizumab, alemtuzumab, ocrelizumab, and rituximab; moderate: dimethyl fumarate, fingolimod, and cladribine; low: teriflunomide, interferons, and glatiramer acetate). HTE models predicting 1 year change in BPF were built using a weighted linear mixed effects model with low-efficacy DMT as the reference. RESULTS: Analyses included 581 high-, 183 moderate-, and 106 low-efficacy DMT-treated patients. The mean and median number of brain MRI observations per treatment period were 2.9 and 3.0, respectively. Risk model performance R2=0.55. After PS weighting, covariate standardized mean differences were <10 %, indicating excellent balance across measured variables. Changes in BPF between baseline and follow-up were found to be statistically significant (p < 0.001), suggesting a pathological change. Patients with low brain atrophy risk had a similar outcome regardless of DMT selection. In patients with high brain atrophy risk, high- and moderate-efficacy DMTs performed similarly, while a 2-fold worse BPF change was predicted for patients selecting low-efficacy DMTs (p < 0.001). Similar results were observed in a sensitivity analysis adjusting for pseudoatrophy effects in a sub-population of patients treated with natalizumab. CONCLUSIONS: The relative benefit of selecting higher efficacy treatments may vary depending on patients' baseline brain atrophy risk. Poor outcomes are predicted in individuals with high baseline risk who are treated with low-efficacy DMTs.

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OBJECTIVE: We describe outcomes following onasemnogene abeparvovec monotherapy for patients with ≥four survival motor neuron 2 (SMN2) gene copies in RESTORE, a noninterventional spinal muscular atrophy patient registry. METHODS: We evaluated baseline characteristics, motor milestone achievement, post-treatment motor function, use of ventilatory/nutritional support, and adverse events as of December 22, 2022. RESULTS: At data cutoff, 19 patients in RESTORE had ≥four SMN2 copies and were treated with onasemnogene abeparvovec monotherapy (n=12 [63.2%] four copies; n=7 [36.8%] >four copies). All patients were identified by newborn screening and were reported as asymptomatic at diagnosis. Median age at onasemnogene abeparvovec administration was 3.0 months. Median time from treatment to last recorded visit was 15.4 months, with a range of post-treatment follow-up of 0.03-39.4 months. All 12 children who were assessed for motor development achieved new milestones, including standing alone (n=2) and walking alone (n=5). Five children reported one or more treatment-emergent adverse events (one Grade 3 or greater). No deaths or use of ventilatory/nutritional support were reported. CONCLUSIONS: Real-world findings from the RESTORE registry indicate that patients with ≥four SMN2 gene copies treated with onasemnogene abeparvovec monotherapy demonstrated improvements in motor function. Adverse events experienced by these patients were consistent with previously reported findings.

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Aim: Sacituzumab-govitecan (Sgov) is a new antibody-drug conjugate recently approved for metastatic triple negative breast cancer (mTNBC), so there are still few data published in the real-world setting.Materials & methods: This study was to analyze the effectiveness and safety of Sgov in mTNBC of patients from the three main hospitals of a city and to compare with the pivotal ASCENT-trial. A total of 46 patients were included, all women diagnosed with mTNBC, with a median age of 52 years and Eastern Cooperative Oncology Group performance status 0-1 71.8% of patients.Results: Sgov effectiveness data seem to be slightly inferior than expected. Furthermore, it is observed that patients with an Eastern Cooperative Oncology Group of two or higher benefit significantly less from treatment with the drug. Safety profile of Sgov is acceptable.

What is this article about? Sacituzumab-govitecan (Sgov) is a new drug recently approved for metastatic triple negative breast cancer, so there is still little data on patients receiving treatment outside of a clinical trial.What were the results? At the cut-off date, 33 patients (71.7%) had withdrawn from treatment with Sgov, and 21 patients (45.7%) had died. Disease remained stable or shrank in 23.9% of patients (n = 11). Median time to disease progression was 4.1 months (IC 95%: 2.6­5.6) and median overall survival 11.0 months (95% CI: 6.1­15.9), with a median duration of follow-up of 6.6 months (interquartile range: 2.1­10.5). Patients with ECOG 0­1 (better performance status) show a higher overall survival than patients with higher ECOG (Not reached [95% CI: NR-NR] vs 3.8 [95% CI: 2.7­4.9]; p = 0.009). No patients stopped treatment due to side effects and almost two thirds of patients did not have to have their dose reduced or delayed due to adverse events (AE). Asthenia (71.7%) and anemia (60.9%) were the most frequent AE. No patient characteristics were identified that predicted less or worse toxicity.What do the results of the study mean? This suggests that sacituzumab-govitecan effectiveness data seem to be slightly worse than expected, even more when performance status before starting Sgov is poor (ECOG 2). AE profile of Sgov is acceptable.

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BACKGROUND: The phase 3 CheckMate 274 trial demonstrated superiority of adjuvant nivolumab over placebo after radical surgery in patients with high-risk urothelial carcinoma (UC). However, real-world data on the efficacy and safety profile of adjuvant nivolumab in Japan have not been reported. METHODS: This retrospective study enrolled patients with high-risk UC who received adjuvant nivolumab therapy following radical surgery between 2022 and 2024 at our institution. We evaluated immune-related adverse events (irAEs) according to the Common Terminology Criteria for Adverse Events, version 5.0. Kaplan-Meier curves were used to assess disease-free survival (DFS) and overall survival (OS). RESULTS: Thirty-three patients with high-risk UC receiving adjuvant nivolumab therapy following radical surgery were identified, and median follow-up was 11 months. Three patients experienced grade 3 irAEs, and 8 discontinued adjuvant nivolumab therapy due to irAEs. No grade 4 or 5 irAEs were observed. Eight patients have completed 1 year of treatment, and nine are currently on treatment. Nine patients had recurrences and one died of cancer. Of the nine patients with recurrences, six relapsed while on adjuvant nivolumab therapy, two relapsed after completing 1 year of treatment, and one relapsed after discontinuation of irAE. The 1- and 2-year OS rates were 100% and 90%, respectively, and median OS was not reached. The 1- and 2-year DFS rates were 70% and 60%, respectively, and median DFS was 26 months. CONCLUSIONS: Adjuvant nivolumab appears to have some efficacy in Japanese patients. Since this is a postoperative adjuvant therapy, careful patient selection is warranted.

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Background: The Observational Medical Outcomes Partnership (OMOP) common data model (CDM) that is developed and maintained by the Observational Health Data Sciences and Informatics (OHDSI) community supports large scale cancer research by enabling distributed network analysis. As the number of studies using the OMOP CDM for cancer research increases, there is a growing need for an overview of the scope of cancer research that relies on the OMOP CDM ecosystem. Objectives: In this study, we present a comprehensive review of the adoption of the OMOP CDM for cancer research and offer some insights on opportunities in leveraging the OMOP CDM ecosystem for advancing cancer research. Materials and Methods: Published literature databases were searched to retrieve OMOP CDM and cancer-related English language articles published between January 2010 and December 2023. A charting form was developed for two main themes, i.e., clinically focused data analysis studies and infrastructure development studies in the cancer domain. Results: In total, 50 unique articles were included, with 30 for the data analysis theme and 23 for the infrastructure theme, with 3 articles belonging to both themes. The topics covered by the existing body of research was depicted. Conclusion: Through depicting the status quo of research efforts to improve or leverage the potential of the OMOP CDM ecosystem for advancing cancer research, we identify challenges and opportunities surrounding data analysis and infrastructure including data quality, advanced analytics methodology adoption, in-depth phenotypic data inclusion through NLP, and multisite evaluation.

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BACKGROUND: Vitamin-K antagonists (VKAs) have widely been replaced by non-VKA oral anticoagulants (NOACs). This includes Austria, Germany and Switzerland, where as VKA, instead of warfarin, the much longer-acting phenprocoumon is used, which was not compared to NOACs in clinical trials. METHODS: Using administrative data from a large German health insurance, we included all anticoagulation-naïve patients with a first prescription of a NOAC or VKA between 2012 and 2020. We analysed overall survival, major adverse cardiac and cerebrovascular events, major thromboembolic events and major bleeding. RESULTS: Overall, 570,137 patients were included (apixaban: 26.9%, dabigatran: 4.6%, edoxaban: 8.8%, rivaroxaban: 39.1% and VKA: 20.7% of these 99.4% phenprocoumon). In the primary analysis using a 1:1 propensity score matching-cohort (PSM-cohort), a significantly higher overall mortality was found for apixaban, edoxaban and rivaroxaban (all p < 0.001) but not for dabigatran (p = 0.13) compared to VKA. In this PSM-cohort, 5-year mortality was 22.7% for apixaban versus 12.7% for VKA, 19.5% for edoxaban versus 11.4% for VKA, 16.0% for rivaroxaban versus 12.3% for VKA (all p < 0.001) and 13.0% for dabigatran versus 12.8% for VKA (p = 0.06). The observed effect was confirmed in sensitivity analyses using un-weighted and three different weighted Fine-Gray regression models on the basis of the entire cohort. CONCLUSIONS: In this large real-world analysis, apixaban, edoxaban and rivaroxaban, but not dabigatran, were associated with worse survival compared to VKA. These findings, consistent with a few other studies including phenprocoumon, cast profound doubts on the unreflected, general use of NOACs. Randomized trials should assess whether phenprocoumon might actually be superior to NOACs.

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Objective: Clinical research networks facilitate collaborative research, but data sharing remains a common barrier. Materials and Methods: The TriNetX platform provides real-time access to electronic health record (EHR)-derived, anonymized data from 173 healthcare organizations (HCOs) and tools for queries and analysis. In 2022, 4 pediatric HCOs worked with TriNetX leadership to found the Pediatric Collaboratory Network (PCN), facilitated via a multi-institutional data-use agreement (DUA). The DUA enables collaborative study design and execution, with institutional review board-approved transfer of complete datasets for further analyses on a per-protocol basis. Results and Discussion: Of the 41.2 million children with TriNetX records, the PCN represents nearly 10%. The PCN assisted several early-career investigators to bring study concepts from conception to an international scientific meeting presentation and journal submission. Conclusion: The PCN facilitates EHR vendor-agnostic multicenter pediatric research on the global TriNetX platform. Continued growth of the PCN will advance knowledge in pediatric health.

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The innovative use of real-world data (RWD) can answer questions that cannot be addressed using data from randomized clinical trials (RCTs). While the sponsors of RCTs have a central database containing all individual patient data (IPD) collected from trials, analysts of RWD face a challenge: regulations on patient privacy make access to IPD from all regions logistically prohibitive. In this research, we propose a double inverse probability weighting (DIPW) approach for the analysis sponsor to estimate the population average treatment effect (PATE) for a target population without the need to access IPD. One probability weighting is for achieving comparable distributions in confounders across treatment groups; another probability weighting is for generalizing the result from a subpopulation of patients who have data on the endpoint to the whole target population. The likelihood expressions for propensity scores and the DIPW estimator of the PATE can be written to only rely on regional summary statistics that do not require IPD. Our approach hinges upon the positivity and conditional independency assumptions, prerequisites to most RWD analysis approaches. Simulations are conducted to compare the performances of the proposed method against a modified meta-analysis and a regular meta-analysis.

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The results of observational studies using real-world data, known as real-world evidence, have gradually started to be used in drug development and decision-making by policymakers. A good quality management system-a comprehensive system of process, data, and documentation to ensure quality-is important in obtaining real-world evidence. A risk-based approach is a common quality management system used in interventional studies. We used a quality management system and risk-based approach in an observational study on a designated intractable disease. Our multidisciplinary team assessed the risks of the real-world data study comprehensively and systematically. When using real-world data and evidence to support regulatory decisions, both the quality of the database and the validity of the outcome are important. We followed the seven steps of the risk-based approach for both database selection and research planning. We scored the risk of two candidate databases and chose the Japanese National Database of designated intractable diseases for this study. We also conducted a quantitative assessment of risks associated with research planning. After prioritizing the risks, we revised the research plan and outcomes to reflect the risk-based approach. We concluded that implementing a risk-based approach is feasible for an observational study using real-world data. Evaluating both database selection and research planning is important. A risk-based approach can be essential to obtain robust real-world evidence.

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WHAT IS THIS SUMMARY ABOUT?: This is a summary of an article about an ongoing study called the BICSTaR study.The BICSTaR study includes people with HIV (human immunodeficiency virus) who are taking a medicine called bictegravir/emtricitabine/tenofovir alafenamide (shortened to B/F/TAF). B/F/TAF is a single tablet that contains 3 different drugs for the treatment of HIV. The drugs work together to reduce the levels of HIV so that the virus can no longer be detected by a blood test.People taking part in the study are adults with HIV living in Europe, Canada, Israel, Japan, South Korea, Singapore and Taiwan. People take 1 tablet of B/F/TAF once a day. They are either taking B/F/TAF as their first treatment for HIV, or they have switched to B/F/TAF from another HIV treatment.Researchers looked at how well B/F/TAF worked and how safe it was in people who took B/F/TAF for a year. WHAT ARE THE KEY TAKEAWAYS?: Researchers found that B/F/TAF worked well in almost all people in the study by reducing levels of HIV in the blood. The virus could not be found in the blood of more than 9 out of 10 (94%) people who were taking B/F/TAF as their first HIV medicine and more than 9 out of 10 people (97%) who had taken another HIV medicine before starting B/F/TAF. This is known as having an 'undetectable viral load' and is a major goal for HIV treatment success. Researchers did not find any evidence of HIV developing resistance to B/F/TAF, which might stop B/F/TAF from working properly.Around 1 out of 10 people (13%) had side effects (any unwanted sign or symptom that people have when taking a medicine that researchers think might be caused by the medicine) that might have been caused by B/F/TAF. Most of these side effects were not classified as serious. Less than 1 out of 100 (0.1%) people had serious side effects that might have been caused by B/F/TAF. Only 6 out of 100 people stopped taking B/F/TAF due to side effects caused by B/F/TAF. As a result, more than 9 out of 10 people (95%) took B/F/TAF for at least 1 year. WHAT WERE THE MAIN CONCLUSIONS REPORTED BY THE RESEARCHERS?: B/F/TAF worked well in people with HIV in this study. Most people (around 9 out of 10) did not have any side effects.

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Background: In China, the 2022-2023 influenza season began earlier and was characterized by higher levels of influenza activity and co-circulation of various respiratory pathogens compared with seasons before the coronavirus disease 2019 (COVID-19) pandemic. Timely and precise estimates of influenza vaccine effectiveness (IVE) against infections can be used to guide public health measures. Methods: A test-negative study was conducted to estimate IVE against laboratory-confirmed influenza using data from the CHinese Electronic health Records Research in Yinzhou (CHERRY) study that prospectively integrated laboratory, vaccination, and health administrative data in Yinzhou, southern China. We included patients who presented influenza-like illness and received nucleic acid tests and/or antigen tests between October 2023 and March 2024. Estimates of IVE were adjusted for age, gender, month of specimen submitted, chronic comorbidities, and hospitalization status. Results: A total of 205 028 participants, including 96 298 influenza cases (7.6% vaccinated) and 108 730 influenza-negative controls (13.4% vaccinated), were eligible for this analysis. The estimates of IVE were 49.4% (95% CI, 47.8%-50.9%), 41.9% (95% CI, 39.8%-44.0%), and 59.9% (95% CI, 57.9%-61.9%) against overall influenza, influenza A, and influenza B, respectively. A lower IVE was observed for individuals aged 7-17 years (38.6%), vs 45.8% for 6 months-6 years, 46.7% for 18-64 years, and 46.1% for ≥65 years. Vaccination reduced the risk of infection by 44.4% among patients with chronic comorbidities. IVEs varied by epidemic weeks with the changes in influenza activity levels and the switch of dominant influenza strains. Conclusions: Influenza vaccination in the 2023-2024 season was protective against infection for the entire population.

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Objective: Type 2 diabetes (T2DM) poses a significant public health challenge, with pronounced disparities in control and outcomes. Social determinants of health (SDoH) significantly contribute to these disparities, affecting healthcare access, neighborhood environments, and social context. We discuss the design, development, and use of an innovative web-based application integrating real-world data (electronic health record and geospatial files), to enhance comprehension of the impact of SDoH on T2 DM health disparities. Methods: We identified a patient cohort with diabetes from the institutional Diabetes Registry (N = 67,699) within the Duke University Health System. Patient-level information (demographics, comorbidities, service utilization, laboratory results, and medications) was extracted to Tableau. Neighborhood-level socioeconomic status was assessed via the Area Deprivation Index (ADI), and geospatial files incorporated additional data related to points of interest (i.e., parks/green space). Interactive Tableau dashboards were developed to understand risk and contextual factors affecting diabetes management at the individual, group, neighborhood, and population levels. Results: The Tableau-powered digital health tool offers dynamic visualizations, identifying T2DM-related disparities. The dashboard allows for the exploration of contextual factors affecting diabetes management (e.g., food insecurity, built environment) and possesses capabilities to generate targeted patient lists for personalized diabetes care planning. Conclusion: As part of a broader health equity initiative, this application meets the needs of a diverse range of users. The interactive dashboard, incorporating clinical, sociodemographic, and environmental factors, enhances understanding at various levels and facilitates targeted interventions to address disparities in diabetes care and outcomes. Ultimately, this transformative approach aims to manage SDoH and improve patient care.

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Background: The acceptance of telemedicine in the German health care system is growing. This also extends to gynecological applications such as the prescription of contraceptives. This study investigates the contraceptive use and adherence of patients using a direct-to-consumer (DTC) prescription platform for oral contraception. Methods: A retrospective cross-sectional study was conducted using anonymized data obtained from a DTC prescription platform between May 2021 and March 2023. The patient-reported outcome was evaluated after 3 months through a follow-up questionnaire. Results: In total, 8,065 patient records were available and 1,008 patients responded to the follow-up questionnaire. Patients were mostly taking combined oral contraceptives (COCs) and only 6% were taking a progestin-only pill (POP). Even in patient populations at higher risk, such as smokers and obese women, the proportion of POP users was less than 20%. Over 90% of users reported that they took the pill without any intake errors, with the main intake error being forgotten intake. Overall, 23% of patients reported adverse events while taking the pill, with POP users reporting more adverse events than COC users (36.7% vs. 22.2%). Over 70% of patients regularly attended cervical screening. Conclusions: Users of a DTC prescribing platform exhibit strong adherence, with over 90% successfully taking oral contraceptive pills. The platform effectively identifies absolute contraindications but could enhance recommendations for contraceptives in the presence of relative contraindications. For healthy women familiar with pill usage, DTC platforms offer a viable and convenient alternative to traditional doctor's office prescriptions.

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AIMS/INTRODUCTION: The study aim was to investigate sulfonylurea prescription patterns in elderly patients (age ≥65 years) with type 2 diabetes mellitus in Japan. Sulfonylurea use among older adults has been insufficiently examined, despite the associated risks of hypoglycemia. MATERIALS AND METHODS: This retrospective cross-sectional survey entailed analysis of Japanese pharmacy data, extracted from the Musubi database, for patients (age 20-100 years) prescribed sulfonylureas between November 2022 and October 2023. Dose distribution, adherence to the Diabetes Treatment Guidelines for the Elderly 2023 and coprescription of other diabetes medications were investigated. RESULTS: Of the total 91,229 patients, 80.1% were prescribed glimepiride, 16.3% gliclazide and 3.6% glibenclamide. In patients aged ≥65 years, exceeding the recommended dose (>1 mg/day for glimepiride, >40 mg/day for gliclazide) was numerically higher for glimepiride (25.0%) than for gliclazide (7.8%). The most common prescribing patterns were quadruple therapy with a sulfonylurea, a dipeptidyl peptidase-4 inhibitor, an sodium-glucose transporter 2 inhibitor and a biguanide in patients aged 65 to <75 years, and dual therapy with a sulfonylurea and a dipeptidyl peptidase-4 inhibitor in patients aged ≥75 years. Unfortunately, glinide was coprescribed for 338 (0.5%) of elderly patients. Insulin was coprescribed for 3,682 (5.6%) of elderly patients. CONCLUSIONS: Analysis of real-world sulfonylurea prescription data found guideline non-adherence, namely, excessive prescription of glimepiride, use of glibenclamide in elderly patients, and common coprescription with dipeptidyl peptidase-4 inhibitors. These findings might provide an opportunity to reconsider the treatment of patients with type 2 diabetes mellitus who are over-prescribed sulfonylureas to reduce residual risks, such as hypoglycemia.

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Japanese guidelines recommend angiotensin II receptor blockers (ARBs) and angiotensin-converting enzyme inhibitors (ACEIs) as first-line therapy in hypertensive patients with chronic kidney disease (CKD) and proteinuria, but calcium channel blockers in patients with stage G4-5 CKD aged ≥75 years; however, the implementation of these guidelines in clinical practice is unclear. We investigated the actual use of these agents in this patient population. We conducted a cross-sectional study using the DeSC database, which includes anonymous information from various health insurance systems in Japan. A total of 34,362 hypertensive patients aged <75 years with CKD stage G1-G5 with urinary protein ≥1+ or aged ≥75 years with CKD stage G1-G3 with urinary protein ≥1+, for whom Japanese guidelines recommend first-line ARBs/ACEIs, were included in the analysis. Prescription rates of ARBs and ACEIs were calculated overall and separately for each age group and glomerular filtration rate category. The mean participant age was 65.8 ± 14.8 years, including 24,585 patients (72%) <75 years and 9777 (28%) ≥75 years. Of these, 9529 were prescribed ARBs/ACEIs (prescription rate 28%). The prescription rate was lower in patients aged <75 years with CKD stage G1-G5 (prescription rate 23%) compared with patients aged ≥75 years old with CKD stage G1-G3 (prescription rate 41%) (p < 0.001). Patients with CKD stage G1 had the lowest prescription rates for ARBs/ACEIs in both age categories. These results indicate that, despite guideline recommendations, ARBs/ACEIs are insufficiently prescribed for patients with hypertension associated with CKD with proteinuria. ARBs and ACEIs were only used in 28% of hypertensive patients aged<75 years (CKD stage G1-G5) or aged ⩾75 years (CKD stage G1-G3), with urinary protein ⩾1+, for whom Japanese guidelines recommend ARBs/ACEIs. The prescription rate was lower in the younger compared with the older patients.

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BACKGROUND: Treatment guidelines were developed early in the pandemic when much about COVID-19 was unknown. Given the evolution of SARS-CoV-2, real-world data can provide clinicians with updated information. The objective of this analysis was to assess mortality risk in patients hospitalized for COVID-19 during the Omicron period receiving remdesivir+dexamethasone versus dexamethasone alone. METHODS: A large, multicenter US hospital database was used to identify hospitalized adult patients, with a primary discharge diagnosis of COVID-19 also flagged as "present on admission" treated with remdesivir+dexamethasone or dexamethasone alone from December 2021 to April 2023. Patients were matched 1:1 using propensity score matching and stratified by baseline oxygen requirements. Cox proportional hazards model was used to assess time to 14- and 28-day in-hospital all-cause mortality. RESULTS: A total of 33 037 patients were matched, with most patients ≥65 years old (72%), White (78%), and non-Hispanic (84%). Remdesivir+dexamethasone was associated with lower mortality risk versus dexamethasone alone across all baseline oxygen requirements at 14 days (no supplemental oxygen charges: adjusted hazard ratio [95% CI]: 0.79 [0.72-0.87], low flow oxygen: 0.70 [0.64-0.77], high flow oxygen/non-invasive ventilation: 0.69 [0.62-0.76], invasive mechanical ventilation/extracorporeal membrane oxygen (IMV/ECMO): 0.78 [0.64-0.94]), with similar results at 28 days. CONCLUSIONS: Remdesivir+dexamethasone was associated with a significant reduction in 14- and 28-day mortality compared to dexamethasone alone in patients hospitalized for COVID-19 across all levels of baseline respiratory support, including IMV/ECMO. However, the use of remdesivir+dexamethasone still has low clinical practice uptake. In addition, these data suggest a need to update the existing guidelines.

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BACKGROUND: Recognizing the limitations of pre-market clinical data, regulatory authorities have embraced total product lifecycle management with post-market surveillance (PMS) data to assess medical device safety and performance. One method of proactive PMS involves the analysis of real-world data (RWD) through retrospective review of electronic health records (EHR). Because EHRs are patient-centered and focused on providing tools that clinicians use to determine care rather than collecting information on individual medical products, the process of transforming RWD into real-world evidence (RWE) can be laborious, particularly for medical devices with broad clinical use and extended clinical follow-up. This study describes a method to extract RWD from EHR to generate RWE on the safety and performance of embolization coils. METHODS: Through a partnership between a non-profit data institute and a medical device manufacturer, information on implantable embolization coils' use was extracted, linked, and analyzed from clinical data housed in an electronic data warehouse from the state of Indiana's largest health system. To evaluate the performance and safety of the embolization coils, technical success and safety were defined as per the Society of Interventional Radiology guidelines. A multi-prong strategy including electronic and manual review of unstructured (clinical chart notes) and structured data (International Classification of Disease codes), was developed to identify patients with relevant devices and extract data related to the endpoints. RESULTS: A total of 323 patients were identified as treated using Cook Medical Tornado, Nester, or MReye embolization coils between 1 January 2014 and 31 December 2018. Available clinical follow-up for these patients was 1127 ± 719 days. Indications for use, adverse events, and procedural success rates were identified via automated extraction of structured data along with review of available unstructured data. The overall technical success rate was 96.7%, and the safety events rate was 5.3% with 18 major adverse events in 17 patients. The calculated technical success and safety rates met pre-established performance goals (≥ 85% for technical success and ≤ 12% for safety), highlighting the relevance of this surveillance method. CONCLUSIONS: Generating RWE from RWD requires careful planning and execution. The process described herein provided valuable longitudinal data for PMS of real-world device safety and performance. This cost-effective approach can be translated to other medical devices and similar RWD database systems.

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