Real-world Assessment Research Articles

A real-world assessment of the treatment-related symptoms and associated burden among patients with lung cancer receiving chemotherapy.

e23159 Background: For patients (pts) with metastatic non-small cell lung cancer (NSCLC), treatment with chemotherapy has shown improved survival, symptom control, and increased quality of life. In clinical trials, the adverse event profile associated with these treatments has been identified. Less explored is the real-world experience of treatment-related symptoms and treatment burden. The objective of this study is to evaluate treatment-related symptoms and treatment burden among chemotherapy-treated NSCLC pts through an electronic remote symptom monitoring system. Methods: Pts diagnosed with NSCLC who received chemotherapy at U.S. academic cancer centers were enrolled in the Carevive’s Patient Reported Outcomes Mobile Platform (Carevive PROmpt®) from September 2020 - June 2023. 174 pts were enrolled. Weekly PRO-CTCAE-derived surveys assessed severity, frequency, and interference for 16 symptoms: anxiety, constipation, cough, decreased appetite, diarrhea, fatigue, general pain, insomnia, mouth/throat sores, muscle pain, nausea, numbness and tingling, rash, sadness, shortness of breath, and vomiting. The GP5 assessed weekly overall side effect treatment bother. Results: The mean age of NSCLC pts was 67.3 (SD = 9.2) years, with 51% female, and 12% African American. The majority of pts, 126 (72%), were enrolled at or within 1 month of the start of treatment. Ninety-one (52%) received chemotherapy and 83 (48%) received chemotherapy in combination. At enrollment, cough, fatigue, and shortness of breath was reported by > 40% of pts receiving chemotherapy, with constipation, decreased appetite, general pain, and numbness/tingling occurring in > 20% of pts. Over 40% of pts reported being at least “somewhat” bothered by the side effects of treatment. Pts with decreased appetite, nausea, and numbness/tingling were more likely to rate symptoms as “somewhat” or “very much” bothersome. No differences were noted for those with combination regimens. Conclusions: For real-world chemotherapy pts, the most frequent symptoms at enrollment were cough, fatigue, shortness of breath, decreased appetite, general pain, and numbness/tingling. Patients with decreased appetite and numbness/tingling were more likely to note higher treatment bother. Results show the feasibility of assessing symptom burden and corresponding patient bother in a real-world NSCLC population, which will have increasing importance increasing use of new treatment modalities with and without cytotoxic chemotherapy. [Table: see text]

Real-world treatment patterns and clinical outcomes among patients with HR+/HER2- advanced/metastatic breast cancer receiving palbociclib in Brazil: IRIS study.

e13088 Background: Palbociclib was approved for hormone receptor-positive (HR+) and human epidermal growth factor receptor 2-negative (HER2-) advanced/metastatic breast cancer (aBC/mBC) in Brazil in 2018. Worldwide studies explore palbociclib's real-world use and outcomes; however, this data is not available for Brazilian patients. IRIS Brazil study aimed to describe demographic/clinical characteristics, treatment and clinical outcomes of adult female patients who have received palbociclib as first-line treatment for HR+/HER2- aBC/mBC in private healthcare institutions in Brazil. Methods: This retrospective descriptive study included females aged 18 years or older diagnosed with HR+/HER2- aBC/mBC and those who had received palbociclib as their initial line of therapy. The study analyzed variables such as demographics, clinical characteristics, treatment history, palbociclib combinations, and clinical outcomes. Progression-free survival (PFS) and overall survival (OS) were calculated using Kaplan-Meier estimates at 6- and 12-month time points. Results: A total of 121 patients were included in the study. The mean age was 54.4 years, and 82 (67.7%) were menopausal at diagnosis. A total of 51 patients (42.1%) were treated with palbociclib + fulvestrant, while 67 (55.8%) were treated with palbociclib + aromatase inhibitors. The majority of patients (n = 79; 65.3%) did not require any dose adjustments. Among the 40 patients (33.1%) who required dose adjustments, the main reason was adverse events/toxicity (n = 36; 90%). The PFS rates at 6 and 12 months were 78% and 60%, respectively. The OS rates at 6 and 12 months were 86% and 70%, respectively. Conclusions: Results from this initial real-world assessment of clinical outcomes in Brazil suggest that Palbociclib combinations exhibit favorable effectiveness in treating HR+/HER2- aBC/mBC disease, as measured by PFS and OS rates.

Real-world Assessment of Red Blood Cell (RBC) Health Biomarkers in Sickle Cell Disease

Real-world Assessment of Red Blood Cell (RBC) Health Biomarkers in Sickle Cell Disease

Real-world assessment of treatment-related symptoms and associated burden among African American and Caucasian patients receiving chemotherapy in the US.

11102 Background: In the treatment of breast cancer, research has identified racial differences in the ability to receive the full dose of prescribed chemotherapy which may be due in part to cancer-treatment symptom burden. In clinical trials, evaluation by race is challenging due to enrollment disparities, with African Americans (AA) accounting for 7% of trial participants, but comprising 12% of the US total population. The objective of this study is to evaluate treatment-related symptoms and treatment burden among patients treated with chemotherapy through an electronic remote symptom monitoring system, and explore whether there were any racial differences. Methods: Patients (pts) diagnosed with multiple myeloma, breast, ovarian, or lung cancer in academic cancer practices were enrolled in Carevive’s Patient Reported Outcomes Mobile Platform (Carevive PROmpt) between September 2020 and June 2023. A total of 1000 pts received chemotherapy alone or in combination. PRO-CTCAE-derived weekly surveys assessed severity, frequency, and interference for 16 symptoms: anxiety, constipation, cough, decreased appetite, diarrhea, fatigue, general pain, insomnia, mouth/throat sores, muscle pain, nausea, numbness and tingling, rash, sadness, shortness of breath, and vomiting. The GP5 assessed weekly overall burden from treatment. Results: The mean age of pts was 60.9 (SD=12.8) years, with 75% female, and 20% AA. At the time of enrollment, fatigue was reported by roughly 48% of AA and Caucasian (C) pts. Muscle pain was noted in 38% of AA pts, followed by general pain (33%), and anxiety (30%), with constipation, decreased appetite, insomnia, nausea, and numbness/tingling occurring in over 20% of pts. A different pattern was observed in C pts, with anxiety occurring in 38%, followed by cough, decreased appetite, general pain, insomnia, muscle pain, nausea, and numbness/tingling present in 20% of pts. The proportion of pts at least “somewhat” bothered by the side effects of treatment were similar (44% AA, 45% C). Conclusions: This study enabled evaluation of symptom burden in cancer in a population closer in racial representation to the real world than in clinical trials; with AA representation being three-fold that seen in clinical trials. Consistent with a qualitative study of AA breast cancer pts, fatigue was identified as the most frequent symptom among those treated with chemotherapy either alone or in combination in the real world. Racial differences in the proportions of pts reporting pain (muscle or general) and anxiety suggest that treating physicians should consider these symptoms to ensure they do not interfere with patients’ ability to receive full chemotherapy courses. Additionally, the levels of anxiety reported in patients highlights the importance of considering concomitant psycho-oncology treatments in cancer therapy.

Real-world assessment of longitudinal opioid use and healthcare resource utilization in patients undergoing colorectal resection

Liposomal bupivacaine (LB) has been used in multimodal pain management regimens to improve postsurgical analgesia. This retrospective cohort analysis assessed clinical and economic outcomes of LB vs non-LB analgesia in minimally invasive colorectal resection surgery using real-world patient data from the IQVIA linkage claims databases. Patients who received LB were 1:1 matched to patients who did not receive LB (non-LB) via propensity scores. Outcomes included opioid use during the perioperative (2 weeks before surgery to 2 weeks after discharge), continued (>2 weeks to 3 months after discharge), and persistent (>3 months to 6 months after discharge) periods and healthcare resource utilization (HRU) during the first 3 months after discharge. Mean opioid consumption was lower in the LB (n = 4397) versus non-LB (n = 4397) cohort perioperatively (483 vs 538 morphine milligram equivalents [MMEs]; P = 0.001) and after discharge within ∼3 months (222 vs 328 MMEs; P < 0.0001) and 3–6 months (245 vs 384 MMEs; P < 0.0001). The LB cohort had shorter mean length of stay (5.2 vs 5.7 days; P < 0.0001) and fewer inpatient readmissions (odds ratio [OR], 0.71; P < 0.0001), emergency department visits (OR, 0.78; P < 0.0001), and outpatient/office visits (OR, 0.91; P = 0.028) than the non-LB cohort 3 months after discharge. These data suggest use of LB in minimally invasive colorectal resection surgery may reduce perioperative and postdischarge opioid use as well as HRU. Although additional studies are needed to confirm these findings, this analysis provides valuable real-world data from large claims databases to evaluate clinical and economic outcomes that complement other types of retrospective and prospective studies.

A Real-World Assessment of Stage I Lung Cancer Through Electronic Nose Technology

IntroductionElectronic nose (E-nose) technology has reported excellent sensitivity and specificity in the setting of lung cancer screening. However, the performance of E-nose specifically for early-stage tumors remains unclear. Therefore, the aim of our study was to assess the diagnostic performance of E-nose technology in clinical stage I lung cancer. MethodsThis phase IIc trial (NCT04734145) included patients diagnosed with a single greater than or equal to 50% solid stage I nodule. Exhalates were prospectively collected from January 2020 to August 2023. Blinded bioengineers analyzed the exhalates, using E-nose technology to determine the probability of malignancy. Patients were stratified into three risk groups (low-risk, [<0.2]; moderate-risk, [≥0.2–0.7]; high-risk, [≥0.7]). The primary outcome was the diagnostic performance of E-nose versus histopathology (accuracy and F1 score). The secondary outcome was the clinical performance of the E-nose versus clinicoradiological prediction models. ResultsBased on the predefined cutoff (<0.20), E-nose agreed with histopathologic results in 86% of cases, achieving an F1 score of 92.5%, based on 86 true positives, two false negatives, and 12 false positives (n = 100). E-nose would refer fewer patients with malignant nodules to observation (low-risk: 2 versus 9 and 11, respectively; p = 0.028 and p = 0.011) than would the Swensen and Brock models and more patients with malignant nodules to treatment without biopsy (high-risk: 27 versus 19 and 6, respectively; p = 0.057 and p < 0.001). ConclusionsIn the setting of clinical stage I lung cancer, E-nose agrees well with histopathology. Accordingly, E-nose technology can be used in addition to imaging or as part of a “multiomics” platform.

Filtering walking actigraphy data in children with unilateral cerebral palsy: A preliminary study.

This study aimed to determine whether filtering out walking-related actigraphy data improves the reliability and accuracy of real-world upper extremity activity assessment in children with unilateral cerebral palsy. Twenty-two children aged 4-12 years diagnosed with unilateral cerebral palsy were included in this study, which was drawn from a two-phase randomized controlled trial conducted from July 2021 to December 2022. Data were collected from a tertiary hospital in Seoul, Republic of Korea. Participants were monitored using tri-axial accelerometers on both wrists across three time points (namely, T0, T1, and T2) over 3 days; interventions were used between each time point. Concurrently, an in-laboratory study focusing on walking and bimanual tasks was conducted with four participants. Data filtration resulted in a reduction of 8.20% in total data entry. With respect to reliability assessment, the intra-class correlation coefficients indicated enhanced consistency after filtration, with increased values for both the affected and less-affected sides. Before filtration, the magnitude counts for both sides showed varying tendencies, depending on the time points; however, they presented a consistent and stable trend after filtration. The findings of this research underscore the importance of accurately interpreting actigraphy measurements in children with unilateral cerebral palsy for targeted upper limb intervention by filtering walking-induced data.

Responding to the real problem of sustainable resuscitation skills with real assessment. Mixed-methods evaluation of an authentic assessment programme.

The retention of resuscitation skills is a widespread concern, with a rapid decay in competence frequently following training. Meanwhile, training programmes continue to be disconnected with real-world expectations and assessment designs remain in conflict with the evidence for sustainable learning. This study aimed to evaluate a programmatic assessment pedagogy which employed entrustment decision and the principles of authentic and sustainable assessment (SA). We conducted a prospective sequential explanatory mixed methods study to understand and address the sustainable learning challenges faced by final-year undergraduate paramedic students. We introduced a programme of five authentic assessments based on actual resuscitation cases, each integrating contextual elements that featured in these real-life events. The student-tutor consensus assessment (STCA) tool was configured to accommodate an entrustment scale framework. Each test produced dual student led and assessor scores. Students and assessors were surveyed about their experiences with the assessment methodologies and asked to evaluate the programme using the Ottawa Good Assessment Criteria. Eighty-four students participated in five assessments, generating dual assessor-only and student-led results. There was a reported mean score increase of 9% across the five tests and an 18% reduction in borderline or below scores. No statistical significance was observed among the scores from eight assessors across 420 unique tests. The mean student consensus remained above 91% in all 420 tests. Both student and assessor participant groups expressed broad agreement that the Ottawa criteria were well-represented in the design, and they shared their preference for the authentic methodology over traditional approaches. In addition to confirming local sustainability issues, this study has highlighted the validity concerns that exist with conventional resuscitation training designs. We have successfully demonstrated an alternative pedagogy which responds to these concerns, and which embodies the principles of SA, quality in assessment practice, and the real-world expectations of professionals.

Effectiveness of BNT162b2 COVID-19 primary series vaccination in children aged 5–17 years in the United States: a cohort study

BackgroundCOVID-19 vaccines are authorized for use in children in the United States; real-world assessment of vaccine effectiveness in children is needed. This study’s objective was to estimate the effectiveness of receiving a complete primary series of monovalent BNT162b2 (Pfizer-BioNTech) COVID-19 vaccine in US children.MethodsThis cohort study identified children aged 5–17 years vaccinated with BNT162b2 matched with unvaccinated children. Participants and BNT162b2 vaccinations were identified in Optum and CVS Health insurance administrative claims databases linked with Immunization Information System (IIS) COVID-19 vaccination records from 16 US jurisdictions between December 11, 2020, and May 31, 2022 (end date varied by database and IIS). Vaccinated children were followed from their first BNT162b2 dose and matched to unvaccinated children on calendar date, US county of residence, and demographic and clinical factors. Censoring occurred if vaccinated children failed to receive a timely dose 2 or if unvaccinated children received any dose. Two COVID-19 outcome definitions were evaluated: COVID-19 diagnosis in any medical setting and COVID-19 diagnosis in hospitals/emergency departments (EDs). Propensity score-weighted hazard ratios (HRs) and 95% confidence intervals (CIs) were estimated with Cox proportional hazards models, and vaccine effectiveness (VE) was estimated as 1 minus HR. VE was estimated overall, within age subgroups, and within variant-specific eras. Sensitivity, negative control, and quantitative bias analyses evaluated various potential biases.ResultsThere were 453,655 eligible vaccinated children one-to-one matched to unvaccinated comparators (mean age 12 years; 50% female). COVID-19 hospitalizations/ED visits were rare in children, regardless of vaccination status (Optum, 41.2 per 10,000 person-years; CVS Health, 44.1 per 10,000 person-years). Overall, vaccination was associated with reduced incidence of any medically diagnosed COVID-19 (meta-analyzed VE = 38% [95% CI, 36–40%]) and hospital/ED–diagnosed COVID-19 (meta-analyzed VE = 61% [95% CI, 56–65%]). VE estimates were lowest among children 5–11 years and during the Omicron-variant era.ConclusionsReceipt of a complete BNT162b2 vaccine primary series was associated with overall reduced medically diagnosed COVID-19 and hospital/ED–diagnosed COVID-19 in children; observed VE estimates differed by age group and variant era.RegistrationThe study protocol was publicly posted on the BEST Initiative website (https://bestinitiative.org/wp-content/uploads/2022/03/C19-VX-Effectiveness-Protocol_2022_508.pdf).

Inconsistencies in rater-based assessments mainly affect borderline candidates: but using simple heuristics might improve pass-fail decisions

IntroductionResearch in various areas indicates that expert judgment can be highly inconsistent. However, expert judgment is indispensable in many contexts. In medical education, experts often function as examiners in rater-based assessments. Here, disagreement between examiners can have far-reaching consequences. The literature suggests that inconsistencies in ratings depend on the level of performance a to-be-evaluated candidate shows. This possibility has not been addressed deliberately and with appropriate statistical methods. By adopting the theoretical lens of ecological rationality, we evaluate if easily implementable strategies can enhance decision making in real-world assessment contexts.MethodsWe address two objectives. First, we investigate the dependence of rater-consistency on performance levels. We recorded videos of mock-exams and had examiners (N=10) evaluate four students’ performances and compare inconsistencies in performance ratings between examiner-pairs using a bootstrapping procedure. Our second objective is to provide an approach that aids decision making by implementing simple heuristics.ResultsWe found that discrepancies were largely a function of the level of performance the candidates showed. Lower performances were rated more inconsistently than excellent performances. Furthermore, our analyses indicated that the use of simple heuristics might improve decisions in examiner pairs.DiscussionInconsistencies in performance judgments continue to be a matter of concern, and we provide empirical evidence for them to be related to candidate performance. We discuss implications for research and the advantages of adopting the perspective of ecological rationality. We point to directions both for further research and for development of assessment practices.

Research on the state of areas in Ukraine affected by military actions based on remote sensing data and deep learning architectures

The invasion of Ukraine by the Russian Federation and the escalation of military actions in the regions have led to significant damage to residential buildings, civilian infrastructure, various critical infrastructure objects, dams, and extensive pollution of the territories. In this context, the tasks of remote sensing using satellite imagery and aerial observation arise to analyze the impact and conduct an economic assessment of damage in these areas. This work investigates and employs deep neural network (DNNs) models in computer vision (CV) tasks (classification, segmentation) and combines their derivatives, such as convolutional networks (CNNs) and vision transformer models (ViTs), to enhance the accuracy of damage assessment. ViTs have demonstrated significant success, often surpassing traditional CNNs, and have potential applications in remote sensing for damage assessment and the protection of critical infrastructure. The research conducted in this work confirms the importance of applying such technologies in environments where labeled data are rare or non-existent, particularly evaluating the use of DNNs, including CNNs and ViTs, in analyzing regions affected by military actions using synthetic aperture radar (SAR) and multispectral images. The aim and subject of this research also include reviewing the possibilities of combining CNNs and ViTs to improve the speed of image feature extraction, landscape detection, and the detection of complex structural contours of objects, where data are usually insufficient. The results of this study provide a critical review of the application of CNNs and ViTs in remote sensing, identifying significant gaps and challenges, especially in the context of the economic consequences of destruction due to military actions. The technical aspects of using CNNs and transformer-based models for complex CV tasks and transfer learning under data-scarce conditions, as well as the challenges in analyzing large volumes of geophysical data, are considered. The conclusions emphasize the transformational potential of DNNs, especially transformers, in remote sensing under conflict and disaster conditions. Their adaptability and accuracy in various environments underscore their utility in both strategic military and humanitarian contexts, establishing a practical standard for their application in key real, real-world scenario-based territory condition assessment.

Introducing re-weighted range voting in clinical practice guideline prioritization: Development and testing of the re-weighted priority-setting (REPS) tool.

We aimed to develop and test a tool based on the re-weighted range voting mechanism to prioritize items (i.e. key questions) in a priority-setting assessment for clinical practice guidelines. The secondary aim was to provide methodological context of the tool. We iteratively developed the tool and used qualitative methods (i.e. think-aloud and semi-structured interviews) to test the tool's usability and make adjustments accordingly. An observational approach was used to test the tool's outcome satisfaction in a real-world priority-setting assessment within a rare-disease guideline of a European Reference Network and under four different conditions in the tool. Four guideline methodologists tested the usability of the tool. The real-world testing was performed with a guideline panel consisting of a core working group, five expertise working groups, and a working group with patient representatives. Thirty-one panel members assigned scores in the priority-setting assessment. Seventeen panel members rated the priority-setting outcome, and sixteen panel members rated the outputs generated under the four conditions. Upon initial use, guideline methodologists found the tool to be quite overwhelming. However, with some initial effort they were able to easily identify the tool's structure. Based on observations and feedback, the tool was further refined and user guidance was developed. Guideline panel members expressed (high) satisfaction with the priority-setting outcome. They particularly preferred the condition when using mean subgroup scores as input or employing aggressive penalties in the weighting method to determine the outputs. The tool generates a ranked list of items and offers flexibility for different choices in priority-setting assessments as long as its input format requirements are met. Although it is not a consensus method, the tool assists in narrowing down a set of priority items. Additional steps in the priority-setting assessment can lead to a consensus being reached regarding the final outcome.

Real-world assessment of anaphylaxis and eosinophilic esophagitis with 12 SQ house dust mite SLIT-tablet sublingual immunotherapy

Sublingual immunotherapy (SLIT) with 12 SQ house dust mile SLIT-tablet (HDM SLIT-tablet) for dust mite-induced perennial allergic rhinitis is reported as effective and safe. Although serious allergic reactions (SARs) and eosinophilic esophagitis (EoE) have infrequently occurred under trial conditions, the safety of HDM SLIT-tablet challenge under real-world conditions is unknown. Our aim was to estimate the incidence of SARs and EoE due to HDM SLIT-tablet challenge. Through use of administrative data from Kaiser Permanente Southern California, this prospective observational study identified patients newly administered HDM SLIT-tablet with follow-up until SLIT discontinuation or end of study. Suspected cases of SARs and EoE were detected by using International Classification of Diseases, 10th Revision, diagnosis and Current Procedural Terminology procedure codes and medication dispensing records. A3-member clinical review committee of allergists adjudicated suspected reactions. The incidence rate of confirmed SARs and EoE per 1000 person years of exposure were determined. A total of 521 patients (93.9% adult and 6.1% pediatric) were exposed to HDM SLIT-tablet challenge from January 2018 through May 2023, for 440.4 person years of exposure. The patients' average age (SD) was 39.3 (14.1) years, 58.7% were female, 44.3% were non-Hispanic White, 40.3% had asthma, and 15.0% had gastroesophageal reflux disease. ASAR occurred in 1 adult patient, and during initial HDM SLIT-tablet challenge, SARs occurred in 2 pediatric adolescents, for an overall incidence of 6.8 SARs per 1000 patient years (95% CI= 2.2-21.1). EoE occurred in 1 adult patient, for an overall incidence of 2.3 cases of EoE per 1000 patient years (95% CI= 0.3-16.1). This real-world study demonstrated that SARs and EoE were infrequent events with HDM SLIT-tablet use, supporting the safety of HDM SLIT-tablets and need for physician supervision with initial challenge.

Real-World Assessment of Personalized Approach With Voglibose Fixed-Dose Combination in Type 2 Diabetes Mellitus.

Post-prandial hyperglycemia (PPHG) remains a complex aspect in the management of type 2 diabetes mellitus (T2DM) in the Indian population due to uncertainty in the optimal utilization of alpha-glucosidase inhibitors (AGIs) either as standalone therapy or in combination, whether initiated initially or as a sequential therapy. This was a post-approval, observational, multicentric clinical study conducted at 50 centers according to principles of the International Council for Harmonisation of Technical Requirements of Pharmaceuticals for Human Use Guideline for Good Clinical Practice (ICH-GCP) and Declaration of Helsinki and local ethics approval. Descriptive and analytical statistics were applied for conclusion and categorical variables using SPSS version 29.0.1.0 (171) (Armonk, NY: IBM Corp.). Protocol analyses of 515 cases revealed baseline demographics as follows: age 57.35±10.04 years, weight 72.86±10.92 kg, and BMI 28.33±6.07 kg/m2. Comorbidities included hypertension (N=169, 32.82%), thyroid disorders (N=99, 19.22%), and heart failure (N=92, 17.86%). Concomitant oral antidiabetics (OADs) prescribed as DPP4i (9.50%), SGLT2i (19.20%), and DPP4i+SGLT2i (7.20%). Study drug reduced glycosylated hemoglobin (HbA1c) by 13.77% (1.25% mean change, p<0.01), fasting blood glucose (FBG) by 23.69% (44.61 mg/dL mean change, p<0.01), post-prandial blood glucose (PPBG) by 24.57% (70.46 mg/dL mean change, p<0.01), and body weight by 4.43% (3.21 kg mean change, p<0.01) over 12 weeks. A total of 161 patients accomplished targeted PPBG of <180 mg/dL (119.13 mg/dL mean change, p<0.01). Regression analysis considering PPBG and HbA1c ≤7.5% showed a weak correlation between them (R-value=0.13, R-squared value=0.02), whereas between PPBG and HbA1c ≤9% yielded moderate positive correlation (R-value=0.53, R-squared value=0.28). There were no adverse events reported or analyzed during the observation period. Voglibose fixed-dose combination (FDC) demonstrates significant effectiveness at the initial dosage when started early in the management of T2DM and high PPBG levels. Moreover, it exhibits good tolerability, thereby contributing to higher compliance among Indian patients who consume a high-carbohydrate diet.

124 Craniotomy Versus Craniectomy for Traumatic Acute Subdural Hematoma Coarsened Exact Matched Analysis of Outcomes

INTRODUCTION: Previous studies comparing craniotomy (CO) versus decompressive hemicraniectomy (DC) for acute subdural hematoma (aSDH) evacuation after traumatic brain injury have not found significant association between surgical approach and patient outcome. We hoped to provide a more granular and real-world assessment of outcomes. METHODS: One hundred thirty-eight patients underwent CO (N = 76) or DC (N = 62) for traumatic aSDH. DC patients were on average 21.4 years younger (P &lt; 0.001), more likely to be male (80.6% vs 60.5%, P = 0.011), and present with GCS = 8 (64.5% vs 36.8%, P = 0.001). Age (P &lt; 0.001), EDH (P = 0.01), skull fracture (P = 0.001), and cisternal effacement (P = 0.02) were independently associated with surgical approach. After coarsened exact matching, DC (P = 0.008), older age (P = 0.007), male sex (P = 0.04), and IVH (P = 0.02), were independently associated with inpatient mortality. Multivariate Cox proportional-hazards regression demonstrated that DC was independently associated with mortality at 90-days (P = 0.001) and 1-year post-operation (P = 0.003) for matched patients. RESULTS: One hundred thirty-eight patients underwent CO (N = 76) or DC (N = 62) for traumatic aSDH. DC patients were on average 21.4 years younger (P &lt; 0.001), more likely to be male (80.6% vs 60.5%, P = 0.011), and present with GCS = 8 (64.5% vs 36.8%, P = 0.001). Age (P &lt; 0.001), EDH (P = 0.01), skull fracture (P = 0.001), and cisternal effacement (P = 0.02) were independently associated with surgical approach. After coarsened exact matching, DC (P = 0.008), older age (P = 0.007), male sex (P = 0.04), and IVH (P = 0.02), were independently associated with inpatient mortality. Multivariate Cox proportional-hazards regression demonstrated that DC was independently associated with mortality at 90-days (P = 0.001) and 1-year post-operation (P = 0.003) for matched patients. CONCLUSIONS: aSDH patients who receive surgical evacuation via DC as opposed to CO are younger, more likely to be male, and have worse clinical exam. After controlling for patient differences via coarsened exact matching, DC is an independent predictor of mortality at discharge, as well as at 90-days and 1-year after surgery.

A Real-world Assessment of Healthcare Resource Utilization and Costs among Patients with Agitation Related to Alzheimer's Dementia Living in Private Homes and Residential Facilities

A Real-world Assessment of Healthcare Resource Utilization and Costs among Patients with Agitation Related to Alzheimer's Dementia Living in Private Homes and Residential Facilities

Abstract 978: Functional characterization and a real-world clinical laboratory pilot of the Foundation for the National Institutes of Health’s (FNIH) circulating tumor DNA (ctDNA) quality control materials (QCMs)

Abstract Background: Analysis of ctDNA now permits assessment of targetable mutations (e.g. EGFR L858R/Ex19del) using noninvasive blood tests. There are several FDA-approved tests using digital PCR (ddPCR) or next-generation sequencing (NGS) and many more laboratory developed tests in use. The FNIH Biomarkers Consortium identified the absence of validated standards to qualify the performance of ctDNA assays as a critical need and set out to aid in the characterization of QCMs. Here we report on a commutability study comparing the performance of three commercial QCMs to clinical samples at or above LoD (limit of detection) and update on a real-world assessment of these QCMs by 11 clinical laboratories spanning 4 continents and 3 assay modalities. Methods: QCMs were provided by Horizon Discovery (H), Thermo Fisher (T) and SeraCare (S). Clinical samples with known EGFR L858R and Ex19del mutations were prepared by diluting specimens at known concentrations into cell-free DNA (cfDNA) from healthy donors. The functional characterization compared detection of the mutations in QCM to clinical samples and consisted of 2 parts: (1) Evaluation of EGFR L858R/Ex19del (range 0.1% to 5% allele frequency [AF]) by ddPCR, amplicon and hybrid-capture NGS; (2) Evaluation near the LoD of the amplicon NGS assay at the assay LoD95 of 0.3% AF. For the clinical pilot, participants were sent QCM pools formulated at 0.5% and 1% AF by ddPCR. Some participants also tested clinical specimens with EGFR L858R and Ex19del at 0.5% and 1% AF by ddPCR. All participants were blinded to AF and identity of the manufacturer. Results: For Part 1, QCM results from hybrid-capture NGS of EGFR L858R were similar to ddPCR; for Ex19del, QCM AF trended lower compared to ddPCR. In contrast, L858R clinical samples showed a strong positive bias compared to ddPCR. For amplicon NGS, QCMs performed similarly to clinical samples, with amplicon NGS AF closest to ddPCR AF for T QCM. For Part 2, the observed hit rates mostly approximated expected values. For H QCM, observed LoD95 (10% for EGFR L858R; 50% for Ex19del) and subsequent dilution hit rates were lower than expected. For the clinical pilot, median Ex19del AF of amplicon NGS were higher than for hybrid-capture NGS for both the 1% QCM formulations and 0.5% QCM formulations. Median QCM L858R AF were similar for amplicon and hybrid-capture NGS, with greatest contributor to variance being inter-lab differences. Conclusions: This project, unprecedented in the number of participants and analytical rigor, revealed unexpected differences in performance of both assays and QCMs. These findings merit consideration by laboratories that rely on QCMs to develop and perform assays for ctDNA testing as diagnostics. Citation Format: Cloud P. Paweletz, Thomas D. Forbes, Grace A. Heavey, Laura M. Yee, Hua-Jun He, Zhiyong He, Dana E. Connors, Daniel Stetson, Susan Keating, Kenneth D. Cole, Gary Kelloff, Robert T. McCormack, Mickey P. Williams, Chris Karlovich. Functional characterization and a real-world clinical laboratory pilot of the Foundation for the National Institutes of Health’s (FNIH) circulating tumor DNA (ctDNA) quality control materials (QCMs) [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 1 (Regular Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(6_Suppl):Abstract nr 978.

Abstract 6474: Real-world assessment of turn-around time for actionable variant testing in NSCLC in the community setting

Abstract Introduction: Rapid turnaround times (TAT) is a key parameter when evaluating the performance of cell free (cf) nucleic acid testing in clinical oncology practice. The purpose of this study was to evaluate the impact of the real-world factors on TAT for actionable variants in NSCLC, detected using commercially available molecular diagnostic tests. These test services are run in a single, centralized laboratory site and utilized primarily by practices in the community across the United States. Methods: In this study, de-identified reference data was reviewed for a ddPCR test that evaluates hotspot mutations including Single Nucleotide Variants (SNV), Insertions and Deletions (INDEL), and gene fusions. The NGS panel additionally tests for Copy Number Amplification (CNA) mutations. Specimen processing was initiated when the specimen was entered into the Laboratory Information System (LIS) and ended when the test result was ready for release. The turnaround time evaluation excluded holidays, and weekends unless the start or end-point occurred on a non-business day. Results: In total, 35,441 specimen results were analyzed. Specimens were typically received in the laboratory within 24 hours of being shipped (88% in 24 hours). Tests by ddPCR included 30,490 (86%) results, while NGS testing accounted for 4,951 (14%) of results. The average analytic lab processing TAT for the ddPCR and NGS test ranged between 2 and 3 days (ddPCR median = 24.5 hours; NGS panel median = 69.7 hours). When no information was missing on the specimen test request form, the turn-around time for the ddPCR tests averaged 30.2 hours and NGS panel averaged 66.2 hours. The percentage of missing or inaccurate information was similar when test requests were initially received. The largest impact factor impacting initiation of specimen processing was information related annotation of the stage of cancer, followed by demographic information related to patient and specimen identifiers. Conclusion: The focus on rapid turnaround times from specimen collection to results released is critical in oncology. Site and patient information, shipping methods, technologies and workflows used in the laboratory are all part of the test process and impact the release of potentially actionable results. Our study has highlighted that rapid turnaround of results is highly feasible for molecular tests being sent out from community practices across the US to a centralized reference laboratory. Specifically, actionable variant results related to NSCLC were successfully detected in the laboratory and were ready for delivery in 2 to 3 days. Automated methods for parsing the information necessary for test requests and delivery will continue to be important in maintaining rapid test result turnaround times. Citation Format: Brittany D'Alessio, Adam Stephen, Colin Cochran, Cole Hager, Zachariah Velasco, Amanda Weaver, Leisa Jackson, Janice Riley, Alisa Tubbs, Gary A. Pestano. Real-world assessment of turn-around time for actionable variant testing in NSCLC in the community setting [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 1 (Regular Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(6_Suppl):Abstract nr 6474.

Development of a new computer simulated environment to screen cognition: assessing the feasibility and acceptability of Leaf Café in younger and older adults

BackgroundExisting traditional cognitive screening tools for dementia have various limitations, including overreliance on tests assessing verbal memory and, to a lesser extent, on some aspects of executive functioning. Comprehensive neuropsychological assessment is sensitive to impairment but time-intensive and expensive. Virtual reality may provide a dynamic and unique understanding of cognitive performance and increase the ecological validity of cognitive assessment. The use of virtual reality in screening for cognitive function in older persons is promising, but evidence for its use remains sparse.ObjectiveOur primary aim was to examine the feasibility and acceptability of a newly developed, virtual reality assessment module, ‘Leaf Café’, a computer-based program that assesses cognition in an engaging, efficient, and ecologically relevant way. The secondary aim was to assess the ability of the module to discriminate between performances of younger and older adults.MethodsA cross-sectional study was carried out in Sydney, Australia, targeting adults aged 18 years and above. Participants completed a traditional cognitive screening tool (Telephone Interview for Cognitive Status-Modified, TICS-M) and Leaf Café, a low-immersive virtual reality module designed to evaluate learning and memory, perceptual-motor function, and executive functioning. The total performance score for each participant, ranging from 0 to 180, was correlated with their cognitive performance assessed by TICS-M, using Pearson’s correlation coefficient. Following module completion, participants were presented with an open and closed-question survey to capture their perceptions, attitudes, and feedback on the module, encompassing practicality, acceptability, and enjoyment. Both descriptive and content analyses were employed to interpret the obtained data.ResultsA sample of 131 participants (mean age 54.9 years, SD = 20.8, range 20–85) took part. The majority were female (71.8%) and born in an English-speaking country (75.8%). The mean amount of time spent in the module was 32.8 min (SD = 13.3) with a mean module score of 107.6 (SD = 38.7). Most participants completed the highest level (5; 80.5%). There was a significant correlation between Leaf Café total scores with TICS-M cognitive scores overall, and for both younger (aged 18–64 years) and older adult (aged 65 + years) groups. No significant difference was found on performance between age groups on TICS-M performance, however, younger adults had significantly better performance on the Leaf Café module than older adults (M = 124.1 vs 95.9; p < .001). Participants had similar response proportions regarding user experience with most agreeing that the module was easy to use (84%) and to navigate (85%). Compared with younger adults, older adults had lower rates of agreement on the module’s design (36.8% vs 64.3%; p = .020) and support experienced (20.5% vs 53.6%; p = .007). Participants highlighted the significance of practicality and the cognitive challenges presented by the module, in terms of memory strain and user interface concerns. Feedback encompassed different opinions on the usefulness of music, with suggestions for improvements centred around clearer instructions, varied game dynamics, and considerations for diverse user needs.ConclusionsLeaf Café is a feasible and acceptable tool to be used for screening for cognitive impairment in older adults and has real-world assessment value. Further verification on the game’s utility in detecting cognitive impairment is required.

Leveraging local neighborhood features in 3D unstructured point cloud data for geometry-based automated damage delineation

In a post-earthquake environment, manual inspection is resource-intensive and subjective. This article presents a real-world damage assessment study of the Improved Iterative Bi-Level Thresholding (IIBLT) algorithm. Damaged regions are delineated using IIBLT based on the local point curvature. The methodology is tested using a unique dataset: Mono Temporal Disaster City 3D Dataset (C3DO). C3DO is a point cloud data generated by a 3D reconstruction of a controlled post-earthquake environment, which presents challenges for concrete façade damage assessment algorithms. Results from IIBLT demonstrated a 16% increase in Matthews Correlation Coefficient (MCC) and Intersection over Union (mGIoU), compared to the current state-of-the-art methods. This corresponds to improving accuracy and completeness in defect region identification without the dependence on knowledge of damaged regions. Results from this study highlight the novel contributions of the IIBLT algorithm including the elimination of parameter tuning, and demonstrate the applicability of the novel iterative framework for concrete surface defect delineation at large.