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e13663 Background: Artificial intelligence (AI) in oncology, including computer vision and radiomics, has expanded rapidly with substantial federal support. However, the extent to which growth in funding and publications is accompanied by growth in prospective interventional evaluation is not well quantified. We conducted an ecosystem-level analysis to compare temporal trends in federal awards, peer-reviewed publications, and interventional clinical trial registrations in AI-oncology. Methods: We performed a cross-sectional landscape analysis spanning 2015–2025 using three public data sources: (1) federally funded extramural awards indexed in NIH RePORTER (including NIH/NCI and other agencies where available); (2) interventional studies registered on ClinicalTrials.gov; and (3) PubMed-indexed publications. Records were identified using a consistent Boolean query requiring AI-related terms (“artificial intelligence,” “machine learning,” “deep learning,” “neural network,” “large language model,” “computer vision,” “natural language processing,” “radiomics”) AND oncology-related terms (“cancer,” “oncology,” “tumor,” “neoplasm,” “carcinoma,” “melanoma,” “leukemia,” “lymphoma”). Annual absolute counts were extracted (without normalization) to preserve volume differences across the ecosystem. We summarized growth over time and calculated publication-to-trial and grant-to-output ratios. Results: Between 2015 and 2025, AI-oncology federal awards totaled 11,503. PubMed-indexed AI-oncology publications increased 38-fold from 558 (2015) to 21,414 (2025), totaling 81,782. Interventional AI-oncology trial registrations increased from 2 (2015) to 84 (2025), totaling 372 across the period. Overall, the publication-to-interventional-trial ratio was 220:1; interventional trial registrations represented 0.45% of publication volume. For every federal award, approximately 7 publications were produced, but only 0.03 interventional trials were registered. Conclusions: Across 2015–2025, AI-oncology funding and publications increased dramatically, while interventional trial registrations remained comparatively sparse. This persistent publication-to-trial gap highlights a translational bottleneck and supports targeted initiatives to accelerate prospective validation, such as dedicated funding mechanisms, multi-center evaluation networks, and trial-ready implementation infrastructure, to realize clinical benefit from AI in cancer care.

e12644 Background: Standard neoadjuvant therapy for HER2-positive early breast cancer combines multi-agent chemotherapy with HER2-targeted antibodies and is associated with substantial toxicity. HER2-directed antibody–drug conjugates (ADCs) may enable de-escalation. We compared efficacy and toxicity of neoadjuvant ADC-based regimens given without concurrent chemotherapy versus standard chemotherapy-based regimens, including by platinum use in the control arm. Methods: We searched PubMed, Embase, Cochrane CENTRAL, Scopus, and Web of Science through December 18, 2024 for randomized controlled trials in early/locally advanced HER2-positive breast cancer. Eligible trials compared neoadjuvant ADC-based regimens without concurrent chemotherapy versus standard chemotherapy-based regimens. Outcomes were pathologic complete response (pCR; per trial definition), grade ≥3 adverse events (AEs), and treatment discontinuation. Odds ratios (ORs) were pooled and heterogeneity was summarized with I². Results: Nine randomized trials (n = 2,021; mean age 50.7±13.7) were included. There was no significant difference in pCR between groups (OR 0.93, 95% CI 0.62–1.40; I² = 59.8%). ADC-based regimens were associated with fewer grade ≥3 AEs (OR 0.30, 95% CI 0.11–0.84; I² = 91.5%). Treatment discontinuation did not differ (OR 1.10, 95% CI 0.39–3.12; I² = 85.8%). Findings were consistent when stratified by platinum use in the control regimen. Conclusions: In HER2-positive early breast cancer, neoadjuvant ADC-based regimens without concurrent chemotherapy achieve pCR comparable to standard chemotherapy while substantially reducing severe toxicity without increasing discontinuation, independent of platinum use. Confirmation in phase III trials with mature survival endpoints is needed.

AbstractVon Willebrand factor (VWF) level and/or function is altered in von Willebrand disease (VWD), the most common heritable bleeding disorder worldwide. Laboratory assessment of VWF is continually evolving. Historically, the primary method for the assessment of VWF platelet-binding activity was the ristocetin cofactor assay (VWF:RCo). Contemporary alternative measures of VWF platelet-binding activity include VWF:GPIbR (recombinant; using ristocetin), VWF:GPIbM (recombinant; gain-of-function mutant), and monoclonal antibody. Recently, the American Society of Hematology, International Society on Thrombosis and Haemostasis, National Hemophilia Foundation, and World Federation of Hemophilia collaboration issued guidelines recommending the use of newer assays of VWF platelet-binding activity (VWF: GPIbM, VWF: GPIbR) over VWF:RCo, given known limitations of the VWF:RCo assay. Despite this recommendation, the newer VWF:GPIbM and VWF:GPIbR assays are not United States Food and Drug Administration cleared, limiting their availability in the United States. We sought to assess assay utilization trends, agreement of VWF testing methods, and imprecision of VWF testing (based on assigned sample type) from the College of American Pathologists Proficiency Testing Surveys. The analysis confirms that, while VWF antigen testing has low imprecision, the various VWF activity assays have significant interassay variability, with VWF:RCo showing greater imprecision than the newer GPIb-binding assays. The overall trends in assay utilization reflect the barriers to complete compliance with modern VWD diagnostic guidelines in North America.

AbstractVon Willebrand factor (VWF) level and/or function is altered in von Willebrand disease (VWD), the most common heritable bleeding disorder worldwide. Laboratory assessment of VWF is continually evolving. Historically, the primary method for the assessment of VWF platelet-binding activity was the ristocetin cofactor assay (VWF:RCo). Contemporary alternative measures of VWF platelet-binding activity include VWF:GPIbR (recombinant; using ristocetin), VWF:GPIbM (recombinant; gain-of-function mutant), and monoclonal antibody. Recently, the American Society of Hematology, International Society on Thrombosis and Haemostasis, National Hemophilia Foundation, and World Federation of Hemophilia collaboration issued guidelines recommending the use of newer assays of VWF platelet-binding activity (VWF: GPIbM, VWF: GPIbR) over VWF:RCo, given known limitations of the VWF:RCo assay. Despite this recommendation, the newer VWF:GPIbM and VWF:GPIbR assays are not United States Food and Drug Administration cleared, limiting their availability in the United States. We sought to assess assay utilization trends, agreement of VWF testing methods, and imprecision of VWF testing (based on assigned sample type) from the College of American Pathologists Proficiency Testing Surveys. The analysis confirms that, while VWF antigen testing has low imprecision, the various VWF activity assays have significant interassay variability, with VWF:RCo showing greater imprecision than the newer GPIb-binding assays. The overall trends in assay utilization reflect the barriers to complete compliance with modern VWD diagnostic guidelines in North America.

SummaryBackgroundPrevious studies have demonstrated an association between nonselective beta‐blockers (NSBBs) and lower risk of hepatocellular carcinoma (HCC) in cirrhosis. However, there has been no population‐based study investigating the risk of HCC among cirrhotic patients treated using carvedilol.AimsTo determine the risk of HCC among cirrhotic patients with NSBBs including carvedilol.MethodsThis retrospective cohort study utilised the Cerner Health Facts database in the United States from 2000 to 2017. Kaplan‐Meier estimate, Cox proportional hazards regression, and propensity score matching (PSM) were used to test the HCC risk among the carvedilol, nadolol, and propranolol groups compared with no beta‐blocker group.ResultsThe final cohort comprised 107 428 eligible patients. The 100‐month cumulative HCC incidence of NSBBs was significantly lower than the no beta‐blocker group (carvedilol (11.24%) vs no beta‐blocker (15.69%), nadolol (27.55%) vs no beta‐blocker (32.11%), and propranolol (26.17%) vs no beta‐blocker (28.84%) (P values < 0.0001). NSBBs were associated with a significantly lower risk of HCC (Hazard ratio: carvedilol 0.61 (95% CI 0.51‐0.73), nadolol 0.74 (95% CI 0.63‐0.87), propranolol 0.75 (95% CI 0.66‐0.84) after PSM in the multivariate cox analysis. In subgroup analysis, NSBBs reduced the risk of HCC in cirrhosis with complications and non‐alcoholic cirrhosis.ConclusionsNSBBs, including carvedilol, were associated with a significantly decreased risk of HCC in patients with cirrhosis when compared with no beta‐blocker regardless of complications status. Future randomised‐controlled studies comparing the incidence of HCC among NSBBs should elucidate which NSBB would be the best option to prevent HCC in cirrhosis.

Context.— Assessing direct oral anticoagulant (DOAC) drug levels by reliable laboratory assays is necessary in a number of clinical scenarios. Objective.— To evaluate the performance of DOAC-specific assays for various concentrations of dabigatran and rivaroxaban, assess the interlaboratory variability in measurement of these DOACs, and investigate the responsiveness of the routine clotting assays to various concentrations of these oral anticoagulants. Design.— College of American Pathologists proficiency testing survey data from 2013 to 2016 were summarized and analyzed. Results.— For dabigatran, the interlaboratory coefficient of variation (CV) of ecarin chromogenic assay was broad (ranging from 7.5% to 29.1%, 6.3% to 15.5%, and 6.8% to 9.0% for 100-ng/mL, 200-ng/mL, and 400-ng/mL targeted drug concentrations, respectively). The CV for diluted thrombin time for dabigatran was better overall (ranging from 11.6% to 17.2%, 9.3% to 12.3, and 7.1% to 11.2% for 100 ng/mL, 200 ng/mL, and 400 ng/mL, respectively). The rivaroxaban-calibrated anti-Xa assay CVs also showed variability (ranging from 11.5% to 22.2%, 7.2% to 10.9%, and 6.4% to 8.1% for 50-ng/mL, 200-ng/mL, and 400-ng/mL targeted drug concentrations, respectively). The prothrombin time (PT) and activated partial thromboplastin time (aPTT) showed variable dose- and reagent-dependent responsiveness to DOACs: PT was more responsive to rivaroxaban and aPTT to dabigatran. The undiluted thrombin time showed maximum prolongation across all 3 dabigatran concentrations, making it too sensitive for drug-level monitoring, but supporting its use as a qualitative screening assay. Conclusions.— DOAC-specific assays performed reasonably well. While PT and aPTT cannot be used safely to determine DOAC degree of anticoagulation, a normal thrombin time excludes the presence of dabigatran.

AbstractSince 1986, the American Society for Apheresis (ASFA) has published practice guidelines on the use of therapeutic apheresis in the Journal of Clinical Apheresis (JCA) Special Issue. Since 2007, updated guidelines have been published every 3 years to reflect current evidence based apheresis practice with the most recent edition (8th) published in 2019. With each edition, the guidelines are reviewed and updated based on any newly published literature since the last review. The PEXIVAS study, an international, randomized controlled trial comparing therapeutic plasma exchange (TPE) vs no TPE and standard vs reduced dose steroid regimen on the primary composite outcome of end stage renal disease or death in patients with ANCA‐associated vasculitis (AAV), was published in February 2020. This study represents the largest study on the role of therapeutic apheresis in AAV published to date and prompted the JCA Special Issue Writing Committee to reassess the current AAV fact sheet for updates based on this newly available evidence. This interim fact sheet summarizes current ASFA recommendations for the evidence‐based use of therapeutic apheresis in AAV and supersedes the recommendations published in the 2019 guidelines.

AbstractImportanceNeuromyelitis optica/neuromyelitis optica spectrum disorder patients' response to therapeutic plasma exchange (TPE) is currently incompletely characterized.ObjectiveOur study aims to understand the clinical status improvement of neuromyelitis optica/neuromyelitis optica spectrum disorder patients treated with TPE.Design, Setting, and ParticipantsThis is a multicenter retrospective study conducted between 1 January 2003 and 31 July 2017 at 13 US hospitals performing apheresis procedures. Subjects studied were diagnosed with neuromyelitis optica/neuromyelitis optica spectrum disorder who received TPE during presentation with acute disease.Main Outcomes and MeasuresThe primary outcome was clinical status improvement in patients treated with TPE. Secondary measures were procedural and patient characteristics associated with response to treatment.ResultsWe evaluated 114 patients from 13 institutions. There was a female predilection. The largest ethnic group affected was non‐Hispanic Caucasian. The average age of diagnosis was 43.1 years. The average time to diagnosis was 3.1 years. On average, five procedures were performed during each treatment series. The most commonly performed plasma volume exchange was 1.0 to 1.25 using 5% albumin as replacement fluid. Most patients (52%) did not require an additional course of TPE and noted “mild” to “moderate” clinical status improvement. Maximal symptom improvement appeared by the fourth or fifth TPE treatment.Conclusion and RelevanceTPE improved the clinical status of patients. Adults responded more favorably than children. Procedural characteristics, including number of TPEs, plasma volume exchanged, and replacement fluid used, were similar between institutions. TPE was well‐tolerated and had a low severe adverse event profile.