Journal of Health Economics and Outcomes Research最新文献

{"title":"Faster but Not Truer, Part 2: The Regulatory Record, the Economics, and the TRACE-HEOR Reporting Standard.","authors":"Onur Baser","doi":"10.36469/001c.171942","DOIUrl":"10.36469/001c.171942","url":null,"abstract":"<p><strong>Background: </strong>Part 1 of this series found that measured gains from artificial intelligence (AI) in health economics and outcomes research (HEOR) concentrate in tasks with observable ground truth and disappear where the quantity of interest is a counterfactual. What regulators, health technology assessment (HTA) bodies, and sponsors have said about that division has not been assembled in one place.</p><p><strong>Methods: </strong>We assembled the primary regulatory, HTA, industry, and journal policy record through September 2026, requiring for each claim a named primary source and, where a quantity was reported, its provenance and whether the reporting party had a commercial interest. We downloaded the Food and Drug Administration (FDA) AI-enabled medical device list in full, tabulated published economic evaluations of AI, and set reported industry efficiency gains against disclosed use in submissions. We then evaluated the field against the task-based productivity framework of Nobel laureate Daron Acemoglu and derived a reporting standard from the documented failure modes.</p><p><strong>Results: </strong>The FDA has authorized 1614 AI-enabled devices, 1230 (76.2%) in radiology, rising from 18 in 2016 to 335 in 2025, and has received more than 500 drug and biologic submissions containing AI components since 2016; its January 2025 credibility assessment guidance remains a draft and excludes discovery and operational uses. Industry sources report time savings of 40% to 97% in regulatory writing and approximately 80% in payer dossier drafting, but every one of these figures comes from a single-arm comparison without a concurrent control and was reported by the company that deployed or sold the tool. AI use was disclosed in 3 of 1391 HTA documents (0.2%) and 0.6% of one journal's submissions, against surveys reporting use between 25% and 81%. Nineteen economic evaluations of AI have been published against 1614 authorized devices. No regulator or HTA body has been documented accepting or rejecting an AI-derived endpoint as such.</p><p><strong>Conclusions: </strong>Current disclosure instruments ask whether AI wrote the text. Reviewers need to know whether AI selected the evidence, defined the cohort, derived the endpoint, or generated the code, and whether the recommendation would change without it.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 2","pages":"161-177"},"PeriodicalIF":2.5,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13676314/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149216261","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Faster but Not Truer, Part 1: A Systematic Scoping Review of Artificial Intelligence in Health Economics and Outcomes Research.","authors":"Onur Baser","doi":"10.36469/001c.171937","DOIUrl":"10.36469/001c.171937","url":null,"abstract":"<p><strong>Background: </strong>Artificial intelligence (AI) is being introduced into every stage of the health economics and outcomes research (HEOR) production line, from literature screening to causal estimation. The field lacks a defensible count of what has been done, and bibliometric claims about AI adoption circulate without measured error rates.</p><p><strong>Methods: </strong>Following PRISMA-ScR, we searched PubMed for AI terms co-occurring with HEOR terms for publications from 2010 through 2025, retrieved 2642 records, and included 2476 after screening. Records were classified into 8 nonexclusive application domains and 1 of 6 method categories using a published term dictionary. To quantify classification error, an independent large language model classifier, blinded to the dictionary labels and given only title and abstract, reclassified a random sample of 300 included records. We report per label counts, agreement, Cohen kappa, prevalence-adjusted bias-adjusted kappa, precision, recall, F1, and a precision-adjusted corpus size with exact binomial intervals. We separately assembled the primary performance evidence for each HEOR workflow, requiring a quantitative metric from a primary study.</p><p><strong>Results: </strong>Included publications rose from 29 in 2010 to 852 in 2025, a factor of 29.4, with the inflection at 2018 to 2019 rather than at the release of ChatGPT. AI reached 6.0% of all indexed HEOR output in 2025 while HEOR fell to 0.86% of all indexed medical AI output. Economic evaluation was the most frequently assigned domain (1559/2476, 63.0%), followed by real-world data and causal inference (496, 20.0%) and governance, reporting, and validity (494, 20.0%). Generative AI appeared in no record before 2021 and in 103 in 2025. Only 154 of 300 sampled records (51.3%; 95% confidence interval [CI], 45.7-56.9) were judged genuinely HEOR relevant, implying a precision-adjusted corpus of 1271 studies (95% CI 1131-1410). Agreement was substantial for real-world data and causal inference (kappa, 0.729) but negligible for economic evaluation (kappa, 0.144), where dictionary precision was 29.6%. Of 2476 records, 38 (1.5%) were randomized trials and 17 (0.7%) validation studies. The replication package is publicly available.</p><p><strong>Conclusions: </strong>The literature is growing faster than its own measurement infrastructure. Measured gains concentrate in tasks with observable ground truth, and predictive performance repeatedly fails to predict evidentiary performance.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 2","pages":"148-160"},"PeriodicalIF":2.5,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13676315/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149216343","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Natural History of Rare Tumor Risk Syndromes: A Scoping Review to Inform Cost-Effectiveness Models.","authors":"Carolina Di Fabrizio, Judite Gonçalves, Zillur R Shabuz, Andrew Titman, Paula Boaventura, Svetlana Bajalica Lagercrantz, Barbara Peleteiro, Sara Pereira, Liliana Sousa, Stefan Aretz, Paula Soares, Janneke Schuurs Hoeijmakers, Jean Christophe Thery, Hildegunn Hoberg Vetti, Judith Balmaña, Joan Brunet, Carla Oliveira, Céu Mateus","doi":"10.36469/001c.167564","DOIUrl":"10.36469/001c.167564","url":null,"abstract":"<p><strong>Background: </strong>Hereditary cancer syndromes account for approximately 10% of all cancers. Many of these, classified as rare tumor risk syndromes (RTRS), lack comprehensive data on natural history, lifetime cancer risk, and the long-term impact of surveillance. These evidence gaps limit the development of decision-analytic disease progression models and cost-effectiveness analyses needed to support healthcare decision-making.</p><p><strong>Objective: </strong>To map the available evidence on the natural history, cancer risk, and surveillance strategies in selected RTRS to inform disease progression models and future economic evaluations.</p><p><strong>Methods: </strong>A scoping review was conducted on 8 RTRS: Birt-Hogg-Dubé syndrome, familial gastrointestinal stromal tumor, familial melanoma, hereditary diffuse gastric cancer, hereditary leiomyomatosis and renal cell cancer, Li-Fraumeni syndrome, Peutz-Jeghers syndrome, and PTEN hamartoma tumor syndrome. Systematic searches of PubMed, Embase, and relevant gray literature sources were performed between April and June 2026 for studies published since 2010.</p><p><strong>Results: </strong>After screening 16 631 records, 80 studies were included in the review. Findings indicate significant heterogeneity in data availability and surveillance effectiveness. Some RTRS have relatively well-defined surveillance recommendations, whereas others remain poorly characterized. Across studies, cancer incidence was markedly higher and occurred earlier than in the general population, with lifetime cancer risks approaching 100% in certain syndromes. However, only 44% of studies reported follow-up protocols and fewer than 12% compared surveillance with expectant management. Limited data on postdiagnosis survival further complicates economic evaluations of alternative care strategies.</p><p><strong>Conclusions: </strong>The evidence base for RTRS is heterogeneous, particularly regarding longitudinal outcomes and comparative surveillance strategies. These limitations restrict the parameterization of disease progression models and the evaluation of the cost-effectiveness of surveillance strategies, leaving substantial uncertainty about optimal management strategies. Significant evidence gaps remain in the natural history and management of RTRS. This review provides a foundation for future cost-effectiveness analyses by mapping existing evidence and identifying critical knowledge gaps in RTRS care.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 2","pages":"135-147"},"PeriodicalIF":2.5,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13666261/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149194747","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Understanding the Evidence Gaps in the Epidemiology and Burden of Lupus Nephritis and Membranous Nephropathy: A Targeted Literature Review.","authors":"Francesca Barion, Vasileios Pardalidis, Alisha Robert, Catherine Kielar","doi":"10.36469/001c.166255","DOIUrl":"https://doi.org/10.36469/001c.166255","url":null,"abstract":"<p><strong>Background: </strong>Lupus nephritis (LN) and membranous nephropathy (MN) are associated with progression to chronic kidney disease and end-stage renal disease (ESRD), but evidence describing the full disease burden across countries remains limited and fragmented.</p><p><strong>Objectives: </strong>This targeted literature review (TLR) aimed to characterize the disease burden of LN and MN, including epidemiology and clinical, humanistic, and economic outcomes, and identify unmet needs and current knowledge gaps.</p><p><strong>Methods: </strong>A targeted, nonsystematic search of MEDLINE and EMBASE was conducted for English-language articles published between January 2013 and August 2023 in the United States (US), the United Kingdom, Germany, France, China, and Japan.</p><p><strong>Results: </strong>Searches identified 26 relevant articles for LN and 11 for MN. LN epidemiology studies were limited to the US; incidence was estimated at 1:100 000, with higher risk in older individuals, females, and African Americans. MN is rare, with reported annual incidences of 10 to 12 per million in North America and 2 to 17 per million in Europe. Prevalence appears to be increasing for both diseases. LN was associated with substantial progression to ESRD, with 21.1% to 36.8% of patients progressing within 5 to 10 years. ESRD represented a major economic driver, with US monthly costs of approximately $15 000 per patient with LN. MN burden data were more limited but indicated risk of severe long-term renal complications and ESRD. Humanistic burden data were sparse; reported health-related quality-of-life impacts include renal symptom domains not captured by generic instruments, indicating a need for disease-specific measures.</p><p><strong>Conclusions: </strong>Despite evidence of substantial burden, important gaps remain. Epidemiological data were geographically limited, with incidence varying widely, potentially reflecting differences in populations, treatment eras, and ethnicity. Long-term data beyond 5 to 10 years were limited, and robust syntheses evaluating prognostic factors influencing progression to ESRD were lacking. The absence of renal-specific patient-reported outcome measures and inconsistent clinical definitions further limits comparability and treatment value assessment. LN and MN are associated with significant clinical, economic, and humanistic burden, especially for patients with ESRD, but certainty and generalizability are limited by the focused TLR methods and heterogeneous evidence base. More robust data would help characterize long-term outcomes and support more informed value assessments.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 2","pages":"120-134"},"PeriodicalIF":2.5,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13651649/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149167117","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Early Economic Model of Extracorporeal Photopheresis and Other Treatments of Chronic Lung Allograft Dysfunction After Lung Transplantation.","authors":"Stephen Rice, Diarmuid Coughlan, Luke Vale, Carole Cohen, Christian Benden, David Bennett, Mark Greer, Jasvir Parmar, Adrien Tissot, Robin Vos, Myriam A Perez, Martin Carby, Richard Thompson, Karthik Santhanakrishnan, Andrew J Fisher","doi":"10.36469/001c.163450","DOIUrl":"10.36469/001c.163450","url":null,"abstract":"<p><strong>Background: </strong>Extracorporeal photopheresis (ECP) is a technology to treat chronic lung allograft dysfunction (CLAD) following lung transplantation. CLAD leads to significantly higher mortality rates.</p><p><strong>Objective: </strong>The objective of this study was to develop an early economic model that can evaluate the potential cost-effectiveness of treatments for patients with a confirmed diagnosis of CLAD and to evaluate a plausible cost-effectiveness range for ECP.</p><p><strong>Methods: </strong>The literature was reviewed and clinical experts were consulted to develop a Markov model with change in percent forced expiratory volume in 1 second (%FEV1) states, defining rates of lung function decline. The literature and clinical experts were also consulted to determine plausible transition probabilities between %FEV1 states and resource use. Probabilities of dying were estimated by calibrating the model to published survival curves. Treatment response was obtained from cohort studies. EQ-5D values and unit costs were obtained from published sources.</p><p><strong>Results: </strong>In the base case analysis, the incremental cost-effectiveness ratio (ICER) estimates for ECP ranged from £33 343/QALY to £88 278/QALY. This does not account for risk of bias in the implicit relative risk estimate in the model or for scenario combinations. The ICER was lower in the bronchiolitis obliterans syndrome population than in the restrictive allograft syndrome population and in earlier CLAD stages. The ICER was lower if ECP was provided for a course of treatment and there was a persistent protective effect from lung function decline after cessation of treatment.</p><p><strong>Conclusion: </strong>There was very little quality evidence to inform the decision model. The results included a wide range of ICERs. Key determinants include the therapeutic benefit of ECP after treatment cessation, response rates with standard care, and CLAD stage mortality rates.The economic model can be updated as new evidence emerges from ongoing clinical trials. For the National Institute of Health and Care Excellence in England, ECP may qualify as a highly specialized technology, for which the cost-effectiveness threshold may be £100 000/QALY or higher.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 2","pages":"104-111"},"PeriodicalIF":2.5,"publicationDate":"2026-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13613274/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149061664","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Adherence and Follow-Up Colonoscopy After Multitarget Stool DNA Screening in a Regional Healthcare System.","authors":"Amanda Hanson, Shrey Gohil, Ayman Abdelmoity, Mohamed Omar, Adam Kazberuk, Jordan Karlitz, Mallik Greene","doi":"10.36469/001c.168717","DOIUrl":"10.36469/001c.168717","url":null,"abstract":"<p><strong>Background: </strong>Multitarget stool DNA (mt-sDNA) testing is an established noninvasive colorectal cancer (CRC) screening option for average-risk adults, but real-world completion of the screening cascade through colonoscopy remains incompletely characterized.</p><p><strong>Objectives: </strong>This study aimed to evaluate mt-sDNA adherence, test positivity, and follow-up colonoscopy completion in a regional healthcare system and identify factors associated with screening cascade completion.</p><p><strong>Methods: </strong>This retrospective cohort study analyzed mt-sDNA test kits ordered for average‑risk, screening‑eligible individuals in the Spartanburg Regional Healthcare System (South Carolina, USA) from January 2016 to June 2023. Analyses were conducted at the test-kit level; patients could contribute more than one eligible test during the study period. Eligible tests were from patients aged 45 to 85 years with ≥6 months of prior continuous insurance coverage and no high-risk CRC conditions. Outcomes were adherence (kit return within 365 days), test positivity among adherent test kits, and follow‑up colonoscopy within 365 days among eligible positive cases. Multivariable logistic regression and Cox proportional hazards regression models evaluated factors associated with adherence and time to colonoscopy.</p><p><strong>Results: </strong>Of 8439 included test kits, 5733 (67.9%) were returned within 1 year. Among returned test kits, 1018 (17.8%) yielded a positive mt-sDNA result. Of 853 positive tests eligible for follow-up assessment, 596 (69.9%) were for patients who completed follow-up colonoscopy. Adherence to mt-sDNA testing was associated with non-Medicaid insurance, higher income, digital outreach, prior test return, and lower comorbidity burden. Time to colonoscopy was shorter for patients in the middle vs lowest income group.</p><p><strong>Discussion: </strong>Medicaid insurance was the strongest insurance-related predictor of nonadherence, with rates approximately 20 percentage points below those with commercial or Medicare-type coverage. Digital outreach and prior test return history were among the independent predictors of adherence. Income was the primary independent predictor of time to follow-up colonoscopy, potentially reflecting financial barriers to diagnostic follow-up.</p><p><strong>Conclusions: </strong>In this real-world cohort study, approximately two-thirds of tests were returned and 70% of eligible positive tests completed follow-up colonoscopy within 1 year. These findings identify potentially modifiable socioeconomic and outreach-related factors associated with screening cascade completion and support targeted interventions for lower-income and Medicaid-insured populations.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 2","pages":"94-103"},"PeriodicalIF":2.5,"publicationDate":"2026-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13602028/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149039223","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Validity and Predictors of Medical Claims Coding for the Identification of Patients with Obesity.","authors":"Effie L Kuti, Emma Richard, Kevin Schott, Christopher L Crowe, Vincent Willey, Bonnie Donato","doi":"10.36469/001c.164460","DOIUrl":"10.36469/001c.164460","url":null,"abstract":"<p><strong>Background: </strong>The evolving obesity treatment landscape requires examining clinical and economic outcomes, but ICD-10-CM coding inconsistencies hinder patient identification in claims databases.</p><p><strong>Objectives: </strong>This study aims to validate obesity diagnosis codes and identify factors influencing their utilization.</p><p><strong>Methods: </strong>This retrospective cohort study utilized claims and clinical data from the Healthcare Integrated Research Database (HIRD®) to identify adults with a BMI of 30 kg/m² or higher, spanning the period from June 2021 to October 2022. The study examined the use of ICD-10-CM diagnosis codes for obesity across two distinct timeframes: a 6-month period before and after the index date for prevalence and modeling purposes, and a 60-day window around the index date for validity calculations. For validation, the sensitivity, specificity, positive predictive value (PPV), and negative predictive value (NPV) of the ICD-10-CM codes were measured, classifying patients as true or false positives or negatives based on their BMI and coding presence. Multivariable logistic regression was used to identify factors predicting obesity coding, guided by clinical reasoning, statistical results, and refined through backward elimination regression.</p><p><strong>Results: </strong>Out of 2 218 115 patients with available BMI data during the relevant period, 966 427 (44%) had a BMI of 30 kg/m² or above. The study identified 380,606 true positives, 17 422 false positives, 1 234 266 true negatives, and 585 821 false negatives, resulting in a PPV of 95.6%, a NPV of 67.8%, a specificity of 98.6%, and a sensitivity of 39.4%. The prevalence of obesity coding was 54%. Increased likelihood of coding was associated with being female, over the age of 24, non-White, insured by Medicare Advantage, having lower socioeconomic status, higher BMI, and receiving weight management interventions.</p><p><strong>Conclusion: </strong>ICD-10-CM codes demonstrate high specificity and PPV but low sensitivity, indicating that while coded cases are likely true obesity, many patients with obesity are not captured in claims.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 2","pages":"87-93"},"PeriodicalIF":2.5,"publicationDate":"2026-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13499625/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148794097","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Acute Kidney Injury on Hospitalization Outcomes in Patients with Chronic Kidney Disease Undergoing Cardiac Procedures with Cardiopulmonary Bypass in the United States.","authors":"Nicolas Pope, Abby Greenblatt, Thainá Jehá, Juliana Meyers, Christine M Solinsky, Rakesh C Arora, Yan Wang","doi":"10.36469/001c.163558","DOIUrl":"10.36469/001c.163558","url":null,"abstract":"<p><strong>Background: </strong>Acute kidney injury (AKI) is a common complication after cardiac surgery and is associated with more infections, prolonged recovery, longer hospital stays, increased readmission rates, greater costs, and higher mortality. Patients with pre-existing chronic kidney disease (CKD) are at particularly high risk, with this risk increasing as renal function worsens. Damage caused by AKI may be irreversible, despite a return to baseline GFR, compounding stress on remaining nephrons. Understanding risk in these populations may ultimately help to improve prevention and outcomes.</p><p><strong>Objective: </strong>Evaluate the impact of AKI on outcomes and healthcare costs among adults with CKD undergoing cardiac surgery with cardiopulmonary bypass (CPB).</p><p><strong>Methods: </strong>Multi-institutional, retrospective, observational study using the Healthcare Cost and Utilization Project Nationwide Readmissions Database, 2016-2020 datasets. The total adult cardiac surgery population were those with CKD who underwent cardiac surgery requiring CPB (reporting incidence); from which a subpopulation of adults with severe CKD (stage 3 or 4; based on ICD-10-CM codes during index hospitalization) was assessed (impact of AKI). Patients were categorized as follows, based on AKI status during index hospitalization: no AKI, AKI, or AKI requiring dialysis (a subpopulation of AKI).</p><p><strong>Results: </strong>AKI incidence during initial hospitalization was 55.9% and 77.3% in patients with CKD stage 3 and 4, respectively. AKI requiring dialysis occurred in 3.4% and 14.5% of CKD 3 and 4 patients, respectively. One-third of all AKI cases and half of AKI requiring dialysis cases occurred in patients with CKD stage 3 or 4. In patients with CKD stage 3 or 4, multivariate analysis showed AKI was associated with increased inpatient days, increased risk of 180-day readmission, higher hospitalization costs, and greater odds of in-hospital mortality. Patients experienced the greatest burden when AKI required dialysis.</p><p><strong>Conclusions: </strong>Incidence of AKI is higher in patients with preexisting CKD stage 3 or 4 vs 1 or 2 and those without CKD. The burden of AKI is elevated in patients with CKD undergoing cardiac surgery with CPB; targeted interventions are needed to prevent poor outcomes.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 2","pages":"77-86"},"PeriodicalIF":2.5,"publicationDate":"2026-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13477740/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148761686","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Natural History and Treatment Outcomes of Congenital Thrombotic Thrombocytopenic Purpura: A Retrospective Longitudinal Cohort Study.","authors":"Paul Coppo, Marie Scully, Johanna A Kremer Hovinga, Bérangère S Joly, Agnès Veyradier, Ping Du, Parth Patwari, Linda T Wang, Björn Mellgård, Hamid Ferdosi, Ragy Saad","doi":"10.36469/001c.164543","DOIUrl":"https://doi.org/10.36469/001c.164543","url":null,"abstract":"<p><strong>Background: </strong>Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra-rare, life-threatening thrombotic disorder. The real-world clinical trajectory and management of cTTP are not well defined.</p><p><strong>Objectives: </strong>Describe patient characteristics, quantify the incidence and prevalence of clinical manifestations and disease-related complications, and describe treatment patterns and treatment-related outcomes of patients with cTTP.</p><p><strong>Methods: </strong>In this retrospective, multinational, longitudinal cohort study, data were abstracted from the medical records of patients with severe hereditary ADAMTS13 deficiency (<10% activity) at 9 participating sites across Europe and the United States. Eligible patients experienced an index event (cTTP diagnosis, an acute TTP event or TTP manifestation, a cTTP-related clinical event, or prophylaxis) between January 1, 2009, and December 31, 2017. Data collection was from January 1, 2009, to December 31, 2020. A post hoc analysis was conducted among patients who matched inclusion criteria for a phase 3 trial (NCT03393975), and who received plasma-based therapy (PBT) prophylaxis at least once per month, up to 3 times per week.</p><p><strong>Results: </strong>Medical records from 78 patients (61 [78.2%] female) were included in the study. The mean (SD) follow-up was 8.1 (3.1) years. Ninety-two acute TTP events were recorded in 55 (70.5%) patients (overall event rate, 0.145 events per person-year). Eighty (87.0%) acute TTP events occurred in the absence of prophylactic treatment, of which 16 (20.0%) resulted in organ damage (based on physicians' assessment). Twelve (13.0%) acute TTP events occurred during prophylaxis; none resulted in organ damage. Sixty-four TTP manifestations were recorded in 29 (37.2%) patients (overall event rate, 0.101 events per person-year). Of the 25 patients in the post hoc analysis, 4 experienced 9 acute TTP events while receiving PBT prophylaxis.</p><p><strong>Conclusions: </strong>Our findings suggest that acute TTP events occurred less frequently during periods of prophylaxis but also highlight prophylaxis' intrinsic limitations in preventing ongoing manifestations, cumulative organ damage, and the logistical and safety burdens associated with repeated plasma infusions. Patients with cTTP bear a substantial clinical and disease burden associated with acute TTP events and organ damage. Despite treatment with PBT prophylaxis, some patients still experienced acute TTP events, suggesting that more effective treatments may be needed.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 2","pages":"55-66"},"PeriodicalIF":2.5,"publicationDate":"2026-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13476067/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148759380","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluating AI Performance in Systematic Literature Reviews for HEOR: A Case Study.","authors":"Jing Wang-Silvanto, Mansee Jajoo, He Guo, Rishi Ohri, Judith Nelissen, Carolina Casañas I Comabella","doi":"10.36469/001c.165173","DOIUrl":"10.36469/001c.165173","url":null,"abstract":"<p><strong>Background: </strong>Systematic literature reviews (SLRs) are central to evidence generation in health economics and outcomes research, but traditional SLR workflows are labor-intensive. Artificial intelligence (AI) is increasingly being explored to support evidence synthesis tasks.</p><p><strong>Objectives: </strong>To describe methods used to assess AI performance in screening and data extraction in SLRs, and to provide recommendations on how to ensure appropriate use of AI in literature reviews.</p><p><strong>Methods: </strong>We conducted an AI-assisted SLR that mirrored a traditional SLR performed by humans only and assessed the performance of the AI tools employed. AI was used to screen titles/abstracts, screen full texts, and conduct data extraction. Using the traditional SLR as the benchmark, AI performance for title/abstract screening was evaluated in terms of accuracy, recall, and precision at predefined screening milestones, followed by the accuracy assessment of full-text screening by AI. Data items extracted by AI were verified by humans and categorized as correct, incomplete, missing, incorrect, or requiring human checks. The average accuracy rate calculated on the basis of correct data items extracted was used as an indicator of AI performance in data extraction.</p><p><strong>Results: </strong>During title/abstract screening, accuracy and recall remained high but precision was low, increasing false positives. Full-text screening identified a minority of studies included in the traditional SLR. Mean extraction accuracy was 72.93% (range, 57.69%-88.46%). No data were hallucinated by the AI model used.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 2","pages":"46-54"},"PeriodicalIF":2.5,"publicationDate":"2026-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13453151/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148701313","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}