Journal of comparative effectiveness research最新文献

{"title":"A protocol for three observational cohort studies evaluating adverse outcomes, excess costs and repeat procedures after surgery for breast cancer in the USA.","authors":"Sam E Wing, Yuki Liu, Jonathan C Johnson, Kristin J Moore, Feibi Zheng","doi":"10.57264/cer-2025-0013","DOIUrl":"10.57264/cer-2025-0013","url":null,"abstract":"<p><p><b>Aim:</b> Surgical therapy for early-stage breast cancer, including lumpectomy and mastectomy, are common treatments for early-stage breast cancer. Despite having favorable survival outcomes, these procedures can lead to repeat surgeries, adverse outcomes, excess costs and potentially aggressive resections. This is a protocol for a study aims to evaluate three main areas: the risk factors, costs and complications of re-operations following lumpectomy ('Lumpectomy Cohort'), the identification of patients potentially overtreated with mastectomy ('Mastectomy Cohort') and the cost and healthcare resource utilization associated with nipple necrosis following nipple-sparing mastectomy (NSM) ('Nipple Necrosis Cohort'). <b>Materials & methods:</b> A retrospective cohort analysis will be conducted using Optum's de-identified Market Clarity Data (2007-2023), which integrates medical and pharmacy claims with electronic health records. Patients will be included based on specific procedure and diagnosis codes, with additional data extracted from unstructured clinical notes using natural language processing. The study will analyze patient demographics, baseline health, surgical details, and outcomes, including costs, complications, reoperations and mortality. Data will be analyzed descriptively, with Kaplan-Meier analyses for time-to-event outcomes and Wilcoxon Signed Rank tests for cost comparisons. <b>Results:</b> Preliminary cohorts are expected to include 26,472 lumpectomy patients, 16,836 mastectomy patients and 6828 NSM patients with 541 cases of nipple necrosis. <b>Conclusion:</b> This study will provide comprehensive insights into the patient journey - highlighting the costs and patient outcomes following lumpectomy, mastectomy and NSM - potentially guiding better clinical decision-making and resource allocation.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250013"},"PeriodicalIF":2.5,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12308534/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144626433","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Value assessment for health services and procedures: a call to action in a new political climate.","authors":"Meng Li, Peter Neumann","doi":"10.57264/cer-2025-0090","DOIUrl":"10.57264/cer-2025-0090","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250090"},"PeriodicalIF":2.5,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12308532/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144659306","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Crossing borders: the need for empirical evidence of real-world evidence transportability in oncology.","authors":"Caitlin Clunie-O'Connor, Per-Olof Thuresson, Elizabeth Masters, Aliki Taylor, Mats Rosenlund, Philani Mpofu, Blythe Adamson","doi":"10.57264/cer-2025-0053","DOIUrl":"10.57264/cer-2025-0053","url":null,"abstract":"<p><strong>What is this article about?: </strong>This article discusses the challenges of using non local real-world evidence (RWE) in health technology assessments (HTA) when local data are unavailable, insufficient, or inappropriate. HTA organizations often prefer data collected locally or regionally, but the lack of suitable data in many markets has increased interest in understanding data 'transportability' - whether data from one country or population can be used to predict outcomes in another. Established in 2024, the Flatiron Fostering Oncology RWE Use Cases and Methods (FORUM) research consortium is exploring when and how non-local RWE can be effectively applied, with initial work focused on lung cancer, breast cancer and multiple myeloma.</p><p><strong>What does the evidence suggest so far?: </strong>Initial studies suggest RWE from the US could predict outcomes in other countries with proper adjustment for population and treatment differences. Recent research in advanced non-small cell lung cancer demonstrated that adjusted US data provided comparable survival to real observed outcomes in Canada and the UK. This limited evidence base indicates that non-local RWE can help inform decision-making when local data is unavailable.</p><p><strong>What studies are needed next?: </strong>The FORUM consortium is expanding research to other cancer types and countries to better understand RWE transportability. Future studies will focus on comparing outcomes across diverse healthcare systems, identifying key variables for adjustment and developing guidelines for when and how non-local data can be used. These efforts aim to create a framework for the use of global RWE in oncology HTA decision-making.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250053"},"PeriodicalIF":2.5,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12308535/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144600617","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost impact of Bruton's tyrosine kinase inhibitor selection in Medicare patients with chronic lymphocytic leukemia.","authors":"Adam S Kittai, Dipen A Patel, Jason Shafrin, Nadine Zawadzki, Vikram S Shetty, Yazan K Barqawi, Joanna M Rhodes","doi":"10.57264/cer-2025-0035","DOIUrl":"10.57264/cer-2025-0035","url":null,"abstract":"<p><p><b>Aim:</b> To estimate cost savings associated with covalent Bruton's tyrosine kinase inhibitor (cBTKi) choice in patients with treatment-naive (TN) and relapsed/refractory (RR) chronic lymphocytic leukemia (CLL) from a Medicare perspective. <b>Materials</b> <b>&</b> <b>methods:</b> An economic model with Markov structure simulated outcomes in patients with CLL initiating ibrutinib, acalabrutinib or zanubrutinib monotherapy. Modeled population included TN and RR patients who had no prior cBTKi. Treatments were dosed per US FDA label and efficacy assumed identical across cBTKis. Cumulative grade ≥3 adverse event (AE) rates were drawn from extended follow-up of cBTKi phase III clinical trials at similar duration. Costs included drug price per 2024 wholesale acquisition cost and AE management medical costs from literature, adjusted for Medicare reimbursement. Outcomes were total change in payer cost over 1, 3 and 5 years. <b>Results:</b> A cohort of 13,726 patients with CLL was modeled (44% TN, 56% RR). Acalabrutinib's aggregate grade ≥3 AE rate was 25.8% points less in TN patients (35.8% vs 61.6%) and 8.0% points less in RR patients (75.0% vs 83.0%) compared with ibrutinib, and 20.6% points less in TN patients (35.8% vs 56.4%) and 11.1% points less in RR patients (75.0% vs 86.1%) compared with zanubrutinib. Acalabrutinib saved $15,478 more per patient versus ibrutinib in year 1 due to lower treatment cost (-$12,076) and lower AE cost (-$3402). Acalabrutinib also saved $1901 more per patient versus zanubrutinib as acalabrutinib higher treatment cost (+$1663) was offset by lower AE cost (-$3563). Across all patients, acalabrutinib saved $212 million more versus ibrutinib and $26 million more versus zanubrutinib from a Medicare perspective. Acalabrutinib cost savings persisted over 3 and 5 years. <b>Conclusion:</b> Acalabrutinib yielded cost savings versus ibrutinib and zanubrutinib for patients with CLL in Medicare due to lower treatment cost versus ibrutinib and fewer grade ≥3 AEs versus both ibrutinib and zanubrutinib.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250035"},"PeriodicalIF":2.5,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12308542/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144368899","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hospital readmission among patients with unruptured intracranial aneurysms undergoing stent-assisted endovascular coiling using the ENTERPRISE^® 2 stent versus Neuroform^® Atlas stent.","authors":"Reade DeLeacy, Rahul Khanna, Emilie Kottenmeier, Yiran Rong","doi":"10.57264/cer-2025-0096","DOIUrl":"https://doi.org/10.57264/cer-2025-0096","url":null,"abstract":"<p><p><b>Aim:</b> Stent-assisted endovascular coiling is a safe and effective treatment for unruptured intracranial aneurysms (UIAs). This study compared 180-day inpatient readmission and cost among patients with UIA who underwent stent-assisted coiling (SAC) using the ENTERPRISE^® 2 or Neuroform^® Atlas stent. <b>Materials & methods:</b> In this retrospective cohort study, adults with UIA undergoing SAC were identified in the Premier Healthcare Database (2016-2022) and grouped based on the stent used: ENTERPRISE 2 or Neuroform Atlas. Outcomes included all-cause and UIA-related inpatient readmission in the 180 days following treatment, index admission and supply cost. Inverse probability of treatment weighting of propensity score method balanced the two cohorts on study covariates. A weighted generalized estimating equation model assessed study outcomes. <b>Results:</b> A total of 1017 patients were included (ENTERPRISE 2, n = 126; Neuroform Atlas, n = 891). Hospital and patient characteristics except race were well-balanced after weighting. Patients treated with ENTERPRISE 2 versus Neuroform Atlas were 55% less likely to have an all-cause inpatient readmission in the 180-day follow-up period (odds ratio 0.45, 95% CI: 0.20-0.98, p = 0.04). Further, the ENTERPRISE 2 cohort had significantly lower index supply cost ($20,442 vs $27,561, exponentiated ratio 0.74, 95% CI: 0.61-0.90, p = 0.002) compared with the Neuroform Atlas cohort. No significant differences were observed in UIA-related inpatient readmission or total index admission cost between cohorts. <b>Conclusion:</b> Among patients with UIA undergoing SAC, the use of ENTERPRISE 2 stent was associated with a significantly reduced risk of all-cause inpatient hospital readmission and significantly lower index supply cost compared with the Neuroform Atlas stent.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250096"},"PeriodicalIF":2.5,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144760213","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-term effectiveness and safety of ravulizumab in paroxysmal nocturnal hemoglobinuria: a plain language summary.","authors":"Celso Arrais, Natalia Carnelutto, Jheremy Reyes","doi":"10.57264/cer-2025-0022","DOIUrl":"10.57264/cer-2025-0022","url":null,"abstract":"<p><strong>What is this summary about?: </strong>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease where red blood cells break apart because they lack certain protective proteins. This makes them easy targets for the immune system, which can also affect other blood cells. PNH can lead to blood clots and serious organ damage. Ravulizumab and eculizumab block C5, part of the immune system that destroys blood cells in PNH. Ravulizumab is given every 8 weeks, reducing treatment burden compared to eculizumab's 2-week schedule. Ravulizumab was studied in two 26-week trials (301 and 302) for PNH. One compared it to eculizumab in participants new to treatment, and the other looked at switching from eculizumab. Both medicines were equally effective and well-tolerated. This summary covers the extension period - an extra phase after the main study - of studies 301 and 302, to see if ravulizumab stayed safe and effective over up to 2 years. Participants on ravulizumab continued treatment, while those on eculizumab switched to ravulizumab. It also includes an additional analysis from studies 301 and 302 on breakthrough intravascular hemolysis (BTH), where red blood cells can break apart during treatment due to insufficient medication or health issues such as infections.</p><p><strong>What were the results?: </strong>A total of 434 people continued taking ravulizumab during the extension period. For up to 2 years, 90-95% of participants maintained a low level of LDH, a blood marker of red blood cell breakdown, indicating that PNH was under control. Most participants did not need blood transfusions. The improvement in participants' quality of life lasted over the 2 years. Ravulizumab was well tolerated. In studies 301 and 302, ravulizumab had a lower risk of BTH compared to eculizumab.</p><p><strong>What do the results of the studies mean?: </strong>This study shows that ravulizumab remains safe and effective for longterm PNH treatment.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250022"},"PeriodicalIF":2.5,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12308549/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144560263","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Access in all areas? A round up of developments in market access and health technology assessment: part 8.","authors":"Sreeram V Ramagopalan, Annie Jullien Pannelay","doi":"10.57264/cer-2025-0091","DOIUrl":"10.57264/cer-2025-0091","url":null,"abstract":"<p><p>In this update, we discuss the pricing paradox of combination therapies in health technology assessment; examine the Inflation Reduction Act's impact on pharmaceutical innovation and analyze the revised Dutch economic evaluation guidelines.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250091"},"PeriodicalIF":2.5,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12308533/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144310007","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Rivaroxaban versus low-molecular-weight heparins for short- and long-term prognosis in patients with deep vein thrombosis after spontaneous intracranial hemorrhage.","authors":"Jingfan Li, Qi Song, Gaopan Zhang, Guannan Tong, Chengyu Bian, Huimei Zhang, Hulin Zheng, Yafei Wang","doi":"10.57264/cer-2025-0030","DOIUrl":"10.57264/cer-2025-0030","url":null,"abstract":"<p><p><b>Aim:</b> Anticoagulation is the cornerstone of deep vein thrombosis (DVT) treatment, but in patients with intracerebral hemorrhage, it requires a delicate balance between preventing thrombosis and minimizing the risk of rebleeding. To assess the effects of rivaroxaban on short- and long-term clinical prognosis in patients with DVT who have suffered spontaneous intracranial hemorrhage (sICH). <b>Materials & methods:</b> The study retrospectively enrolled 327 sICH patients with DVT from 11 October 2019 to 18 September 2023. The primary outcomes were defined as recurrent sICH, bleeding-related events and mortality within 90 days and 1 year. Multivariate logistic regression was conducted to evaluate the association between rivaroxaban and clinical outcomes based on inverse probability of treatment weighting. <b>Results:</b> Of the included patients, 230 received low-molecular-weight heparins (LMWH) and 97 received rivaroxaban. The reoccurrence rate of sICH was 1.30 and 2.06% in the LMWH and rivaroxaban groups, respectively. Bleeding rates were 8.70% in the LMWH group and 5.15% in the rivaroxaban group. The mortality was less frequent in patients received rivaroxaban than LMWH, following up 90 days (8.25 vs 15.65%, p = 0.07) and 1 year (10.42 vs 25.22%, p = 0.003). Multivariate and inverse probability of treatment weighting-adjusted analyses confirmed the association of rivaroxaban with reduced 1-year mortality and better functional recovery. <b>Conclusion:</b> Rivaroxaban use in DVT patients after sICH was associated with lower long-term mortality and better functional independence, without significantly increasing the risk of sICH recurrence or bleeding complications. These findings should be interpreted with caution and require confirmation through prospective randomized trials.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250030"},"PeriodicalIF":2.5,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12308541/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144608544","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world phosphorodiamidate morpholino oligomer treatment patterns in Duchenne muscular dystrophy: a claims-based analysis.","authors":"Shannon Grabich, Brian Ung, Aalok Nadkar, Kathryn DeYoung, James Signorovitch, Aravindhan Veerapandiyan","doi":"10.57264/cer-2025-0037","DOIUrl":"10.57264/cer-2025-0037","url":null,"abstract":"<p><p><b>Aim:</b> Phosphorodiamidate morpholino oligomers (PMOs) are exon-skipping therapies administered through once-weekly intravenous infusions used to treat Duchenne muscular dystrophy (DMD). This study assessed treatment patterns among patients with DMD receiving PMOs using administrative claims data while accounting for limitations in claims data for these therapies. <b>Materials & methods:</b> This study used Inovalon^® public and private closed claims data (1 June 2016-31 March 2024). Male patients with ≥1 claim for a PMO approved for DMD in the US (eteplirsen, casimersen, golodirsen and viltolarsen) were included. Index date was the first PMO claim. All available follow-up data were used to assess continuous PMO claims coverage, ≥60-day and ≥30-day gaps in PMO claims and PMO re-initiation after a gap. Adherence during 1 year after index was measured using proportion of days covered (PDC). Treatment patterns were also assessed in patients stratified by baseline algorithm-defined nonambulatory status (inferred from claims). <b>Results:</b> Among 397 patients included, median (IQR) follow-up time was 788 (484, 1109) days. Gaps in PMO claims coverage occurred in 190 (47.9%) and 254 (64.0%) patients using ≥60-day and ≥30-day gaps, respectively, among whom 110 (57.9%) and 176 (69.3%) had PMO re-initiation. Using ≥60-day and ≥30-day gap lengths, median (IQR) time to first gap in PMO claims was 25.5 (22.3, 32.9) months and 13.5 (10.2, 17.7) months, respectively and median (IQR) time to PMO re-initiation (not including gap time) was 4.4 (2.8, 8.7) months and 2.5 (1.7, 3.2) months. Median (IQR) PDC was 78.8% (38.8, 94.0) during 1 year after index. PMO treatment patterns were generally similar in patients stratified by algorithm-defined nonambulatory status. <b>Conclusion:</b> In an analysis of administrative claims data, adherence to PMO treatment for DMD was high. For patients with a gap in PMO claims, most subsequently re-initiated treatment, indicating lower discontinuation rates than previously reported.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250037"},"PeriodicalIF":2.5,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12308544/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144659305","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Matching-adjusted indirect comparison between garadacimab and lanadelumab for the long-term prophylactic treatment of patients with hereditary angioedema.","authors":"Sarah Walsh, Anja Haltner, Meaghan Bartlett, John Sears, Yinglei Li, Maebh Kelly, Simona Gavata-Steiger, Chiara Nenci, Iris Jacobs, Ingo Pragst, Neelanjana Ray, Imtiaz A Samjoo","doi":"10.57264/cer-2024-0237","DOIUrl":"10.57264/cer-2024-0237","url":null,"abstract":"<p><p><b>Aim:</b> This study aimed to estimate the relative efficacy between garadacimab 200 mg once monthly (200 QM) and two dosing regimens of lanadelumab (300 mg once every 2 weeks [300 Q2W] and 300 mg once every 4 weeks [300 Q4W]) in adolescent/adult patients with hereditary angioedema (HAE) using matching-adjusted indirect comparisons (MAICs), in the absence of head-to-head randomized controlled trials. <b>Materials & methods:</b> Individual patient data were available from the phase II (NCT03712228) and the phase III VANGUARD (NCT04656418) trials investigating garadacimab, and published summary-level data from the phase III HELP trial investigating lanadelumab (NCT02586805). The primary outcome was time-normalized number of HAE attacks. Secondary efficacy outcomes included time-normalized number of HAE attacks requiring on-demand treatment, time-normalized number of moderate and/or severe HAE attacks, and proportion of attack-free patients. Quality of life (QoL) was also assessed via change from baseline in AE-QoL total score. <b>Results:</b> Compared with lanadelumab 300 Q2W, garadacimab 200 QM statistically significantly reduced number of moderate and/or severe HAE attacks (rate ratio [RR]; 95% confidence interval: 0.25; 0.07, 0.84) and improved AE-QoL score (mean difference: -17.38; -33.67, -1.08). Compared with lanadelumab 300 Q4W, garadacimab 200 QM showed statistically significant improvements in all outcomes: HAE attacks (RR: 0.29; 0.13, 0.63), attacks requiring on-demand treatment (RR: 0.29; 0.13, 0.66), moderate and/or severe HAE attacks (RR: 0.15; 0.05, 0.49), proportion of attack-free patients (hazard ratio: 3.25; 1.45, 7.29), and AE-QoL score (mean difference: -21.29; -37.39, -5.18). <b>Conclusion:</b> These MAICs showed improved efficacy and QoL with garadacimab compared with lanadelumab across multiple endpoints. These findings demonstrate that garadacimab may provide improved therapeutic benefit compared with lanadelumab in the long-term prophylactic treatment of patients with HAE.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240237"},"PeriodicalIF":2.5,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12308543/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144600618","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}