Journal of comparative effectiveness research最新文献

{"title":"A novel real-world data methodology for lymphoma outcome classification: the real-world Lugano study.","authors":"Richard Scott Swain, Andrew Klink, Parisa Asgarisabet, Kristin M Zimmerman Savill, Bindu Kalesan, Alexandrina Balanean, Harlen Hays, Jill Kaufman, Lindsay McAllister, Courtney Omary, Hsing-Ting Yu, JaLyna Laney, Nicholas C Richardson, Catherine C Lerro, Fatima Rizvi, Jonathon Vallejo, Kun Wang, Marc R Theoret, Donna R Rivera, Bruce A Feinberg","doi":"10.57264/cer-2025-0134","DOIUrl":"10.57264/cer-2025-0134","url":null,"abstract":"<p><p><b>Aim:</b> In oncology trials, blinded independent central review (BICR) is the standard for treatment response classification. Real-world data methodologies that align with BICR may reduce misclassification in real-world evidence (RWE) studies and enhance reproducibility, increasing value of RWE. We aimed to develop and validate a novel real-world data-based methodology - real-world Lugano (rwLugano) - for assessing lymphoma response to align with clinical trials. <b>Materials & methods:</b> We conducted a retrospective, multisite chart abstraction study using Cardinal Health Practice Research Network (PRN) sites to identify adults with diffuse large B-cell lymphoma initiating first-line (1L) therapy from 1 January 2015, through 31 December 2022, in US community oncology. Sites collected patient characteristics and PET/CT scans at baseline and first response. Two radiologists independently classified responses; a medical oncologist adjudicated discordances. <b>Results:</b> We compared initial treatment responses using three methods: physician-charted from electronic health records, rwLugano-derived per Lugano 2014 and BICR-adjudicated per Lugano 2014. Agreement was assessed via percentage concordance, kappa (κ), and multivariable generalized linear mixed modeling for assigning complete response (CR). Among 178 patients, CR rates were 63.5% (physician-charted), 81.5% (rwLugano) and 83.1% (BICR). Compared with BICR, rwLugano showed higher agreement (87.9%, κ = 0.52) than physician-charted (77.0%, κ = 0.40). The generalized linear mixed modeling analyses identified clinical factors associated with concordance: for physician charted assessments, greater numbers of extranodal sites increased agreement with BICR (OR 1.92), while MYC mutation (OR 0.38) and anemia (OR 0.37) reduced agreement. For rwLugano, nonprivate insurance was associated with higher agreement (odds ratio [OR]: 4.40), whereas MYC mutation reduced agreement (OR: 0.26). <b>Conclusion:</b> rwLugano improves real-world lymphoma response classification, aligning with BICR and supporting more accurate, reproducible RWE for clinical and regulatory decision-making. Using methods BICR and rwLugano may provide opportunities to minimize outcome misclassification and improve comparability of clinical trial and clinical practice approaches.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250134"},"PeriodicalIF":2.5,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13044812/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147443625","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":"Ataluren for the treatment of people living with nonsense mutation Duchenne muscular dystrophy: a plain language summary of Study 041.","authors":"Shiwen Wu, Dmitry Vlodavets, Alexandra Prufer de Queiroz Campos Araujo, Jill Castle","doi":"10.57264/cer-2025-0184","DOIUrl":"10.57264/cer-2025-0184","url":null,"abstract":"<p><strong>What is this summary about?: </strong>This article describes results from Study 041. Study 041 was a clinical study of ataluren, a treatment for people living with nonsense mutation Duchenne muscular dystrophy (nmDMD for short). Over time, people living with nmDMD experience muscle loss and reduced muscle strength and function (decline in muscle function). The researchers wanted to know whether 72 weeks of treatment with ataluren slowed the decline in muscle function in a large group of people living with nmDMD. To determine this, they compared the effects of ataluren with the effects of a placebo, which is a treatment that looks the same as the study treatment but has no active ingredients. The study also looked at the safety of ataluren over the 72 weeks of treatment. Ataluren has previously been compared with placebo in 48-week long clinical trials.</p><p><strong>What were the results?: </strong>In this study, 359 people took at least one dose of ataluren or placebo, even if they later switched or didn't stick to the treatment plan. Over 72 weeks, ability to walk and physical function declined less in people receiving ataluren than in people receiving placebo. This means that their physical abilities were maintained for longer if they took ataluren. The number of side effects were generally similar between people who took ataluren and those who took placebo.</p><p><strong>What do the results of study 041 mean?: </strong>These results help to confirm that 72 weeks of treatment with ataluren (compared with 72 weeks of placebo treatment) slows down the decline in muscle function experienced by people living with nmDMD. Results also confirmed that side effects reported were similar with ataluren and placebo. These results likely show that people who receive ataluren can maintain their independence for a longer time. This study is the largest phase 3 clinical study of people living with nmDMD that has been done so far. <b>ClinicalTrials.gov, NCT number:</b> NCT03179631.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250184"},"PeriodicalIF":2.5,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13044809/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147512474","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":"Indirect treatment comparison of iptacopan versus pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and residual anemia despite C5 inhibitor treatment.","authors":"Austin Kulasekararaj, Phillip Scheinberg, Maria-Magdalena Balp, Olivier Somenzi, Anggie Wiyani, Josefin Snellman, Sarah Walsh, Katharina Pannagl, Jason Steenkamp, Régis Peffault de Latour","doi":"10.57264/cer-2025-0167","DOIUrl":"10.57264/cer-2025-0167","url":null,"abstract":"<p><p>This study conducted an indirect treatment comparison (ITC) of iptacopan, the first oral monotherapy targeting factor B, versus pegcetacoplan, a subcutaneous infusion targeting complement component 3, in patients with paroxysmal nocturnal hemoglobinuria (PNH) and residual anemia despite complement component 5 inhibitors (C5i). An unanchored matching adjusted ITC was performed, using phase III APPLY-PNH and PEGASUS studies, identified by a systematic literature review. The outcomes were: change from baseline (CfB) in hemoglobin (Hb), with and without post-transfusion data, CfB in lactate dehydrogenase (LDH), CfB in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score, transfusion avoidance, and serious adverse events. Two adjustment scenarios were analyzed: (A) baseline Hb, sex, transfusion history ≤12 months, and (B) scenario A plus reticulocyte count, baseline LDH and age. After matching and adjusting, baseline characteristics were similar across trials. Iptacopan showed greater Hb improvements than pegcetacoplan with post-transfusion data (mean difference - scenario A: 1.05 g/dl; p < 0.001, scenario B: 0.76 g/dl; p = 0.018) and without (A: 1.31 g/dl; p < 0.001, B: 1.01 g/dl; p = 0.014). Odds of transfusion avoidance were higher with iptacopan (A: odds ratio [OR] = 9.17, p = 0.013; B: OR = 12.71, p = 0.009). No significant difference was noted in CfB in LDH and FACIT-Fatigue score. These findings suggest that iptacopan may improve Hb levels and reduce transfusion dependency compared with pegcetacoplan in PNH patients with residual anemia despite C5i and must be interpreted in the context of ITCs.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250167"},"PeriodicalIF":2.5,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13044902/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147480725","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":"Lefamulin versus omadacycline for community acquired bacterial pneumonia: a systematic review and anchored indirect treatment comparison using moxifloxacin as the common comparator.","authors":"Meiyu Wu, Sheng Han, Hongchao Li","doi":"10.57264/cer-2025-0188","DOIUrl":"10.57264/cer-2025-0188","url":null,"abstract":"&lt;p&gt;&lt;p&gt;&lt;b&gt;Aim:&lt;/b&gt; Community-acquired bacterial pneumonia (CABP) remains a major cause of morbidity and mortality worldwide, particularly among elderly and susceptible populations. Escalating antimicrobial resistance among prevalent CABP pathogens in China, combined with safety limitations of existing regimens, underscores the urgent need for novel therapeutic strategies. Lefamulin (LEF) and omadacycline (OMA), recently approved in mainland China, offer promising alternatives, but direct comparative evidence is lacking. This study aims to indirectly compare the clinical efficacy and safety outcomes of LEF versus OMA in the treatment of CABP and to explore subgroup differences in high-risk populations. &lt;b&gt;Materials & methods:&lt;/b&gt; A systematic literature review was conducted across PubMed, Embase, the Cochrane Library and ClinicalTrials.gov from inception through March 2024, limited to English-language studies, to identify phase III randomized controlled trials evaluating LEF or OMA in adults with CABP. The Bucher method was used for the indirect comparison, with effect estimates reported as risk ratios (RRs) and 95% CIs. Similarities in trial design and populations supported the transitivity assumption. Primary end points were early clinical response (ECR), investigator-assessed clinical response (IACR) at test of cure (TOC) and treatment-emergent adverse events leading to death. Subgroup analyses were further stratified by patient age (elderly patients), presence of comorbidities and causative pathogens. &lt;b&gt;Results:&lt;/b&gt; Three randomized controlled trials involving 2063 patients were included in this study. LEF and OMA demonstrated comparable efficacy in terms of ECR (RR: 1.01, 95% CI: 0.93-1.09) and in terms of IACR at TOC (RR: 0.95, 95% CI: 0.88-1.02). The relative risk of treatment-emergent adverse events leading to death in the LEF group compared with the OMA group was 0.67 (95% CI: 0.15-3.02), with no statistically significant difference observed. In subgroup analysis, LEF demonstrated statistically significant superiority over OMA in treating patients with &lt;i&gt;Haemophilus influenzae&lt;/i&gt; infections (RR: 1.28, 95% CI: 1.03-1.60). No other subgroups reached statistical significance. LEF showed a numerical trend toward favoring in multiple subgroups, including the elderly, patients with comorbidities, and those infected with specific pathogens, particularly in the ECR analysis. Meanwhile, OMA demonstrated potential numerical advantages in a few subgroups defined by comorbidities or specific pathogens for IACR at TOC. &lt;b&gt;Conclusions:&lt;/b&gt; Both LEF and OMA have been shown to be effective and safe in treating CABP. LEF demonstrated significant benefit in &lt;i&gt;Haemophilus influenzae&lt;/i&gt; infections and consistently favorable trends in high-risk or specific infected subgroups. OMA also shows favorable trends in certain patient groups. These findings highlight the need to further accumulate additional clinical data or real-world evidence to support future c","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250188"},"PeriodicalIF":2.5,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13044819/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147443558","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":"Health utility by Psoriasis Area and Severity Index response status after biologic induction therapy in Chinese patients with moderate to severe psoriasis.","authors":"Lu Jian, Liang Zhang, Guo Zhou, Xingzhi Wang, Kun Hu, Yongfang Duan, Mi Zhang, Liang Tan, Wendong Chen, Bingfa Li, Yehong Kuang","doi":"10.57264/cer-2025-0122","DOIUrl":"10.57264/cer-2025-0122","url":null,"abstract":"<p><p><b>Aim:</b> Psoriasis severely affects quality of life, particularly in patients with moderate to severe psoriasis. This study aims to estimate health utility by Psoriasis Area and Severity Index (PASI) response after biologic induction therapy in Chinese patients with moderate to severe psoriasis. <b>Materials & methods:</b> Based on an established mapping algorithm between the Dermatology Life Quality Index and EuroQol 5 Dimension 5 Level utility score, the retrospective Dermatology Life Quality Index total scores before and after biologic induction therapy in 512 patients were converted into health utility scores to assess the associations between PASI response and post-induction utility using a multivariate generalized linear regression model. The constructed regression model was further applied to estimate post-induction utility by PASI response for a representative patient cohort, including 300 moderate to severe psoriasis patients from five tertiary hospitals. <b>Results:</b> When compared with baseline utility, the post-induction utility increased significantly irrespective of PASI response status. The multivariate regression analysis indicated that higher PASI response status was significantly correlated with higher utility score when compared with PASI <50 (coefficient: 0.050-0.124, p < 0.001). Relative to the baseline utility before treatment (0.653) in the representative patient cohort, the estimated post-induction utility increased by 0.174 for PASI <50, 0.224 for PASI 50-74, 0.275 for PASI 75-89, 0.280 for PASI 90-99 and 0.298 for PASI 100, respectively. <b>Conclusion:</b> As the extent of PASI improvement increases in Chinese patients with moderate to severe psoriasis, their health utility values rise significantly. However, the improvement in quality of life above PASI 75 is relatively limited when compared with PASI 75.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250122"},"PeriodicalIF":2.5,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13044821/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147498292","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":"Comparative effectiveness of oral antibiotics to treat uncomplicated urinary tract infections in male outpatients.","authors":"Karl Madaras-Kelly, Jeremy Boyd, Laura Bond","doi":"10.57264/cer-2025-0163","DOIUrl":"10.57264/cer-2025-0163","url":null,"abstract":"<p><p><b>Aim:</b> New IDSA guidelines define uncomplicated UTI (uUTI) as infection limited to the bladder in both men and women. This study compared the effectiveness of β-lactams, nitrofurantoin and trimethoprim/sulfamethoxazole (TMP/SMX) to fluoroquinolones for outpatient uUTI treatment in men. <b>Materials & methods:</b> A retrospective cohort of adult male outpatients diagnosed with uUTI during 2019-2021 in the Department of Veterans Affairs system was created. Inclusion required an Emergency Department, Urgent/Primary Care visit with ICD-10 documentation of UTI and a prescription of interest dispensed. Patients with recent UTI, hospitalization, infectious co-diagnosis, temperature >99.9 F, pyelonephritis or prostatitis, or for whom asymptomatic bacteriuria treatment was appropriate were excluded. Overlap weighting propensity scores and generalized estimating equation models assessed the relative risk of a subsequent UTI-related visit or hospitalization within 3-30 days with a new antibiotic dispensed. <b>Results:</b> A total of 45,442 males (mean [SD]) age 71.6 (12.7) years were treated at 130 VA medical centers. Treatment n (%) included: β-lactams 17,655 (38.9%), nitrofurantoin 8394 (18.5%), TMP/SMX 9709 (21.4%) and fluoroquinolones 9684 (21.3%). UTI-related return visits occurred in 5453 (12.0%) and UTI-related hospitalization occurred in 1431 (3.1%). The adjusted relative risk (aRR, [95% CI]) of a return visit compared with fluoroquinolones was higher for β-lactams (1.22, [1.02, 1.48]) and nitrofurantoin (1.47, [1.23, 1.74]) but not for TMP/SMX (0.99, [0.80, 1.23]). The aRR for UTI-related hospitalization was not different for β-lactams (1.06, [0.80, 1.40] or TMP/SMX [0.80, [0.56, 1.15], but was lower for nitrofurantoin [0.60, [0.41, 0.89]). <b>Conclusion:</b> Compared with fluoroquinolones, β-lactam and nitrofurantoin prescribed for outpatient uUTI were associated with modestly increased UTI-related return visits but not hospitalization in men.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250163"},"PeriodicalIF":2.5,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13044810/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147512559","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":"Comparison of two-stage methods for count data in Mendelian randomization: a simulation study.","authors":"Mary Appah, Riku Takei, Vinodh Srinivasasainagendra, Sandeep C Vejandla, Richard J Reynolds, Tony R Merriman, Hemant K Tiwari","doi":"10.57264/cer-2025-0081","DOIUrl":"10.57264/cer-2025-0081","url":null,"abstract":"<p><p><b>Aim:</b> Mendelian randomization (MR) is an instrumental variable (IV) method that utilizes genetic variants to establish causality between risk factors and outcomes in observational studies. These methods were primarily developed under assumptions appropriate for continuous or approximately normally distributed variables. However, in many biomedical and clinical studies, exposures and outcomes are naturally recorded as counts, such as the number of disease episodes or clinical events. Despite this, two-stage MR methods are applied to count data without a clear understanding of their validity under such settings. While individual-level MR methods like two-stage predictor substitution (TSPS) and two-stage residual inclusion (TSRI) are common, their comparative performance for count exposures and outcomes remains unclear. <b>Materials & methods:</b> We conducted the first systematic evaluation of TSPS and TSRI for count data using Poisson and negative binomial models across realistic MR scenarios. Simulations varied instrument strength (IS), confounding, sample size and also focused on invalid instruments. Performance was assessed by bias, root mean square error (RMSE), 95% confidence interval (CI) coverage, CI width and Type I error rate. To demonstrate practical application, we applied these methods to investigate the causal relationship between alcohol consumption and gout attacks using empirical data. <b>Results:</b> Our results revealed that across all scenarios, TSRI with the Poisson model produced the most stable estimates with lower bias and RMSE. TSRI achieved near-nominal coverage and narrower CI across varying IS and confounding levels, maintaining Type I error close to 0.05. IS significantly impacted performance, with IS = 0.5 yielding estimates closer to the true values, while weaker instruments (IS = 0.1) led to higher RMSE and bias. Increasing sample size in the presence of invalid and weak genetic variants increased the bias. In additional simulations with multiple weak instruments, TSRI continued to outperform TSPS, yielding lower bias or RMSE, narrow CI width and near-nominal coverage across sample sizes. In our application, alcohol consumption was causally associated with an estimated 11.6-12.7% increase in the expected number of gout attacks per year per unit increase in alcohol intake, although the presence of an invalid single nucleotide polymorphism likely biased this estimate. <b>Conclusion:</b> This study advances MR methodology by clarifying how TSPS and TSRI behave with count exposures and outcomes, providing practical guidance for valid instrument selection and reliable causal inference in MR studies involving count data.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250081"},"PeriodicalIF":2.5,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13044820/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147473946","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-effectiveness analysis of the use of faricimab in diabetic macular edema in China.","authors":"Fenghao Shi, Shuhua Tan, Yamin Shu, Tianyi Liu, Yan Xia, Christian Buehrer, Sheng Han","doi":"10.57264/cer-2025-0190","DOIUrl":"10.57264/cer-2025-0190","url":null,"abstract":"<p><p><b>Aim:</b> Diabetic macular edema (DME) is one of the leading causes of vision impairment in diabetic patients. The aim of this study was to evaluate the cost-effectiveness of faricimab (Vabysmo^®), the first bispecific antibody targeting VEGF-A and ANG-2, for treating DME in China. <b>Materials & methods:</b> We conducted a cost-effectiveness analysis from the perspective of the Chinese health system using a Markov model to simulate long-term outcomes in DME patients. The model population was based on the Chinese subgroup from the RHINE trial, with a mean starting age of 58.7 years. Faricimab, administered according to a personalized treatment interval regimen following four initial monthly injections, was compared with aflibercept (every 8 weeks) and with ranibizumab and conbercept (both following a pro-re nata dosing regimen). The model incorporated clinical efficacy, adverse events, quality-adjusted life years and direct costs. Extensive sensitivity analyses (one-way and probabilistic) were performed to assess the robustness of the findings. <b>Results:</b> Over 20 years, faricimab gained 0.42, 0.61, 0.80 incremental quality-adjusted life years compared with aflibercept, conbercept, ranibizumab, while reducing treatment costs by 33,315 Chinese Yuan (CNY), 59,061 CNY and 12,314 CNY, respectively. Sensitivity analyses confirmed the robustness of these results, consistently demonstrating faricimab as a dominant and cost-effective treatment option for DME. <b>Conclusion:</b> This first Chinese cost-effectiveness study of faricimab for DME shows it enhances visual outcomes and reduces costs, supporting its adoption and providing evidence for healthcare decision-making on reimbursement and resource allocation.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250190"},"PeriodicalIF":2.5,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13044818/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147473865","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-word evidence on healthcare resource use and associated costs in on-demand users of replacement therapies in von Willebrand disease in France: the FORvWARD study.","authors":"Benoit Polack, Virginie Nerich, Claire Marant Micallef, Marc Trossaërt, Christine Biron-Andreani, Nadège Bornier, Catherine Chatelanaz, Franck Favre-Besse, Julien Beoletto, Mélanie Née, Manon Belhassen, Cinira Lefèvre, Gérard de Pouvourville","doi":"10.57264/cer-2025-0128","DOIUrl":"10.57264/cer-2025-0128","url":null,"abstract":"<p><p><b>Background:</b> Real-world data about use of Von Willebrand factor (VWF) concentrates to manage on-demand patients with Von Willebrand disease (VWD) are scarce. <b>Aim:</b> To describe and compare patients' characteristics, treatment patterns, healthcare resource use and associated costs of patients with VWD using VWF concentrates. <b>Materials & methods:</b> Using the French healthcare claims database, we included adult patients with ≥1 reimbursement for a replacement therapy (RT) containing VWF concentrate between 1 January 2017 and 30 September 2021 and followed them from first RT dispensation to 31 December 2021. Treatment patterns, healthcare resource use and associated costs of RT on-demand users were evaluated over each 30-days exposure period (EP) starting the first day of each hospital stay with ≥1 RT administration. In- and out-hospital RT doses and FVIII, number of general practitioner and nurse visits, in- and out-hospital RT dispensings and length of hospitalizations and their costs were described and compared across RTs using adjusted Generalized Estimating Equation models accounting for confounding factors. <b>Results:</b> Among 2540 on-demand RT users, WILFACTIN^® was the main RT used, followed by VONCENTO^®, VEYVONDI^®, EQWILATE^® and WILSTART^®. Overall, the mean total RT dose was 12,962 IU and the mean cost was €21,034/EP. Compared with VEYVONDI^®-treated EP, WILFACTIN^®-treated EP had significantly longer stay duration, had more out-hospital RT dose and had higher overall and in-hospital costs; VONCENTO^®-treated EP had more overall and in-hospital RT dose, and had higher in-hospital and RT-related costs. <b>Conclusion:</b> This first real-world study suggests that VEYVONDI^® seems to be a cost-saving RT compared with other RT. Future studies including clinical data should provide further evidence.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250128"},"PeriodicalIF":2.5,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13044817/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147498302","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":"Performance of stenting in femoropopliteal disease: a systematic literature review and meta-analysis of proportions.","authors":"Yann Gouëffic, Antonia Bosworth Smith, Fabian Distler, Anya Lissina, Juliane Hafermann, Rhodri Saunders, Andrew Holden, Sabine Steiner","doi":"10.57264/cer-2025-0152","DOIUrl":"10.57264/cer-2025-0152","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate the performance of four stent types (BMS, bare metal stents; Eluvia, a polymer-based paclitaxel-eluting stent; Viabahn, a covered stent; and Zilver PTX, a polymer-free paclitaxel-coated stent) in femoropopliteal lesions at 12 and 24 months using a meta-analysis of proportions. <b>Materials & methods:</b> This systematic review (PROSPERO CRD42024528559) used PubMed to identify relevant single-arm and comparative studies (with ≥50 patients/study) published between 1 January 2009 and 1 July 2024. Data on patient/lesion characteristics and outcomes were extracted. Subgroup analyses were based on lesion length (<150 mm vs ≥150 mm) and study quality. A random-intercept logistic regression model was used to pool the data. The 95% CI around the pooled effect was calculated using Knapp-Hartung adjustments. <b>Results:</b> Data were extracted from 141 of the 870 screened studies, corresponding to 35,897 patients. The mean patient age was 70.9 (range: 63.3-80.0) years; 69.6% were male. The overall mean lesion length was 153.1 (range: 37-330) mm. Although all stent types performed well in the main analysis, Eluvia exhibited consistently high primary patency and low target lesion revascularization rates at both timepoints and across lesion lengths. Mortality rates for all stent types were stable for short lesions but more variable for long lesions. <b>Conclusion:</b> All stents used in short lesions performed well; however, Eluvia also demonstrated reliable performance in long lesions.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250152"},"PeriodicalIF":2.5,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13044811/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147390213","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}