Drugs - Real World Outcomes最新文献

{"title":"Treatment Patterns in Patients with Late-Onset Myasthenia Gravis in Japan: An Insurance Claims Database Study.","authors":"Akiyuki Uzawa, Makoto Samukawa, Shingo Konno, Izumi Mishiro, Kentaro Taki, Hiroshi Todaka, Céline Quelen, Katarzyna Jabłońska, Renata Majewska, Yohei Ohashi","doi":"10.1007/s40801-026-00552-y","DOIUrl":"10.1007/s40801-026-00552-y","url":null,"abstract":"<p><strong>Background and objective: </strong>This study evaluated treatment patterns and oral corticosteroid (OCS) use in Japanese patients with myasthenia gravis, comparing late-onset (mean age 79.8 years) and early/adult-onset (mean age 61.3 years) cohorts. It also explored treatment frequency rates.</p><p><strong>Methods: </strong>We conducted a retrospective analysis of the DeSC Healthcare claims database (2014-21). Adults with myasthenia gravis starting immunosuppressant therapy and with ≥ 2 years of follow-up were included. Patients were stratified by age group (< 75 and ≥ 75 years) and by exposure to early fast-acting treatment (EFT). Outcomes included OCS dose trajectories, time to achieve a maintenance dose of ≤ 5 mg/day, and the frequency of myasthenia gravis cases treated with immunotherapy.</p><p><strong>Results: </strong>Early fast-acting treatment use was lower in the late-onset cohort (31/148 patients; 20.9%) than in the early-onset cohort (42/180; 28.0%). Initial and maximum OCS doses and time spent > 20 mg/day tended to be lower in late-onset patients. The OCS target was achieved more often and sooner in late-onset patients (median 5.1 vs 14.9 months), particularly in those without EFT. The frequency of treated myasthenia gravis cases was higher in late-onset patients (7.09 vs 3.97 per 100,000).</p><p><strong>Conclusions: </strong>Late-onset patients were less likely to receive EFT, received lower OCS doses yet reached the OCS target earlier, which may reflect physician caution with OCS exposure and good response to treatments. The findings highlight timely identification of the need for EFT, safer OCS-sparing strategies, and clearer clinical guidance in patients with late-onset myasthenia gravis.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"225-239"},"PeriodicalIF":2.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13287361/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147728508","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":"Regional Trends in the Opioid Epidemic Using Safety Signals of Drug Abuse and Dependence: A Disproportionality Analysis Using VigiBase.","authors":"Bohyun Suh, Hyung Suk Oh, Ju-Young Shin, Ju Hwan Kim","doi":"10.1007/s40801-026-00550-0","DOIUrl":"10.1007/s40801-026-00550-0","url":null,"abstract":"<p><strong>Background: </strong>The opioid epidemic, once a major concern in the United States, has escalated worldwide through three waves driven by prescription and synthetic opioids like oxycodone and fentanyl. Regional patterns of misuse and dependence remain unclear due to data heterogeneity. To address this, we compared adverse events reporting for these opioids using VigiBase.</p><p><strong>Methods: </strong>We analyzed \"drug abuse and dependence\" adverse events using VigiBase, a global pharmacovigilance database containing more than 40 million Individual Case Safety Reports (ICSRs). A regional comparison of opioid-related adverse event reporting was performed, with oxycodone and fentanyl as the primary drugs and morphine and tramadol as comparators. Adverse events were defined by Standardized MedDRA Query (SMQ) criteria and cases were stratified by three periods (1990-2009, 2010-2012, 2013-2024) and regions (Americas, Western Pacific, Europe). Opioid-related death was additionally assessed as a secondary outcome. Disproportionality analysis was conducted to identify safety signals, using the reporting odds ratio (ROR). Signals were considered significant if ROR > 2, χ² > 4, and more than three reports were observed.</p><p><strong>Results: </strong>Oxycodone-related reports peaked in 2005 (n = 8395), 2011 (n = 10,444), and 2021 (n = 45,025), while fentanyl peaked in 2011 (n = 11,451) and 2017 (n = 32,704). The safety signals for oxycodone were found across all predefined periods in the Americas (1990-2009: ROR 4.40, n = 9102; 2010-2012: ROR 2.13, n = 2299; 2013-2024: ROR 5.20, n = 108,068), and in two periods in the Western Pacific (2010-2012: ROR 6.53, n = 81; 2013-2024: ROR 5.94, n = 1066). No signals were detected for oxycodone in Europe, and fentanyl did not meet signal criteria in any region or period in the primary analyses.</p><p><strong>Conclusions: </strong>Oxycodone showed consistent safety signals in the Americas and Western Pacific. The absence of signals for fentanyl and in Europe may reflect under-reporting, highlighting the need for improved surveillance of illicit opioid use.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"201-210"},"PeriodicalIF":2.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13287511/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147638369","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":"The Relevance of Lenvatinib in Advanced Hepatocellular Carcinoma: Insights from a Prospective Observational Cohort in South India.","authors":"Joe Jose, Shibu V Ignatious, Thattungal Manoharan Anoop","doi":"10.1007/s40801-026-00554-w","DOIUrl":"10.1007/s40801-026-00554-w","url":null,"abstract":"<p><strong>Background and objective: </strong>Lenvatinib is a multi-kinase inhibitor approved as a first-line therapy for advanced hepatocellular carcinoma. In India and other low- and middle-income countries, immune checkpoint inhibitor-based regimens are not universally accessible, and real-world data on lenvatinib are limited. We evaluated the effectiveness, safety, and predictors of outcomes of first-line lenvatinib in Indian patients with advanced hepatocellular carcinoma.</p><p><strong>Methods: </strong>This prospective observational cohort included consecutive patients with advanced hepatocellular carcinoma treated with first-line lenvatinib at the Regional Cancer Centre, Kerala, India, between March 2021 and May 2024. Lenvatinib was started at 8 or 12 mg once daily based on weight. Overall survival and progression-free survival were estimated using the Kaplan-Meier method. Cox regression analysis identified independent predictors of outcomes. Toxicities were graded using Common Terminology Criteria for Adverse Events Version 5.0.</p><p><strong>Results: </strong>A total of 112 patients were enrolled; median age was 62 years, and 88% were male. The predominant etiologies were non-viral (83%), mainly non-alcoholic steatohepatitis and alcohol-related liver disease. Most patients (79 %) had Barcelona Clinic Liver Cancer stage C disease, with nearly half showing macroscopic portal vein invasion. At a median follow-up of 30.8 months, median overall survival was 13.3 months (95% confidence interval [CI] 10.6-16.6) and median progression-free survival was 8.1 months (95% CI 6.5-9.8). Multivariate analysis showed improved overall survival with non-viral etiology (hazard ratio [HR] 0.49, 95% CI 0.28-0.86, p = 0.014), radiological response (HR 0.40, 95% CI 0.22-0.71, p = 0.002), and subsequent therapy (HR 0.36, 95% CI 0.22-0.60, p < 0.001), whereas portal vein invasion predicted worse overall survival (HR 1.67, 95% CI 1.06-2.61, p = 0.026). Grade ≥3 toxicities occurred in 38% of patients, mainly dermatologic, and no treatment-related deaths occurred.</p><p><strong>Conclusions: </strong>Lenvatinib demonstrated favorable survival outcomes and manageable toxicity in advanced hepatocellular carcinoma in our cohort, with superior effectiveness in non-viral disease. Given some evidence suggesting that immune checkpoint inhibitors may be less effective in non-viral hepatocellular carcinoma, along with the high costs and lack of accessibility, lenvatinib could still be a viable first-line option for non-viral hepatocellular carcinoma in resource-constrained settings. This is even more relevant with the rising trend of non-viral related hepatocellular carcinoma in low- and middle-income countries.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"253-264"},"PeriodicalIF":2.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13287363/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147728495","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 Evidence for Drug Value Assessment in China: Learning from International Experiences.","authors":"Abraham G Hartzema, Adel Gehchan, Jennifer D Guo, Jianwei Xuan","doi":"10.1007/s40801-026-00557-7","DOIUrl":"10.1007/s40801-026-00557-7","url":null,"abstract":"<p><p>In China, the national medical insurance system has undergone rapid development over the past two decades. The National Healthcare Security Administration was established to manage the growing healthcare expenditure. Since then, this administration has maintained the drug formulary, known as the National Reimbursement Drug List. The list comprises Category A (essential and cost-effective drugs) and Category B (effective drugs with relatively higher cost profiles), with the Commercial Insurance Innovative Drug list (referred to as Category C) added in 2025. China has made progress in adopting real-world evidence into healthcare decision making. Real-world evidence helps address key uncertainties related to safety, effectiveness, and long-term outcomes to complement clinical trial findings using real-world data from routine clinical practice. Evolving international experiences provide practical insights into the use of real-world evidence to support drug reimbursement and reassessment decisions. This paper proposes practical strategies supporting the integration of real-world evidence into China's drug value assessment to better inform national formulary management and reimbursement pathways.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"153-157"},"PeriodicalIF":2.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13287191/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148162145","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":"Comparison of Opioid Utilization in Older Adults with Rheumatoid Arthritis before and after Initiating Biological or Targeted Synthetic Disease-Modifying Antirheumatic Drugs.","authors":"Yinan Huang, Shadi Bazzazzadehgan, Liang-Yuan Lin, Omkar Ghodke, Sebastian Bruera, Kaustuv Bhattacharya, Sujith Ramachandran, John P Bentley, Sandeep K Agarwal, Yi Yang","doi":"10.1007/s40801-025-00537-3","DOIUrl":"10.1007/s40801-025-00537-3","url":null,"abstract":"<p><strong>Introduction: </strong>Opioid use is common in rheumatoid arthritis (RA) for pain management; however, evidence of opioid-associated adverse events is increasing. While biological (b) or targeted synthetic (ts) disease-modifying antirheumatic drugs (DMARDs) improve functional outcomes such as pain, little is known about their impact on opioid utilization patterns. This study investigated opioid utilization before and after b/tsDMARD initiation and assessed effect modification by sex.</p><p><strong>Methods: </strong>Using 5% Medicare claims data from 2012 to 2020, this cohort study included older adults (≥ 65 years) with RA who initiated b/tsDMARDs (first prescription = index date), and had continuous Medicare Parts A, B, and D, but not Part C enrollment, during 12 months before and after initiation. The outcomes of interest were any opioid use and long-term opioid therapy (LTOT). McNemar's test was performed to compare outcomes before and after b/tsDMARD initiation. Sex-based differences in changes in opioid use after b/tsDMARD initiation were also evaluated.</p><p><strong>Results: </strong>The study cohort included 3585 individuals with RA initiating b/tsDMARDs; most were female (75.87%) with a mean (SD) age of 73.15 (5.99) years. Following b/tsDMARD initiation, any opioid use decreased significantly from 2094 (58.41%) to 2017 (56.26%) (p = 0.015). However, LTOT use increased significantly from 733 (20.45%) to 900 (25.10%) (p < 0.001), following b/tsDMARD initiation. No evidence of sex differences in the association between b/tsDMARD initiation and opioid utilization was identified.</p><p><strong>Conclusions: </strong>Initiating b/tsDMARDs was associated with a modest reduction in any opioid use. However, LTOT use in RA remained persistently high. The impact of different b/tsDMARD initiation on opioid utilization patterns needs further investigation.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"51-61"},"PeriodicalIF":2.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13003035/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145818348","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 Experience with Selexipag in Patients with Pulmonary Arterial Hypertension: Treatment Patterns and Outcomes across Baseline Risk Strata (SPHERE and EXPOSURE Studies).","authors":"Pilar Escribano Subias, Murali M Chakinala, Kelly M Chin, Eva-Maria Didden, Gurinderpal Doad, Harrison W Farber, Sean Gaine, Anna R Hemnes, Kristin B Highland, Tobias J Lange, Vallerie McLaughlin, Audrey Muller, Mohammad Rahman, Tatiana Remeňová, Stefan Söderberg, Apoorva Venkatesh, Nick H Kim","doi":"10.1007/s40801-025-00536-4","DOIUrl":"10.1007/s40801-025-00536-4","url":null,"abstract":"<p><strong>Background: </strong>Treatment strategies for pulmonary arterial hypertension (PAH) depend on patients' 1-year mortality risk; however, real-world practices and outcomes remain inadequately described.</p><p><strong>Objective: </strong>We aimed to investigate real-world experience with selexipag, an oral selective prostacyclin I2 receptor agonist used to treat PAH.</p><p><strong>Methods: </strong>This analysis of two large, prospective, observational studies: SPHERE (NCT03278002; USA; completed) and EXPOSURE (EUPAS19085; Europe and Canada; ongoing, data cutoff November 2021) included adults newly initiating selexipag with follow-up available.</p><p><strong>Results: </strong>Of 1366 selexipag users, 894 met eligibility criteria. At selexipag initiation, the median (Q1-Q3) age was 61 (48-70) years and the time since diagnosis was 2.7 (0.8-7.4) years. Most patients initiated selexipag within triple oral combination therapy (67%) with WHO-FC III symptoms (61%). During a median 10.8 (Q1-Q3: 3.3-18.6) months of selexipag exposure, 388 (43%) patients discontinued selexipag, mostly as a result of tolerability (n = 173, 19%) and death (n = 69, 8%). The median individualised dose was 800 μg twice-daily (n = 742). One-year mortality risk at selexipag initiation (assessable for 591 patients using a four-strata method): 22% were low-risk, 40% intermediate-low, 29% intermediate-high, and 9% high-risk. From low to high risk: 86%, 73%, 57% and 45% were free from all-cause hospitalisation at 1 year, and 1-year survival was 100%, 96%, 91%, and 59%, respectively.</p><p><strong>Conclusions: </strong>Despite current guidelines, over half of patients started selexipag almost 3 years after diagnosis, and 38% were already intermediate-high or high risk of 1-year mortality. Lower hospitalisation rates and better survival were observed for selexipag-treated patients in the low and intermediate-low risk groups versus higher risk groups.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"85-100"},"PeriodicalIF":2.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13003052/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145951224","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":"Health Equity Concerns in People with Sickle Cell Disease and Recurrent Vaso-Occlusive Crises: Results from an International Survey Study.","authors":"Adriana Boateng-Kuffour, Lauren Lilly, Jennifer Drahos, Melanie Calvert, Ashley Valentine, Anthony Mason, Nanxin Li, Zahra Pakbaz, Farrukh Shah, Nick Ainsworth, Antony P Martin","doi":"10.1007/s40801-025-00531-9","DOIUrl":"10.1007/s40801-025-00531-9","url":null,"abstract":"<p><strong>Background: </strong>Vaso-occlusive crises (VOCs) are a hallmark of sickle cell disease (SCD). Individuals with SCD often report stigma and negative healthcare provider (HCP) attitudes when seeking treatment. This study examines health equity concerns and perceived barriers to care among adults with recurrent VOCs.</p><p><strong>Methods: </strong>A prospective survey was conducted from May to November 2022 in the US, UK, France, Germany, and Italy. Adults (≥ 18 years) with recurrent VOCs completed a health equity survey and patient-reported outcome measures (PROMs) at month 6. Participants were categorized as experiencing either unfair or fair treatment based on their response to whether they reported ever having been treated unfairly by an HCP due to their race or ethnicity. PROMs were scored, and analyses included Pearson's Chi-squared test and two-sample t-tests.</p><p><strong>Results: </strong>Among 110 participants, most were female (75.5%), Black/African American (93.5%), and US residents (59.1%). In the past year, 66.7% had ≥ 4 VOCs, and 85.3% used opioids. Most (68.6%) believed they would receive better care if they were of a different race/ethnicity, and 64.7% felt HCPs did not believe their symptoms. About 30% waited > 60 min for emergency department check-in, with additional delays before treatment. Key barriers included reported HCP lack of empathy (58.9%) and SCD knowledge (55.9%). Pain significantly impacted daily activities, with all outcomes worse in the Unfair treatment group.</p><p><strong>Conclusions: </strong>Findings highlight significant health equity concerns and barriers to care for adults with SCD and recurrent VOCs, underscoring unmet needs and the humanistic burden in this population.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"29-40"},"PeriodicalIF":2.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13003058/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145563022","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":"Time to Treatment Discontinuation and Cost Effectiveness of Third-Line Therapies in Advanced Colorectal Cancer: Real-World Evidence from the NIH All of Us Research Program.","authors":"Patrick J Kiel, Mark W McGiffin, Todd C Skaar, Michael A Preston","doi":"10.1007/s40801-026-00539-9","DOIUrl":"10.1007/s40801-026-00539-9","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Treatment options for patients with advanced colorectal cancer who progress after standard therapies remain limited, and real-world evidence on treatment durability and economic outcomes in later lines of therapy is needed to inform clinical and policy decision making. This study evaluated real-world outcomes, healthcare utilization, and cost effectiveness of immune checkpoint inhibitors (ICI) compared with regorafenib or trifluridine/tipiracil (Rego/Tri) in third-line (3L) advanced colorectal cancer (CRC), leveraging the nationally distributed NIH All of Us Research Program.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We conducted a retrospective cohort cost-utility analysis using All of Us Version 8 Curated Data Repository. Eligible adults (≥ 18 years) with advanced CRC initiated ICI (pembrolizumab, nivolumab ± ipilimumab) or Rego/Tri during 2017-2023. Outcomes included time to treatment discontinuation (TTD), healthcare utilization, costs, and quality-adjusted life years (QALYs; base-case utility = 0.75). Costs were estimated from CMS 2023 schedules (Part B ASP for infused agents, Part D unit costs for orals; inpatient/outpatient fee schedules for non-drug services), applied per cycle and proportionally for partial cycles. Analyses were conducted from the US Medicare payer perspective, with the time horizon limited to observed TTD and no discounting applied (&lt; 1 year). Incremental cost-effectiveness ratios (ICERs), incremental net monetary benefit (INMB), and cost-effectiveness acceptability curves (CEACs) were estimated using nonparametric bootstrapping (1000 replicates). Sensitivity analyses varied utility weights (0.6-1.0).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Among 82 patients (ICI n = 50; Rego/Tri n = 32), median TTD was significantly longer with ICI (8.0 vs 1.4 months; HR 0.49, 95% CI 0.30-0.79; p = 0.0037). Non-drug utilization costs were not significantly different, but ICI drug costs were higher (US$207,558 vs US$119,805; p = 0.001). Mean total costs were US$476,729 for ICI versus US$264,225 for Rego/Tri (p = 0.018). QALYs were higher for ICI (0.84 vs 0.43; p &lt; 0.001). The ICER was US$518,000 per QALY gained. CEAC analysis demonstrated ~ 0% probability of cost effectiveness at US$150,000/QALY, ~ 50% at US$500,000/QALY, and ~ 80% at ≥ US$1,000,000/QALY. Sensitivity analyses yielded consistent findings.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;In this exploratory analysis using the novel NIH All of Us Research Program, ICI therapy was associated with longer treatment duration and greater QALYs but substantially higher costs compared with Rego/Tri. ICI was unlikely to be cost effective at conventional US thresholds but may be favorable at higher willingness-to-pay values. These findings highlight both the promise of leveraging the All of Us Research Program for nationally distributed, demographically diverse pharmacoeconomic research and the challenges of interpreting real-world cost effectiveness in small cohorts. As the datas","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"121-131"},"PeriodicalIF":2.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13003027/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147431483","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":"Effectiveness and Safety of the Novel Selective Urate Reabsorption Inhibitor Dotinurad After Switching from Febuxostat in Patients with Stage B/C Heart Failure.","authors":"Tsutomu Murakami, Yuki Watanabe, Norihito Nakamura, Shigemitsu Tanaka, Yuji Ikari","doi":"10.1007/s40801-025-00526-6","DOIUrl":"10.1007/s40801-025-00526-6","url":null,"abstract":"<p><strong>Background: </strong>Hyperuricemia is a common comorbidity in patients with cardiovascular diseases and chronic kidney disease, often requiring long-term urate-lowering therapy. Febuxostat, a xanthine oxidase inhibitor, has raised cardiovascular safety concerns. Dotinurad, a selective urate reabsorption inhibitor, has emerged as a potential alternative, but clinical evidence in patients with Stage B/C heart failure remains limited.</p><p><strong>Methods: </strong>This single-center retrospective study evaluated 30 patients with Stage B (n = 10) and C (n = 20) heart failure and hyperuricemia who were switched from febuxostat (10, 20, or 40 mg) to dotinurad (0.5, 1.0, or 2.0 mg, respectively), based on the prior febuxostat dose. Laboratory and urinary parameters were assessed at baseline and at follow-up (median 65 days [56-84] after switching).</p><p><strong>Results: </strong>In Stage B heart failure, serum uric acid showed a non-significant trend toward reduction (5.8 [4.4-6.3] to 5.3 [4.8-7.5] mg/dL, p = 0.09), whereas in Stage C heart failure, serum uric acid increased significantly (5.1 [4.6-6.6] to 5.4 [4.8-6.8] mg/dL, p = 0.02). The proportion of patients achieving serum uric acid ≤ 6.0 mg/dL was maintained (Stage B: 60.0 to 50.0%; Stage C: 70.0 to 75.0%). Urinary uric acid excretion increased, while urinary pH remained stable in both groups. The uricosuric effect of dotinurad was evident with or without concomitant use of sodium-glucose cotransporter 2 inhibitors. No adverse events, including cardiovascular events, urolithiasis, or gout flares, were observed.</p><p><strong>Conclusions: </strong>Switching from febuxostat to dotinurad may be effective and safe over the short term in patients with Stage B/C heart failure and hyperuricemia.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"1-6"},"PeriodicalIF":2.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13003072/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145631262","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":"Use of Potentially Inappropriate Medication and Association with Falls During Hospitalisation: An Analysis Based on Electronic Health Records (POLAR_MI project).","authors":"Louisa Redeker, Miriam Kesselmeier, Beate Mussawy, Steffen Grabe, Marietta Rottenkolber, Torsten Thalheim, Florian Schmidt, Thomas Peschel, Alexander Strübing, Daniel Neumann, André Scherag, Markus Loeffler, Sven Schmiedl, Petra Thürmann","doi":"10.1007/s40801-025-00528-4","DOIUrl":"10.1007/s40801-025-00528-4","url":null,"abstract":"<p><strong>Background: </strong>In an ageing population, drug-related problems, including potentially inappropriate medications (PIMs), are increasingly relevant. PIM lists are important tools for improving medication safety in older adults.</p><p><strong>Objective: </strong>As part of the POLAR_MI (POLypharmacy, drug interActions, Risks) project, this study aimed to assess the prevalence of PIM and its association with falls in patients aged ≥ 65 years, based on the methods and processes of the Medical Informatics Initiative Germany.</p><p><strong>Methods: </strong>A retrospective, distributed analysis of electronic health records (EHRs) (2018-2021) was conducted at ten German university hospitals. PIMs were defined using PRISCUS and EU(7)-PIM lists. Falls were identified using documented fractures as a surrogate. Multivariable logistic regression modelling was applied, adjusting for confounders including age, gender, Charlson Comorbidity Index (CCI) and fall-risk-increasing drugs (FRIDs).</p><p><strong>Results: </strong>A total of 166,126 cases (median age: 76 years; 45.8% women) were analysed. According to PRISCUS, 12.8% of cases had at least one PIM, with amitriptyline most common (1.1% of all cases). According to EU(7)-PIM, 45.6% of cases were affected, with apixaban most frequent (8.6%). An association between falls and at least one PIM (PRISCUS: adjusted odds ratio [aOR] 1.21, 95% confidence interval [CI] 0.94-1.55; EU(7)-PIM: aOR 1.01, 95% CI 0.83-1.22) could not be observed. However, FRIDs were associated with falls (PRISCUS: aOR 2.31, 95% CI 1.64-3.27; EU(7)-PIM: aOR 2.43, 95% CI 1.69-3.48).</p><p><strong>Conclusions: </strong>In this large multicentre EHR analysis, PIMs were common in older patients, but an association with an increased likelihood of falls could not be observed. In contrast, the well-known association with FRIDs was confirmed. The ongoing digitalisation of German hospitals enables such large-scale data analyses for risk assessment and supports improvements in patient safety.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"15-27"},"PeriodicalIF":2.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13003043/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145548703","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}