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Search Results (810)

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Keywords = real-world evidence studies

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15 pages, 6966 KiB  
Article
A Concise Grid-Based Model Revealing the Temporal Dynamics in Indoor Infection Risk
by Pengcheng Zhao and Xiaohong Zheng
Buildings 2025, 15(15), 2786; https://doi.org/10.3390/buildings15152786 - 6 Aug 2025
Abstract
Determining the transmission routes of pathogens in indoor environments is challenging, with most studies limited to specific case analyses and pilot experiments. When pathogens are instantaneously released by a patient in an indoor environment, the peak infection risk may not occur immediately but [...] Read more.
Determining the transmission routes of pathogens in indoor environments is challenging, with most studies limited to specific case analyses and pilot experiments. When pathogens are instantaneously released by a patient in an indoor environment, the peak infection risk may not occur immediately but may instead appear at a specific moment during the pathogen’s spread. We developed a concise model to describe the temporal crest of infection risk. The model incorporates the transmission and degradation characteristics of aerosols and surface particles to predict infection risks via air and surface routes. Only four real-world outbreaks met the criteria for validating this phenomenon. Based on the available data, norovirus is likely to transmit primarily via surface touch (i.e., the fomite route). In contrast, crests of infection risk were not observed in outbreaks of respiratory diseases (e.g., SARS-CoV-2), suggesting a minimal probability of surface transmission in such cases. The new model can serve as a preliminary indicator for identifying different indoor pathogen transmission routes (e.g., food, air, or fomite). Further analyses of pathogens’ transmission routes require additional evidence. Full article
(This article belongs to the Special Issue Development of Indoor Environment Comfort)
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16 pages, 300 KiB  
Review
SGLT2 Inhibitors and GLP-1 Receptor Agonists in PAD: A State-of-the-Art Review
by Alfredo Caturano, Damiano D’Ardes, Paola Giustina Simeone, Gianfranco Lessiani, Nicoletta Di Gregorio, Lorenzo Andreetto, Davide Grassi, Carla Serra, Francesca Santilli, Maria Teresa Guagnano, Fabio Piscaglia, Claudio Ferri, Francesco Cipollone and Andrea Boccatonda
J. Clin. Med. 2025, 14(15), 5549; https://doi.org/10.3390/jcm14155549 - 6 Aug 2025
Abstract
Sodium–glucose co-transporter-2 inhibitors (SGLT2is) and GLP-1 receptor agonists (GLP-1 RAs) are now established as cornerstone therapies for patients with type 2 diabetes mellitus (T2DM), given their cardiovascular and renal protective properties. However, their use in patients with peripheral artery disease (PAD) remains controversial [...] Read more.
Sodium–glucose co-transporter-2 inhibitors (SGLT2is) and GLP-1 receptor agonists (GLP-1 RAs) are now established as cornerstone therapies for patients with type 2 diabetes mellitus (T2DM), given their cardiovascular and renal protective properties. However, their use in patients with peripheral artery disease (PAD) remains controversial due to concerns raised in early trials about potential increases in lower limb complications, particularly amputations. This narrative review examines current evidence on the association between SGLT2is and GLP-1 RAs in PAD-related outcomes, including limb events, amputation risk, and cardiovascular and renal endpoints. Drawing from randomized controlled trials, real-world cohort studies, and systematic reviews, we provide an integrated perspective on the safety and utility of SGLT2is and GLP-1 RAs in individuals with PAD, highlight patient selection considerations, and identify areas for future investigation. Full article
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10 pages, 235 KiB  
Article
Efficacy and Safety of Anti-Respiratory Syncytial Virus Monoclonal Antibody Nirsevimab in Neonates: A Real-World Monocentric Study
by Maria Costantino, Mariagrazia Bathilde Marongiu, Maria Grazia Corbo, Anna Maria Della Corte, Anna Rita Frascogna, Angela Plantulli, Federica Campana, Luigi Fortino, Emanuela Santoro, Emilia Anna Vozzella, Walter Longanella, Giovanni Boccia, Amelia Filippelli and Francesco De Caro
Vaccines 2025, 13(8), 838; https://doi.org/10.3390/vaccines13080838 (registering DOI) - 6 Aug 2025
Abstract
Background: RSV remains a leading cause of infant hospitalization worldwide, and the recently approved nirsevimab could represent an effective and safe prophylactic strategy to prevent severe infections in the general neonatal population. Objectives: We conducted a retrospective observational monocentric pilot study in a [...] Read more.
Background: RSV remains a leading cause of infant hospitalization worldwide, and the recently approved nirsevimab could represent an effective and safe prophylactic strategy to prevent severe infections in the general neonatal population. Objectives: We conducted a retrospective observational monocentric pilot study in a mixed preterm/term birth cohort to add real-world evidence of the efficacy and safety of nirsevimab in preventing severe RSV infection. Methods: We included a total of 2035 consecutive infants admitted to the Neonatal Unit, University Hospital “San Giovanni di Dio e Ruggi d’Aragona”, Salerno, Italy, from November 2024 to April 2025. We evaluated 30-day safety profiles and season-wide RSV infection rates, and the outcomes were also compared to newborns’ birth rate in the two previous seasons (2022–2023 and 2023–2024). Results: After the introduction of nirsevimab, a lower RSV infection rate was reported compared to previous seasons, and no adverse effects were observed. Compared to previous seasons, the clinical outcomes were more favorable, as only one unvaccinated neonate with RSV infection required invasive ventilation. Conclusions: In this real-world analysis, we demonstrated a good short-term safety profile of nirsevimab, as well as a potentially high efficacy in the general neonatal population with lower RSV infection incidence. However, future studies are needed to better assess its long-term safety and season-wide efficacy. Full article
(This article belongs to the Collection Research on Monoclonal Antibodies and Antibody Engineering)
16 pages, 3766 KiB  
Article
Evaluation of Energy and CO2 Reduction Through Envelope Retrofitting: A Case Study of a Public Building in South Korea Conducted Using Utility Billing Data
by Hansol Lee and Gyeong-Seok Choi
Energies 2025, 18(15), 4129; https://doi.org/10.3390/en18154129 - 4 Aug 2025
Abstract
This study empirically evaluates the energy and carbon reduction effects of an envelope retrofit applied to an aging public building in South Korea. Unlike previous studies that primarily relied on simulation-based analyses, this work fills the empirical research gap by using actual utility [...] Read more.
This study empirically evaluates the energy and carbon reduction effects of an envelope retrofit applied to an aging public building in South Korea. Unlike previous studies that primarily relied on simulation-based analyses, this work fills the empirical research gap by using actual utility billing data collected over one pre-retrofit year (2019) and two post-retrofit years (2023–2024). The retrofit included improvements to exterior walls, roofs, and windows, aiming to enhance thermal insulation and airtightness. The analysis revealed that monthly electricity consumption was reduced by 14.7% in 2023 and 8.0% in 2024 compared to that in the baseline year, with corresponding decreases in electricity costs and carbon dioxide emissions. Seasonal variations were evident: energy savings were significant in the winter due to reduced heating demand, while cooling energy use slightly increased in the summer, likely due to diminished solar heat gains resulting from improved insulation. By addressing both heating and cooling impacts, this study offers practical insights into the trade-offs of envelope retrofitting. The findings contribute to the body of knowledge by demonstrating the real-world performance of retrofit technologies and providing data-driven evidence that can inform policies and strategies for improving energy efficiency in public buildings. Full article
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25 pages, 861 KiB  
Article
Designing a Board Game to Expand Knowledge About Parental Involvement in Teacher Education
by Zsófia Kocsis, Zsolt Csák, Dániel Bodnár and Gabriella Pusztai
Educ. Sci. 2025, 15(8), 986; https://doi.org/10.3390/educsci15080986 (registering DOI) - 2 Aug 2025
Viewed by 340
Abstract
Research highlights a growing demand for active, experiential learning methods in higher education, especially in teacher education. While the benefits of parental involvement (PI) are well-documented, Hungary lacks tools to effectively prepare teacher trainees for fostering family–school cooperation. This study addresses this gap [...] Read more.
Research highlights a growing demand for active, experiential learning methods in higher education, especially in teacher education. While the benefits of parental involvement (PI) are well-documented, Hungary lacks tools to effectively prepare teacher trainees for fostering family–school cooperation. This study addresses this gap by introducing a custom-designed board game as an innovative teaching tool. The game simulates real-world challenges in PI through a cooperative, scenario-based framework. Exercises are grounded in international and national research, ensuring their relevance and evidence-based design. Tested with 110 students, the game’s educational value was assessed via post-gameplay questionnaires. Participants emphasized the strengths of its cooperative structure, realistic scenarios, and integration of humor. Many reported gaining new insights into parental roles and strategies for effective home–school partnerships. Practical applications include integrating the game into teacher education curricula and adapting it for other educational contexts. This study demonstrates how board games can bridge theory and practice, offering an engaging, effective medium to prepare future teachers for the challenges of PI. Full article
(This article belongs to the Section Teacher Education)
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62 pages, 4641 KiB  
Review
Pharmacist-Driven Chondroprotection in Osteoarthritis: A Multifaceted Approach Using Patient Education, Information Visualization, and Lifestyle Integration
by Eloy del Río
Pharmacy 2025, 13(4), 106; https://doi.org/10.3390/pharmacy13040106 - 1 Aug 2025
Viewed by 151
Abstract
Osteoarthritis (OA) remains a major contributor to pain and disability; however, the current management is largely reactive, focusing on symptoms rather than preventing irreversible cartilage loss. This review first examines the mechanistic foundations for pharmacological chondroprotection—illustrating how conventional agents, such as glucosamine sulfate [...] Read more.
Osteoarthritis (OA) remains a major contributor to pain and disability; however, the current management is largely reactive, focusing on symptoms rather than preventing irreversible cartilage loss. This review first examines the mechanistic foundations for pharmacological chondroprotection—illustrating how conventional agents, such as glucosamine sulfate and chondroitin sulfate, can potentially restore extracellular matrix (ECM) components, may attenuate catabolic enzyme activity, and might enhance joint lubrication—and explores the delivery challenges posed by avascular cartilage and synovial diffusion barriers. Subsequently, a practical “What–How–When” framework is introduced to guide community pharmacists in risk screening, DMOAD selection, chronotherapeutic dosing, safety monitoring, and lifestyle integration, as exemplified by the CHONDROMOVING infographic brochure designed for diverse health literacy levels. Building on these strategies, the P4–4P Chondroprotection Framework is proposed, integrating predictive risk profiling (physicians), preventive pharmacokinetic and chronotherapy optimization (pharmacists), personalized biomechanical interventions (physiotherapists), and participatory self-management (patients) into a unified, feedback-driven OA care model. To translate this framework into routine practice, I recommend the development of DMOAD-specific clinical guidelines, incorporation of chondroprotective chronotherapy and interprofessional collaboration into health-professional curricula, and establishment of multidisciplinary OA management pathways—supported by appropriate reimbursement structures, to support preventive, team-based management, and prioritization of large-scale randomized trials and real-world evidence studies to validate the long-term structural, functional, and quality of life benefits of synchronized DMOAD and exercise-timed interventions. This comprehensive, precision-driven paradigm aims to shift OA care from reactive palliation to true disease modification, preserving cartilage integrity and improving the quality of life for millions worldwide. Full article
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12 pages, 855 KiB  
Article
Application of Integrative Medicine in Plastic Surgery: A Real-World Data Study
by David Lysander Freytag, Anja Thronicke, Jacqueline Bastiaanse, Ioannis-Fivos Megas, David Breidung, Ibrahim Güler, Harald Matthes, Sophia Johnson, Friedemann Schad and Gerrit Grieb
Medicina 2025, 61(8), 1405; https://doi.org/10.3390/medicina61081405 - 1 Aug 2025
Viewed by 146
Abstract
Background and Objectives: There is a global rise of public interest in integrative medicine. The principles of integrative medicine combining conventional medicine with evidence-based complementary therapies have been implemented in many medical areas, including plastic surgery, to improve patient’s outcome. The aim [...] Read more.
Background and Objectives: There is a global rise of public interest in integrative medicine. The principles of integrative medicine combining conventional medicine with evidence-based complementary therapies have been implemented in many medical areas, including plastic surgery, to improve patient’s outcome. The aim of the present study was to systematically analyze the application and use of additional non-pharmacological interventions (NPIs) of patients of a German department of plastic surgery. Materials and Methods: The present real-world data study utilized data from the Network Oncology registry between 2016 and 2021. Patients included in this study were at the age of 18 or above, stayed at the department of plastic surgery and received at least one plastic surgical procedure. Adjusted multivariable logistic regression analyses were performed to detect associations between the acceptance of NPIs and predicting factors such as age, gender, year of admission, or length of hospital stay. Results: In total, 265 patients were enrolled in the study between January 2016 and December 2021 with a median age of 65 years (IQR: 52–80) and a male/female ratio of 0.77. Most of the patients received reconstructive surgery (90.19%), followed by hand surgery (5.68%) and aesthetic surgery (2.64%). In total, 42.5% of the enrolled patients accepted and applied NPIs. Physiotherapy, rhythmical embrocations, and compresses were the most often administered NPIs. Conclusions: This exploratory analysis provides a descriptive overview of the application and acceptance of NPIs in plastic surgery patients within a German integrative care setting. While NPIs appear to be well accepted by a subset of patients, further prospective studies are needed to evaluate their impact on clinical outcomes such as postoperative recovery, pain management, patient-reported quality of life, and overall satisfaction with care. Full article
(This article belongs to the Section Surgery)
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26 pages, 315 KiB  
Article
Development of a Multicultural Leadership Promotion Program for Youth in Thailand’s Three Southern Border Provinces
by Kasetchai Laeheem, Punya Tepsing and Khaled Hayisa-e
Youth 2025, 5(3), 82; https://doi.org/10.3390/youth5030082 - 1 Aug 2025
Viewed by 117
Abstract
Thailand’s southern border provinces need youth-focused multicultural leadership programs integrating local religious–cultural elements, community involvement, and long-term evaluation to enhance social cohesion and sustainable development. This study aimed to develop and evaluate a program to foster multicultural leadership among youth in Thailand’s three [...] Read more.
Thailand’s southern border provinces need youth-focused multicultural leadership programs integrating local religious–cultural elements, community involvement, and long-term evaluation to enhance social cohesion and sustainable development. This study aimed to develop and evaluate a program to foster multicultural leadership among youth in Thailand’s three southern border provinces. The research was conducted in two phases. The first phase involved synthesizing key multicultural leadership characteristics, designing a structured program and assessing its relevance and coherence through expert evaluation. The second phase focused on empirical validation by implementing the program with 22 selected youth participants, employing repeated-measures analysis of variance to assess its effectiveness. Additionally, experts evaluated the program’s validity, appropriateness, cost-effectiveness, utility, and feasibility. The resulting program, “EARCA”, comprises five core components: Experiential Exposure, Active Exploration & Engagement, Reflective Thinking & Analysis, Concept Integration & Synthesis, and Application & Extension. Expert assessments confirmed its appropriateness at the highest level, with a consistency index ranging from 0.8 to 1.0. Statistical analyses demonstrated significant improvements in all dimensions of multicultural leadership among participants. Furthermore, the program was rated highly accurate, appropriate, cost-effective, practical, and feasible for real-world implementation. These findings offer valuable insights for policymakers and practitioners seeking to enhance multicultural leadership development through structured, evidence-based interventions. Full article
10 pages, 710 KiB  
Article
CPAP Use and Retinal Disease Risk in Obstructive Apnea: A Cohort Study
by Dillan Cunha Amaral, Pedro Lucas Machado Magalhães, Muhammad Alfatih, Bruna Gabriel Miranda, Hashem Abu Serhan, Raíza Jacometti, Bruno Fortaleza de Aquino Ferreira, Letícia Sant’Ana, Diogo Haddad Santos, Mário Luiz Ribeiro Monteiro and Ricardo Noguera Louzada
Vision 2025, 9(3), 65; https://doi.org/10.3390/vision9030065 - 1 Aug 2025
Viewed by 148
Abstract
Obstructive sleep apnea (OSA) is a common condition associated with intermittent hypoxia, systemic inflammation, and vascular dysfunction; mechanisms implicated in retinal disease pathogenesis. This real-world retrospective cohort study used data from the TriNetX Research Network to assess whether continuous positive airway pressure (CPAP) [...] Read more.
Obstructive sleep apnea (OSA) is a common condition associated with intermittent hypoxia, systemic inflammation, and vascular dysfunction; mechanisms implicated in retinal disease pathogenesis. This real-world retrospective cohort study used data from the TriNetX Research Network to assess whether continuous positive airway pressure (CPAP) therapy reduces retinal disease incidence among adults with OSA and BMI between 25.0 and 30.0 kg/m2. After 1:1 propensity score matching, 101,754 patients were included in the analysis. Retinal outcomes included diabetic retinopathy (DR), age-related macular degeneration (AMD), retinal vein occlusion (RVO), and central serous chorioretinopathy (CSC). CPAP use was associated with a modest but statistically significant reduction in DR (3.2% vs. 3.4%, RR: 0.922, p = 0.016) and AMD (2.1% vs. 2.3%, RR: 0.906, p = 0.018), while no significant differences were found for RVO or CSC. These findings support prior evidence linking CPAP to improved retinal microvascular health and suggest a protective effect against specific retinal complications. Limitations include a lack of data on CPAP adherence, OSA severity, and imaging confirmation. Still, this study highlights the importance of interdisciplinary care between sleep and eye health, and the need for further prospective studies to validate CPAP’s role in preventing retinal disease progression in OSA patients. Full article
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20 pages, 586 KiB  
Article
Implementing High-Intensity Gait Training in Stroke Rehabilitation: A Real-World Pragmatic Approach
by Jennifer L. Moore, Pia Krøll, Håvard Hansen Berg, Merethe B. Sinnes, Roger Arntsen, Chris E. Henderson, T. George Hornby, Stein Arne Rimehaug, Ingvild Lilleheie and Anders Orpana
J. Clin. Med. 2025, 14(15), 5409; https://doi.org/10.3390/jcm14155409 - 31 Jul 2025
Viewed by 281
Abstract
Background: High-intensity gait training (HIT) is an evidence-based intervention recommended for stroke rehabilitation; however, its implementation in routine practice is inconsistent. This study examined the real-world implementation of HIT in an inpatient rehabilitation setting in Norway, focusing on fidelity, barriers, and knowledge [...] Read more.
Background: High-intensity gait training (HIT) is an evidence-based intervention recommended for stroke rehabilitation; however, its implementation in routine practice is inconsistent. This study examined the real-world implementation of HIT in an inpatient rehabilitation setting in Norway, focusing on fidelity, barriers, and knowledge translation (KT) strategies. Methods: Using the Knowledge-to-Action (KTA) framework, HIT was implemented in three phases: pre-implementation, implementation, and competency. Fidelity metrics and coverage were assessed in 99 participants post-stroke. Barriers and facilitators were documented and categorized using the Consolidated Framework for Implementation Research. Results: HIT was delivered with improved fidelity during the implementation and competency phases, reflected by increased stepping and heart rate metrics. A coverage rate of 52% was achieved. Barriers evolved over time, beginning with logistical and knowledge challenges and shifting toward decision-making complexity. The KT interventions, developed collaboratively by clinicians and external facilitators, supported implementation. Conclusions: Structured pre-implementation planning, clinician engagement, and external facilitation enabled high-fidelity HIT implementation in a real-world setting. Pragmatic, context-sensitive strategies were critical to overcoming evolving barriers. Future research should examine scalable, adaptive KT strategies that balance theoretical guidance with clinical feasibility to sustain evidence-based practice in rehabilitation. Full article
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11 pages, 608 KiB  
Article
Both Alcoholic and Non-Alcoholic Liver Cirrhosis Are Associated with an Increased Risk of HF—A Cohort Study Including 75,558 Patients
by Karel Kostev, Jamschid Sedighi, Samuel Sossalla, Marcel Konrad and Mark Luedde
J. Cardiovasc. Dev. Dis. 2025, 12(8), 295; https://doi.org/10.3390/jcdd12080295 - 31 Jul 2025
Viewed by 172
Abstract
The objective of the present study was to evaluate the association between liver cirrhosis (LC) and subsequent Heart failure (HF). This retrospective cohort study utilized data from the Disease Analyzer database (IQVIA) and included adults with a first-time diagnosis of LC in 1293 [...] Read more.
The objective of the present study was to evaluate the association between liver cirrhosis (LC) and subsequent Heart failure (HF). This retrospective cohort study utilized data from the Disease Analyzer database (IQVIA) and included adults with a first-time diagnosis of LC in 1293 general practices in Germany between January 2005 and December 2023. A comparison cohort without liver diseases was matched to the cirrhosis group using 5:1 propensity score matching. Univariable Cox proportional hazards models were used to assess the association between alcoholic vs. non-alcoholic LC and HF. The final study cohort included 5530 patients with alcoholic LC and 27,650 matched patients without liver disease, as well as 7063 patients with non-alcoholic LC and 35,315 matched patients without liver disease. After up to 10 years of follow-up, HF was diagnosed in 20.9% of patients with alcoholic LC compared to 10.3% of matched cohort, and in 23.0% of patients with non-alcoholic LC, compared to 14.2% in matched cohort. Alcoholic LC (Hazard Ratio (HR): 2.07 (95% CI: 1.85–2.31) and non-alcoholic LC (HR: 1.70; 95% CI: 1.56–1.82) were associated with an increased risk of HF. The association was also stronger in men than in women. LC, both alcoholic and non-alcoholic, is significantly associated with an increased long-term risk of HF. The association is particularly pronounced in patients with alcoholic cirrhosis and in men. To the best of the authors’ knowledge, this is the first real-world evidence for the positive association between LC and subsequent HF from Europe. Full article
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13 pages, 762 KiB  
Article
Implementation of Medical Therapy in Different Stages of Heart Failure with Reduced Ejection Fraction: An Analysis of the VIENNA-HF Registry
by Noel G. Panagiotides, Annika Weidenhammer, Suriya Prausmüller, Marc Stadler, Georg Spinka, Gregor Heitzinger, Henrike Arfsten, Guido Strunk, Philipp E. Bartko, Georg Goliasch, Christian Hengstenberg, Martin Hülsmann and Noemi Pavo
Biomedicines 2025, 13(8), 1846; https://doi.org/10.3390/biomedicines13081846 - 30 Jul 2025
Viewed by 402
Abstract
Background/Objectives: Real-world evidence shows alarmingly suboptimal utilization of guideline directed medical therapy (GDMT) in heart failure with reduced ejection fraction (HFrEF). One of the barriers of GDMT implementation appears to be concerns about the potential development of drug-related adverse events (AEs), particularly in [...] Read more.
Background/Objectives: Real-world evidence shows alarmingly suboptimal utilization of guideline directed medical therapy (GDMT) in heart failure with reduced ejection fraction (HFrEF). One of the barriers of GDMT implementation appears to be concerns about the potential development of drug-related adverse events (AEs), particularly in high-risk patients. This study aimed to evaluate whether advanced HFrEF (AHF) patients can be up-titrated safely and whether AHF predisposes individuals to the occurrence of putatively drug-related AEs. Methods: A total of 373 HFrEF patients with documented baseline, 2 months, and 12 months visits were analyzed for utilization and target dosages (TDs) of HF drugs. Successful up-titration and AEs were evaluated for different stages of HF reflected by N-terminal pro-B type natriuretic peptide (NT-proBNP) (<1000 pg/mL, 1000–2000 pg/mL, >2000 pg/mL). Results: A stepwise increase in HF medications was observed for all drug classes during follow-up. At 12 months, 73%, 75%, 62%, 86%, and 45% of patients received ≥90% of TDs of beta-blockers (BBs), renin–angiotensin system inhibitors (RASis), mineralocorticoid receptor antagonists (MRAs), sodium–glucose cotransporter-2 inhibitors (SGLT2 i), and triple-therapy, respectively. Predictors of successful up-titration in logistic regression were baseline HF drug TDs, estimated glomerular filtration rate (eGFR), and potassium, but not NT-proBNP or age. The development of AEs was rare, with hyperkalemia as the most common event (34% at 12 months). AEs were comparable in all stages of HF. However, the development of hyperkalemia was more frequent in patients with higher NT-proBNP and also accounted for most cases of incomplete up-titration. Conclusions: This study suggests that with dedicated protocols and frequent visits, GDMT can be successfully implemented across all stages of HFrEF, including patients with AHF. Full article
(This article belongs to the Special Issue Advanced Research on Heart Failure and Heart Transplantation)
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16 pages, 1138 KiB  
Review
Cardiac Myosin Inhibitors in the Treatment of Hypertrophic Cardiomyopathy: Clinical Trials and Future Challenges
by Arnold Kukowka and Marek Droździk
Biomolecules 2025, 15(8), 1098; https://doi.org/10.3390/biom15081098 - 29 Jul 2025
Viewed by 343
Abstract
Hypertrophic cardiomyopathy (HCM) is a prevalent and often underdiagnosed genetic cardiac disorder characterized by left ventricular hypertrophy and, in many cases, dynamic left ventricular outflow tract obstruction (LVOTO). The development of cardiac myosin inhibitors (CMIs) represents an emerging therapeutic approach in the pharmacological [...] Read more.
Hypertrophic cardiomyopathy (HCM) is a prevalent and often underdiagnosed genetic cardiac disorder characterized by left ventricular hypertrophy and, in many cases, dynamic left ventricular outflow tract obstruction (LVOTO). The development of cardiac myosin inhibitors (CMIs) represents an emerging therapeutic approach in the pharmacological management of obstructive HCM (oHCM). This review offers an integrated and up-to-date synthesis of the cardiac myosin inhibitor class, with a focus on mavacamten, aficamten, and the broader landscape of emerging agents. It also highlights recent clinical trial outcomes, pharmacokinetic and pharmacogenetic considerations, and potential future directions in therapy. Furthermore, we incorporate the most recent data up to May 2025, including late-breaking trial results and real-world safety findings, aiming to provide clinicians with a practical and comprehensive perspective on this evolving drug class. A narrative review was conducted by systematically searching PubMed, Scopus, Google Scholar, and ClinicalTrials.gov for English-language articles and trials published between January 2016 and May 2025. Keywords included “cardiac myosin inhibitor”, mavacamten”, “aficamten”, “MYK-224”, and “hypertrophic cardiomyopathy.” Inclusion criteria encompassed clinical trials and comprehensive reviews specifically addressing CMIs in cardiac applications. CMIs such as mavacamten and aficamten have demonstrated significant clinical benefits in reducing LVOT gradients, improving exercise capacity, and alleviating symptoms in patients with oHCM. Mavacamten is currently approved for clinical use, while aficamten is in advanced regulatory review. Comparative data suggest potential advantages of aficamten in the onset of action, pharmacokinetic profile, and tolerability. Emerging evidence supports the exploration of CMIs in pediatric populations, heart failure with preserved ejection fraction (HFpEF), and non-obstructive HCM (nHCM), although results are still preliminary. Cardiac myosin inhibitors offer a novel, pathophysiology-targeted approach to managing oHCM. While mavacamten has established efficacy, next-generation agents like aficamten may offer improved safety and versatility. Further long-term studies are needed to clarify their role across broader patient populations. Full article
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14 pages, 487 KiB  
Article
Sex-Based Differences in Clinical Presentation, Management, and Outcomes in Patients Hospitalized with Pulmonary Embolism: A Retrospective Cohort Study
by Benjamin Troxler, Maria Boesing, Cedrine Kueng, Fabienne Jaun, Joerg Daniel Leuppi and Giorgia Lüthi-Corridori
J. Clin. Med. 2025, 14(15), 5287; https://doi.org/10.3390/jcm14155287 - 26 Jul 2025
Viewed by 264
Abstract
Background/Objectives: Pulmonary embolism (PE) remains a major cause of morbidity and mortality. Despite advances in care, its nonspecific symptoms pose diagnostic and therapeutic challenges. Emerging evidence suggests sex-based differences in PE presentation, management, and outcomes, yet real-world data from European settings remain [...] Read more.
Background/Objectives: Pulmonary embolism (PE) remains a major cause of morbidity and mortality. Despite advances in care, its nonspecific symptoms pose diagnostic and therapeutic challenges. Emerging evidence suggests sex-based differences in PE presentation, management, and outcomes, yet real-world data from European settings remain scarce. This study aimed to investigate sex differences in clinical presentation, diagnostic workup, therapeutic interventions, and outcomes among hospitalized PE patients. Methods: We conducted a retrospective cohort study including all adult patients (≥18 years) admitted with a main diagnosis of acute PE at the Cantonal Hospital Baselland between January 2018 and December 2020. Data were extracted from electronic medical records and included demographics, comorbidities, symptoms, diagnostics, treatments, and outcomes. Sex-based comparisons were performed using univariate analyses. Results: Among 197 patients, 54% were women. Compared to men, women were more often admitted by ambulance (42% n = 45 vs. 24% n = 22, p = 0.009), had more frequent tachycardia (38% n = 41 vs. 23% n = 21, p = 0.024), and received lysis therapy more often (10% n = 11 vs. 2% n = 2, p = 0.023). DVT was more frequently diagnosed in women when sonography was performed (82% n = 49 vs. 64% n = 34, p = 0.035). Men had higher rates of B symptoms, smoking, and family history of PE. Women had longer hospital stays and were more frequently discharged to rehabilitation facilities. No sex differences were found in in-hospital mortality, 6-month rehospitalization, or adherence to diagnostic guidelines. Conclusions: This study reveals sex-based differences in PE presentation and management, suggesting potential disparities in care pathways. Further research is needed to promote equitable, personalized treatment strategies. Full article
(This article belongs to the Special Issue Pulmonary Embolism: Clinical Advances and Future Opportunities)
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13 pages, 640 KiB  
Article
Transforming Patient Experience: Real-World Impact of Mepolizumab on Symptom Burden in Chronic Rhinosinusitis with Nasal Polyps—A Multicenter Perspective
by Alfonso García-Piñero, Tomás Pérez-Carbonell, María-José Gómez-Gómez, Encarna Domenech-Campos, Fernando Martinez-Expósito, Noelia Muñoz-Fernández, Jordi Calvo-Gómez, Carmen García-Navalón, Lucas Fito-Martorell, Felip Ferrer-Baixauli, Ainhoa García-Lliberós, Nezly Mosquera-Lloreda, Chakib Taleb, Carlos Zac-Romero, Cecilia López-Valdivia, Juan Pardo-Albiach and Miguel Armengot-Carceller
J. Clin. Med. 2025, 14(15), 5248; https://doi.org/10.3390/jcm14155248 - 24 Jul 2025
Viewed by 418
Abstract
Background: Chronic rhinosinusitis with nasal polyps (CRSwNP) is a chronic upper airway disease that may involve different inflammatory endotypes, although in Western populations it is most commonly associated with type 2 inflammation. CRSwNP has a significant impact on the patient’s quality of [...] Read more.
Background: Chronic rhinosinusitis with nasal polyps (CRSwNP) is a chronic upper airway disease that may involve different inflammatory endotypes, although in Western populations it is most commonly associated with type 2 inflammation. CRSwNP has a significant impact on the patient’s quality of life. The recommended appropriate medical therapy is effective in controlling CRSwNP symptoms in many patients; however, a subset continues to exhibit persistent type 2 inflammation, evidenced by recurrent nasal polyps, elevated eosinophil counts, or the need for systemic corticosteroids or surgery. Monoclonal antibodies have recently become a novel and personalized treatment that can help refractory patients restore disease control. Objective: The present study aims to evaluate the effectiveness of mepolizumab in real-world settings in a diverse patient population, focusing on assessing the impact of this therapy on patient-reported outcomes after six months of treatment. Methods: This is a multicenter, observational study of CRSwNP patients treated with mepolizumab carried out in five hospitals located in Spain. Adult patients with a diagnosis of uncontrolled CRSwNP were included in the study. The change in the nasal polyp score (NPS) was the main clinical endpoint. Changes in the Sinonasal Outcome Test (SNOT-22), nasal congestion and smell impairment visual analogue scale scores, and blood and nasal polyp tissue eosinophil counts were among other endpoints included. Results: In total, 47 patients were included, and 91% were asthmatic. The nasal polyp score (0–8) was reduced significantly in the cohort (mean change: −2.56, p < 0.0001). The mean SNOT-22 score improved 25.29 points. Nasal congestion (−3.57, p < 0.0001) and smell impairment (−4.0, p < 0.0001) visual analog scale scores (0–10) showed a significant improvement. Blood and tissue eosinophil median counts showed significant reductions versus baseline of 86% and 26%, respectively. Among those patients with asthma, the asthma control test score achieved a median value of 24 points. Conclusions: This study provides real-world evidence supporting the effectiveness of mepolizumab in managing CRSwNP in patients with features suggestive of type 2 inflammation. The observed improvements in patient-reported outcomes, nasal polyp burden, and asthma control suggest that mepolizumab may be a valuable therapeutic option for this patient population. Full article
(This article belongs to the Section Otolaryngology)
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