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Real-World Effectiveness of Budesonide Orodispersible Tablets for Eosinophilic Oesophagitis: The Importance of Patience and Education.

Budesonide orodispersible tablets (BOT) have been shown to be efficacious for eosinophilic oesophagitis (EoE) in clinical trials. This study evaluated the real-world effectiveness of BOT in a retrospective cohort of 94 patients across six tertiary centres. 61% of patients achieved histological remission and 39% clinicohistological remission following induction therapy. Adverse events were reported by 16% of patients and 17% were noted to have incorrect technique of administration. BOT was demonstrated to have lower rates of clinicohistological remission in real-world practice than reported in clinical trials, largely due to incorrect technique and cessation due to adverse events.

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  • Journal IconAlimentary pharmacology & therapeutics
  • Publication Date IconMay 14, 2025
  • Author Icon Sarah Taylor + 15
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Prospective multicenter real-world outcomes of Suction Technology Utility in Mini-PCNL Study (STUMPS) in modern-day practice: formulation of the global STUMPS registry on behalf of the endourology section of the European Association of Urology and the suction mini-PCNL collaborative study group.

To present outcomes of a registry to understand the practice patterns, resource utilization, and nuances of suction mini-percutaneous nephron lithotripsy (SM-PCNL). Data from 30 centers in 21 countries were prospectively collected (March-November 2024). SM-PCNL was defined as PCNL using a suction nephrostomy sheath of size 14-22 Fr. with any lithotripsy device. There were no instructions on how to perform the surgical procedure. Stone features and stone-free status were assessed using an unenhanced CT scan. Data are presented as median/interquartile range and frequency/proportion. 1707 patients were included and 42.4% of them were males. Most were first-time stone formers. Median age was 50years. Median stone volume was 1700 mm3. Surgery was commonly performed using a single access tract (92.9%) and in supine position (56.5%). The fluoroscopy-only puncture was used as the most common access (70.7%), followed by the combination of fluoroscopy and ultrasound (25.1%). Median operation time was 45min. The most common sheath was Clearpetra (27.8%). Thulium fiber laser was the most frequent energy used (26.2%). A tubeless procedure with a stent was employed in 47.0% of cases. Most common complications were fever managed by observation (7.3%), fever requiring antibiotics (3.3%), blood transfusion (1.1%), and sepsis (0.2%). Median hospitalization was 3days. 30-day CT scan showed zero fragments in 82.4% of patients. Reintervention was performed in 2.6% of cases. This registry outlines the various equipment, peri-operative strategies, complications, and outcomes of SM-PCNL performed in real-world practice, providing valuable data on the nuances of performing such surgery.

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  • Journal IconWorld journal of urology
  • Publication Date IconMay 13, 2025
  • Author Icon Vineet Gauhar + 42
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Face-based machine learning diagnostics: applications, challenges and opportunities

Traditional medical diagnostic methods face bottlenecks such as high cost, poor accessibility, and delayed diagnosis in genetic syndromes, neurological disorders, psychiatric disorders, and endocrine disorders. Face-based machine learning (ML) technology provides a new path for early screening of diseases by analyzing facial phenotypes, dynamic expressions, facial skin, and 3D structural abnormalities, and is gradually becoming a clinically assisted screening tool. This paper provides a comprehensive overview of the applications, advances, and challenges of the technology. We summarize the range of diseases for which facial diagnosis is applicable and describe the basic process and related techniques for face-based ML diagnostic systems. In addition, this paper organizes the resources of current publicly available facial medical datasets and clarifies their disease coverage and sample size. Finally, possible future solutions to challenges hindering widespread adoption in clinical practice such as data bias, privacy, interpretability, generalizability, clinical value, and resource constraints are discussed. This review aims to provide researchers with a comprehensive foundation that integrates clinical perspectives, technological insights, and practical resources, to facilitate the development and successful implementation of face-based ML diagnostics in real-world clinical practice.

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  • Journal IconArtificial Intelligence Review
  • Publication Date IconMay 13, 2025
  • Author Icon Jie Song + 8
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Use of fluorine-18-fluorodeoxyglucose positron emission tomography/computed tomography in diagnosing giant cell arteritis in a Western Australian tertiary centre: a 36-month analysis.

Fluorine-18-fluorodeoxyglucose positron emission tomography/computed tomography (18F-FDG-PET/CT) has been established as a beneficial diagnostic tool in large vessel vasculitis. There have been limited studies of its use in real-world clinical practice. We examined patients at Royal Perth Hospital (RPH) with suspected giant cell arteritis (GCA) from January 2019 to 2022 who had 18F-FDG-PET/CT, and analysed its diagnostic accuracy compared to temporal artery biopsy (TAB) and clinical diagnosis at 6 months. Patients referred to RPH Rheumatology with suspected GCA from January 2019 to 2022 were identified as having had 18F-FDG-PET/CT for diagnostic evaluation. 18F-FDG-PET/CT results were dichotomous into positive (consistent with GCA) or negative, and compared to TAB and clinical diagnosis of GCA at 6 months by a consultant rheumatologist. A total of 32 patients had 18F-FDG-PET/CT for diagnostic purposes; nine of 32 18F-FDG-PET/CT scans were positive. Compared to TAB, 18F-FDG-PET/CT had a sensitivity of 50%, a specificity of 93%, a positive predictive value (PPV) of 86%, a negative predictive value (NPV) of 68% and accuracy of 61.5%. Compared to clinical diagnosis, 18F-FDG-PET/CT had a sensitivity of 41%, a specificity of 100%, a PPV of 100%, an NPV of 44% and accuracy of 59.4%. Only one 18F-FDG-PET/CT (11%) had cranial artery involvement. The median time taking glucocorticoids (GCs) was 7 days (interquartile range (IQR) 0-22.5 days) for positive 18F-FDG-PET/CT scans, versus 13 days (IQR 8-39 days) for negative 18F-FDG-PET/CT scans. The median time from request to scan was 29 days (IQR 31.5-77 days) for 18F-FDG-PET/CT on 14 or more days of GCs. 18F-FDG-PET/CT is more likely to be positive if it is performed within 7 days of steroid commencement. 18F-FDG-PET/CT had lower sensitivity and higher specificity than reported in clinical studies. In clinical practice, the significant delays from steroid start to scan time may reduce diagnostic sensitivity. A negative 18F-FDG-PET/CT scan did not negate the need for further investigations.

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  • Journal IconInternal medicine journal
  • Publication Date IconMay 13, 2025
  • Author Icon Madeleine Venables + 4
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Trends in device utilization and procedural outcomes of endovascular treatment of acute ischemic stroke: A 10-year review of individual institutional experience.

BackgroundMost of endovascular thrombectomy (EVT) procedures are performed outside of clinical trials, thus the data available from such trials is often not reflective of real-world practice. How specific indications for EVT or new techniques evolve or impact procedural outcomes in individual patients remains largely unknown.MethodsFrom 2013 to 2023, demographic, clinical and procedural data was collected on consecutive first 20 EVT procedures at the beginning of each year. Trends in utilization of thrombectomy devices and correlations with procedural outcomes were investigated.ResultsThere was a significant difference in the distribution of target occlusion sites (<0.001) with a trend towards a higher proportion of distal occlusions treated with EVT in more recent years. Aspiration-first approach to EVT became more frequent whereas the frequency of stent retriever-first approach decreased (p < 0.001). Average duration of the EVT procedure decreased dramatically (p < 0.001, r = 0.7). In 2013-2015 versus 2022-2023, mean duration of thrombectomy was 128 and 41 min, respectively. However, the number of passes, rates of first pass effect, final TICI 2b/3 and TICI 2c/3 reperfusion remained largely unchanged (p > 0.1 for each metric).ConclusionsOur analysis demonstrates continuous changes in target population of patients with acute stroke treated with EVT and procedural techniques used by operators. While the average duration of the procedure has decreased dramatically, its technical success has remained largely unchanged. This indicates continuous unmet need for future innovation in this field of neurointervention.

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  • Journal IconInterventional neuroradiology : journal of peritherapeutic neuroradiology, surgical procedures and related neurosciences
  • Publication Date IconMay 13, 2025
  • Author Icon Amy Letavay + 6
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Effect of pulmonary artery pressure-guided management on outcomes of patients with heart failure outside clinical trials: A systematic review and meta-analysis of real-world evidence with the CardioMEMS Heart Failure System.

Pulmonary artery pressure (PAP)-guided heart failure (HF) management using the CardioMEMS HF System improves patient outcomes in randomized controlled trials (RCTs). Our aim was to systematically assess its impact on outcomes of HF patients receiving the device in a non-RCT setting. PubMed, ClinicalTrials.gov and Cochrane Clinical Trial Collection were searched on 8 January 2025. Studies were eligible if they included: (i) pre/post-comparison of outcomes in patients receiving CardioMEMS; (ii) adult patients diagnosed with HF; (iii) follow-up ≥6 months, and (iv) report of the primary outcome, i.e. HF hospitalization (HFH) rates. Other outcomes were changes in PAP, New York Heart Association (NYHA) class and quality of life. Quantitative analysis was performed by generating forest plots and calculating pooled means, mean differences and incidence rate ratio (IRR) by random-effect models, as appropriate. Eight studies with a total of 3306 patients were included. Four studies were single-arm, open-label, industry-funded studies and four real-world practice studies. Four studies were performed in the US and four in Europe. Significant decreases in systolic PAP (-7.8 mmHg [-10.1 mmHg; -5.6 mmHg]), mean PAP (-5.2 mmHg [-6.7 mmHg; -3.8 mmHg]) and diastolic PAP (-4.4 mmHg [-5.5 mmHg; -3.3 mmHg]) were demonstrated. One year after CardioMEMS implantation, 56% [43%; 67%] of patients were NYHA class I/II with EQ-5D-5L visual analogue scale scores being significantly improved from baseline (7.2 [3.5; 10.9]). PAP-guided HF management was associated with a significant, 61% decrease in HFHs at 1 year (IRR 0.39 [0.31; 0.47]). Heart failure management with CardioMEMS leads to lower PAP, improved functional status and quality of life, and decrease of HFHs at 1 year in patients receiving the device in a real-life, non-RCT setting. PROSPERO database registration number CRD42025635206.

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  • Journal IconEuropean journal of heart failure
  • Publication Date IconMay 13, 2025
  • Author Icon Chris J Kapelios + 4
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العوامل المؤثرة على الرضا الوظيفي لممارسي العلاقات العامة في المشاريع الكبرى لصندوق الاستثمارات العامة وعلاقته بالأداء

The research examines the variables which affect job satisfaction for publicity practitioners who work on principal Public Investment Fund projects (Diriyah, Neom, Giddiya, Red Sea and Rodhn). The research has used both descriptive-analytical methods to conduct their study through structured questionnaires with 54 practitioner participants. Numerous internal organizational aspects demonstrated statistical relationships with job satisfaction based on the findings of this study including organizational culture and work environment and training opportunities and administrative leadership. The investigation has verified that professionals at higher levels of job satisfaction demonstrate better professional performance results. The research points out several major factors affecting job satisfaction that emerge from inadequate upper-level leadership communication and insufficient resources and excessive workloads and restricted career advancement opportunities. Stress and burnout tend to develop when employees face these workplace factors in large-scale environments that pressure them through demanding tasks. Organizations must establish transparent work environments focused on motivation through promotion of staff participation while maintaining fair remuneration systems. The findings demonstrate an urgent need to establish both open communication systems and repetitive professional development schemes. The research results benefit both scholarly and real-world practices by providing valuable guidelines for those who should enhance employee engagement along with organizational effectiveness. The study offers important findings that future investigations in internal communication and human resource management can refer to. The study concludes that organizations should establish a strategic internal communication system to enhance both work commitment and enduring job satisfaction of their employees.

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  • Journal IconInternational Journal of Educational Sciences and Arts
  • Publication Date IconMay 12, 2025
  • Author Icon Abdulaziz Al Mahmoud
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Empowering Generalist Material Intelligence with Large Language Models.

Large language models (LLMs) are steering the development of generalist materials intelligence (GMI), a unified framework integrating conceptual reasoning, computational modeling, and experimental validation. Central to this framework is the agent-in-the-loop paradigm, where LLM-based agents function as dynamic orchestrators, synthesizing multimodal knowledge, specialized models, and experimental robotics to enable fully autonomous discovery. Drawing from a comprehensive review of LLMs' transformative impact across representative applications in materials science, including data extraction, property prediction, structure generation, synthesis planning, and self-driven labs, this study underscores how LLMs are revolutionizing traditional tasks, catalyzing the agent-in-the-loop paradigm, and bridging the ontology-concept-computation-experiment continuum. Then the unique challenges of scaling up LLM adoption are discussed, particularly those arising from the misalignment of foundation LLMs with materials-specific knowledge, emphasizing the need to enhance adaptability, efficiency, sustainability, interpretability, and trustworthiness in the pursuit of GMI. Nonetheless, it is important to recognize that LLMs are not universally efficient. Their substantial resource demands and inconsistent performance call for careful deployment based on demonstrated task suitability. To address these realities, actionable strategies and a progressive roadmap for equitably and democratically implementing materials-aware LLMs in real-world practices are proposed.

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  • Journal IconAdvanced materials (Deerfield Beach, Fla.)
  • Publication Date IconMay 12, 2025
  • Author Icon Wenhao Yuan + 3
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Augmented reality microscopy to bridge trust between AI and pathologists

Diagnostic certainty is the cornerstone of modern medicine and critical for maximal treatment benefit. When evaluating biomarker expression by immunohistochemistry (IHC), however, pathologists are hindered by complex scoring methodologies, unique positivity cut-offs and subjective staining interpretation. Artificial intelligence (AI) can potentially eliminate diagnostic uncertainty, especially when AI “trustworthiness” is proven by expert pathologists in the context of real-world clinical practice. Building on an IHC foundation model, we employed pathologists-in-the-loop finetuning to produce a programmed cell death ligand 1 (PD-L1) CPS AI Model. We devised a multi-head augmented reality microscope (ARM) system overlayed with the PD-L1 CPS AI Model to assess interobserver variability and gauge the pathologists’ trust in AI model outputs. Using difficult to interpret regions on gastroesophageal biopsies, we show that AI-assistance improved case agreement between any 2 pathologists by 14% (agreement on 77% vs 91%) and among 11 pathologists by 26% (agreement on 43% vs 69%). At a clinical cutoff of PD-L1 CPS ≥ 5, the number of cases diagnosed as positive by all 11 pathologists increased by 31%. Our findings underscore the benefits of fully engaging pathologists as active participants in the development and deployment of IHC AI models and frame the roadmap for trustworthy AI as a bridge to increased adoption in routine pathology practice.

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  • Journal Iconnpj Precision Oncology
  • Publication Date IconMay 12, 2025
  • Author Icon Sunil Badve + 22
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Clinical Characteristics and Safety Profiles of Japanese Psoriasis Patients Who Continued Apremilast Treatment for 6 and 12 Months: A Post Hoc Analysis of an Apremilast Postmarketing Surveillance Study.

Apremilast is a phosphodiesterase 4 inhibitor approved for moderate to severe psoriasis in Japan. Apremilast significantly improved Physician's Global Assessment (PGA) and Dermatology Life Quality Index (DLQI) both at 6 and 12 months in a previously published primary post-surveillance study. Here, we performed a post hoc analysis of the surveillance data to evaluate patient characteristics, effectiveness, and safety among psoriasis patients who continued apremilast for 6 and 12 months. The PMS included 992 patients, of whom 646 of 992 patients continued treatment for 6 months and 509 of 992 patients subsequently continued treatment for 12 months. Baseline characteristics between these groups were similar. Among 992 patients, the treatment persistence rate was 65.1% at 6 months and 51.3% at 12 months after the start of apremilast treatment. PGA 0/1 response was 47.9% at 6 months and 60.8% at 12 months, whereas DLQI 0/1 responses at 6 months and 12 months were 38.5% and 58.7%, respectively. Among 646 patients who continued apremilast for 6 months, diarrhea was reported in 60 patients (9.3%), nausea in 35 patients (5.4%), and headache in 11 (1.7%) patients, which were mainly observed within the first month since treatment initiation. In 509 patients who continued apremilast for 12 months, diarrhea was reported in 43 patients (8.5%), nausea in 24 patients (4.7%), and headache in 6 (1.2%) patients; similar frequencies of these adverse reactions were observed within 6 months and between 6 and 12 months of follow-up. It is important to continue apremilast by appropriately managing diarrhea and nausea in real-world practice.

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  • Journal IconThe Journal of dermatology
  • Publication Date IconMay 10, 2025
  • Author Icon Mamitaro Ohtsuki + 8
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Making better clinical decisions: How doctors can recognise and reduce bias and noise in medical practice

Clinical reasoning and decision-making are essential to medical practice, where poor clinical judgement can lead to serious diagnostic errors, suboptimal use of diagnostic tests and investigations, flawed management strategies and ultimately, adverse patient outcomes. It is known that in real-world clinical environments, clinicians often rely on intuitive judgements for decision-making, due to natural proclivities for pattern-recognition and retrieval of pre-existing illness scripts, as well as out of practical necessity to be efficient in fast-paced work environments with high patient volume and cognitive load. Yet, an over-reliance on intuitive judgements can result in cognitive errors. While biases associated with heuristics-based or intuitive thought processes are often discussed in literature, there is also a lesser entity of noise that affects consistency and reliability of clinical decision-making. In this article, we highlight the importance of learning foundational principles of clinical reasoning and understanding how cognitive errors happen in medical training, and suggest a series of educational pedagogies and workplace-based interventions that could help to cultivate and optimise medical decision-making in real-world practice.

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  • Journal IconAnnals of the Academy of Medicine, Singapore
  • Publication Date IconMay 9, 2025
  • Author Icon Isaac Ks Ng + 6
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A Review of Artificial Intelligence Techniques for Wheat Crop Monitoring and Management

Artificial intelligence (AI) techniques, particularly machine learning and deep learning, have shown great promise in advancing wheat crop monitoring and management. However, the application of AI in this domain faces persistent challenges that hinder its full potential. Key limitations include the high variability of agricultural environments, which complicates data acquisition and model generalization; the scarcity and limited diversity of labeled datasets; and the substantial computational demands associated with training and deploying deep learning models. Additionally, difficulties in ground-truth generation, cloud contamination in remote sensing imagery, coarse spatial resolution, and the “black-box” nature of deep learning models pose significant barriers. Although strategies such as data augmentation, semi-supervised learning, and crowdsourcing have been explored, they are often insufficient to fully overcome these obstacles. This review provides a comprehensive synthesis of recent advancements in AI for wheat applications, critically examines the major unresolved challenges, and highlights promising directions for future research aimed at bridging the gap between academic development and real-world agricultural practices.

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  • Journal IconAgronomy
  • Publication Date IconMay 9, 2025
  • Author Icon Jayme Garcia Arnal Barbedo
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Effectiveness of Urea and Tolvaptan in the Treatment of Hypotonic Hyponatremia

Objective: The objective of this study was to compare the effectiveness of urea and tolvaptan in the treatment of plasma sodium levels in patients with hypotonic hyponatremia. Methods: This was an observational, longitudinal, and retrospective study including all adult patients who received treatment with urea or tolvaptan for hypotonic hyponatremia from 1 April 2014 to 31 October 2023 at the Department of Internal Medicine, Virgen del Rocío University Hospital, Seville, Spain. Results: Forty-seven (55.3%) patients received urea and 38 (44.7%) tolvaptan. The drugs were prescribed for the treatment of syndrome of inappropriate antidiuresis (SIAD) in 59 (69.4%) patients. The mean blood sodium level at the start of treatment was 123.5 ± 6.2 mEq/L. Overall, 61.7% and 63.2% of patients treated with urea and tolvaptan, respectively, achieved a normal blood sodium level (p = 0.89), although the time to have their sodium levels corrected differed between both groups: 41.7 ± 76 days with urea and 21 ± 23.9 days with tolvaptan (p = 0.038). The following were significant in the multivariate study: Initial sodium value (p = 0.037), absolute sodium improvement (p = 0.041), and percentage sodium improvement (p = 0.033). Among patients with SIAD, 69.5% achieved a normal sodium level; this figure was 45.5% for patients with heart failure. Three patients reported side adverse events in the urea group and none in the tolvaptan group. Conclusions: Our data, reflecting real-world practice and follow-up of patients with hypotonic hyponatremia, suggest that both urea and tolvaptan are safe, well-tolerated, and have a similar effectiveness in correcting blood sodium levels in patients with hypotonic hyponatremia, overall and secondary to SIAD, though treatment with tolvaptan achieved this goal earlier than urea.

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  • Journal IconJournal of Clinical Medicine
  • Publication Date IconMay 9, 2025
  • Author Icon Juan Delgado-Cuesta + 6
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Efficacy and safety of combination therapy with lenvatinib and pembrolizumab in elderly patients with advanced endometrial cancer

Background. Currently, women over 65 years of age comprise a quarter of endometrial cancer (EC) patients, but this population remains underrepresented in clinical trials. The combination therapy with lenvatinib plus pembrolizumab has demonstrated a significant improvement in progression-free survival and overall survival in patients with mismatch repair system proficiency (pMMR/MSS) after a single prior treatment line. However, data on the efficacy and safety of this regimen in real-world clinical practice, especially in elderly patients, are limited.Aim. To describe the efficacy and safety of EC therapy with lenvatinib and pembrolizumab in elderly patients.Materials and methods. In this retrospective single-center study conducted at a Moscow hospital (December 2020 – March 2024), we collected data on the efficacy and safety of lenvatinib and pembrolizumab therapy in 26 patients over 65 years of age. Patients with pathologically confirmed EC without evidence of microsatellite instability were included. The primary endpoint of the study was progression-free survival; additionally, we evaluated the clinical characteristics of the patients, analyzed the objective response rate and the incidence of adverse events.Results. The median age was 69 (65–81) years, 23 % of patients were older than 75 years. The performance status of 92.3 % of patients was classified as ECOG 0–1, endometrial adenocarcinoma was the most frequently detected type (61.5 % of cases), serous carcinoma was reported in 10 (38.5 %) patients. The median number of prior treatment lines was 3 (2–6). The objective response rate reached 25 %, and the disease control rate was 75 %. The median progression-free survival was 5.67 (1.4–35.3) months. Adverse events of any grade were noted in 24 (92.3 %) patients. Lenvatinib dose reduction was performed in 76.9 % of cases. The most frequent adverse events were arterial hypertension (in 16 (61.5 %) patients), fatigue (in 5 (19.2 %) patients), and diarrhea (in 3 (11.5 %) patients).Conclusion. In this real-world clinical practice study in Russia involving patients with recurrent and metastatic EC without mismatch repair system deficiency (pMMR/MSS) over 65 years of age who received lenvatinib plus pembrolizumab, the efficacy and safety of the treatment were comparable to those reported in earlier studies, suggesting the feasibility of combination therapy in this population with appropriate attention to performance status and management of adverse events. Additional studies are required to evaluate the efficacy and safety of the combination therapy of lenvatinib and pembrolizumab in patients with a more severe functional status.

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  • Journal IconTumors of female reproductive system
  • Publication Date IconMay 8, 2025
  • Author Icon M V Volkonskiy + 13
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Pediatric Chronic Kidney Disease: Mind the Gap Between Reality and Expectations

Pediatric chronic kidney disease (CKD) is a growing concern that often originates early in life, yet significant challenges remain in translating clinical guidelines into real-world practice. World Kidney Day 2025 highlights the importance of early detection, but the three levels of preventive strategies commonly recommended for adults may not be directly applicable to children. Unlike adult CKD, primary prevention in pediatrics should focus on prenatal, neonatal, and early-life factors such as congenital anomalies of the kidney and urinary tract (CAKUT), preterm birth, maternal health, and environmental exposures. Secondary prevention, involving early detection through screening, is crucial, yet the effectiveness of mass urinary screening in children remains a subject of global debate. Several key challenges persist, including the accurate estimation of glomerular filtration rate (eGFR), consistent definition and diagnosis of pediatric hypertension, identification of reliable biomarkers, and targeted screening in specific pediatric populations. Although clear guidelines exist to manage CKD progression and enhance quality of life, a critical gap remains between what is known and what is practiced. Closing this gap requires robust evidence to inform best practices, improve health-related quality of life, and advance pediatric kidney replacement therapies. To protect and improve kidney health for every child worldwide, these challenges must be acknowledged, and sustainable, evidence-based solutions must be developed and implemented without further delay.

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  • Journal IconChildren
  • Publication Date IconMay 8, 2025
  • Author Icon Chien-Ning Hsu + 3
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Evaluation of the effectiveness and safety of avapritinib in real-world Spanish cases with gastrointestinal stromal tumor and D842V-PDGFRA mutation.

Gastrointestinal stromal tumors (GISTs) are the most common sarcoma subtype. Patients with unresectable or metastatic GISTs harboring the D842V mutation in the PDGFRA gene have a poor prognosis due to intrinsic resistance to imatinib and all other approved tyrosine kinase inhibitors. Avapritinib, targeting this mutation, is the first agent approved for patients with unresectable or metastatic GIST that have the PDGFRA D842V mutation. This study assesses the effectiveness and safety of avapritinib in real-world clinical scenarios involving Spanish patients with this mutation. The AVARWE study is a descriptive, retrospective, multicenter observational study of 21 patients treated with avapritinib across 13 Spanish centers from June 9, 2023, to December 18, 2023. Data collected included patient demographics, disease characteristics, treatment history, and response rates based on RECIST criteria. The main outcomes, progression-free survival (PFS) and overall survival (OS), were measured, with safety assessed through adverse events documentation according to CTCAE criteria. Median PFS 35.6 was months and median OS was 42.2 months, with survival rates at 1, 5, and 3 years demonstrating avapritinib effectiveness. The objective response rate was 76.2% for partial response and 4.8% for complete response. Avapritinib enabled surgical intervention in previously unresectable cases and was generally well-tolerated, with manageable adverse events. Avapritinib extends PFS and OS among patients with PDGFRA D842V-mutant GIST in real-world practice, mirroring pivotal trial outcomes. Its substantial activity supports its use as a first-line therapy for this subgroup. The manageable safety profile reinforces avapritinib viability for routine use. Given the rarity of these cases, it is advised to consult sarcoma-expert units.

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  • Journal IconThe oncologist
  • Publication Date IconMay 8, 2025
  • Author Icon Isaac Nuñez Hernández + 14
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Long-Term Testosterone Shows Cardiovascular Safety in Men with Testosterone Deficiency in Electronic Health Records

Abstract Objective Our objective is to examine the association between cardiovascular (CV) safety and long-term testosterone therapy (TTh) in men with testosterone deficiency (TD) in real-world. Method We extracted the electronic health records of 2,683 adult men with TD from 3 healthcare systems from 1/1/2013-7/1/2022. We matched TTh and non-TTh groups by 1:1 ratio based on age, race, Charlson Comorbidity Index, and serum testosterone level via propensity score. We used the intent-to-treat analysis using Kaplan-Meier curves and Cox regressions to examine CV risk for major adverse cardiovascular events (MACE). We also explored the impact of TTh on diabetes and hyperlipidemia development and progression. We compared the 928 TTh patients to the 928 untreated patients with a median follow-up of 3 years for both groups. Results After matching, BMI, DBP, hyperlipidemia, hypertension, depression, and anxiety were statistically significant different between treatment and control cohorts. The log-rank test for the cumulative MACE incidence was comparable (p-value&amp;gt;0.05). There were no statistically significant associations between TTh use and CV risk in the univariate Cox regression (HR [95% CI]: 1.01 [0.75-1.36]) and Cox regressions adjusted by the pre-existing MACE (HR [95% CI]: 0.98 [0.72-1.32]) and other baseline covariates (HR [95% CI]: 0.93 [0.68-1.26]). No statistically significant associations were found between TTh and diabetes. For hyperlipidemia, TTh group presented statistically significant improvement on LDL and total cholesterol. Conclusion TTh use among men with TD was not associated with increased CV risk in real-world clinical practice.

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  • Journal IconJournal of the Endocrine Society
  • Publication Date IconMay 7, 2025
  • Author Icon Yilu Lin + 4
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Excision margins and prognosis in cutaneous melanoma: retrospective population-based cohort study.

The impact of surgical excision margin size on the survival of patients with cutaneous melanoma (CM) is controversial. The aim of this study was to assess the impact of excision margins on patient outcomes. This retrospective population-based follow-up study analysed data from patients presenting with CM without extranodal disease. The linear distance of CM from the excision margin was assessed microscopically. Regarding overall and melanoma-specific survival estimates, Kaplan-Meier analysis, the Cox proportional hazards model, and the subdistribution hazard ratio of the Fine-Gray model were applied. Of 3262 patients, a total of 2698 (82.7%) with pathological (p) T1-2 and 564 (17.3%) with pT3-4 CM underwent wide local excision. Multivariable analyses, adjusting for melanoma incidence cohort, age, sex, anatomical site, ulceration, histotype, mitotic count, and tumour-infiltrating lymphocytes, demonstrated that, in pT1-2 CM, surgical excision > 1.1 cm resulted in significant CM-specific survival benefit. In pT4 disease, a CM distance to the surgical margins of < 1.6 cm significantly lowered CM-specific survival. This population-based study conducted in real-world clinical practice found that the prognosis of patients with pT1-2 CM may benefit from excision margins > 1.1 cm. In pT3-4 CM, margins < 1.6 cm were significantly associated with lower CM-specific survival rates. These findings highlight the need for further prospective studies to evaluate the safety of surgical excision margins in managing primary CM.

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  • Journal IconBJS open
  • Publication Date IconMay 7, 2025
  • Author Icon Alessandra Buja + 10
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Lublin Comorbidity of Adiposity Study (LUCAS 1.0 BMI) - quantifying the success of a region-wide overweight and obesity treatment program: a retrospective real‑world data analysis.

Obesity has emerged as a global health epidemic, which carries significant implications for both individual health and healthcare systems worldwide. Numerous reports have shown the health advantages associated with a 5-10% reduction of one's initial body weight, but it remains unclear whether the data from clinical trials translate to real-world clinical practice. In our retrospective analysis, we evaluated the effectiveness of a multifactorial obesity treatment, including pharmacotherapy, in Polish conditions. The objective was to assess the efficacy of this multimodal treatment in achieving a 5-10% weight reduction over 3-6 months. The patient cohort included 1114 adults: 243 (22%) men and 871 (78%) women, aged 16-80 years, diagnosed with obesity and overweight in a team-based obesity treatment program with one-day-stay admissions. Each patient underwent a tailored multifactorial obesity treatment regimen, which incorporated dietary adjustments, physical activity, psychological support, and pharmacotherapy. Weight reduction was observed in 868 (78%) patients. Of these, 635 (57%) achieved a 5% weight reduction, and 379 (34%) achieved a 10% reduction. No significant gender differences were found in weight reduction. Unlike clinical trials, weight reduction was similar between those receiving only behavioral treatment and those also on medication. The profile of a good responder in real-world conditions included high adherence to recommendations and follow-up visits, and absence of type 2 diabetes. Our study demonstrates that obesity can be effectively managed through individualized multimodal treatment. Nonetheless, this process demands sustained effort and necessitates individualized therapy overseen by a multidisciplinary therapeutic team.

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  • Journal IconEndokrynologia Polska
  • Publication Date IconMay 7, 2025
  • Author Icon Monika Lenart-Lipińska + 3
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An international real-world study of primary vitreoretinal lymphoma from the Australasian lymphoma alliance and collaborators.

Primary vitreoretinal lymphoma (PVRL) is a high-grade extranodal non-Hodgkin lymphoma, with limited prospective data to inform practice. High rates of central nervous system (CNS) relapse contribute to its poor prognosis. This international multicentre retrospective cohort study aimed to characterise real-world contemporary practice and outcomes in PVRL (2010-2022). Sixty patients were included from 11 centres across Australia, Singapore, Canada and the United Kingdom. Most patients had systemic therapy included in their initial management (63%) either alone or in combination with local therapies; 13% had upfront autologous stem cell transplantation (ASCT). The overall response rate was 78%. With a median follow-up of 68 months, the median progression-free survival (PFS) was 25 months, with a median overall survival (OS) of 73 months. Neither incorporation of systemic therapy into initial treatment nor upfront ASCT demonstrated a statistically significant impact on PFS or OS. The 5-year cumulative incidence of CNS relapse was 33%, with front-line systemic therapy being the only predictive factor for CNS relapse in a multivariate model, hazard ratio of 0.30 (95% CI 0.09-0.98, p = 0.05). Concerning heterogeneity in real-world approaches to diagnosis, staging and management approaches were identified. Further international collaborative efforts are required to address the unmet need in this rare entity.

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  • Journal IconBritish journal of haematology
  • Publication Date IconMay 6, 2025
  • Author Icon Catherine Tang + 17
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