Review of Challenges in Performing Real-World Evidence Studies for Nonprescription Products.

In recent years, regulatory authorities have signaled a willingness to consider real-world evidence (RWE) data to support applications for new claims and indications for pharmaceuticals. Historically, RWE studies have been the domain of prescription drugs, driven by the fact that clinical data on patients are routinely captured in medical records, claims databases, registries, etc. However, RWE reports of nonprescription drugs and supplements are relatively sparse due to methodological gaps in this area.

Current advances in neurocritical care.

This review summarizes the current research advances and guideline updates in neurocritical care. For the therapy of ischemic stroke, the extended treatment time window for thrombectomy and the emergence of novel thrombolytic agents and strategies have brought greater hope for patient recovery. Minimally invasive hematoma evacuation and goal-directed bundled management have shown clinical benefits in treating cerebral hemorrhage.

Expert insights on Hodgkin's lymphoma development in an activated PI3K delta syndrome patient undergoing leniolisib treatment.

Activated PI3K delta syndrome (APDS) is a primary immunodeficiency that is caused by mutations in the PI3K signalling pathway resulting in either gain-of-function or loss-of-function phenotypes of APDS 1 and 2. Malignancy is one of the most serious complications associated with APDS patients, with the most commonly occurring of these being lymphoma, and is the most common cause of death in APDS patients. Management of APDS is complex and variable due to the heterogeneous nature of the disease and ranges from antimicrobial and immunosuppressant agents to haematopoetic stem cell transplantation.

Development of a Complex Intervention for Promoting Participation in Resistance Exercise Among Community-Dwelling Frail Older Adults in China: A Multimethod Qualitative Study.

Background And Objectives: Regular resistance exercise (RE) showed a promising effect in reducing frailty in older adults. However, the participation of RE among this population remains low. This study was, therefore, aimed at developing a complex intervention tailored to community-dwelling frail older adults in China to promote participation in RE and reduce frailty ultimately.

A biomarker framework for liver aging: the Aging Biomarker Consortium consensus statement.

In human aging, liver aging per se not only increases susceptibility to liver diseases but also increases vulnerability of other organs given its central role in regulating metabolism. Total liver function tends to be well maintained in the healthy elderly, so liver aging is generally difficult to identify early. In response to this critical challenge, the Aging Biomarker Consortium of China has formulated an expert consensus on biomarkers of liver aging by synthesizing the latest scientific literature, comprising insights from both scientists and clinicians.

Recommendations on Rapid Diagnostic Point-of-care Molecular Tests for Respiratory Infections in the United Arab Emirates.

Traditional testing methods in the Middle East Region, including the United Arab Emirates (UAE), particularly the testing of Respiratory Syncytial Virus (RSV), influenza, group A streptococcus (GAS), and COVID-19 have the potential to be upgraded to new and advanced diagnostics methods that improve lead time to diagnosis, consumption of healthcare resources and patient experience. In addition, based on the research, it was reported that there is an underreporting of respiratory cases, overuse of antibiotics, and prolonged hospitalizations which is posing pressure on UAE healthcare stakeholders. A literature review was done exploring UAE's current diagnostic practices, recommended guidelines, diagnostic gaps, and challenges in RSV, GAS, Influenza, and COVID-19.

Environment scan of generative AI infrastructure for clinical and translational science.

This study reports a comprehensive environmental scan of the generative AI (GenAI) infrastructure in the national network for clinical and translational science across 36 institutions supported by the CTSA Program led by the National Center for Advancing Translational Sciences (NCATS) of the National Institutes of Health (NIH) at the United States. Key findings indicate a diverse range of institutional strategies, with most organizations in the experimental phase of GenAI deployment. The results underscore the need for a more coordinated approach to GenAI governance, emphasizing collaboration among senior leaders, clinicians, information technology staff, and researchers.

Assessing hard and loose "endpoints": comparison of patient and expert Bristol Stool Scale scoring of 2280 fecal samples.

Background: Stool consistency is an important outcome measure to evaluate in the investigation of several gastrointestinal diseases. The Bristol Stool Scale (BSS) is one of the most commonly used tools for evaluation of stool consistency. BSS ranges from 1-7 and each score is assigned to a given consistency of the feces.

Decentralized clinical trials are better for the participants and for the planet: the case study of a double-blind randomized controlled trial in Singapore (PROMOTE study).

Introduction: Novel technologies have enabled the decentralization of many aspects of clinical trials, but little research has been done on the impact of these changes on the participant experience, trial operations, or the environment.

Methods: A fully decentralized clinical trial conducted in Singapore is used as a case study to evaluate the operational outcomes, environmental impact (via life cycle assessment), and participants experience (qualitative interviews) of the decentralized model compared to a traditional study with in-person visits.

Results: The decentralized study achieved high participant retention rates (97%) and high completion rates for clinical data, even for biological samples.

Real-world usage and effectiveness of recombinant factor IX Fc in haemophilia B from the B-SURE study in France.

Background: More real-world data are needed to complement existing phase III studies on the efficacy and safety of recombinant factor IX Fc fusion protein (rFIXFc) in people with haemophilia B.

Objectives: We report final data from the B-SURE study, evaluating the real-world usage and effectiveness of rFIXFc in France.

Methods: Previously treated patients (all ages/severities) received on-demand or prophylactic rFIXFc during B-SURE.

Identifying enablers and barriers to teleultrasound use for remote settings: A scoping review.

Introduction/purpose: Teleultrasound connects expert point-of-care ultrasound (POCUS) users with remote community and rural sites. Evolving technologies including handheld devices, upgraded image quality, and the ability to transmit over low bandwidth connections increase POCUS education, accessibility, and clinical integration. Potential teleultrasound venues include low-resource settings, prehospital care, and austere environments (high altitudes, microgravity, conflict zones, etc.

Cleft Lip and Palate Research in the United Kingdom: Advances in Clinical Psychological Knowledge and Future Directions.

In 2012, the James Lind Alliance (JLA) worked with individuals with cleft lip and/or palate (CL/P), their families and clinicians to identify priority areas for future research. This article reviews progress conducted in the United Kingdom in the 3 JLA priorities most closely related to Clinical Psychology. It then builds upon the original priorities to identify 4 future directions, based on contemporary literature and in-depth discussions between clinical and research experts.

Impact of cariprazine on body weight and blood pressure among adults with bipolar I disorder, schizophrenia, or major depressive disorder in a real-world setting.

Background: Atypical antipsychotics are a common treatment for serious mental illness, but many are associated with adverse effects, including weight gain and cardiovascular issues, and real-world experience may differ from clinical trial data. Cariprazine has previously demonstrated a favorable safety and tolerability profile in clinical trials. Here, we evaluated the effects of cariprazine on body weight and blood pressure for bipolar I disorder (BP-I), schizophrenia, or as adjunctive treatment for major depressive disorder (MDD) using real-world data.

Enhancing the understanding of safety and the quality of patient care among medical and health sciences students in interprofessional climate: an interventional study.

Background: Interprofessional education (IPE) plays an essential role in improving healthcare outcomes through achieving shared understanding. Unfortunately, most healthcare professionals have not received training for patient safety (PS) in an interprofessional setting, which can meet the societal medical needs. This study aimed to foster the understanding of senior medical, dental, pharmacy and health sciences students about PS and quality of care at the University of Sharjah (UoS) in UAE.

Screening Young Adult Cancer Patients for Vocational Rehabilitation Services: A Vocational Rehabilitation Readiness Screening Tool.

Purpose: We aimed to develop an online vocational rehabilitation (VR) readiness screening (VRRS) tool for young adults diagnosed with cancer. VR readiness was defined as being physically and cognitively ready to enter or return to work or school.

Methods: We developed an initial VRRS tool informed by previous studies, a scoping review to determine such a tool had not already been developed, and consultation with subject matter experts.

International myeloma working group immunotherapy committee recommendation on sequencing immunotherapy for treatment of multiple myeloma.

T-cell redirecting therapy (TCRT), specifically chimeric antigen receptor T-cell therapy (CAR T-cells) and bispecific T-cell engagers (TCEs) represent a remarkable advance in the treatment of multiple myeloma (MM). There are several products available around the world and several more in development targeting primarily B-cell maturation antigen (BCMA) and G protein-coupled receptor class C group 5 member D (GRPC5D). The relatively rapid availability of multiple immunotherapies brings the necessity to understand how a certain agent may affect the safety and efficacy of a subsequent immunotherapy so MM physicians and patients can aim at optimal sequential use of these therapies.

A prospective pragmatic evaluation of automatic trial matching tools in a molecular tumor board.

Publicly available trial matching tools can improve the access to therapeutic innovations, but errors may expose to over-solicitation and disappointment. We performed a pragmatic non-interventional prospective evaluation on sequential patients at the Molecular Tumor Board of Centre Leon Berard. During 10 weeks in 2024, we analysed 157 patients with four clinical trial matching tools from the 19 screened: Klineo, ScreenAct, Trialing and DigitalECMT.

Guidelines International Network: Principles for Use of Artificial Intelligence in the Health Guideline Enterprise.

Description: Artificial intelligence (AI) has been defined by the High-Level Expert Group on AI of the European Commission as "systems that display intelligent behaviour by analysing their environment and taking actions-with some degree of autonomy-to achieve specific goals." Artificial intelligence has the potential to support guideline planning, development and adaptation, reporting, implementation, impact evaluation, certification, and appraisal of recommendations, which we will refer to as "guideline enterprise." Considering this potential, as well as the lack of guidance for the use of AI in guidelines, the Guidelines International Network (GIN) proposes a set of principles for the development and use of AI tools or processes to support the health guideline enterprise.

Severe bradycardia in patients with obstructive sleep apnoea and good early response to CPAP.

Background: Obstructive sleep apnoea (OSA) may lead to heart rhythm abnormalities including bradycardia. Our aim was to ascertain clinical and echocardiographic parameters in patients with OSA in whom severe bradycardia was detected in an outpatient setting, as well as to evaluate the efficacy of CPAP therapy on heart rate normalization at the early stages of treatment.

Methods: Fifteen patients mild, moderate or severe OSA and concomitant bradycardia were enrolled.

Mindful self-compassion for lung cancer (MSC-LC): Incorporating perspectives of lung cancer patients, clinicians, and researchers to create an adapted intervention to reduce lung cancer stigma.

Background: Stigma is a pervasive and distressing problem experienced frequently by lung cancer patients, and there is a lack of psychosocial interventions that target the reduction of lung cancer stigma. Mindful self-compassion (MSC) is an empirically supported intervention demonstrated to increase self-compassion and reduce feelings of shame and distress in non-cancer populations. However, there are several anticipated challenges for delivering MSC to lung cancer patients, and modifications may be needed to improve acceptability, appropriateness, and feasibility.

The ketamine chameleon: history, pharmacology, and the contested value of experience.

Introduction: Since its synthesis in 1962, ketamine has been widely used in diverse medical contexts, from anesthesia to treatment-resistant depression. However, interpretations of ketamine's subjective effects remain polarized. Biomedical frameworks typically construe the drug's experiential effects as dissociative or psychotomimetic, while psychedelic paradigms emphasize the potential therapeutic merits of these non-ordinary states.

Multicenter validation of secondary hemophagocytic lymphohistiocytosis diagnostic criteria.

Background: Five fulfilled hemophagocytic lymphohistiocytosis (HLH)-2004 criteria, and the HScore are widely used and recommended by international expert consensus to diagnose secondary HLH. Both diagnostic scores have never been validated in heterogeneous patient cohorts of secondary HLH patients. We aimed to systematically optimize and validate diagnostic criteria of secondary HLH using a multicenter approach.

Exploring the role of T cells in Alzheimer's and other neurodegenerative diseases: Emerging therapeutic insights from the T Cells in the Brain symposium.

This proceedings article summarizes the inaugural "T Cells in the Brain" symposium held at Columbia University. Experts gathered to explore the role of T cells in neurodegenerative diseases. Key topics included characterization of antigen-specific immune responses, T cell receptor (TCR) repertoire, microbial etiology in Alzheimer's disease (AD), and microglia-T cell crosstalk, with a focus on how T cells affect neuroinflammation and AD biomarkers like amyloid beta and tau.

Personalized Photoprotection: Expert Consensus and Recommendations From a Delphi Study Among Dermatologists.

Background: Recommending comprehensive personalized photoprotection requires an accurate assessment of the patient's skin, including phototype, lifestyle, exposure conditions, environmental factors, and concomitant cutaneous conditions as well as deep knowledge of the available options: sunscreen ingredients (type of filters, spectrum coverage, sun protection factor, enhanced active ingredients), oral photoprotection, and other methods of sun protection and avoidance.

Objectives: To establish consensus-based recommendations endorsed by an international panel of experts for personalized medical photoprotection recommendations that are applicable globally.

Methods: A two-round Delphi study was designed to determine the degree of agreement and relevance of aspects related to personalized medical photoprotection.