Multiple Perspectives on the Need for Real-World Evidence to Inform Regulatory and Health Technology Assessment Decision-Making: Scoping Review and Stakeholder Interviews.

Purpose: Real-world evidence (RWE) is increasingly considered in regulatory and health technology assessment (HTA) decision-making, though perspectives on its relevance may vary. Expanding on a recent review regarding regulatory decisions, this study aimed to identify factors influencing the need for RWE in HTA decision-making, confirm and enrich factors with stakeholder views, and evaluate similarities and differences between regulatory and HTA needs.

Methods: Previous scoping review methodology was used to identify factors influencing the need for RWE in HTA decision-making.

The Metabolic Treatabolome and Inborn Errors of Metabolism Knowledgebase therapy tool: Do not miss the opportunity to treat!

Inborn errors of metabolism (IEMs) are rare genetic conditions with significant morbidity and mortality. Technological advances have increased therapeutic options, making it challenging to remain up to date. A centralized therapy knowledgebase is needed for early diagnosis and targeted treatment.

Neuroimaging of postoperative pediatric cerebellar mutism syndrome: a systematic review.

Background: Postoperative pediatric cerebellar mutism syndrome (ppCMS) poses serious morbidity after posterior fossa tumor surgery. Neuroimaging studies aim to understand its pathophysiology, yet these vary in methodology and outcome measures. Therefore, we systematically reviewed the current literature to evaluate the evidence for differences in neuroimaging features between children with and without ppCMS.

Longitudinal association between nailfold capillaroscopy and incident interstitial lung disease: A EUSTAR database analysis.

Objectives: To evaluate (1) the association between nailfold capillaroscopy pattern and 5-year risk for incident interstitial lung disease and (2) the association between transition in nailfold capillaroscopy pattern and risk of incident interstitial lung disease.

Methods: Data of adult patients from the EUSTAR database fulfilling the ACR-EULAR criteria with a disease duration ⩽5 years, having a scleroderma pattern at nailfold capillaroscopy with high-resolution computed tomography confirmed absence of interstitial lung disease (i.e.

Health recommender systems to facilitate collaborative decision-making in chronic disease management: A scoping review.

Objective: Health recommender systems (HRSs) are increasingly used to complement existing clinical decision-making processes, but their use for chronic diseases remains underexplored. Recognizing the importance of collaborative decision making (CDM) and patient engagement in chronic disease treatment, this review explored how HRSs support patients in managing their illness.

Methods: A scoping review was conducted using the framework proposed by Arksey and O'Malley, advanced by Levac et al.

Symptom management in isocitrate dehydrogenase mutant glioma.

According to the 2021 World Health Organization classification of CNS tumors, gliomas harboring a mutation in isocitrate dehydrogenase (mIDH) are considered a distinct disease entity, typically presenting in adult patients before the age of 50 years. Given their multiyear survival, patients with mIDH glioma are affected by tumor and treatment-related symptoms that can have a large impact on the daily life of both patients and their caregivers for an extended period of time. Selective oral inhibitors of mIDH enzymes have recently joined existing anticancer treatments, including resection, radiotherapy, and chemotherapy, as an additional targeted treatment modality.

Reliability and validity of four cognitive interpretation bias measures in the context of social anxiety.

People with social anxiety disorder tend to interpret ambiguous social information in a negative rather than positive manner. Such interpretation biases may cause and maintain anxiety symptoms. However, there is considerable variability in the observed effects across studies, with some not finding a relationship between interpretation biases and social anxiety.

The challenge of preventing gastric cancer in patients under surveillance for familial adenomatous polyposis.

Several extra-colonic manifestations, including duodenal polyposis and desmoid tumors, are well-described manifestations in familial adenomatous polyposis (FAP). More recently, an increase in gastric cancer diagnoses has been observed in FAP. This case series presents nine patients with FAP who were diagnosed with gastric cancer at our FAP expertise center, of whom eight were diagnosed between 2017 and 2023, while before 2017 the only diagnosis of gastric cancer was in 2001.

New gantry angle-dependent beam control optimization with Elekta linear accelerator for VMAT delivery.

Introduction: This paper describes a method to improve gantry-dependent beam steering for Elekta traveling wave linear accelerators by applying the measured and filtered beam servo corrections to the existing lookup table (LUT). Beam steering has a direct influence on the treatment accuracy by affecting the beam symmetry and position. The presented method provides an improved LUT with respect to the default Elekta method to reduce treatment delivery interruptions.

AI as an intervention: improving clinical outcomes relies on a causal approach to AI development and validation.

The primary practice of healthcare artificial intelligence (AI) starts with model development, often using state-of-the-art AI, retrospectively evaluated using metrics lifted from the AI literature like AUROC and DICE score. However, good performance on these metrics may not translate to improved clinical outcomes. Instead, we argue for a better development pipeline constructed by working backward from the end goal of positively impacting clinically relevant outcomes using AI, leading to considerations of causality in model development and validation, and subsequently a better development pipeline.

Endocrine dysfunction in long-term survivors of pediatric head and neck rhabdomyosarcoma.

Objective: Survivors of pediatric head and neck rhabdomyosarcoma (HNRMS) are at risk of developing endocrinopathies following local treatment, resulting from radiation damage to the pituitary gland, hypothalamus, or thyroid gland, often at a young age. Our aim was to determine the prevalence of endocrine dysfunction in long-term HNRMS survivors and compare the prevalence of anterior pituitary insufficiency (API) among different local treatment strategies: external beam radiation with photons, external beam radiation with protons, microscopically radical surgery combined with external irradiation, and macroscopic radical surgery combined with brachytherapy.

Design And Methods: Head and neck rhabdomyosarcoma survivors treated between 1993 and 2017, with ≥2 years of follow-up, without recurrent disease or secondary malignancy were eligible for this study.

Long-term mortality of Dutch COVID-19 patients admitted to the intensive care medicine: a retrospective analysis from a national quality registry.

Objective: To describe the 12-month mortality of Dutch COVID-19 intensive care unit patients, the total COVID-19 population and various subgroups on the basis of the number of comorbidities, age, sex, mechanical ventilation, and vasoactive medication use.

Methods: We included all patients admitted with COVID-19 between March 1, 2020, and March 29, 2022, from the Dutch National Intensive Care (NICE) database. The crude 12-month mortality rate is presented via Kaplan-Meier survival curves for each patient subgroup.

Moderating factors in psilocybin-assisted treatment affecting mood and personality: A naturalistic, open-label investigation.

Rationale: Psychedelic-assisted therapy is increasingly applied within mental health treatment.

Objectives: This study focused on factors moderating changes in the acute and long-term effects of an individual psilocybin-assisted program on depression, anxiety, PTSD and personality structures by including demographic factors, subjective experience and degree of mystical type experiences during the dosing, as well as emotional breakthrough and personal growth after the program.

Methods: At baseline, 1 week and 3 months after the psilocybin program participants completed the Generalized Anxiety Disorder Assessment (GAD-7), Patient Health Questionnaire (PHQ-9), PTSD Checklist for DSM-5 (PCL-5) and NEO Five-Factor Inventory-3 (NEO-FFI-3).

The bcc coating of Lennard-Jones crystal nuclei vanishes with a change of local structure detection algorithm.

Since the influential work of ten Wolde, Ruiz-Montero, and Frenkel [Phys. Rev. Lett.

Views and opinions of the general public about the reimbursement of expensive medicines in the Netherlands.

Objectives: Solidarity-based healthcare systems are being challenged by the incremental costs of new and expensive medicines for cancer and rare diseases. To regulate reimbursement of such drugs, the Dutch government introduced a policy instrument known as the Coverage Lock (CL) in 2015. Little is known about the public opinion regarding such policy instruments and their consequences, i.

Negative effects by mineral accretion technique on the heat resilience, growth and recruitment of corals.

Restoration and artificial reefs can assist the recovery of degraded reefs but are limited in scalability and climate resilience. The Mineral Accretion Technique (MAT) subjects metal artificial reefs to a low-voltage electrical current, thereby creating a calcium-carbonate coating. It has been suggested that corals on MAT structures experience enhanced health and growth.

Differences in eHealth Access, Use, and Perceived Benefit Between Different Socioeconomic Groups in the Dutch Context: Secondary Cross-Sectional Study.

Background: There is a growing concern that digital health care may exacerbate existing health disparities. Digital health care or eHealth encompasses the digital apps that are used in health care. Differences in access, use, and perceived benefits of digital technology among socioeconomic groups are commonly referred to as the digital divide.

Sensory alterations and immunological changes during the chronification of postsurgical pain: a study protocol for a prospective observational cohort study.

Introduction: Chronic postsurgical pain (CPSP) represents a widely underdiagnosed and often poorly treated medical problem, affecting 10-50% of all surgical patients, exhibiting neuropathic features in 35-60%. It is hypothesised that surgery-induced tissue damage and the subsequent immune response cause sensory alterations in the early postoperative period, ultimately leading to a chronic neuropathic or nociplastic pain state. The 'Sensory Changes and Immunological parameters in Postsurgical pain' study (SCIP-Pain study) was designed to test this hypothesis and identify sensory alterations and changes in the immunological response that are related to the development of CPSP with neuropathic features.

Clinical validation of an artificial intelligence-based decision support system for diagnosis and risk stratification of heart failure (STRATIFYHF): a protocol for a prospective, multicentre longitudinal study.

Introduction: Heart failure (HF) is a complex clinical syndrome. Accurate risk stratification and early diagnosis of HF are challenging as its signs and symptoms are non-specific. We propose to address this global challenge by developing the STRATIFYHF artificial intelligence-driven decision support system (DSS), which uses novel analytical methods in determining the risk, diagnosis and prognosis of HF.

Mouse strain-specific responses along the gut-brain axis upon fecal microbiota transplantation from children with autism.

Several factors are linked to the pathophysiology of autism spectrum disorders (ASD); however, the molecular mechanisms of the condition remain unknown. As intestinal problems and gut microbiota dysbiosis are associated with ASD development and severity, recent studies have focused on elucidating the microbiota-gut-brain axis' involvement. This study aims to explore mechanisms through which gut microbiota might influence ASD.

The Lebanese health economic evaluation guideline.

Background: Economic evaluation guidelines (EEGs) serve as a valuable tool to assist appraisers in making consistent and transparent recommendations, standardize EE studies, enhance their quality, and minimize methodological uncertainties. As other LMICs, Lebanon aims for UHC where EEG is a necessity. This paper aims to report on the Lebanese health EEG (LEEG) and its reference case, including the intermediate results leading to the final decisions.