Publications by authors named "Bourgeois F"

Background: Patient notes contain substantial information but are difficult for computers to analyse due to their unstructured format. Large-language models (LLMs), such as Generative Pre-trained Transformer 4 (GPT-4), have changed our ability to process text, but we do not know how effectively they handle medical notes. We aimed to assess the ability of GPT-4 to answer predefined questions after reading medical notes in three different languages.

View Article and Find Full Text PDF
Article Synopsis
  • Cell and gene therapies (CGTs) have shown potential for treating hard-to-treat conditions, but their development is risky for pharmaceutical companies due to uncertainties in clinical trials and regulatory approval.
  • An analysis of CGT products from 1993 to 2023 found that only 5.3% received regulatory approval, with higher chances for products with orphan designation and lower chances for oncology-related therapies.
  • Both CAR T cell therapies and AAV gene therapies showed a similar approval likelihood of 13.6%, highlighting that approval rates vary by product type and therapeutic indication.
View Article and Find Full Text PDF

Purpose: United States healthcare systems have enacted varied adolescent online patient portal policies. No prior work has established whether these policies are acceptable to adolescents and parents.

Methods: Mixed-method Delphi approach with 18 parents and 18 adolescents with and without chronic illness.

View Article and Find Full Text PDF

Objective: To examine the relationship between the Child Opportunity Index (COI) and unintentional poisonings in a geographically diverse pediatric population.

Study Design: We conducted a retrospective, case-control study of children ≤ 6 years of age who had emergency department encounters for poisonings from January 1, 2016, to December 31, 2021. Data were obtained from the Pediatric Health Information System database.

View Article and Find Full Text PDF

Countries with small and/or less-resourced regulatory authorities that operate outside of a larger medical product regulatory system face a regulatory strategy dilemma. These countries may rely on foreign well-resourced regulators by recognising the regulatory decisions of large systems and following suit (regulatory reliance); alternatively, such countries may extend formal decision recognition to regulators in multiple other jurisdictions with similar oversight and public health goals, following a system which we call regulatory pluralism. In this policy comment, we discuss three potential limitations to regulatory pluralism: (i) regulatory escape, in which manufacturers exploit regulatory variation and choose the lowest regulatory threshold for their product; (ii) increased fragmentation and complexity for countries adopting this approach, which may, in turn, lead to inconsistent processes; and (iii) loss of international bargaining power in developing regulatory policies.

View Article and Find Full Text PDF

Background: The existing data on enrollment trends of historically underserved racial and ethnic children in clinical trials are limited.

Objective: We sought to evaluate documentation and representation of race and ethnicity in pediatric asthma clinical trials in the United States.

Methods: This is a cross-sectional study of United States-based interventional trials studying pediatric asthma that were completed between 2008 and 2022 and registered on ClinicalTrials.

View Article and Find Full Text PDF

The 3CL protease (3CL, M) plays a key role in the replication of the SARS-CoV-2 and was validated as therapeutic target by the development and approval of specific antiviral drugs (nirmatrelvir, ensitrelvir), inhibitors of this protease. Moreover, its high conservation within the coronavirus family renders it an attractive therapeutic target for the development of anti-coronavirus compounds with broad spectrum activity to control COVID-19 and future coronavirus diseases. Here we report on the design, synthesis and structure-activity relationships of a new series of small covalent reversible inhibitors of the SARS-CoV-2 3CL.

View Article and Find Full Text PDF
Article Synopsis
  • Consensus statements are important in medicine and public health, but not all use solid evidence to support their claims.
  • Some statements rely on expert panels, which can be biased if many members share the same opinions or interests, especially without a thorough review of evidence.
  • A recent case about COVID-19 showed that many panel members had strong connections to groups pushing for strict COVID measures without revealing these biases, highlighting the need for clear conflicts of interest to ensure trustworthiness.
View Article and Find Full Text PDF

When the primary endpoints in randomized clinical trials require long term follow-up or are costly to measure, it is often desirable to assess treatment effects on surrogate instead of clinical endpoints. Prior to adopting a surrogate endpoint for such purposes, the extent of its surrogacy on the primary endpoint must be assessed. There is a rich statistical literature on assessing surrogacy in the overall population, much of which is based on quantifying the proportion of treatment effect on the primary endpoint that is explained by the treatment effect on the surrogate endpoint.

View Article and Find Full Text PDF

Background: Sickle cell disease (SCD) is one of the most frequent inherited diseases in the world. Over the last decades, in high-income countries, an important decrease in mortality have been observed due to the improvement of care. However, children with SCD can become critically ill and require admission in Pediatric Intensive Care Units (PICU).

View Article and Find Full Text PDF

The utilization of the human immune system as a therapeutic modality has materialized in the form of novel biologics known as immune effector cells (IECs). However, currently approved IECs rely on autologous cells for manufacturing that are funneled through costly centralized supply chains leading to long wait times and potentially increased mortality. Alternative models for manufacturing at or near the point-of-care in a distributed and local approach are being proposed to overcome such a bottleneck.

View Article and Find Full Text PDF

Objective: To identify perceived benefits, problems, facilitators, and barriers to adolescent online patient portal use.

Study Design: Qualitative, semi-structured interviews with dyads of parents and adolescents with or without chronic illness. The study team purposively sampled for racial and ethnic minorities and fathers.

View Article and Find Full Text PDF

Background: Web-based patient portals are tools that could support adolescents in managing their health and developing autonomy. However, informatics administrators must navigate competing interests when developing portal access policies for adolescents and their parents.

Objective: We aimed to assess the perspectives of informatics administrators on guiding principles for the development of web-based health care portal access policies in adolescent health care.

View Article and Find Full Text PDF

Importance: Concerns over the mental health of young people have been increasing over the past decade, especially with the rise in mental health burden seen during the COVID-19 pandemic. Examining trends in mental health-related outpatient visits provides critical information to elucidate contributing factors, identify vulnerable populations, and inform strategies to address the mental health crisis.

Objective: To examine characteristics and trends in mental health-related outpatient visits and psychotropic medication use among US adolescents and young adults.

View Article and Find Full Text PDF

Objectives: Accurate and timely diagnosis relies on close collaboration between patients/families and clinicians. Just as patients have unique insights into diagnostic breakdowns, positive patient feedback may also generate broader perspectives on what constitutes a "good" diagnostic process (DxP).

Methods: We evaluated patient/family feedback on "what's going well" as part of an online pre-visit survey designed to engage patients/families in the DxP.

View Article and Find Full Text PDF

Triazolinediones are an important class of derivatization agents that have found application in various research disciplines. Their unique reactivity often allows precise and selective tagging of relevant molecular scaffolds to facilitate structural elucidation, tracking in biological systems, and stabilization of labile compounds. Recent research efforts mainly focused on the development of novel fluorescent and ionizable or isotopically labeled tags improving the quantification and identification of the parent molecule by suitable analytical methods.

View Article and Find Full Text PDF

Background: Patients and families at risk for health disparities may also be at higher risk for diagnostic errors but less likely to report them.

Objectives: This study aimed to explore differences in race, ethnicity, and language preference associated with patient and family contributions and concerns using an electronic previsit tool designed to engage patients and families in the diagnostic process (DxP).

Methods: Cross-sectional study of 5,731 patients and families presenting to three subspecialty clinics at an urban pediatric hospital May to December 2021 who completed a previsit tool, codeveloped and tested with patients and families.

View Article and Find Full Text PDF