Impact of Islet Transplantation on Diabetes Complications and Mortality in Patients Living With Type 1 Diabetes.

Objective: This study aimed to evaluate the impact of islet transplantation (IT) on diabetes complications, death, and cancer incidence.

Research Design And Methods: This retrospective, multicenter, cohort study included patients from three IT clinical trials (intervention group) and from the French health insurance claims database Système National des Données de Santé (SNDS) (control group). Two cohorts of IT recipients were analyzed: IT recipients after kidney transplantation (IAK) and IT recipients alone (ITA).

Perceptions of contribution of the placebo effects to drug efficacy in community pharmacy practice: A cross-sectional survey.

Introduction: The placebo effect, a well-documented phenomenon in clinical practice, remains complex and multifaceted. While the perception of placebo effects has been assessed among various healthcare professionals, pharmacists - recognized experts in treatment effects and effectiveness - have not been specifically studied. This cross-sectional study aimed to evaluate the knowledge and perceptions of healthcare professionals working in community pharmacies in France, Switzerland, and Belgium regarding placebo effects.

Electronic pharmaceutical record for best possible medication history at preoperative evaluation to prevent postoperative adverse events: a quasi-experimental study.

Background: Access to reliable data about patient's medications before surgery represents a challenge for reducing the risk of postoperative adverse events (AE) potentially related to preoperative treatment.

Objective: To evaluate the impact on AE of a nationwide ambulatory electronic pharmaceutical record (EPR) used by a pharmacist for best possible medication history (BPMH), associated with the preoperative evaluation.

Methods: This quasi-experimental comparative interventional study included 750 adult patients with an available EPR, admitted to the preoperative clinic for elective orthopaedic surgery, between April 2014 and April 2017.

Trial-level surrogacy of non-high-density and low-density lipoprotein cholesterol reduction on the clinical efficacy of statins.

LDL cholesterol (LDL - c) and non-HDL cholesterol (non-HDL-c) are prognostic factors of cardiovascular risk. However, their validity as trial-level surrogates for cardiovascular outcomes is debated. This study aimed to determine whether LDL - c and non-HDL-c are reliable surrogates for cardiovascular events in statin trials, and to explore discrepancies in previous studies.

Are Causal Statements Reported in Pharmacovigilance Disproportionality Analyses Using Individual Case Safety Reports Exaggerated in Related Citations? A Meta-epidemiological Study.

Background: Previous meta-epidemiological surveys have found considerable misinterpretation of results of disproportionality analyses. We aim to explore the relationship between the strength of causal statements used in title and abstract conclusions of pharmacovigilance disproportionality analyses and the strength of causal language used in citing studies.

Methods: On March 30, 2022, we selected the 30 disproportionality studies with the highest Altmetric Attention Scores.

Priors and decision thresholds in phase 2 and phase 3 randomized controlled trials evaluating drug efficacy using Bayesian methods: a systematic review.

Objectives: To describe the priors and decision thresholds in phase 2 and 3 randomized controlled trials (RCTs) evaluating drug efficacy using Bayesian methods.

Study Design And Setting: A systematic review of phase 2 and 3 RCTs evaluating drug efficacy through Bayesian inference was conducted across the MEDLINE, EMBASE, and Cochrane databases, with no date restrictions until September 2022. The type of prior used for the analysis of the primary endpoint and its characteristics (type and parameters of the distribution, justification, and sensitivity analysis), the use of a posterior probability decision threshold defined a priori, and its value, were extracted.

Methodological expectations for demonstration of health product effectiveness by observational studies.

The issue of assessing the effectiveness of health technologies (drugs, devices, etc.) through observational studies is becoming increasingly important as registration and market access agencies consider them in their evaluation process. In this context, observational studies must be able to provide real demonstrations of a level of reliability comparable to those produced by the conventional randomized controlled trial (RCT) approach.

Micro- and macrovascular function in the highest city in the world: a cross sectional study.

Background: Since vascular responses to hypoxia in both healthy high-altitude natives and chronic mountain sickness (a maladaptive high-altitude pathology characterised by excessive erythrocytosis and the presence of symptoms-CMS) remain unclear, the role of inflammation and oxidative/nitrosative stress on the endothelium- and - responses in both the micro- and macrocirculation, in healthy Andeans at different altitudes and in CMS patients, was examined.

Methods: 94 men were included: 18 lowlanders (LL), 38 healthy highlanders permanently living at 3800 m (n = 21-HL-3800) or in La Rinconada, the highest city in the world (5100-5300 m) (n = 17-HL-5100/No CMS). Moreover, 14 participants with mild (Mild CMS) and 24 with moderate to severe CMS (Mod/Sev CMS) were recruited.

Regional Heterogeneity of the Results of Glucagon-Like Peptide 1 Receptor Agonist Trials in Type 2 Diabetes: A Reanalysis of Individual Participant Data.

Objective: Multiregional trials are designed under the assumption that treatment effect applies to the entire target population, yet several factors may introduce geographic heterogeneity in treatment effect. We explored whether such variations exist in trials assessing the efficacy of glucagon-like peptide 1 receptor agonists (GLP-1RAs) in major cardiovascular events (MACE) in type 2 diabetes.

Research Design And Methods: A systematic search of Medline and the Cochrane Library was conducted from inception until 30 June 2020.

The evidence base of the 10 most prescribed drugs in England, France, and the United States: a scoping review.

Objectives: To evaluate whether there is evidence of efficacy of the most commonly used medications in their primary indications.

Study Design And Setting: This scoping review was executed using the Cochrane Library and MEDLINE databases up to May 2023. The 10 most prescribed medications in England, France, and the United States were identified using country-specific public databases.

Assessing the benefit-risk balance of drugs. Some lessons from the COVID pandemic.

Introduction: Drug efficacy and effectiveness are assessed respectively through clinical trials and pharmaco-epidemiological studies. However, relative and absolute benefits of drugs are distinct measures that must be considered in relation to the baseline risk of disease incidence, complication or progression. On the other hand, adverse drug reactions are independent of the basic risk but depend on the characteristics of the population treated.

Mouse models of diabetes-related ulcers: a systematic review and network meta-analysis.

Background: Diabetic foot ulcers (DFUs) are a common complication of diabetes, associated with important morbidity. Appropriate animal models of DFUs may improve drug development, and subsequently the success rate of clinical trials. However, while many models have been proposed, they are extremely heterogeneous, and no standard has emerged.

Radiological diagnosis of hepatocellular carcinoma does not preclude biopsy before treatment.

Background & Aims: The diagnosis of hepatocellular carcinoma (HCC) in patients with cirrhosis relies on non-invasive criteria based on international guidelines. The advent of systemic therapies warrants reconsideration of the role of biopsy specimens in the diagnosis of HCC. Accordingly, we investigated the diagnostic performance of the LI-RADS 2018 and the AASLD 2011 criteria.

Data Monitoring Committees and clinical trials: From scientific justification to organisation.

Clinical trials often last several months or even several years. As the trial progresses, it can be tempting to find out whether the data obtained already answers the question posed at the start of the trial in order to stop inclusions or monitoring earlier. However, knowing and taking into account interim results can sometimes compromise the integrity of the results, which is counterproductive.

Use of Bisphosphonates and the Risk of Skin Ulcer: A National Cohort Study Using Data from the French Health Care Claims Database.

Introduction: Previous pre-clinical and pharmacovigilance disproportionality analyses highlighted a safety signal of cutaneous ulcer with bisphosphonate use. Therefore, our objective is to evaluate this risk and assess whether unmeasured confounding factors could explain this association.

Methods: This study is a population-based cohort study from a representative sample (1/97th) of the French health insurance claims database: Echantillon Généraliste des Bénéficiaires (EGB) from 2006 to 2019.

Assessment of digital perfusion as a surrogate outcome in Raynaud's phenomenon clinical trials.

Objectives: Measurement of digital perfusion, sometimes coupled with a cold challenge, has been widely used as an objective outcome in trials evaluating drug therapies in RP, in addition to patient-reported outcomes or to establish the proof-of-concept in preliminary studies. However, whether digital perfusion is a valid surrogate for clinical outcomes in RP trials has never been explored. The principal aim of this study was to evaluate the potential surrogacy of digital perfusion, by combining individual-level and trial-level data.

Individualized Net Benefit estimation and meta-analysis using generalized pairwise comparisons in N-of-1 trials.

Background: The Net Benefit (Δ) is a measure of the benefit-risk balance in clinical trials, based on generalized pairwise comparisons (GPC) using several prioritized outcomes and thresholds of clinical relevance. We extended Δ to N-of-1 trials, with a focus on patient-level and population-level Δ.

Methods: We developed a Δ estimator at the individual level as an extension of the stratum-specific Δ, and at the population-level as an extension of the stratified Δ.