Raphaël Porcher
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View article: Tool for Implementing TArget Trial emulatioN (TITAN): an open-access design assistant
Tool for Implementing TArget Trial emulatioN (TITAN): an open-access design assistant Open
Background: Target trial emulation offers a structured framework to reduce bias in observational studies by emulating the design of a hypothetical target randomised controlled trial (RCT). Despite a growing interest for this framework, the…
View article: Factors influencing the trustworthiness of non-randomized studies of interventions: a survey of international experts
Factors influencing the trustworthiness of non-randomized studies of interventions: a survey of international experts Open
Background Perceived trustworthiness of research may be influenced by factors beyond the risk of bias, including study-related characteristics, research context, and external circumstances. Identifying these factors is essential for gaugin…
View article: Effect of Baseline Dialysis and Plasma Exchange on Renal Prognosis in Patients With Antineutrophil Cytoplasmic Autoantibody–Associated Vasculitis
Effect of Baseline Dialysis and Plasma Exchange on Renal Prognosis in Patients With Antineutrophil Cytoplasmic Autoantibody–Associated Vasculitis Open
View article: Methods and reporting of studies assessing the impact of adherence to clinical practice guidelines: a scoping review
Methods and reporting of studies assessing the impact of adherence to clinical practice guidelines: a scoping review Open
View article: Evaluating virtual-control-augmented trials for reproducing treatment effect from original RCTs
Evaluating virtual-control-augmented trials for reproducing treatment effect from original RCTs Open
This study investigates the use of virtual patient data to augment control arms in randomised controlled trials (RCTs). Using data from the IST and IST3 trials, we simulated RCTs in which the recruitment in the control arms would stop afte…
View article: Primary Sjögren’s Disease: a review of unmet need, outcome measures, therapeutic advances and health economic impacts. Lessons from the NEw Clinical Endpoints in primary Sjögren’s Syndrome: an Interventional Trial based on stratifYing patients (NECESSITY) Innovative Health Initiative (IHI)
Primary Sjögren’s Disease: a review of unmet need, outcome measures, therapeutic advances and health economic impacts. Lessons from the NEw Clinical Endpoints in primary Sjögren’s Syndrome: an Interventional Trial based on stratifYing patients (NECESSITY) Innovative Health Initiative (IHI) Open
Primary Sjögren's disease (pSjD) is an autoimmune rheumatic disease involving exocrine glands and associated with high symptom burden (dryness, fatigue, pain), systemic features and salivary gland dysfunction. B-cell hyperactivity is commo…
View article: Food additive mixtures and type 2 diabetes incidence: Results from the NutriNet-Santé prospective cohort
Food additive mixtures and type 2 diabetes incidence: Results from the NutriNet-Santé prospective cohort Open
Background Mixtures of food additives are daily consumed worldwide by billions of people. So far, safety assessments have been performed substance by substance due to lack of data on the effect of multiexposure to combinations of additives…
View article: Optimal treatment regimes for the net benefit of a treatment
Optimal treatment regimes for the net benefit of a treatment Open
We develop a mathematical framework to define an optimal individualized treatment rule (ITR) within the context of prioritized outcomes in a randomized controlled trial. Our optimality criterion is based on the framework of generalized pai…
View article: Decision‐Making Criteria and Methods for Initiating Late‐Stage Clinical Trials in Drug Development From a Multi‐Stakeholder Perspective: A Scoping Review
Decision‐Making Criteria and Methods for Initiating Late‐Stage Clinical Trials in Drug Development From a Multi‐Stakeholder Perspective: A Scoping Review Open
The decision‐making process in drug development involves “go/no‐go” decisions, particularly at the transition from early to late‐stage trials. While the decisions are solely made by drug developers, they must take into account the perspect…
View article: On the Concepts, Methods, and Use of “Probability of Success” for Drug Development Decision‐Making: A Scoping Review
On the Concepts, Methods, and Use of “Probability of Success” for Drug Development Decision‐Making: A Scoping Review Open
Drug development is a lengthy process with considerable uncertainty at each milestone. Several trials are needed to progress to confirmatory evaluation and establish a positive benefit–risk balance. One of the critical milestones is the de…
View article: A scoping review of robustness concepts for machine learning in healthcare
A scoping review of robustness concepts for machine learning in healthcare Open
View article: Methods to Estimate When to Stop a Treatment and to Compare Treatment Durations Using Observational Data: A Methodological Review
Methods to Estimate When to Stop a Treatment and to Compare Treatment Durations Using Observational Data: A Methodological Review Open
View article: Methods of nivolumab administration in advanced melanoma: A comparison of patients’ clinical outcomes treated with flat dose or weight‐adjusted dose, a multicenter observational study
Methods of nivolumab administration in advanced melanoma: A comparison of patients’ clinical outcomes treated with flat dose or weight‐adjusted dose, a multicenter observational study Open
Background Nivolumab obtained approval in advanced melanoma (AM) with weight‐adjusted dose (WAD) administration (3 mg/kg/2 weeks). In 2018, the dosage regimen was changed to flat dose (FD) administration (240 mg/2 weeks or 480 mg/4 weeks) …
View article: Methodological expectations for demonstration of health product effectiveness by observational studies
Methodological expectations for demonstration of health product effectiveness by observational studies Open
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. I…
View article: When to stop immunotherapy for advanced melanoma: the emulated target trials
When to stop immunotherapy for advanced melanoma: the emulated target trials Open
This work was supported by a grant from Bristol Myers Squibb, Merck Sharp Dhome, Pierre Fabre, Novartis, Sun Pharm, Regeneron, Sanofi, Nektar, Therapeutics and Oncyte.
View article: Benchmarking covariates balancing methods, a simulation study
Benchmarking covariates balancing methods, a simulation study Open
Causal inference in observational studies has advanced significantly since Rosenbaum and Rubin introduced propensity score methods. Inverse probability of treatment weighting (IPTW) is widely used to handle confounding bias. However, newer…
View article: Glucocorticoids versus glucocorticoids plus cyclophosphamide in eosinophilic granulomatosis with polyangiitis with poor-prognosis factors
Glucocorticoids versus glucocorticoids plus cyclophosphamide in eosinophilic granulomatosis with polyangiitis with poor-prognosis factors Open
View article: G-computation for increasing performances of clinical trials with individual randomization and binary response
G-computation for increasing performances of clinical trials with individual randomization and binary response Open
In a clinical trial, the random allocation aims to balance prognostic factors between arms, preventing true confounders. However, residual differences due to chance may introduce near-confounders. Adjusting on prognostic factors is therefo…
View article: Two forced expiratory volume in 1 s trajectories with distinct prognoses in pulmonary Langerhans cell histiocytosis
Two forced expiratory volume in 1 s trajectories with distinct prognoses in pulmonary Langerhans cell histiocytosis Open
Background Lung function outcomes in pulmonary Langerhans cell histiocytosis (PLCH) patients are variable and difficult to predict. Our goal was to identify different forced expiratory volume in 1 s (FEV 1 ) trajectories in these patients …
View article: The PERMIT guidelines for designing and implementing all stages of personalised medicine research
The PERMIT guidelines for designing and implementing all stages of personalised medicine research Open
View article: Attentes méthodologiques pour la démonstration de l’efficacité des produits de santé par les études observationnelles
Attentes méthodologiques pour la démonstration de l’efficacité des produits de santé par les études observationnelles Open
View article: Characteristics of non-randomised studies of drug treatments: cross sectional study
Characteristics of non-randomised studies of drug treatments: cross sectional study Open
Objective To examine the characteristics of comparative non-randomised studies that assess the effectiveness or safety, or both, of drug treatments. Design Cross sectional study. Data sources Medline (Ovid), for reports published from 1 Ju…
View article: European Study of Cerebral Aspergillosis treated with Isavuconazole (ESCAI): A study by the ESCMID Fungal Infection Study Group
European Study of Cerebral Aspergillosis treated with Isavuconazole (ESCAI): A study by the ESCMID Fungal Infection Study Group Open
Background Cerebral aspergillosis (CA) is associated with high mortality. According to the European Conference on Infections in Leukemia and the European Society of Clinical Microbiology and Infectious Diseases guidelines, the recommended …
View article: Long-term functional course of Sjögren's disease-associated interstitial lung disease
Long-term functional course of Sjögren's disease-associated interstitial lung disease Open
Background Interstitial lung disease (ILD) is common in primary Sjögren's disease (pSD); its functional course is poorly known. Our aim was to characterise the long-term functional course and prognosis in patients with pSD-ILD. We determin…
View article: Editorial: Uses of Generative Artificial Intelligence in Clinical Orthopaedics and Related Research ®—An Update
Editorial: Uses of Generative Artificial Intelligence in Clinical Orthopaedics and Related Research ®—An Update Open
Last year, Clinical Orthopaedics and Related Research® partnered with three other leading orthopaedic journals—JBJS, BJJ, and JOR—to articulate some preliminary standards about using artificial intelligence (AI) applications in the creatio…
View article: Estimating individualized treatment effects using an individual participant data meta-analysis
Estimating individualized treatment effects using an individual participant data meta-analysis Open
View article: Do machine learning methods lead to similar individualized treatment rules? A comparison study on real data
Do machine learning methods lead to similar individualized treatment rules? A comparison study on real data Open
Identifying patients who benefit from a treatment is a key aspect of personalized medicine, which allows the development of individualized treatment rules (ITRs). Many machine learning methods have been proposed to create such rules. Howev…
View article: Immunomodulators for immunocompromised patients hospitalized for COVID-19: a meta-analysis of randomized controlled trials
Immunomodulators for immunocompromised patients hospitalized for COVID-19: a meta-analysis of randomized controlled trials Open
Hellenic Foundation for Research and Innovation.
View article: Serum and Tissue Biomarkers Associated With Composite of Relevant Endpoints for Sjögren Syndrome (CRESS) and Sjögren Tool for Assessing Response (STAR) to B Cell–Targeted Therapy in the Trial of Anti–B Cell Therapy in Patients With Primary Sjögren Syndrome (TRACTISS)
Serum and Tissue Biomarkers Associated With Composite of Relevant Endpoints for Sjögren Syndrome (CRESS) and Sjögren Tool for Assessing Response (STAR) to B Cell–Targeted Therapy in the Trial of Anti–B Cell Therapy in Patients With Primary Sjögren Syndrome (TRACTISS) Open
Objective This study aimed to identify peripheral and salivary gland (SG) biomarkers of response/resistance to B cell depletion based on the novel concise Composite of Relevant Endpoints for Sjögren Syndrome (cCRESS) and candidate Sjögren …
View article: More rapid blood interferon α2 decline in fatal versus surviving COVID-19 patients
More rapid blood interferon α2 decline in fatal versus surviving COVID-19 patients Open
Background The clinical outcome of COVID-19 pneumonia is highly variable. Few biological predictive factors have been identified. Genetic and immunological studies suggest that type 1 interferons (IFN) are essential to control SARS-CoV-2 i…