A5037349205- Health Systems, Economic Evaluations, Quality of Life
- Lymphoma Diagnosis and Treatment
- Chronic Lymphocytic Leukemia Research
- Multiple Myeloma Research and Treatments
- Statistical Methods in Clinical Trials
- Chronic Obstructive Pulmonary Disease (COPD) Research
- Retinoids in leukemia and cellular processes
- Psychometric Methodologies and Testing
- Multiple Sclerosis Research Studies
- Hereditary Neurological Disorders
- Cognitive Abilities and Testing
- Pain Management and Placebo Effect
- Respiratory and Cough-Related Research
- Gastrointestinal Tumor Research and Treatment
- Advanced Causal Inference Techniques
- BRCA gene mutations in cancer
- Pain Mechanisms and Treatments
- Diverse Approaches in Healthcare and Education Studies
- Palliative Care and End-of-Life Issues
- Acute Lymphoblastic Leukemia research
- Advanced Breast Cancer Therapies
- Schizophrenia research and treatment
- Cancer Treatment and Pharmacology
- Rheumatoid Arthritis Research and Therapies
- Biomedical Ethics and Regulation
Pathophysiology, Diagnosis and Treatment of Bone Diseases
2019-2024
Centre Hospitalier Universitaire de Tours
2022
Nantes Université
2021
Sciences, Philosophie, Histoire
2020
Inserm
2020
Background and purpose Guillain-Barré syndrome (GBS) is an autoimmune neurological disorder characterized by muscle weakness. In clinical trials, treatment benefit disease severity are typically measured using clinician-reported outcome measures like the Hughes Functional Grading Scale (HFGS). However, patient-reported measures, such as Rasch-built Overall Disability (R-ODS) may provide additional insight into patient experience during treatment. this study, exploratory analyses of trial...
Introduction The Cohen-Mansfield Agitation Inventory (CMAI) quantifies the frequency of agitation behaviors in elderly persons. This post hoc analysis data from brexpiprazole clinical program aimed to determine a meaningful within-patient change (MWPC) threshold for CMAI Total score among patients with associated dementia due Alzheimer’s disease. Methods Data were included three 12-week, multicenter, randomized, double-blind, placebo-controlled, parallel-arm trials treatment disease (...
Abstract Background The patient-reported outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) is used to assess symptomatic adverse events in oncology trials. Currently, no standard PRO-CTCAE analysis exists. Methods Key methods descriptive and longitudinal modeling using data from an clinical trial, DRiving Excellence Approaches Multiple Myeloma-2 (DREAMM-2), a phase II trial belantamab mafodotin multiple myeloma (NCT03525678), were explored. Descriptive...
Abstract Background As disease-modifying therapies do not reverse the course of multiple sclerosis (MS), assessment therapeutic success involves documenting patient-reported outcomes (PROs) concerning health-related quality life, disease and treatment-related symptoms, impact symptoms on function. Interpreting PRO data going beyond statistical significance to calculate within-patient meaningful change scores. These thresholds are needed for each in order fully interpret data. This analysis...
Trials of disease-modifying therapies (DMTs) for multiple sclerosis (MS) often include patients with minimal disability. Patient-reported outcome instruments used in these trials have not captured physical and psychological treatment effects concomitant observed clinical benefits.To examine whether the Multiple Sclerosis Impact Scale-29 (MSIS-29) captures changes impact MS a sample enrolled Phase 3 ASCLEPIOS studies (ofatumumab vs. teriflunomide).Measurement properties (i.e. item fit,...
Meaningfully interpreting patient-reported outcomes (PRO) results from randomized clinical trials requires that the PRO scores obtained in trial have same meaning across patients and previous applications of instrument. Calibration instruments warrants this property. In Rasch measurement theory (RMT) framework, calibration is performed by fixing item parameter estimates when measuring targeted concept for each individual trial. The used purpose are typically a "calibration" study. But...
Abstract Background This psychometric analysis generated evidence to support the use of Functional Assessment Cancer Therapy item GP5 (GP5) as a measure tolerability and confirms appropriateness categorizing “high side-effect burden” using rating 3 or 4 (score ranges 0–4) in patients with advanced/metastatic RET- mutant medullary thyroid cancer (MTC). Methodology Blinded, pooled interim data from safety population ( n =290) enrolled phase LIBRETTO-531 trial (NCT04211337) were used....
e18587 Background: The PRO-CTCAE is a patient-reported outcome measure of symptomatic toxicity in oncology trials designed to complement CTCAE criteria (clinician’s adverse events [AEs]). Items for evaluation are selected from 78 toxicities the CTCAE. uptake and use has not been uniformly assessed; understanding how best analyze present data important. We reviewed literature on clinical reporting ascertain its current analytical methods inform future analyses. Methods: A review ongoing using...
Topic: 18. Indolent and mantle-cell non-Hodgkin lymphoma - Clinical Background: BRUIN 18001 (NCT03740529) is an ongoing, open-label, multi-center phase 1/2 study investigating the safety efficacy of pirtobrutinib for treatment B-cell malignancies, including mantle cell (MCL). Pirtobrutinib a non-covalent (reversible) Bruton tyrosine kinase inhibitor (BTKi) that has recently received regulatory approval in United States (US) patients with relapsed or refractory MCL after two more lines...
Abstract Background In the analysis of clinical trial endpoints, calibration patient-reported outcomes (PRO) instruments ensures that resulting “scores” represent same quantity measured concept between applications. Rasch measurement theory (RMT) is a psychometric approach guarantees algebraic separation person and item parameter estimates, allowing formal PRO instruments. RMT framework, performed using estimates obtained from previous “calibration” study. But if based on poorly estimated...