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3342. Twenty-five years of novel drug approvals in rheumatology.
作者: Kathryn Henry.;Natalie Anumolu.;Michael Putman.
来源: Rheumatology (Oxford). 2023年62卷12期3801-3803页
The field of rheumatology has experienced dozens of novel drug approvals in the past two and a half decades, but the regulatory mechanisms underpinning these decisions are not well understood. In the USA, the Food and Drug Administration (FDA) evaluates the safety and efficacy of novel drugs through the New Drug Application (NDA) process. When additional content expertise is required to evaluate scientific or technical matters, the FDA may convene Human Drug Advisory Committees. To better understand the landscape of rheumatology NDAs and the FDA use of advisory committees, we performed a review of all rheumatic disease drug applications from 1996 to 2021 that were granted approval by the FDA. Our review identified 31 NDAs, seven of which utilized an advisory committee. The indications for using advisory committees and their influence on ultimate approvals was not clear. Recommendations to improve transparency and increase public trust in FDA decisions are provided.
3343. Assessment of digital perfusion as a surrogate outcome in Raynaud's phenomenon clinical trials.
作者: Alicia Guigui.;Léa Liaigre.;Marc Manceau.;Olivier Gaget.;Jean-Luc Cracowski.;Sophie Blaise.;Charles Khouri.;Matthieu Roustit.
来源: Rheumatology (Oxford). 2024年63卷6期1502-1506页
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.
3346. Temporal trends in mortality in patients with rheumatoid arthritis: a Danish population-based matched cohort study.
作者: Bolette G Soussi.;Kirsten Duch.;René L Cordtz.;Jesper Lindhardsen.;Salome Kristensen.;Christian S Bork.;Asta Linauskas.;Erik B Schmidt.;Lene Dreyer.
来源: Rheumatology (Oxford). 2024年63卷4期1049-1057页
To investigate the 5-year all-cause mortality in patients with RA compared with the general population.
3348. Infection incidence, timing and dose dependency in rheumatoid arthritis patients treated with rituximab: a retrospective cohort study.
作者: Lara D Veeken.;Merel A A Opdam.;Lise M Verhoef.;Calin Popa.;Reinout van Crevel.;Alfons A den Broeder.
来源: Rheumatology (Oxford). 2024年63卷5期1246-1250页
Rituximab (RTX) is a safe and effective treatment for RA. However, there are some concerns about infection risk and preliminary data suggest dose and time dependency. This study aims to determine the infection incidence in a large real-life population of RA patients using RTX, with special focus on (ultra-)low dosing and time since last infusion.
3350. The bone marrow side of axial spondyloarthritis.
作者: Daniele Mauro.;Saviana Gandolfo.;Enrico Tirri.;Georg Schett.;Walter P Maksymowych.;Francesco Ciccia.
来源: Nat Rev Rheumatol. 2023年19卷8期519-532页
Spondyloarthritis (SpA) is characterized by the infiltration of innate and adaptive immune cells into entheses and bone marrow. Molecular, cellular and imaging evidence demonstrates the presence of bone marrow inflammation, a hallmark of SpA. In the spine and the peripheral joints, bone marrow is critically involved in the pathogenesis of SpA. Evidence suggests that bone marrow inflammation is associated with enthesitis and that there are roles for mechano-inflammation and intestinal inflammation in bone marrow involvement in SpA. Specific cell types (including mesenchymal stem cells, innate lymphoid cells and γδ T cells) and mediators (Toll-like receptors and cytokines such as TNF, IL-17A, IL-22, IL-23, GM-CSF and TGFβ) are involved in these processes. Using this evidence to demonstrate a bone marrow rather than an entheseal origin for SpA could change our understanding of the disease pathogenesis and the relevant therapeutic approach.
3352. Gut microbiota alterations are associated with phenotype and genotype in familial Mediterranean fever.
作者: Marion Delplanque.;Nicolas Benech.;Nathalie Rolhion.;Cyriane Oeuvray.;Marjolène Straube.;Chloé Galbert.;Loic Brot.;Thomas Henry.;Yvan Jamilloux.;Léa Savey.;Gilles Grateau.;Harry Sokol.;Sophie Georgin-Lavialle.
来源: Rheumatology (Oxford). 2024年63卷4期1039-1048页
FMF is the most common monogenic autoinflammatory disease associated with MEFV mutations. Disease phenotype and response to treatment vary from one patient to another, despite similar genotype, suggesting the role of environmental factors. The objective of this study was to analyse the gut microbiota of a large cohort of FMF patients in relation to disease characteristics.
3353. The influence of biological DMARDs on aseptic arthroplasty loosening: a retrospective cohort study.
作者: Markus M Schreiner.;Jennifer Straub.;Sebastian Apprich.;Kevin Staats.;Reinhard Windhager.;Daniel Aletaha.;Christoph Böhler.
来源: Rheumatology (Oxford). 2024年63卷4期970-976页
To investigate whether biological DMARDs affect the risk of aseptic loosening after total hip/knee arthroplasty (THA/TKA) in patients with RA.
3354. Physical function and severe side effects matter most to patients with RA (< 5 years): a discrete choice experiment assessing preferences for personalized RA treatment.
作者: Karin Schölin Bywall.;Bente Appel Esbensen.;Marie Heidenvall.;Inger Erlandsson.;Marta Lason.;Mats Hansson.;Jennifer Viberg Johansson.
来源: BMC Rheumatol. 2023年7卷1期17页
Early assessment of patient preferences has the potential to support shared decisions in personalized precision medicine for patients with rheumatoid arthritis (RA). The aim of this study was to assess treatment preferences of patients with RA (< 5 years) with previous experience of inadequate response to first-line monotherapy.
3356. Efficacy and safety of telitacicept in primary Sjögren's syndrome: a randomized, double-blind, placebo-controlled, phase 2 trial.
作者: Dong Xu.;Jianmin Fang.;Shangzhu Zhang.;Cibo Huang.;Chenghui Huang.;Li Qin.;Xiaomei Li.;Meiqing Chen.;Xiumei Liu.;Yi Liu.;Zhijun Li.;Jiankang Hu.;Chunde Bao.;Wei Wei.;Jing Tian.;Xinwang Duan.;Xiaofeng Zeng.
来源: Rheumatology (Oxford). 2024年63卷3期698-705页
To evaluate the efficacy and safety of telitacicept in adult patients with primary SS (pSS) in a phase II randomized double-blind placebo-controlled trial.
3357. Lung ultrasound compared to computed tomography detection and automated quantification of systemic sclerosis-associated interstitial lung disease: preliminary study.
作者: Davide Mohammad Reza Beigi.;Greta Pellegrino.;Michele Loconte.;Nicholas Landini.;Monica Mattone.;Gregorino Paone.;Simona Truglia.;Francesca Romana Di Ciommo.;Ilaria Bisconti.;Marius Cadar.;Katia Stefanantoni.;Valeria Panebianco.;Fabrizio Conti.;Valeria Riccieri.
来源: Rheumatology (Oxford). 2024年63卷5期1240-1245页
Lung ultrasound (LUS) is a promising tool for detecting SSc-associated interstitial lung disease (SSc-ILD). Currently, consensus on the best LUS findings and execution technique is lacking.
3358. Post-COVID condition in patients with inflammatory rheumatic diseases: a prospective cohort study in the Netherlands.
作者: Laura Boekel.;Sadaf Atiqi.;Maureen Leeuw.;Femke Hooijberg.;Yaëlle R Besten.;Rosa Wartena.;Maurice Steenhuis.;Erik Vogelzang.;Casper Webers.;Annelies Boonen.;Martijn Gerritsen.;Willem F Lems.;Sander W Tas.;Ronald F van Vollenhoven.;Alexandre E Voskuyl.;Irene van der Horst-Bruinsma.;Mike Nurmohamed.;Theo Rispens.;Gertjan Wolbink.
来源: Lancet Rheumatol. 2023年5卷7期e375-e385页
Studies on long-term consequences of COVID-19, commonly referred to as post-COVID condition, in patients with inflammatory rheumatic diseases are scarce and inconclusive. Furthermore, classifying patients with inflammatory rheumatic diseases as having post-COVID condition is complicated because of overlapping symptoms. Therefore, we investigated the risk of post-COVID condition and time until recovery, and compared the prevalence of symptoms seen in post-COVID condition, between patients with inflammatory rheumatic diseases and healthy controls, with and without a history of COVID-19.
3359. A proteomics study of rheumatoid arthritis patients on etanercept identifies putative biomarkers associated with clinical outcome measures.
作者: Stephanie F Ling.;Chuan Fu Yap.;Nisha Nair.;James Bluett.;Ann W Morgan.;John D Isaacs.;Anthony G Wilson.;Kimme L Hyrich.;Anne Barton.;Darren Plant.
来源: Rheumatology (Oxford). 2024年63卷4期1015-1021页
Biologic DMARDs (bDMARDs) are widely used in patients with RA, but response to bDMARDs is heterogeneous. The objective of this work was to identify pretreatment proteomic biomarkers associated with RA clinical outcome measures in patients starting bDMARDs.
3360. Improving our understanding of the paradoxical protective effect of obesity on radiographic damage: a large magnetic resonance imaging-study in early arthritis.
作者: Nikolet K den Hollander.;Annette H M van der Helm-van Mil.;Hanna W van Steenbergen.
来源: Rheumatology (Oxford). 2024年63卷4期1007-1014页
Obesity conveys a risk for RA development, while paradoxically, associating with less radiographic progression after RA diagnosis. Using MRI we can study this surprising association in detail from MRI-detected synovitis and osteitis to MRI-detected erosive progression, which precedes radiographic progression. Previous research suggested obesity associates with less osteitis and synovitis. We therefore aimed to (i) validate the previously suggested association between BMI and MRI-detected osteitis/synovitis; (ii) study whether this is specific for ACPA-positive or ACPA-negative RA or also present in other arthritides; (iii) study whether MRI-detected osteitis associates with MRI-detected erosive progression; and (iv) study whether obesity associates with MRI-detected erosive progression.
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